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>>> Psilocybin outperforms other treatments in easing depression in study
UPI News
5-2-24
by Dennis Thompson
HealthDay News
https://www.msn.com/en-us/health/other/psilocybin-outperforms-other-treatments-in-easing-depression-in-study/ar-AA1o2ppJ?OCID=BingNewsSerp
The active chemical in magic mushrooms could prove to be a powerful antidepressant, a new review finds.
Psilocybin outperformed a variety of "control" treatments in easing symptoms of depression, researchers reported Wednesday in the BMJ.
Those control groups received either placebo medications, the dietary supplement niacin (vitamin B), or microdoses of psychedelics.
"This review's findings on psilocybin's efficacy in reducing symptoms of depression are encouraging for its use in clinical practice as a drug intervention for patients with primary or secondary depression, particularly when combined with psychological support and administered in a supervised clinical environment," concluded the research team led by Athina-Marina Metaxa, a master's student with the University of Oxford's Department of Medicine in the U.K.
Depression affects an estimated 300 million people worldwide, an increase of nearly 20% over the past decade, researchers said in background notes.
Psilocybin has shown promise in reducing depression symptoms after one or two doses, with few side effects and no apparent risk of addiction, researchers said.
To provide an overview of where research now stands, a U.K. team examined data from seven clinical trials involving 436 people with depression.
Psilocybin provided a significantly greater change in depression scores than any of the control treatments, results show.
The treatment effects of psilocybin were significantly more powerful among patients who had depression alongside another mental illness, and when participants had previously used psychedelics, researchers found.
"Interestingly, a clear pattern emerged for past use of psychedelics -- the higher the proportion of study participants who had used psychedelics in the past, the higher the post-psilocybin treatment effect observed," the team wrote.
However, more evidence is needed to support psilocybin as an antidepressant, researchers said.
"Real-world" data is also needed, to assess both potential effectiveness as well as potential costs, researchers added.
Trial patients typically receive psilocybin in a calm living room with soothing music, under the supervision of a psychotherapist -- a situation unlikely to occur in typical healthcare settings.
"The combination of these elements makes this a relatively complex and expensive intervention, which could make it challenging to gain approval from regulatory agencies and to gain reimbursement from insurance companies and others," the researchers wrote.
"The high cost associated with the intervention also increases the risk that unregulated clinics may attempt to cut costs by making alterations to the protocol and the therapeutic process, which could have detrimental effects for patients," they added.
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>>> Simulations Plus (NASDAQ:SLP) is a leading modeling and simulation software provider for drug discovery and development. The company’s innovative software platform helps pharmaceutical and biotechnology companies accelerate the drug development process, reduce costs and improve success rates. Simulations Plus’ strong market position, expanding customer base and focus on innovation position are good for continued growth. The increasing complexity of drug discovery, the growing demand for efficient drug development processes and the rising adoption of computer-aided drug design are key megatrends that support Simulations Plus’ long-term prospects.
This is another stock in the healthcare industry sort of in the same boat as TCMD. Do not expect massive returns, but the possible gains are significant enough to include in a high-risk, high-reward basket of stocks. The company beat top-line estimates by nearly 6% in the recent quarter and has some of the best margin and cash positions, with negligible debt and $108 million in cash. Revenue growth is expected to be around 15% annually and 20-30% EPS growth going forward. However, you are paying a hefty premium for this stock, so I do not think life-changing returns are possible. However, it is still one of the AI stock picks worth buying for potential double-digit returns.
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https://finance.yahoo.com/news/ai-bot-predicts-7-stocks-175326496.html
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>>> Amgen, Inc. (NASDAQ:AMGN) - 14-day RSI: 37.92
https://finance.yahoo.com/news/11-oversold-blue-chip-stocks-195219274.html
Number of Hedge Fund Holders: 69
Thousand Oaks, California-based Amgen, Inc. (NASDAQ:AMGN) is a leading biotechnology company discovering, developing, manufacturing, and delivering innovative human therapeutics with a focus on areas of high unmet medical need.
On February 6, Amgen, Inc. (NASDAQ:AMGN) released its financial results for the Q4 2023. Its revenues increased by 20% y-o-y to $8.2 billion, while it generated a net income of $767 million. The normalized EPS for the quarter was recorded at $4.71, which surpassed the consensus by $0.12.
Earlier on October 6, 2023, Amgen, Inc. (NASDAQ:AMGN) completed the acquisition of Horizon Therapeutics plc in an all-cash transaction implying an equity value of nearly $27.8 billion. The acquisition strengthened the company’s inflammation portfolio by adding first-in-class, early-in-lifecycle medicines which treat rare inflammatory diseases.
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>>> Invivyd, Inc. (IVVD), a clinical-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of antibody-based solutions for infectious diseases in the United States. The company's lead product candidate is adintrevimab, a neutralizing antibody that is in Phase 3 clinical trials for the treatment and prevention of coronavirus disease, as well as developing monoclonal antibody candidates, including VYD222 and VYD224, which provides neutralizing protection against SARS-CoV-2. It also has discovery stage candidates for the prevention of seasonal influenza. Invivyd, Inc. has a collaboration agreement with Adimab, LLC for the discovery and optimization of proprietary antibodies; and the Scripps Research Institute to perform research activities to identify vaccine candidates for the prevention, diagnosis or treatment of influenza or beta coronaviruses. The company was formerly known as Adagio Therapeutics, Inc. and changed its name to Invivyd, Inc. in September 2022. Invivyd, Inc. was incorporated in 2020 and is headquartered in Waltham, Massachusetts.
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https://finance.yahoo.com/quote/IVVD/profile
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>>> Regeneron Pharmaceuticals, Inc. (REGN) discovers, invents, develops, manufactures, and commercializes medicines for treating various diseases worldwide. The company's products include EYLEA injection to treat wet age-related macular degeneration and diabetic macular edema; myopic choroidal neovascularization; diabetic retinopathy; neovascular glaucoma; and retinopathy of prematurity. It also provides Dupixent injection to treat atopic dermatitis and asthma in adults and pediatrics; Libtayo injection to treat metastatic or locally advanced cutaneous squamous cell carcinoma; Praluent injection for heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease in adults; REGEN-COV for covid-19; and Kevzara solution for treating rheumatoid arthritis in adults. In addition, the company offers Inmazeb injection for infection caused by Zaire ebolavirus; ARCALYST injection for cryopyrin-associated periodic syndromes, including familial cold auto-inflammatory syndrome and muckle-wells syndrome; and ZALTRAP injection for intravenous infusion to treat metastatic colorectal cancer; and develops product candidates for treating patients with eye, allergic and inflammatory, cardiovascular and metabolic, infectious, and rare diseases; and cancer, pain, and hematologic conditions. The company was incorporated in 1988 and is headquartered in Tarrytown, New York.
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>>> FDA rejects Regeneron lymphoma drug, setting back company’s oncology push
Industry Dive
by Jonathan Gardner
March 25, 2024
https://finance.yahoo.com/news/fda-rejects-regeneron-lymphoma-drug-115500198.html
The Food and Drug Administration declined to approve an experimental lymphoma drug from Regeneron Pharmaceuticals, asking the company to first make more progress with a trial meant to confirm benefits observed in earlier testing, the company said Monday.
Regeneron said the FDA “did not identify any approvability issues” related to the safety, effectiveness or manufacturing of the drug, which is known as odronextamab. However, the agency wants to see study participants enrolled in the “dose finding” as well as “confirmatory” parts of an ongoing Phase 3 trial before it will issue a verdict, the company said.
Should it ultimately win approval, odronextamab would compete for market share with similar, “bispecific” drugs from AbbVie and Roche. Those drugs were greenlit in 2023 and posted sales of $17 million and $65 million, respectively.
Dive Insight:
Since cracking down on “dangling” accelerated approvals for cancer drugs – conditional clearances that weren’t confirmed through further testing – the FDA has been more hesitant to grant new ones. In Regeneron’s case, the drugmaker has been seeking a speedy approval in two types of lymphoma based on studies that measured remission rates but didn’t compare odronextamab’s performance to a placebo.
Accelerated approvals are conditional and granted on smaller trials that are “likely” to predict a benefit for drug recipients. But companies seeking them must back them up with bigger studies that prove those benefits by testing their therapies against a placebo or existing treatment.
Regeneron has already begun such a trial. However, the agency doesn’t believe Regeneron has made enough progress with enrollment yet, prompting a rejection. The FDA must also sign off on the timelines for the study’s completion before Regeneron resubmits its application. Regeneron will share updates later this year, it said in a statement.
Odronextamab is a type of dual-pronged antibody drug that’s become increasingly popular in recent years. It binds to the same targets on immune and malignant cells as AbbVie’s Epkinly and Roche’s Columvi, both of which won accelerated approvals in B cell lymphomas.
In a research note Monday, Sean McCutcheon, an analyst with Raymond James, wrote that he expects an agreement between Regeneron and the FDA once the “confirmatory” portions of the study begin. Still, the setback represents a missed opportunity for Regeneron, which has struggled to compete with more established players in oncology.
“We continue to see odronextamab as a modest opportunity given competitive headwinds, with the delay as a further hurdle to garnering market share,” he wrote. “The [rejection] also further adds to investor pessimism on the company’s execution within oncology.”
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>>> Cardiff Oncology Announces Upcoming Presentations at the AACR Annual Meeting 2024
Cardiff Oncology, Inc.
March 6, 2024
https://finance.yahoo.com/news/cardiff-oncology-announces-upcoming-presentations-210500325.html
SAN DIEGO, March 06, 2024 (GLOBE NEWSWIRE) -- Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage biotechnology company leveraging PLK1 inhibition to develop novel therapies across a range of cancers, today announced publications of five abstracts that will be presented in a poster session at the American Association for Cancer Research (AACR) Annual Meeting, taking place from April 5-10, 2024, in San Diego, California.
“The posters we will be presenting at this year’s AACR meeting represent a broad view of the potential of onvansertib in several different cancer indications, including RAS-mutant mCRC, RAS-wild type mCRC, small cell lung cancer and ovarian cancer,” said Mark Erlander, Ph.D., Chief Executive Officer of Cardiff Oncology. “In RAS-mutated mCRC, we are showing the underlying mechanism through which the combination of onvansertib and bevacizumab targets the hypoxia response pathway. We believe this mechanism explains the strong clinical results we have seen in both our Phase 1b/2 and ONSEMBLE second-line RAS-mutated mCRC clinical trials. The additional posters provide new insights and rationale for future clinical trials in other cancer indications.”
Details on the posters and corresponding abstracts are shown below.
Poster Title: A phase 1b/2 clinical study of onvansertib in combination with FOLFIRI/bevacizumab revealed a new role of PLK1 in regulating the hypoxia pathway in KRAS-mutant colorectal cancer
Session Title: Microenvironment, Immunity, and DNA Repair in Therapeutic Response
Session Date and Time: Monday Apr 8, 2024: 9:00 AM - 12:30 PM PT
Location: Poster Section 27, Poster Board #14, Abstract Number #2031
This abstract presents updated clinical data and biomarker analysis from the Phase 1b/2 study evaluating onvansertib in combination with FOLFIRI/bevacizumab for second-line treatment of KRAS-mutant metastatic colorectal cancer (mCRC) patients. Analysis of patient baseline characteristics revealed superior clinical benefit in patients not exposed to bevacizumab in first-line treatment (Bev-naïve) compared to Bev-exposed patients. Bev-naive patients exhibited higher objective response rates (73.3% versus 15.7%) and longer median progression-free survival (14.9 versus 7.8 months) compared to Bev-exposed patients. Preclinical studies, using KRAS-mutant colorectal cancer mouse models revealed robust antitumor activity of onvansertib in combination with bevacizumab and a novel role of onvansertib in regulating tumor vascularization. Further preclinical investigations showed that PLK1 regulates the hypoxia pathway in KRAS-mutant CRC cells through the modulation of the hypoxia-inducible factor 1 alpha, HIF1a, emphasizing the potential crosstalk between PLK1 and angiogenesis. These findings reinforce the rationale for exploring onvansertib in combination with FOLFIRI/bevacizumab for Bev-naïve mCRC patients with KRAS mutation.
Poster Title: The PLK1 inhibitor, onvansertib, is active as monotherapy and in combination with cetuximab in RAS wild-type colorectal cancer patient-derived xenografts
Session Title: Drug Resistance 2: Ras GTPase
Session Date and Time: Monday Apr 8, 2024: 9:00 AM - 12:30 PM PT
Location: Poster Section 24, Poster Board #12, Abstract Number #1934
This abstract focuses on the preclinical assessment of onvansertib’s antitumor activity in RAS wild-type colorectal cancer, as both a monotherapy and in combination with cetuximab, a monoclonal antibody targeting the epidermal growth factor receptor (EGFR). Employing patient-derived xenograft (PDX) models, the study highlights the robust antitumor efficacy of onvansertib monotherapy, in cetuximab-sensitive and -resistant RAS wild-type colorectal cancer models. Furthermore, the combination of onvansertib and cetuximab was highly effective, resulting in tumor regression in 90% of the PDXs. These compelling data strongly support the clinical development of onvansertib as a potential treatment for RAS wild-type colorectal cancer.
Poster Title: A phase 2, randomized, open-label study of onvansertib in combination with standard-of-care (SoC) versus SoC alone for first-line treatment of RAS-mutant metastatic colorectal cancer(mCRC)
Session Title: Phase II and Phase III Clinical Trials in Progress
Session Date and Time: Tuesday, Apr 9, 2024: 1:30 PM - 5:00 PM PT
Location: Poster Section 50, Poster Board #5, Abstract Number #CT275
This abstract describes a clinical trial in progress.
Poster Title: The PLK1 inhibitor, onvansertib, synergizes with paclitaxel in small cell lung cancer
Session Title: Kinase and Phosphatase Inhibitors 1
Session Date and Time: Sunday Apr 7, 2024: 1:30 PM - 5:00 PM PT
Location: Poster Section 25, Poster Board #16, Abstract Number #606
This abstract outlines preclinical studies showing the promising potential of combining onvansertib with paclitaxel for small cell lung cancer (SCLC). The research reveals significant synergy of the combination in SCLC cell lines and demonstrates its robust antitumor activity in patient-derived xenograft models, including models resistant to the standard therapy cisplatin. Further insights into the combination's mechanism of action will be presented. These findings support that combining onvansertib with paclitaxel could emerge as a highly promising treatment strategy for SCLC.
Poster Title: In vivo anti-tumor activity of onvansertib, a PLK1 inhibitor, combined with gemcitabine or carboplatin in platinum-resistant ovarian carcinoma patient-derived xenograft models
Session Title: Application of Precision Medicine for Cancer Care
Session Date and Time: Sunday Apr 7, 2024: 1:30 PM - 5:00 PM PT
Location: Poster Section 39, Poster Board #13, Abstract Number #945
This abstract explores preclinically the potential of combining onvansertib with the chemotherapeutic agents carboplatin or gemcitabine for platinum-resistant high-grade ovarian carcinoma. Using patient-derived xenografts, we demonstrated robust efficacy for both combinations, coupled with favorable tolerability. These data underscore the potential of onvansertib to improve the efficacy of the standard-of-care carboplatin and gemcitabine for patients with platinum-resistant high-grade ovarian carcinoma.
The abstracts are available on the AACR Online Program and will be published in the online Proceedings of the AACR. Following presentation, the posters will be posted to the "Scientific Presentations" section of the Cardiff Oncology website.
About Cardiff Oncology, Inc.
Cardiff Oncology is a clinical-stage biotechnology company leveraging PLK1 inhibition, a well-validated oncology drug target, to develop novel therapies across a range of cancers. The Company's lead asset is onvansertib, a PLK1 inhibitor being evaluated in combination with standard-of-care (SoC) therapeutics in clinical programs targeting indications such as RAS-mutated metastatic colorectal cancer (mCRC) and metastatic pancreatic ductal adenocarcinoma (mPDAC), as well as in investigator-initiated trials in small cell lung cancer (SCLC) and triple negative breast cancer (TNBC). These programs and the Company's broader development strategy are designed to target tumor vulnerabilities in order to overcome treatment resistance and deliver superior clinical benefit compared to the SoC alone.
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>>> Acurx Pharmaceuticals, Inc. (NASDAQ:ACXP) Q4 2023 Earnings Call Transcript
Published March 19, 2024
by INSIDER MONKEY TRANSCRIPTS
https://www.insidermonkey.com/blog/acurx-pharmaceuticals-inc-nasdaqacxp-q4-2023-earnings-call-transcript-1276747/#q-and-a-session
Acurx Pharmaceuticals, Inc. (NASDAQ:ACXP) Q4 2023 Earnings Call Transcript March 18, 2024
Operator: Greetings and welcome to the Acurx Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Earnings Results and Business Update Call. At this time, all participants are in a listen only mode. A question-and-answer session will follow the formal presentation. [Operator Instructions] As a reminder, this conference is being recorded. I would now like to turn the call over to your host, Robert Shawah, Chief Financial Officer for Acurx Pharmaceuticals. Thank you. You may begin.
Robert Shawah : Thank you, Melissa. Good morning, and welcome to our call. This morning we issued a press release providing financial results and company highlights for the fourth quarter and full year 2023, which is available on our website at acurxpharma.com. Joining me today is David Luci, President and CEO of Acurx; as well as Robert DeLuccia, Executive Chairman. David will give a corporate update and outlook. After that, I’ll provide some highlights of the financials from the quarter and year end of December 31, 2023, and then turn the call back over to Dave for his closing remarks. As a reminder, during today’s call, we’ll be making certain forward-looking statements. These forward-looking statements are based on current information assumptions, estimates and projections about future events that are subject to change and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained in forward-looking statements.
Investors should consider these risks and other information described in our filings made with the Securities and Exchange Commission, including our annual report on Form 10-K, which we filed on Friday, March 15, 2024. You are cautioned not to place undue reliance on these forward-looking statements and Acurx disclaims any obligations to update such statements at any time in the future. This conference call contains time sensitive information that’s accurate only as of the date of this live broadcast today, March 18, 2024. I’ll now turn the call over to Dave Luci. Dave?
David Luci : Thanks Rob. Good morning, everyone, and thanks for joining us to review our financial results for the fourth quarter of 2023 and also to hear some very exciting recent updates. Then we’d be pleased to take any questions. First, I’ll summarize some of our key activities for the fourth quarter of ‘23 or in some cases shortly thereafter. On October 2, 2023, we ended enrollment in our Phase IIb clinical trial of ibezapolstat or IBEZ, our lead antibiotic candidate for the treatment of patients with C. difficile infection or CDI. On November 2, 2023, we reported top line data from the Phase IIb clinical trial including overall results from the full Phase II study, demonstrating and ibezapolstat clinical cure rate at end 10 days oral treatment or EOT of 96%, 25 of 26 patients, which included 100% cure in Phase IIa and 94% cure in Phase IIb compared with vancomycin control arm the standard of which was 14 for 14 or a 100% at end of treatment and 94% was sustained cures.
No safety concerns were reported in either arm of the Phase IIb clinical trial and ibezapolstat was well tolerated in all patients in both the Phase IIa open-label trial and the Phase IIb vancomycin control segment. In consultation with our scientific advisors, the company determined that based on review of aggregate blinded data, the Phase IIb vancomycin control trial segment was terminated early due to success showing high observed clinical cure rates with no emerging safety concerns, clinical comparability was established. We also stated at that further data would be provided as it becomes available on secondary and exploratory endpoints for the Phase IIb trial segment, including sustained clinical cure data at 30 days after EOT and extended clinical cure data 94 days after EOT as well as comparative data on the impact on the patient’s microbiome.
On December 11 ‘23, we announced the sustained clinical cure data. These data showed that in the Phase IIb trial segment 100% or 15 out of 15 of our advantaged patients who were cured at EOT remained cured with no reinfection 30 days later, while vancomycin experienced a reinfection rate of 14.3%, 2 of 14 patients were reinfected.
On January 17, 2024, we announced positive comparative microbiology and microbiome data for the ibezapolstat in CDI patients from the Phase IIb clinical trial segment. Ibezapolstat to outperformed vancomycin showing eradication of fecal C. difficile at day three of treatment in 15 of 16 treated patients versus vancomycin, which had eradication of fecal C. difficile in just 10 of 14 treated patients. Additional data from the Phase IIb clinical trial showed ibezapolstat but not vancomycin consistently observed and allowed regrowth of key gut bacterial species believed to confer health benefits including prevention of recurrent C.difficile infection.
We anticipate that additional data from the secondary and exploratory endpoint will provide further favorable separation these two therapeutic options in our Phase III clinical trial program and ultimately in the marketplace if approved. Additional analysis, regarding other secondary and exploratory endpoints will be forthcoming as data become available. We remain particularly excited about the dual impact of ibezapolstat to treat acute C. difficile infection while appropriately managing the long-term care of each patient’s microbiome, which we believe is exceptional for antibiotic therapy. Having robust preclinical — clinical and manufacturing data to date, we submitted a formidable information package in early February to FDA along with a request for an end of Phase II meeting, which was granted by FDA on Feb 26, and is scheduled to occur in April.
We anticipate discussing our Phase III clinical trial mandate at this meeting and would anticipate documented meeting minutes from FDA sometime in the second quarter this year. We also announced that the European Medicines Agency approved their application to be designated as a small to medium sized enterprise or SME in Europe, which provides for certain benefits including fee reductions and other support from the European Medicines Agency for seeking a marketing authorization in Europe. In November, 2023, we filed an amendment to our shelf registration statement with the Securities and Exchange Commission and put up a $17 million at the market or ATM facility with Alliance Global Partners acting as sales agent to the company. Proceeds from the ATM will be used for general corporate purposes going forward, including our plan Phase III clinical trial mandate.
In October, 2023, at ID Week, Dr. Kevin Garey presented on our behalf with selective spectrum of activity data from our Phase IIa clinical trial. Many of you may recall, Dr. Gary is Professor and Chair University of Houston, College of Pharmacy and the Principal Investigator for our microbiome aspects of the ibezapolstat clinical trial program. Also at ID Week, Bob DeLuccia, our Executive Chairman, presented our new class of novel DNA pol IIIC inhibitors in our preclinical pipeline at the symposium entitled new antimicrobes in the pipeline. Now that’s a lot of activity to digest. So I’ll summarize further as to where we are today. As we speak, we’re preparing to advance ibezapolstat into Phase III clinical trials and anticipate a favorable outcome from our upcoming FDA meeting regarding readiness to proceed and also to obtain agreement on the regulatory pathway for a new drug application filing for marketing approval in the U.S. once Phase III is completed.
We’ve also officially started the regulatory process in Europe will add other territories to the list later this year. The Phase III trials will include U.S. and international sites to enhance overall enrollment and to support international regulatory filings for marketing approval. This will save us a lot of time and money and allow us to expand our ultimate commercial reach. To ensure Phase III clinical trial and enrollment as quickly as possible, we’re adding substantially more clinical trial sites, way above the number we used to conduct our U.S.-only Phase II trials. We’re now getting our arms around the costs and time lines, but our plan is to conduct the required two Phase III registration trials consecutively not concurrently given the size of our company and need to use our financial resources most efficiently.
The time line for conduct of our Phase III trial is not a concern since ibezapolstat will have a rolling 10 years of regulatory exclusivity in the U.S. from the FDA approval date with similar legislation in Europe, the U.K. and Japan. We will continue to seek strategic transaction for the company, including a potential partner for the further development and potential commercialization of ibezapolstat as well as a potential sale merger third-party ex U.S. territorial or licensing arrangement or our strategic transaction alongside in parallel with our preparation for Phase III clinical trials. At this time, we have no commitments from potential partners or others to provide the company with capital, but we started this initiative only recently in February after our Phase IIb data was released.
So basically, we have two formidable plans going forward, and we’re equally excited about partnering M&A and Phase III enrollment as next steps over the next 12 months. As we’ve consistently reported, ibezapolstat continues to outperform in a series of potentially life-threatening infectious disease called C. difficile that the U.S. CDC categorizes as an urgent threat and there’s a need for new classes of antibiotics for initial treatment but also has a low incidence of recurrence. Ibezapolstat also has FDA Fast Track designation for treatment of C. difficile infection. Additionally, we believe ibezapolstat, if approved, could make a favorable impact by reducing the cost burden of current C. difficile infection in our U.S. health care system, which is estimated at $4.7 billion annually.
We do believe the best is yet to come. And now back to our CFO, Rob Shawah to guide you through the highlights of our financial results for the fourth quarter and full year 2023. Rob?
Robert Shawah : Thanks, Dave. Our financial results for the fourth quarter and 12 months ended December 31, 2023, were included in our press release issued earlier this morning. The company ended the year with cash totaling $7.5 million compared to $9.1 million as of December 31, 2022. Subsequent to year-end, the company sold an additional 1,121,793 shares under its ATM financing program with gross proceeds of approximately $4.5 million. Research and development expenses for the three months ended December 31, 2023, were $1.9 million compared to $1.4 million for the three months ended December 31, 2022. The increase was due to timing of Phase IIb trial related costs and an increase in consulting costs. For the year ended December 31, 2023, research and development expenses were $6 million, which is $4.8 million for the year ended December 31, 2022.
The increase to due primarily the Phase IIb costs and an increase in consulting costs. General and administrative expenses for three months ended December 31, 2023 were $3.2 million compared to $1.8 million, where the three months ended December 31, 2022. The increase was due primarily to $800,000 increase in professional fees, a $0.1 million increase in share based compensation and a $0.3 million increase in employee compensation costs. For the year ended December 31, 2023, general and administrative expenses were $8.5 million versus $7.3 million for the year ended December 31, 2022. The amounts reflect an increase in professional fees of $0.5 million, an increase of $0.3 million in share based compensation and an increase of $0.3 million in employee compensation costs.
The company reported a net loss of $5.1 million or $0.37 per diluted share for a three months ended December 31, 2023, compared to an net loss of $3.3 million or $0.28 per diluted share for the three months ended December 31, 2022, and the net loss of $14.6 million or $1.15 per share for the year ended December 31, 2023 compared to a net loss of $12.1 million or $1.12 per diluted share for the year ended December 31, 2022, the reasons previously mentioned. The company had 14,468,229 shares outstanding as of December 31, 2023. With that, I’ll turn the call back over to Dave.
David Luci : Thanks Rob, and to all of you for joining us today. I’ll now ask Bob DeLuccia our Executive Chairman to provide his perspective given Bob manages our R&D program and when Bob is finished, operator, please open the call for questions. Bob?
Robert DeLuccia : Thanks Dave and Rob for updating our stakeholders and thanks to all for the continuing support as we advance ibezapolstat into Phase III clinical trials. It is a very exciting time and really it’s the final step to commercialize ibezapolstat for patients in need of a promising new antibiotic with a novel bactericidal mechanism of action to treat CDI, and many of you will remember that approximately 30,000 people die each year in the U.S. alone from CDI and the annual incidence is more than about 500,000 per year in the United States. But the bottom line here is that if the outcome of our Phase III trials are consistent with our Phase II experience, we’ll have an approvable new drug in our hands. The data are solid, the market is large and our manufacturing cost is low.
We do have a robust preclinical — clinical microbiome safety and CMC evidence package that we’ve submitted to the FDA and as Dave mentioned, they’ve determined it acceptable to grant us an end of Phase II meeting next month to discuss trial design, patient enrollment targets and to confirm our readiness to go forward. So to complete our Phase II international Phase III trials, which will be done sequentially as Dave mentioned and as quickly as possible, we’ve already initiated steps to advance screen potential clinical trial sites, which in addition to North America will cover sites in about 17 other countries, including several in Eastern Central, Northern and Western Europe, totaling about 100 sites, which we believe will accelerate overall enrollment.
We’ll also be including several high enrolling trial sites from the fast enrolling Summit Phase III CDI trial, which was conducted during the height of COVID-19. In addition to global scope of the program compared to Phase II and the strength of our Phase II data, the Phase III protocol will allow more flexibility for inclusion and exclusion criteria as a standard for CDI pivotal registration trials. So we anticipate faster enrollment. Then once we have results from our FDA meeting and documented meeting minutes from the FDA, our next priority will be to submit our plans to the European Medicines Agency or EMA for conducting Phase III clinical trials in Europe. So if approved, we’ll have the first new class of antibiotics approved by FDA in over 30 years with only confirmatory Phase III trials between now and market introduction.
Ibezapolstat as Dave mentioned is fast tracked by FDA, it’s fully patented and with regulatory exclusivity 10 years post-market introduction in the U.S. and similar opportunities for regulatory exclusivity in other geographies to pursue at the appropriate time. And also with recent focus on minimizing effects on the microbiome to lower recurrence of infection, we stand out from other antibiotics since we’ve shown no reinfection in Phase III patients (note - I think he meant Phase 2) who were initially cured of their infection. So in my over 50 years of experience in antibiotic development and marketing. I think, I have got a great rear view mirror and a clear vision to say that we have a winner here for patients with CDI and in general for better public health as well as for our shareholders.
Thanks for letting me comment, Dave.
Robert Shawah : Thank you, Bob. Melissa, we’re now ready to go to Q&A.
Operator: [Operator Instructions] First question is from the line of Jason McCarthy with Maxim Group.
Michael Okunewitch: This is Michael Okunewithch for Jason. I guess to start off, I’d like to see if you could give an idea of what sort of time line you are expecting between getting those meeting minutes back from the FDA and actually launching the first of those Phase III studies?
David Luci: So we think that we will be ready to enroll the first patient in for the international Phase III in the fourth quarter of this year.
Michael Okunewitch: And then, I guess, regarding the international component to the study, could you talk a little bit more about the path to actually getting those sites online? And is this something that you would look to have in place ahead of the launch? Or would you start with what do you have when you usually enroll the study like you are in these international sites as they were approved?
David Luci: We will be a little bit on both. We have a number of the sites already kind of ready to go from the international list. There won’t be any hold up from the international aspect of it. We actually have a consultant who is extremely familiar with Phase III C. diff international trials who’s been guiding us in this regard. But yes, clearly, when our manufactured product is ready, we’ll be ready to go with a large wedge at the very least of the hundred sites.
Michael Okunewitch: And then one last one from me, and I’ll hop back in the queue. With regards to securing international partnerships, do you think this is something with your existing body of data that is compelling enough that you could really progress those conversations now and get a partner or would you expect it to be more after you get that first slot of Phase III data?
David Luci: No, I would expect that the prime time for us to find, I’d say a European partner or a Japanese partner would be — when we start enrolling the first of the two Phase III trials, once we have the Phase III trial mandate completed and we’re ready to find or to file a new drug application, if we haven’t had a partnership by then, I think it would probably not occur until a year after the market introduction, because folks will want to see how well we do. I think that the time is right now. I’ll make one additional comment on the territorial partnerships to carry Michael’s question response a little bit further. As we look at strategic alternatives, there’s M&A full stop and there’s territorial licensing and co-development agreements that I have entered into in the past.
With our share price being where it is, the territorial licensing is a bit easier to consummate because it’s all about the intrinsic value of your drug as opposed to M&A, which you can’t in my view, wholly divorce yourself of your NASDAQ share price. So it may be a more attractive option for our board of directors and but we’ll see what term sheets present themselves.
Operator: Our next question comes from the line of James Molloy with Alliance Global Partners.
James Molloy : The Phase III trials, I know that obviously a lot to go here, but can you walk through expectation which we should expect to see coming out of the end of Phase II? And then the trial designed to sort of the length of time to run is about two years start to finish. So Phase IIb, I know we we’re dealing with COVID then as well. Is that seem reasonable to run these two Phase IIIs from start to finish?
David Luci: So we’re going to address one Phase III and then the idea would be to either do a strategic transaction after that first Phase III or with positive data on the first Phase III, if it’s the same or consistent with our Phase II, get a share price rise and then raise capital for the second Phase III. So we’re targeting a year and a half from start to finish for the first Phase III, and that would have us completing the first Phase III in the second quarter of ‘26. Now I can tell you that we spent a lot of time looking at the Summit Therapeutics experience and Summit enrolled in the teeth of COVID, 750 or so patients in about two years time largely in Eastern Europe, where the behavior patterns weren’t dramatically changed apparently, like they were here in the U.S. So, that’s a public list on clinicaltrial.gov.
So we were able to see exactly the heat patterns from their enrollment. So that’s gone a long way to helping us get comfortable with fixing the enrollment issues we had during COVID with our U.S. only trial.
James Molloy : I thought I’d heard you say earlier in the call that you’re going to run the two trials at the same time obviously misheard that, you’re running back to back.
David Luci: No. Our going in plan is to do them consecutively instead of concurrently for the financial reasons that we discussed. But if we get a partnership, say a European licensing agreement and that brings in and enough money to call an audible along the way and at that point then we would certainly start the second Phase III accordingly.
Operator: [Operator Instructions] Our next question comes from the line of Ed Arce with H.C. Wainwright.
Ed Arce : So a couple from me, firstly on the end of Phase II next month and I apologize I joined late, so maybe you went over this already, but wanted to make sure to review sort of the key objectives in particular what do you not have agreement with the agency yet on that you would seek to get agreement on next month in that meeting? And then secondly, I think you made mention of a wider inclusion exclusion criteria for the Phase III relative to the prior Phase II. And so just wanted to get a little more clarity on that in particular that a view to concerns there might be around the risk that you could change the sort of design of the trial and there could be some disruption from a slightly different patient population.
David Luci: Thank you, Ed. I’ll answer the first part of your question and ask Bob to provide a response for the inclusion and exclusion criteria part of your question. So for the first part of your question, what we hope to agree on with the FDA is the overall protocol designed for our Phase III trial, including the number of patients to be enrolled the notion that we’re going to have a vancomycin control arm like we had in our Phase IIb. Largely, the trial design is 90% plus of what the trail design was for our Phase IIb. So we don’t expect a real lot of drama, but it’s a necessary step in order to kind of be allow to into Phase III, so we’re looking forward to our valuation point that we have to get through. Bob, did you want to add some comment on the inclusion and exclusion criteria?
Robert DeLuccia: I think really two things, which is standard fair for going forward in Phase III is that we’ll have additional patients beyond mild and moderate that are included in the trial. So this is not severe CDI, but a little bit more than moderate according to the IDSA criteria for patient entry into a trial in the study. So we’ve already submitted a framework to the FDA in the meeting package. It’s very straightforward. I’d even say a higher percentage that they’ve said at 90%. We’re pretty much there with the design. It’s the same design basically, it’s Phase II and very standard according to the FDA guidance from October 2022, I believe it is, as to what needs to be included in the Phase III clinical trials. So we’ll be discussing final numbers of patients, as I said before and statistical analysis plan will be finalized at that upcoming FDA meeting as well. Anything else? Does that answer your question?
Ed Arce: Yes, that’s helpful.
Operator: Thank you. Ladies and gentlemen, that concludes our question-and-answer session. And this concludes our call today. We thank you for your interest and participation. You may now disconnect your lines.
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The FDA approved Amarin’s CVD risk-reducing drug, Vascepa, in 2019, having first approved it for triglyceride reduction in 2012. At a cost less than $60/year under my insurance plan, I began taking oral Vascepa capsules, off-label, with prescription in April 2013 at age 76(father died from 2nd heart attack at age 51 in 1959). Never obese, I have lost about 25 lbs while using 20% less LLY insulin to manage my T1-D since 2016. I now weigh the same as I did in my university years, 1955-1961.
PFE sells Vascepa in all Canadian provinces, per agreement.
AMRN’s share price, $0.90, is about even YTD. Share price was $18-25 in 2019. Shareholders will vote on a share repurchase plan in early April 2024 while waiting for revenue-driven earnings from Europe and Asia, and results from the Brave trial (Indication: Alzheimer’s ). Sarissa Capital holds ~ 9.9% of outstanding shares, and views Amarin as under-valued. Will AMRN share price out-perform share price of either LLY or NVO in 2024-2025 in % gain?
Orchard Therapeutics - >>> A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access
CNN
by Brenda Goodman
March 20, 2024
https://www.yahoo.com/lifestyle/fda-approved-lifesaving-therapy-children-194412723.html
A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a wholesale price of $4.25 million, its manufacturer announced Wednesday, making it the world’s most expensive medicine.
Lenmeldy was approved by the US Food and Drug Administration on Monday and is the first therapy for the rare and devastating disease, which typically kills affected children before they turn 7. About 40 children are born with MLD in the US each year.
The wholesale cost isn’t usually what patients pay, but it’s a cost that’s considered and shouldered by public and private health insurance plans, including state Medicaid plans, which cover roughly 4 out of every 10 children in the United States.
Manufacturers of gene therapies say the big prices reflect big benefits — the chance to be free of a disabling or even fatal disease — and they point out that they need to be able to recoup the steep costs of development, testing and manufacturing their products.
Health policy experts say that as the list of gene and cell therapies with eye-popping prices grows, it may strain the ability of states and other insurers to cover their costs, and ultimately limit patient access if plans begin to exclude these therapies as a class from coverage.
Dr. Bobby Gaspar, the co-founder and CEO of Orchard Therapeutics, the company that makes Lenmeldy, said the treatment is “paradigm-shifting medicine and has the potential to stop or slow the progression of this devastating childhood disease with a single treatment.”
“We are committed to enabling broad, expedient and sustainable access to this important therapy for eligible patients with early-onset MLD in the U.S.,” Gaspar said in a statement.
Price of hope
Lenmeldy takes stem cells from someone with MLD and uses a harmless virus to insert working copies of a faulty gene. The repaired cells are then infused back to the patient, where they begin to produce an enzyme that’s lacking in children who have the disease. Some of the cells eventually migrate to the bone marrow, where they continue to live and make new cells that also make the enzyme, providing a long-lasting benefit to patients.
The first patients treated with Lenmeldy have now been followed for more than 12 years, and researchers continue to find gene-modified cells making the missing enzyme, said Orchard Therapeutics’ Gaspar.
“We can’t say at the moment that this will last a lifetime, but what we can say is that there is a longterm durable effect,” Gaspar said.
MLD is an inherited disorder, and children born with it lack an enzyme needed break down fatty substances called sulfatides. The build-up of these fatty materials eventually becomes toxic to nerves, leading to the progressive loss of movement and thinking.
Babies with MLD develop normally for a time and but then typically begin to lose the ability to walk and talk around age 2. The disease advances rapidly, causing children to deteriorate into a vegetative state.
“We are over the moon regarding what this means for other families,” said Kendra Riley, 41, of Phoenix, who has two children with MLD.
For their family, the FDA’s action this week was bittersweet.
Riley’s 5-year-old daughter, Olivia, is in hospice after being diagnosed with MLD as a toddler.
Riley says the first clues that something was wrong came when Olivia was around the age of 2. She began to have trouble walking, and her head started to regularly tilt to the side when she would watch TV.
“We thought we just needed some physical therapy,” Riley said.
“Then the irises of her eyes started vibrating. That’s when we knew something else was going on.”
By the time doctors diagnosed Olivia with MLD, it was too late for the gene therapy to help. But knowing that the condition is inherited, they were able to get their younger daughter, Keira, tested and diagnosed.
Keira, now 4, was the 32nd child in the world to get the therapy, which is most effective before children show symptoms.
The treatment wasn’t yet available in the United States, so the family crowdsourced donations to temporarily relocate to Italy in 2020 to get the therapy. It cost them about $500,000 to live abroad and pay for Keira’s medical care, even though the company provided the gene therapy for free.
“Having this FDA-approved therapy means that if a child does get diagnosed before symptom onset, they have a chance at normal life,” Riley said.
“She is doing amazing. Zero symptoms,” she said of Keira. “You would never know.”
‘Exciting’ drug development
The Boston nonprofit Institute for Clinical and Economic Review, or ICER, which evaluates the cost effectiveness of new drugs, published a final report on Lenmeldy last fall and estimated that the cost of the therapy would match its expected benefits to patients if it was priced between $2.3 million and $3.9 million.
Experts agreed that even such hefty price tag would be worth its results. Children with the disease who aren’t treated usually die within five years of their diagnosis; the oldest patient to have the therapy has now been followed for more than 12 years and appears to be developing normally.
“It’s taking a child who would have had a miserable short life and likely giving them a normal life. And that’s worth a lot of money,” said Dr. David Rind, the chief medical officer for ICER. “This is one of the more exciting drugs that we’ve looked at.”
Still, the Lenmeldy’s price came in hundreds of thousands of dollars higher that even Rind expected.
“I think when you get into numbers this big that people don’t necessarily pay attention when it gets a little bigger, but I do think this price is too high,” Rind said.
ICER gave a range of $2.29 million to $3.94 million — that doesn’t mean that the top of the range is the right price, said Rind “despite the desire by manufacturers to think of it that way.”
“Going $310,000 above the very top of that range is actually a lot of money,” he said.
The last product to top the charts on a per-treatment basis was another gene therapy called Hemgenix, which was approved in 2022 to treat a blood clotting disorder called hemophilia B. Its list price was $3.5 million for a one-time treatment.
Close behind that one is Elvevidys, which was approved in 2023 for muscular dystrophy at $3.2 million, and Skysona, approved in 2022 for a disease related to MLD called adrenoleukodystrophy, which costs $3 million for a one-time dose.
Even with a top tier price, the MLD treatment will not be a blockbuster, Rind noted, because the disease is so rare. Some gene therapies approved for rare diseases have disappeared because the companies that made them couldn’t make enough money to stay in business. Orchard Therapeutics has already struggled to market a previous gene therapy it developed, leaving patients hanging.
Edwin Park, a research professor at the McCourt School of Public Health at Georgetown University, worries about the impact to states, which share the cost of covering patients on Medicaid with the federal government.
Medicaid requires a minimum rebate from drug manufacturers, but without any competition, there probably won’t be much negotiation about the price of the therapy, Park said.
More and more states are closely following drug pipelines after being surprised by the costs of the antiviral medications that cure hepatitis C, he said.
But even paying for one or two children to get Lenmeldy a year could be a sizeable cost for some states to absorb.
“Unless states have allocated appropriately for it, and looked at the drug pipeline, they may not be prepared for what could be significant cost spikes,” Park said.
New possibilities for patients
This latest approval comes as the FDA is working to clear a backlog of cell and gene therapies that have been waiting for its attention since the pandemic. The agency has hired more staff to review the new treatments and set up a new superoffice, the Office of Therapeutic Products, to oversee their regulation.
Lenmeldy has been available in Europe since 2020, but it took four more years to bring it to the US.
Dr. Nicole Verdun, who was hired to head the new FDA initiative last year, said Lenmeldy’s approval is a sign that things are moving in the right direction.
“MLD is a devastating disease that profoundly affects the quality of life of patients and their families,” Verdun said.
“This approval represents important progress in the advancement and availability of effective treatments, including gene therapies, for rare diseases.”
Dr. Barbara Burton, who is an attending physician, of genetics, genomics and metabolism at the Ann & Robert H. Lurie Children’s Hospital of Chicago, said it was a watershed moment for doctors, too.
“For too long, my colleagues and I have consoled families at their most vulnerable times — usually following an arduous diagnostic odyssey, coping with a dire prognosis and being told there were no treatments, and then having to watch their young child slip away.
“With this approval, we are now one significant step closer to ensuring future generations of children, families and healthcare professionals no longer need to experience first-hand the terrible manifestations this disease has on untreated patients,” she said.
The approval of a treatment is already leading to new ways to catch the disease earlier.
Gaspar, of Orchard Therapeutics, noted that the company was supporting studies of newborn screening tests in the hopes of catching and treating the condition earlier, and screening is happening in some US states.
“Over 250,000 babies have been screened them as a result of these initiatives,” Gaspar said, and five babies with MLD have been identified. At least three of those babies have now been treated.
Riley said she wishes screening had been available for Olivia. The 5-year-old is stable for now, but Riley said she lives in fear that Keira could bring germs home.
“The common cold could kill her,” Riley said. “So every time her sister has come home with a cold from school, that could be potentially something that takes her out.”
Olivia can’t walk or talk, and she’s fed through a tube. Riley says she requires round-the-clock care. Physical and occupational therapists come to the house each week “to keep her mind moving. She does understand what’s going on.”
She said that as they have watched Olivia slip away, they’ve come to understand that her life has had profound purpose. But they say they hope that the approval of Lenmeldy means that no other families with MLD will have to lose one child to save another.
“We’ve always called Livvy ‘Keira’s guardian angel on Earth,’ ” Riley said. “She’s here for a reason.”
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>>> Will Novo Nordisk Overtake Eli Lilly as the Most Valuable Healthcare Stock?
David Jagielski
Motley Fool
March 20, 2024
https://finance.yahoo.com/news/novo-nordisk-overtake-eli-lilly-123000972.html
Novo Nordisk (NYSE: NVO) and Eli Lilly (NYSE: LLY) are the top two healthcare stocks in the world based on market cap. Their diabetes and weight loss medications are a big reason these have been among the best healthcare investments to own over the past few years. Today, Eli Lilly is the larger of the two companies, with a market cap of more than $700 billion. Novo Nordisk, however, isn't too far behind, sporting a valuation about $600 billion. And there are some recent catalysts that suggest that gap could shrink in the future.
Why Novo Nordisk could soar higher this year
Novo Nordisk stock is up 28% so far in 2024, and there are a couple of reasons it may become an even hotter in the weeks and months ahead.
On March 8, the Food and Drug Administration approved Novo Nordisk's weight loss treatment, Wegovy, for a new indication: reducing cardiovascular risk in obese or overweight adults. Up until then, the drug was only approved as a treatment for weight loss. The label expansion gives patients another reason to use the drug, which could result in more prescriptions and insurance coverage -- and thus, more revenue.
Novo Nordisk has also been working on a weight loss pill (Wegovy is an injectable) that has been demonstrating encouraging results in clinical trials, showing that it can achieve faster weight loss than Wegovy: 13% after 12 weeks versus just 6% with the injectable treatment. It was an early-stage trial but the data is promising nonetheless.
In 2023, Novo Nordisk's sales increased by 36% (when excluding the impact of foreign exchange) to 232.3 billion Danish kroner ($34.8 billion). Wegovy's sales of 31.3 billion Danish kroner soared by a staggering 420%. And with much more potential on the horizon with a new indication for Wegovy and it entering new markets, Novo Nordisk is nowhere near done growing. Having a weight loss pill in development that could be even better than Wegovy gives investors even more reason to stay bullish on the stock for the long haul.
Could Eli Lilly's stock struggle?
Novo Nordisk is more of a pure-play weight loss and diabetes investment, whereas Eli Lilly's business is much more diversified. And that diversification is one of the reasons the healthcare stock may stumble a bit this year. Recently, regulators delayed making a decision on Eli Lilly's Alzheimer's treatment, donanemab. While it's likely to still obtain approval, the stock fell on the news.
In addition to diabetes treatments, Eli Lilly's top five treatments for the last three months of 2023 featured a breast cancer drug (Verzenio) and psoriasis medication (Taltz). While weight loss is a big growth opportunity for Eli Lilly, with recently approved Zepbound just starting to generate revenue for the business, its operations are certainly broader than Novo Nordisk's.
Another reason the healthcare stock could face pressure this year is that it trades at a lofty 60 times forward earnings, which is based on analyst expectations of where its profits will be in the next year. By comparison, Novo Nordisk trades at a multiple of 39. With a much higher premium, there's more pressure for Eli Lilly to deliver on not just its weight loss and diabetes treatments but on its other drugs as well.
Will Novo Nordisk become the more valuable healthcare stock?
Over the past month, shares of Eli Lilly have fallen by about 3% while Novo Nordisk stock has risen by close to 7%. The gap is shrinking between these two companies and there's a possibility it narrows even more narrow later this year, depending on how these businesses perform.
I don't, however, expect Novo Nordisk to become the more valuable company. Eli Lilly is simply too strong, and with Zepbound in its very early innings of generating revenue and the approval of donanemab still a strong possibility, the recent pullback in price may only prove to be temporary.
Overall, these are two solid healthcare stocks and whichever you decide to invest in may ultimately depend on whether you prefer to focus on the broader and pricier business (Eli Lilly) or a slightly cheaper company whose priorities center around diabetes and weight loss (Novo Nordisk). But with stellar results and exciting futures ahead, both of these stocks can be great buys.
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re: CRDF
I don't see how any analyst writing about CRDF can fail to mention Pfizer's involvement with the company. Pfizer has taken a tiny equity stake in CRDF and Adam Schayowitz (from Pfizer) has been added to the BOD. The trials changed direction after Schayowitz's appointment and Pfizer is now running the lead trial in CRC.
Bladerunner
PIper Sandler raises price target on CRDF from $5 to $7. Stock up 25%.
Bladerunner
CRDF: Change of opinion by "Seeking Alpha" analyst
Cardiff Oncology: Why The Bulls Are Winning Right Now (Rating Upgrade)
Mar. 08, 2024 7:37 PM ET Cardiff Oncology, Inc. (CRDF) Stock 8 Comments2 Likes
Galzus Research
Summary
Cardiff Oncology is focused on developing a drug for colorectal cancer and pancreatic cancer, with promising early activity shown in colorectal cancer patients.
The company is now focused on moving their drug to the first-line setting and has started a clinical trial to evaluate its efficacy.
Cardiff Oncology has a strong financial position with enough funds to operate through Q3 2025, but the success of their drug is heavily dependent on the outcomes of a single trial.
Topline Summary and UpdateCardiff Oncology (NASDAQ: CRDF) is a cancer-focused biotech working on developing a drug that exploits a novel target, particularly in colorectal cancer. In my past articles, I have been lukewarm to slightly pessimistic about their findings presented to date and their overall outlook. But recent news has sent them on a meteoric rise, and the ensuing roller coaster has readers asking me directly for input on this company. So let's examine what happened to lead to this strong rise in value, as well as where I think they might be headed next.
Pipeline UpdatesOnvansertib
Today, the PLK1 inhibitor onvansertib remains the one and only drug in development by CRDF, meaning its entire future rests on the success or failure of this agent. It is being studied mainly in a few forms of gastrointestinal cancer, namely colorectal cancer and pancreatic cancer, both representing critical unmet needs in oncology.
Most of the focus I've given in my previous writings has been for the colorectal cancer program, which has demonstrated some encouraging (though early) activity in the setting of KRAS -mutant, metastatic disease. Updated findings using add-on onvansertib with FOLFIRI-bevacizumab in the second-line setting have since been published in Clinical Cancer Research, showing a 44% response rate among 18 patients, with reasonable toxicity.
Now, the company's focus is on moving onvansertib to the first-line setting, hurdling over the typical path we have taken in targeted therapy development of starting with more "dire" clinical settings, establishing efficacy, and then moving them up. The phase 2 CRDF-004 study is seeking to randomize 90 patients with newly diagnosed, metastatic, KRAS or NRAS mutated CRC to receive first-line doublet chemotherapy plus bevacizumab, with or without one of 2 doses of onvansertib. The primary endpoint is objective response rate. CRDF announced that the first patient was dosed in this study on leap day. Initial top-line findings are anticipated later this year.
The phase 2 ONSEMBLE second-line study has been discontinued to focus on the frontline study, but CRDF provided an update in their most recent guidance that they interpret as evidence that patients who had no prior bevacizumab seemed to have better response rates, as high as 50%. It is worth keeping in mind that the patient numbers are small here, and in the standard of care, most patients with KRAS mutated mCRC are going to receive bevacizumab as part of their care, so it's difficult to interpret these findings with any real de
We've also gotten glimpses of clinical development for onvansertib in non-CRC settings. Most notably, a report of activity back in September suggests preliminary benefit from onvansertib in metastatic pancreatic cancer and extensive-stage small cell lung cancer. In particular, 4 of 21 patients with pancreatic cancer treated with add-on onvansertib (plus nal-IRI and 5-FU) yielding a 19% objective response rate, which tracks favorably with the findings from NAPOLI-1. A single response was observed with onvansertib monotherapy in the 7 patients with SCLC.
Financial OverviewAs of their most recent earnings report, CRDF held $77.4 million in total current assets, including $21.7 million in cash and equivalents, and another $53.2 million in short-term investments. Their reported annual loss was $45.4 million, with a recognized net loss of $41.4 million after interest income and other expenses.
Given these expenses, CRDF has upwards of 2 years of cash and assets on hand to fund activities, with a serious going concern emerging about a year from now at the current burn rate, by my estimation. This is consistent with guidance from the company saying they have funds to operate through Q3 2025.
Strengths and RisksStrength - CRDF's fiscal management focuses on high-priority studies
For a company in the middle of developing a drug, CRDF has a rather low cash burn rate. This could help give them time to get a data readout that propels them to the levels that we've seen recently with the likes of Janux Therapeutics, where relatively small numbers rev up a huge cash raise. Such penny-pinching stewardship is admirable, although there is a catch
Risk - Careful prioritization places most eggs in one basket
CRDF is now resting almost everything on the CRDF-004 study, and this trial is taking a risky step moving things into the frontline setting. Despite it being at the FDA's recommendation (according to the company), the observation in their 2 early studies that bevacizumab-naive patients seemed to derive the most benefit from onvansertib is shaky to me. These are small patient numbers, and I don't feel this is a hypothesis that's been carried through rigorously enough at this point.
That doesn't mean I think they're wrong, and I don't think it's a wantonly reckless step to go first line. If onvansertib really does work best in bev-naive patients, then this should end up being the exact play they need to make. But it's based on an idea that comes from pretty small patient numbers, and CRDF-004 may not generate convincing-enough findings to really show that onvansertib is moving the needle. All investors should be aware of that risk before jumping in.
Bottom-Line Summary: Why the Rating Change?As of late, developmental biotech companies are undergoing a surge of interest, with multiple companies showing promising phase 1 data sending them into market caps of billions. I do not think this is necessarily justified, but it speaks to the power of encouraging early data. CRDF, even after its recent gains, has the opportunity to surge even higher if their anticipated mid-2024 data readout in CRC shows support for their idea that onvansertib has more benefit in patients with no exposure to bevacizumab (which would be true of all first-line patients).
Tackling KRAS would be a huge coup, since there are currently no approved therapies that are specifically beneficial for patients in the frontline setting of mCRC with KRAS mutations. It's only used right now to help determine what therapies patients should not receive.
All said, I'm upgrading my rating to a "strong buy," which means that I think you should take a serious look at their portfolio and consider entering into a limited position. I would warn heavily against overextending, though, since so much of the company's value is now going to be tied to the outcomes of a single trial, and a lot can go wrong there. But it is clear now that the ceiling is too high to ignore for this company.
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Mind Medicine Inc (MNMD) - >>> Single dose of LSD provides immediate and lasting relief from anxiety, study says
CNN
by Sandee LaMotte, CNN
March 7, 2024
https://www.yahoo.com/lifestyle/single-dose-lsd-provides-immediate-120037213.html
A clinical trial’s encouraging results won US Food and Drug Administration breakthrough therapy status for an LSD formulation to treat generalized anxiety disorder, Mind Medicine Inc. announced Thursday. The biopharmaceutical company is developing the drug.
“A breakthrough designation is a recognition that a drug has demonstrated evidence of clinical efficacy in meeting an unmet medical need with morbidity and mortality associated with it,” said Dr. Daniel Karlin, assistant professor of psychiatry at Tufts University School of Medicine in Boston and chief medical officer for MindMed.
MindMed’s MM120 will still go through the standard FDA approval process, including phase III trials.
The designation, however, “is an offer from the agency to engage more closely in drug development,” Karlin said. “It affects timelines of response and our ability to get more interactions with the agency so that we can be sure that we’re in lockstep agreement as we move forward.”
Two other companies have also received FDA breakthrough therapy status: psilocybin for treatment-resistant depression and to MDMA, (3,4-Methyl?enedioxy?methamphetamine) commonly known as ecstasy or molly, for post-traumatic stress disorder or PTSD.
New results on efficacy at 12 weeks
A single dose of MM120 (lysergide d-tartrate) led to a 48% rate of remission from generalized anxiety disorder at 12 weeks following the drug’s administration, according to MindMed.
The MM120 drug also significantly improved clinical signs of generalized anxiety disorder for 65% of patients within three months, according to results of the phase 2b trial designed to test dosage levels, the company said.
Anxiety is the most common mental disorder in the United States, affecting over 40 million people age 18 and older each year, according to the Anxiety and Depression Association of America. Generalized anxiety order is characterized by excessive, ongoing thoughts that are difficult to control and interfere with day-to-day activities.
“The clinical improvement for many patients was more than double what we see with today’s standard of care,” Karlin said. “This occurred at all levels of anxiety, from moderate all the way up to severe.”
Standard of care for generalized anxiety disorder is a combination of cognitive behavioral therapy and medications such as selective serotonin reuptake inhibitors, or SSRIs, and buspirone — both of which work on levels of serotonin in the brain — as well as sedatives called benzodiazepines.
All of these medications need time to work and may require experimentation with various doses, adding time and expense to a patient’s treatment, Karlin said.
Determining proper dosage
The multicenter, randomized, double-blinded trial tested doses of 25, 50, 100 and 200 micrograms compared with a placebo.
“We’re very confident based on the results that 100 micrograms is the right dose to bring into our phase three studies, as we didn’t see any more improvement with 200 micrograms but did see additional adverse effects,” Karlin said.
Professor David Nutt, director of the Neuropsychopharmacology Unit at Imperial College London’s division of brain sciences, who researches psychedelics, said in an email that the study results “are very exciting data in what can be a difficult to treat population (anxiety).”
“They expand the likely utility of psychedelic treatment beyond depression,” said Nutt, who was not involved in the research. “And again, as with the depression trials, a single dose produces enduring effects, probable due to its breaking down persistent negative thought processes.”
While it was not the study’s primary purpose, results did show that MM120 also improved signs of depression, Karlin said. “We saw rapid and robust improvement on depression symptoms in people — depression and anxiety have overlapping disease definitions.”
No use of psychotherapy
Most research with MDMA and psilocybin has relied on the use of trained therapists who meet and establish a rapport with participants before the drug is administered. Those therapists are then on hand during the “trip” to help each person assimilate the experience, thus helping assure the lasting impact of any psychological insights.
The MM120 study, however, was accomplished without the use of psychotherapy during the session. Instead, monitors sat in the room to assure safety, but spent their time “mostly reading books,” Karlin said.
“While prior research has documented the benefits of combining LSD with psychotherapy to alleviate anxiety associated with life-threatening conditions, this groundbreaking study is the first to show that a single dose of LSD … can effectively treat generalized anxiety without the adjunct of psychotherapy,” said psychiatrist Dr. Gabriella Gobbi, a professor and scientist at McGill University Health Centre in Montreal and Canada Research Chair in Therapeutics for Mental Health. She was not involved in the clinical trial.
Compared with experiences with forms of LSD purchased illegally on the street, the study’s grade of MM120 did not appear to induce “bad trips,” Karlin said.
“LSD is difficult to manufacture with high purity and tends to degrade quickly in the presence of light and water,” Karlin said. “We’re manufacturing it to pharmaceutical industry standards, a highly pure version that is also shelf stable. So that’s a critical difference.”
Most adverse effects in the study were rated as mild to moderate by participants, occurring mostly on the day of the study, Karlin said. Those included euphoric feelings, illusions and hallucinations, anxiety, abnormal thinking, headaches, dizziness, nausea, excessive sweating, vomiting, numbness or tingling of the skin, and pupil dilation.
A long history of LSD research
When the MM120 clinical trial began in August 2022, it marked the first time LSD had been studied in a medical setting in over 40 years, Karlin said.
During the 1940s and early 1950s, tens of thousands of patients took LSD and other psychotropics to study their effects on cancer anxiety, alcoholism, opioid use disorder, depression, and post-traumatic stress disorder or PTSD. Researchers began to see psychedelics as possible “new tools for shortening psychotherapy.”
But when Harvard University psychologists Timothy Leary and Richard Alpert were fired from the Harvard Psilocybin Project in 1963 after the university discovered they had been giving LSD to their students, the use of psychedelics for research began to lose its luster.
Leary began to speak out publicly, encouraging young people to take LSD recreationally. He quickly became the face of the drug counterculture movement with his signature message, “Turn on, tune in, drop out.”
No longer administered solely in the relative safety of a lab or psychiatrist’s office, LSD began to feature in horror stories of bad “acid” trips at colleges and concerts — headlines that appeared alongside images of anti-Vietnam protests and Woodstock attendees.
In 1968, the United States outlawed LSD and research projects were shut down or forced underground. Then came the 1970 Controlled Substances Act, signed by President Richard Nixon. It classified all hallucinogenics, including psilocybin, as Schedule I drugs — substances with “no currently accepted medical use” and a high probability of abuse.
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Blade, Nice going with IOVA :o) Also, a nice move with CRDF, and JSPR is having a nice bounce. I'll check out the other picks, Thanks :o)
The only bio stock I've been following lately has been Acurx (ACXP). They have a new antibiotic approach for C. Dif infections, with a small Phase 2b completed, but very limited durability data available. Phase 3 is planned for later this year, but looks like the Phase 2b data may not be sufficient to get a decent partnership deal, which will mean dilution. So a typical biotech, with lots of promise but plenty of risk :o)
'Amtagvi / Lifileucel' ---> You have to wonder how they come up with these names lol. Probably an Ai program, but they are tongue twisters ..
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IOVA announces lifting of clinical hold in NSCLC
Iovance Biotherapeutics Announces FDA has Lifted Clinical Hold on the IOV-LUN-202 Registrational Trial in Non-Small Cell Lung Cancer
Iovance Biotherapeutics, Inc.
Mon, March 4, 2024 at 3:30 AM PST
In this article:
IOVA
-0.60%
Watchlist
Watchlist
Recommendation Rating
Buy
Iovance Biotherapeutics, Inc.
Iovance Biotherapeutics, Inc.
SAN CARLOS, Calif., March 04, 2024 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, today announced that the U.S. Food and Drug Administration (FDA) lifted a partial clinical hold placed on the registrational IOV-LUN-202 trial investigating LN-145 TIL cell therapy in non-small cell lung cancer (NSCLC). In collaboration with the FDA and an independent data monitoring committee, Iovance developed additional safety measures and monitoring. Upon reviewing this proposal, the FDA has cleared Iovance to resume patient enrollment in IOV-LUN-202.
The IOV-LUN-202 trial is investigating LN-145 in patients with advanced (unresectable or metastatic) NSCLC without EGFR, ROS or ALK genomic mutations who were previously treated with chemotherapy and anti-PD-1 therapy and at least one line of an approved targeted therapy if indicated by other actionable tumor mutations. Iovance expects to complete enrollment of approximately 120 patients in the IOV-LUN-202 registrational cohorts in 2025.
Preliminary data from the IOV-LUN-202 trial support the potential benefit of one-time TIL therapy, including the opportunity for more durable responses than available second line chemotherapies. Initial preliminary data were reported in July of 2023. An updated analysis in November of 2023 showed additional ongoing responses and duration of response greater than six months for 71% of the confirmed responders in the trial.
For more information about the IOV-LUN-202 trial please visit www.lungcelltherapy.com.
About Iovance Biotherapeutics, Inc.
Iovance Biotherapeutics, Inc. aims to be the global leader in innovating, developing, and delivering tumor infiltrating lymphocyte (TIL) therapies for patients with cancer. We are pioneering a transformational approach to cure cancer by harnessing the human immune system’s ability to recognize and destroy diverse cancer cells in each patient. The Iovance TIL platform has demonstrated promising clinical data across multiple solid tumors. Iovance’s Amtagvi™ is the first FDA-approved T cell therapy for a solid tumor indication. We are committed to continuous innovation in cell therapy, including gene-edited cell therapy, that may extend and improve life for patients with cancer. For more information, please visit www.iovance.com.
Amtagvi™ and its accompanying design marks, Proleukin®, Iovance®, and IovanceCares™ are trademarks and registered trademarks of Iovance Biotherapeutics, Inc. or its subsidiaries. All other trademarks and registered trademarks are the property of their respective owners.
Bladerunner
gfp,
I'm holding all my IOVA. As you suggest, Lifileucel may (probably?) has applications in other cancers. It certainly looks like a buyout candidate, although that's not a reason to hold the stock.
Bladerunner
Hey gfp,
I don't follow MEIP any longer and haven't for a long time.
My largest position in terms of dollars is IOVA. Lififeucel recently approved and he stock has risen considerable.
In terms of shares, CRDF is my largest position. I was upgraded at Piper Sandler last week with the price target being raised from $5 to $7.
Other large positions of mine include IMMP, BCAB, JSPR, and GRTS.
Bladerunner
>>> Why Iovance Biotherapeutics Stock Is Skyrocketing Today
by Keith Speights
Motley Fool
February 20, 2024
https://finance.yahoo.com/news/why-iovance-biotherapeutics-stock-skyrocketing-160233478.html
Shares of Iovance Biotherapeutics (NASDAQ: IOVA) had skyrocketed by 33.8% as of 10:39 a.m. ET Tuesday. The huge gain came after the company announced on Friday that the Food and Drug Administration (FDA) had granted accelerated approval for Amtagvi (lefileucel) as a treatment for advanced melanoma.
Because of the Presidents' Day holiday, the stock market was closed on Monday. Investors had to wait until Tuesday to push Iovance's share price higher.
Iovance also announced on Tuesday morning the pricing of an underwritten offering to issue just over 23 million new shares for $9.15 per share. That stock sale will generate gross proceeds of around $211 million. Part of the money will be used to support the commercial launch of Amtagvi.
How big is the FDA approval for Iovance?
It's hard to overstate just how important the FDA's approval of Amtagvi is for Iovance. The company is no longer a clinical-stage biotech -- it now has a marketable product. And not just any product: Amtagvi is the first (and so far, only) individualized T-cell therapy to win a thumbs-up from the FDA to treat a solid tumor.
Iovance is also evaluating Amtagvi in late-stage clinical studies targeting cervical cancer and non-small cell lung cancer. Analytics company GlobalData projects that Amtagvi will generate annual global sales of nearly $900 million by 2029.
Is Iovance Biotherapeutics stock a good pick to buy now?
With a market cap of nearly $3.2 billion, much of the anticipated growth for Iovance is already priced into the stock. However, the company could still have more catalysts on the way, including potential regulatory approvals for Amtagvi in other countries. I think Iovance stock remains a good pick to buy for aggressive investors.
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Blade, Just curious about your strategy with Iovance? It sounds like a platform technology, and could have much broader applications in other solid tumors types. I see they announced a financing yesterday, priced at 9.15, but the stock blew right past that level to 15, and is now 16 in after hours.
Anyway, just curious to get your take on Iovance. Thanks for any insights :o)
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Iovance - >>> FDA approves groundbreaking treatment for advanced melanoma
NBC News
by Erika Edwards and Anne Thompson and Marina Kopf
2-16-24
https://www.msn.com/en-us/health/other/fda-approves-groundbreaking-treatment-for-advanced-melanoma/ar-BB1ioIYQ?OCID=ansmsnnews11
The Food and Drug Administration on Friday approved a new cancer therapy that could one day transform the way a majority of aggressive and advanced tumors are treated.
The treatment, called Amtagvi, from Iovance Biotherapeutics, is for metastatic melanoma patients who have already tried and failed other drugs. It’s known as TIL therapy and involves boosting the number of immune cells inside tumors, harnessing their power to fight the cancer.
It’s the first time a cellular therapy has been approved to treat solid tumors. The drug was given a fast-track approval based on the results of a phase 2 clinical trial. The company is conducting a larger phase 3 trial to confirm the treatment’s benefits.
“This is going to be huge,” said Dr. Elizabeth Buchbinder, a senior physician at Dana-Farber Cancer Institute in Boston. Melanoma is “not one of those cancers where there’s like 20 different” possible treatments, she said. “You start running out of options fast.”
Friday’s approval is only for melanoma, the deadliest form of skin cancer, but experts say it holds promise for treating other solid tumors, which account for 90% of all cancers.
“It is our hope that future iterations of TIL therapy will be important for lung cancer, colon cancer, head and neck cancer, bladder cancer and many other cancer types,” said Dr. Patrick Hwu, chief executive of the Moffitt Cancer Center in Tampa, Florida. Moffitt has been involved with Iovance’s clinical trials of TIL therapy.
TIL stands for tumor-infiltrating lymphocytes, which are immune cells that exist within tumors. But there are nowhere nearly enough of those cells to effectively fight off cancer cells. TIL therapy involves, in part, extracting some of those immune cells from the patient’s tumor and replicating them billions of times in a lab, then reinfusing them back into the patient.
It’s similar to CAR-T cell therapy, where healthy cells are taken out of a person’s body and then modified in a lab to fight cancers. That’s usually used for hard-to-treat blood cancers such as leukemia and lymphoma. With TIL therapy, the cells used are already programmed to recognize cancer — no lab modifications needed — they just need a boost in numbers to fight it.
Like CAR-T, TIL therapy is a one-time treatment, though the entire process can take up to eight weeks. The TIL cells are first harvested from the tumor through a minimally invasive procedure and then grown and multiplied in the lab, a process that takes 22 days, according to Iovance.
While that’s happening, patients are given chemotherapy to clear out their immune cells to make room for the billions of new melanoma-fighting TIL cells. Once the TIL cells are reinfused back into the body, patients get a drug called interleukin-2 to further stimulate those cells.
Hwu said that most side effects in patients undergoing TIL therapy are not from the reinfusion of cells, but from the chemotherapy and the interleukin-2. These can include nausea and extreme fatigue, and patients are also vulnerable to other illnesses because the body is depleted of disease-fighting white blood cells.
Putting billions of cells back into the body is not entirely risk-free, however, said Dr. William Dahut, chief scientific officer of the American Cancer Society. It’s possible that the body’s immune system could overreact in what’s known as a cytokine storm, which can cause flu-like symptoms, low blood pressure and organ damage. “There are risks for immune-related side effects, which could be serious,” he said.
Common side effects associated with Amtagvi can include abnormally fast heart rate, fluid buildup, rash, hair loss and feeling short of breath, the FDA said.
Those side effects can be managed, said Dr. Steven Rosenberg, chief of the surgery branch at the National Cancer Institute. “They’re a small price to pay for a growing cancer that would otherwise be be lethal.”
Overall, Dahut said the approval of TIL therapy is “meaningful.”
“What’s nice about this is that patients will receive a wide variety of tumor fighting lymphocytes that will be able to have the capacity to overcome resistance and actually be a living therapy over time, too, to target additional cancer cells should they develop,” Dahut said.
In addition to melanoma, Dahut said that TIL therapy is most likely to be useful in cancers that respond to drugs that “take the brakes off the immune system,” called checkpoint inhibitors.
“Those would be things like non-small cell lung cancer, kidney cancer, maybe bladder cancer, that we know are responsive to immune-based therapies to begin with,” he said. “Many of those patients relapse, so another immune-based therapy that works in a different way, seems to me, the most likely way for this to be effective.”
Much more research is needed, and it may be years before TIL therapy is approved for other types of cancer.
One of Iovance’s clinical trials investigating TIL therapy for non-small cell lung cancer was forced to pause when a participant died. While the death is under investigation, the company said it may have been the result of either chemotherapy or interleukin 2 — therapies meant to knock down each patients’ immune system before they can get the reinfusion of their TIL cells.
The therapy is not expected to work for every metastatic melanoma patient. Clinical trial data that Iovance submitted to the FDA showed that tumors shrank in about a third of patients who received TIL therapy.
Of those patients, about half saw their tumors shrink for at least one year, Dr. Friedrich Graf Finckenstein, chief medical officer of Iovance Biotherapeutics. “Some of these patients even had their tumor completely disappear,” he said.
Another study, conducted in the Netherlands, did a head-to-head analysis of TIL therapy and another form of immunotherapy, called ipilimumab. Twenty percent of the patients who received TIL had complete remissions, compared with 7% of patients who got ipilimumab. Iovance was not involved with the Dutch trial.
The goal of the therapy, Hwu said, “is to get rid of the cancer and have it stay away. These immune cells stay in the body and live in the body for decades.”
The technology has been in development and studied for nearly 40 years. It was Rosenberg who pioneered TIL therapy — first describing how it could shrink melanoma tumors in the New England Journal of Medicine in 1988.
“I’ve been waiting for a very long time to see this given to patients, because I know that it can cure some patients that have metastatic melanoma that cannot be affected by any other treatment,” Rosenberg said.
It’s worked so far for Dan Bennett, 59, of Clermont, Florida. Bennett was diagnosed with melanoma in 2011 after his daughter noticed a suspicious mole on his neck that had changed color.
Despite surgery, chemotherapy and radiation, his cancer kept returning. In 2014, his doctors at Moffitt recommended he try TIL therapy.
“At first, we were pretty leery about it because it was unproven,” Bennett said. Ten years later, Bennett is convinced the TIL therapy is the reason he has survived so long with stage 4 melanoma, which usually has a five-year survival rate of 22.5%.
“I would recommend any experimental drug if it’s your last opportunity,” he said. “You owe it to yourself and your family to do whatever you can to stay alive and to be a productive member of society.”
Buchbinder, the Dana-Faber doctor, was not involved with Iovance’s TIL therapy trial for melanoma, but she is scheduled to begin similar trials with other drugmakers.
“We literally have patients right now waiting for approval because they are hoping they’ll be able to go on it,” Buchbinder said. “It is definitely a practice-changing therapy.”
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>>> Acurx Announces Positive Extended Clinical Cure Data for Ibezapolstat from Phase 2b Clinical Trial in CDI Patients
PR Newswire
Jan 29, 2024
https://finance.yahoo.com/news/acurx-announces-positive-extended-clinical-120000145.html
In the Phase 2b trial, 100% (5 of 5) of ibezapolstat-treated patients who agreed to observation for up to three months following Clinical Cure of CDI experienced no recurrence of infection
Further analyses will be forthcoming as data become available regarding effects on bile acid metabolism and pharmacokinetic data
Preparation is underway for meetings with FDA, European Medicines Agency and other global regulatory agencies and advancement to international Phase 3 clinical trials
Ibezapolstat has previously received FDA QIDP and Fast-Track Designation
STATEN ISLAND, N.Y., Jan. 29, 2024 /PRNewswire/ -- Acurx Pharmaceuticals, Inc. (NASDAQ: ACXP) ("Acurx" or the "Company"), a late-stage biopharmaceutical company developing a new class of small molecule antibiotics for difficult-to-treat bacterial infections, today announced positive extended clinical cure (ECC) data for ibezapolstat (IBZ), its lead antibiotic candidate, from the Company's recently completed Phase 2b clinical trial in patients with CDI. This exploratory endpoint showed that 12 patients who agreed to be followed up to three months following Clinical Cure of their infection, 5 of 5 IBZ patients experienced no recurrence of infection. In the vancomycin control arm of the trial, 7 of 7 patients experienced no recurrence of infection. ECC success is defined as a clinical cure at the TOC visit (i.e., at least 48 hours post EOT) and no recurrence of CDI within the 56 ± 2 days post EOT (ECC56) and 84 ± 2 days post EOT (ECC84) in patients who consented to extended observation.
According to Dr. Garey: "CDI has long-term consequences including poor quality of life for the patient. It's exciting to see that the ibezapolstat Phase 2b study followed patients for an extended time period to assure that the ibezapolstat cure and anti-recurrence effects are long-lasting and durable. This adds to the exciting microbiome data showing replenishment of healthy microbiota that we have seen in our prior studies."
Robert J. DeLuccia, Executive Chairman of Acurx, stated: "This Extended Clinical Cure data showing that no ibezapolstat-treated patient who achieved Clinical Cure and consented to three months of post-treatment observation experienced a recurrence of CDI during this period further strengthens the ibezapolstat lineage of clinical success building on our previously reported Phase 2 results of 96% Clinical Cure and 100% Sustained Clinical Cure along with favorable preservation of the microbiome and no safety signals." He further stated: "We look forward to advancing to Phase 3 clinical trials and are optimistic about achieving non-inferiority versus vancomycin which is the regulatory clinical development hurdle for marketing registration".
David P. Luci, President & CEO of Acurx, stated: "Ibezapolstat continues to demonstrate success compared to a standard of care, oral vancomycin, to treat patients with CDI. We anticipate continued favorable differentiation between the two therapeutic options in 2024. We expect to leverage this success in a $1 billion plus US CDI global market as we move forward with an international Phase 3 clinical trial mandate." He added: "The Company also anticipates its price point for ibezapolstat, if approved, could meet or beat other antibiotics recommended for use in treating patients with CDI, thereby providing the whole package of clinical comparability with microbiome health, safety and cost for patients with this life-threatening disease."
About the Ibezapolstat Phase 2 Clinical Trial
The completed multicenter, open-label single-arm segment (Phase 2a) study was followed by a double-blind, randomized, active-controlled, non-inferiority, segment (Phase 2b) at 28 US clinical trial sites which together comprise the Phase 2 clinical trial.(see https://clinicaltrials.gov/ct2/show/NCT04247542). This Phase 2 clinical trial was designed to evaluate the clinical efficacy of ibezapolstat in the treatment of CDI including pharmacokinetics and microbiome changes from baseline and continue to test for anti- recurrence microbiome properties seen in the Phase 2a trial, including the treatment- related changes in alpha diversity and bacterial abundance and effects on bile acid metabolism.
The completed Phase 2a segment of this trial was an open label cohort of up to 20 subjects from study centers in the United States. In this cohort, 10 patients with diarrhea caused by C. difficile were treated with ibezapolstat 450 mg orally, twice daily for 10 days. All patients were followed for recurrence for 28± 2 days. Per protocol, after 10 patients of the projected 20 Phase 2a patients completed treatment (100% cured infection at End of Treatment), the Trial Oversight Committee assessed the safety and tolerability and made its recommendation regarding early termination of the Phase 2a study and advancement to the Ph2b segment. The Company's Scientific Advisory Board concurred with this recommendation.
In the now completed Phase 2b trial segment, 32 patients with CDI were enrolled and randomized in a 1:1 ratio to either ibezapolstat 450 mg every 12 hours or vancomycin 125 mg orally every 6 hours, in each case, for 10 days and followed for 28 ± 2 days following the end of treatment for recurrence of CDI. The two treatments were identical in appearance, dosing times, and number of capsules administered to maintain the blind. The Company previously reported that the overall observed Clinical Cure rate in the combined Phase 2 trials in patients with CDI was 96% (25 out of 26 patients), based on 10 out of 10 patients (100%) in Phase 2a in the Modified Intent to Treat Population, plus 15 out of 16 (94%) patients in Phase 2b in the Per Protocol Population, who experienced Clinical Cure during treatment with ibezapolstat. Ibezapolstat was well-tolerated, with three patients each experiencing one mild adverse event assessed by the blinded investigator to be drug-related. All three events were gastrointestinal in nature and resolved without treatment. There were no drug-related treatment withdrawals or no drug-related serious adverse events, or other safety findings of concern. In the Phase 2b vancomycin control arm, 14 out of 14 patients experienced Clinical Cure. The Company is confident that based on the pooled Phase 2 ibezapolstat Clinical Cure rate of 96% and the historical vancomycin cure rate of approximately 81% (Vancocin® Prescribing Information, January 2021), we will demonstrate non-inferiority of ibezapolstat to vancomycin in Phase 3 trials in accordance with the applicable FDA Guidance for Industry (October 2022).
The Phase 2b clinical trial segment was discontinued due to success. The Company made this decision in consultation with its medical and scientific advisors and statisticians based on observed aggregate blinded data and other factors, including the cost to maintain clinical trial sites and slow enrollment due to COVID-19 and its aftermath. The Company had determined that the trial performed as anticipated for both treatments, ibezapolstat and the control antibiotic vancomycin (a standard of care to treat patients with CDI), with high rates of clinical cure observed across the trial without any emerging safety concerns. Accordingly, an Independent Data Monitoring Committee was not required to perform an interim analysis of this Phase 2b trial data as originally planned. The Company anticipated that this decision would allow the Company to advance this first-in-class, FDA QIDP/Fast Track-designated antibiotic product candidate to Phase 3 clinical trials more expeditiously.
The Phase 2b trial was originally designed to be a non-inferiority (NI) trial and later amended to include an interim efficacy analysis with review by an Independent Data Monitoring Committee (IDMC). The decision to end the trial early based on blinded clinical observations obviated the need for an interim analysis, IDMC review, and NI assessment. The Company determined, in consultation with its clinical and statistical experts, that presenting clinical cure rates for the primary efficacy endpoint is the most appropriate representation for the clinical activity of ibezapolstat in treating CDI.
In the Phase 2 clinical trial, the Company will also evaluate pharmacokinetics (PK) and microbiome changes and test for anti-recurrence microbiome properties, including the change from baseline in alpha diversity and bacterial abundance, especially overgrowth of healthy gut microbiota Actinobacteria and Firmicute phylum species during and after therapy. Phase 2a data demonstrated complete eradication of colonic C. difficile by day three of treatment with ibezapolstat as well as the observed overgrowth of healthy gut microbiota, Actinobacteria and Firmicute phyla species, during and after therapy. Very importantly, emerging data show an increased concentration of secondary bile acids during and following ibezapolstat therapy which is known to correlate with colonization resistance against C. difficile. A decrease in primary bile acids and the favorable increase in the ratio of secondary-to-primary bile acids suggest that ibezapolstat may reduce the likelihood of CDI recurrence when compared to vancomycin.
About Ibezapolstat
Ibezapolstat is a novel, orally administered antibiotic being developed as a Gram-Positive Selective Spectrum (GPSS™) antibacterial. It is the first of a new class of DNA polymerase IIIC inhibitors under development by Acurx to treat bacterial infections. Ibezapolstat's unique spectrum of activity, which includes C. difficile but spares other Firmicutes and the important Actinobacteria phyla, appears to contribute to the maintenance of a healthy gut microbiome.
In June 2018, ibezapolstat was designated by the U.S. Food and Drug Administration (FDA) as a Qualified Infectious Disease Product (QIDP) for the treatment of patients with CDI and will be eligible to benefit from the incentives for the development of new antibiotics established under the Generating New Antibiotic Incentives Now (GAIN) Act. In January 2019, FDA granted "Fast Track" designation to ibezapolstat for the treatment of patients with CDI. The CDC has designated C. difficile as an urgent threat highlighting the need for new antibiotics to treat CDI.
About Clostridioides difficile Infection (CDI). According to the 2017 Update (published February 2018) of the Clinical Practice Guidelines for C. difficile Infection by the Infectious Diseases Society of America (IDSA) and Society or Healthcare Epidemiology of America (SHEA), CDI remains a significant medical problem in hospitals, in long-term care facilities and in the community. C. difficile is one of the most common causes of health care- associated infections in U.S. hospitals (Lessa, et al, 2015, New England Journal of Medicine). Recent estimates suggest C. difficile approaches 500,000 infections annually in the U.S. and is associated with approximately 20,000 deaths annually. (Guh, 2020, New England Journal of Medicine). Based on internal estimates, the recurrence rate for the antibiotics currently used to treat CDI is between 20% and 40% among approximately 150,000 patients treated. We believe the annual incidence of CDI in the U.S. approaches 600,000 infections and a mortality rate of approximately 9.3%.
About the Microbiome in C. difficile Infection (CDI) and Bile Acid Metabolism
C. difficile can be a normal component of the healthy gut microbiome, but when the microbiome is thrown out of balance, the C. difficile can thrive and cause an infection. After colonization with C. difficile, the organism produces and releases the main virulence factors, the two large clostridial toxins A (TcdA) and B (TcdB). (Kachrimanidou, Microorganisms 2020, 8, 200; doi:10.3390/microorganisms8020200.) TcdA and TcdB are exotoxins that bind to human intestinal epithelial cells and are responsible for inflammation, fluid and mucous secretion, as well as damage to the intestinal mucosa.
Bile acids perform many functional roles in the GI tract, with one of the most important being maintenance of a healthy microbiome by inhibiting C. difficile growth. Primary bile acids, which are secreted by the liver into the intestines, promote germination of C. difficile spores and thereby increase the risk of recurrent CDI after successful treatment of an initial episode. On the other hand, secondary bile acids, which are produced by normal gut microbiota through metabolism of primary bile acids, do not induce C. difficile sporulation and therefore protect against recurrent disease. Since ibezapolstat treatment leads to minimal disruption of the gut microbiome, bacterial production of secondary bile acids continues which may contribute to an anti-recurrence effect. Beneficial effects of bile acids include a decrease in primary bile acids and an increase in secondary bile acids in patients with CDI, which was observed in the Company's Ph2a trial results and previously reported (CID, 2022).
About Acurx Pharmaceuticals, Inc.
Acurx Pharmaceuticals is a late-stage biopharmaceutical company focused on developing a new class of small molecule antibiotics for difficult-to-treat bacterial infections. The Company's approach is to develop antibiotic candidates with a Gram-positive selective spectrum (GPSS®) that blocks the active site of the Gram+ specific bacterial enzyme DNA polymerase IIIC (pol IIIC), inhibiting DNA replication and leading to Gram-positive bacterial cell death. Its R&D pipeline includes antibiotic product candidates that target Gram-positive bacteria, including Clostridioides difficile, methicillin-resistant Staphylococcus aureus (MRSA), vancomycin resistant Enterococcus (VRE) and drug-resistant Streptococcus pneumoniae (DRSP).
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>>> Novo Nordisk A/S (NVO), a healthcare company, engages in the research, development, manufacture, and marketing of pharmaceutical products worldwide. It operates in two segments, Diabetes and Obesity care, and Rare Disease.
The Diabetes and Obesity care segment provides products in the areas of insulins, GLP-1 and related delivery systems, oral antidiabetic products, obesity, glucagon, needles, and other chronic diseases.
The Rare Disease segment offers products in the areas of haemophilia, blood disorders, endocrine disorders, growth disorders, and hormone replacement therapy.
The company has a collaboration agreement with Gilead Sciences, Inc.; and research collaboration with Novo Nordisk to discover cell-specific carriers of nucleic acid therapeutics. The company was founded in 1923 and is headquartered in Bagsvaerd, Denmark.
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https://finance.yahoo.com/quote/NVO/profile?p=NVO
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>>> FDA says cancer treatment CAR-T therapy may increase risk of cancer
NBC News
by Berkeley Lovelace Jr.
January 24, 2024
https://www.yahoo.com/news/fda-says-cancer-treatment-car-020945813.html
The Food and Drug Administration this week told several drugmakers to add a boxed warning — the agency’s strongest safety label — to the prescribing information for a type of cancer treatment called CAR-T therapy, saying the treatment itself may increase a person’s risk of cancer.
Carly Kempler, a spokesperson for the FDA, said that, despite the warning, "the overall benefits of these products continue to outweigh their potential risks."
The agency’s decision to update the labels was based on reports of rare blood cancers in patients who had previously gotten CAR-T therapy, Kempler said. As of Monday, the agency had received 25 reports of the blood cancers in CAR-T patients, she said.
Bruce Levine, a professor in cancer gene therapy at the University of Pennsylvania, said that in addition to the reports submitted to the FDA, two abstracts published late last year in the journal Blood also cited a potential cancer risk associated with CAR-T therapy, which likely “forced the FDA’s hand.”
CAR-T — or chimeric antigen receptor T cell — therapy uses a patient’s own immune cells to treat certain blood cancers, such as leukemia, multiple myeloma and lymphoma. It involves harvesting the immune cells — in this case, T cells — then genetically altering them in a lab to make them target cancer cells, and finally reinfusing them back into the patient.
It’s proven to be highly effective in hard-to-treat cases, experts said. In 2022, doctors who had treated two leukemia patients with CAR-T a decade ago said it was fair to say the therapy had cured the patients of the disease.
“This has been a game changer when we think about treating lymphoma and other diseases,” said Dr. Matthew Frigault, the clinical director of the Massachusetts General Hospital Cellular Immunotherapy Program in Boston.
The first CAR-T therapy, Novartis’ drug Kymriah, received FDA approval in 2017. Since then, another five have been approved.
The makers of the drugs — Bristol Myers Squibb, for Abecma and Breyanzi; Gilead Sciences' Kite Pharma, for Yescarta and Tecartus; Johnson & Johnson's Carvykti; and Novartis, for Kymriah — received letters from the FDA, stating that they must submit proposed label changes in the next 30 days to note that, in rare cases, CAR-T therapy can increase the risk of rare blood cancers.
If the drugmakers disagree, they can instead submit a rebuttal explaining why a change isn’t needed.
In a statement to NBC News, a spokesperson for Novartis said the company has not found “sufficient evidence” to support a link between cancer and its treatment, which has been used in more than 10,000 patients. However, the spokesperson said, the company will work with the FDA to update its label “appropriately.”
Spokespersons for Johnson & Johnson and Gilead Sciences also said the drugmakers would work with the agency to update their labels.
A spokesperson for Bristol Myers Squibb said the company is evaluating “next steps” following the FDA’s notice, although it has not seen any cancer cases associated with its treatment.
“Patient safety is our top priority,” the spokesperson said.
How might CAR-T therapy cause cancer?
Still, there’s the question of how CAR-T could cause cancer — if it does at all.
“We actually don’t know whether this is causal, meaning, we don’t know for a fact that the CAR-T cells in the tumor have led to this,” said Frigault, of Mass General.
CAR-T treatments are still relatively new: Frigault noted that the FDA has required that the makers of the products conduct 15-year follow-up studies to assess the potential risk of secondary cancers following treatment. (Secondary cancers are cancers that can arise from treatment.)
The FDA “is not saying that every single one of the cases they’ve reported has clearly shown CAR-T has led to this,” he said, “but more that there may be an association.”
“This is what the FDA does. They look for a signal,” he added.
If CAR-T does cause cancer, the risk is likely very small, said Dr. Hemant Murthy, a hematology-oncology physician at the Mayo Clinic in Jacksonville, Florida.
More than 27,000 doses of CAR-T therapy have been administered in the U.S., according to the FDA.
“I don’t really see this affecting too much of practice,” Murthy said.
Dr. Saad Usmani, a myeloma physician and cell therapist at Memorial Sloan Kettering, said the label change should support physicians’ current practice of discussing with patients the risk of developing secondary cancers following cancer treatment.
Usmani noted that other cancer treatments, such as radiation and chemotherapy, also carry a risk of secondary cancers.
“The change is expected given the recent reports, albeit very low incidence in such cases,” he said.
Dr. Marcela Maus, an associate professor of medicine at Harvard Medical School and director of the Cellular Immunotherapy Program at Massachusetts General Hospital, said physicians might be more cautious, but it most likely won’t change much in their practice.
“We need to manage the cancer that they have now, so I don’t imagine it being massively different,” she said.
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>>> Acurx Pharmaceuticals to Present at the Microcap Conference, January 31, 2024 - February 1, 2024
PR Newswire
January 23, 2024
https://finance.yahoo.com/news/acurx-pharmaceuticals-present-microcap-conference-120100998.html
STATEN ISLAND, N.Y., Jan. 23, 2024 /PRNewswire/ -- Acurx Pharmaceuticals, Inc. ("Acurx" or the "Company") (NASDAQ: "ACXP"), a late-stage biopharmaceutical company developing a new class of small molecule antibiotics for difficult-to-treat bacterial infections, today announced that David P. Luci, President & Chief Executive Officer, will be presenting at The Microcap Conference, which will take place January 30, 2024-February 1, 2024 at the Ceasars Atlantic City Hotel & Casino in Atlantic City, New Jersey.
Presentation Details:
Wednesday, January 31st, 2 pm ET and
Thursday, February 1st at 11:40 am ET
Interested parties can register to attend here.
About Acurx Pharmaceuticals, Inc.
Acurx Pharmaceuticals is a late-stage biopharmaceutical company focused on developing a new class of small molecule antibiotics for difficult-to-treat bacterial infections. The Company's approach is to develop antibiotic candidates with a Gram-positive selective spectrum (GPSS®) that blocks the active site of the Gram+ specific bacterial enzyme DNA polymerase IIIC (pol IIIC), inhibiting DNA replication and leading to Gram-positive bacterial cell death. Its R&D pipeline includes antibiotic product candidates that target Gram-positive bacteria, including Clostridioides difficile, methicillin-resistant Staphylococcus aureus (MRSA), vancomycin resistant Enterococcus (VRE) and drug-resistant Streptococcus pneumoniae (DRSP).
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>>> Acurx Announces Positive Comparative Microbiology and Microbiome Data for Ibezapolstat from Phase 2b Clinical Trial in CDI Patients
Yahoo Finance
January 17, 2024
https://finance.yahoo.com/news/acurx-announces-positive-comparative-microbiology-120100113.html
Ibezapolstat outperformed vancomycin showing eradication of fecal C. difficile at Day 3 of treatment in 15 of 16 treated patients (94%), versus vancomycin which had eradication of C. difficile in 10 of 14 treated patients (71%).
Ibezapolstat, but not vancomycin, consistently preserved and allowed regrowth of key gut bacterial species believed to confer health benefits including to prevent recurrence of CDI
Further analyses will be forthcoming Q1 2024, as data become available, regarding other endpoints from the Phase 2b trial, including Extended Clinical Cure (ECC) data up to 94 days
Preparation underway for meetings with FDA, European Medicines Agency and other global regulatory agencies and advancement to international Phase 3 clinical trials
Ibezapolstat has previously received FDA QIDP and Fast-Track Designation
STATEN ISLAND, N.Y., Jan. 17, 2024 /PRNewswire/ -- Acurx Pharmaceuticals, Inc. (NASDAQ: ACXP) ("Acurx" or the "Company"), a late-stage biopharmaceutical company developing a new class of small molecule antibiotics for difficult-to-treat bacterial infections, today announced positive comparative microbiology and microbiome data for ibezapolstat, its lead antibiotic candidate, from the Company's recently completed Phase 2b clinical trial in patients with CDI. Data showed that ibezapolstat outperformed vancomycin, a standard of care to treat patients with CDI, with eradication of fecal C. difficile at Day 3 of treatment in 15 of 16 patients (94%) versus vancomycin which had eradication of fecal C. difficile in 10 of 14 patients (71%). In addition, ibezapolstat, but not vancomycin, consistently preserved and allowed regrowth of key gut bacterial species believed to confer health benefits including to prevent CDI recurrence. Further analyses will be forthcoming Q1 2024, as data become available, regarding other endpoints from the Phase 2b trial, including Extended Clinical Cure (ECC) data up to 94 days. Preparation is underway for meetings with FDA, European Medicines Agency and other global regulatory agencies and advancement to international Phase 3 clinical trials.
The Company also announced that a scientific poster will be presented on January 18, 2023 at the Gulf Coast Consortia Antimicrobial Resistance (AMR) Conference in Houston, Texas by Kevin Garey, PharmD, MS, Professor and Chair, University of Houston College of Pharmacy, the Principal Investigator for microbiology and microbiome aspects of the ibezapolstat clinical trial program and Acurx Scientific Advisory Board member. The poster will show comparative data details from the Phase 2b clinical trial entitled: "A Phase 2, Randomized, Double-Blind Study of Ibezapolstat Compared with Vancomycin for the Treatment of Clostridioides difficile Infection."
After the presentation, the poster will be posted on Acurx website: www.acurxpharma.com
According to Dr. Garey: "These results help validate our ongoing scientific investigations into the anti-CDI recurrence effects of ibezapolstat. Our microbiologic findings show that markedly fewer patients treated with ibezapolstat had persistent C. difficile compared to patients treated with vancomycin. He further stated: "Preservation of key health-conferring native gut bacteria, such as Firmicutes, in patients treated with ibezapolstat has now been shown consistently during all clinical studies. These results correlate with prior findings by showing superior preservation of key native gut bacteria compared to vancomycin in CDI patients. Preservation of native gut bacteria during treatment for CDI is believed to be a key component for preventing recurrence of CDI. These findings will need to be further validated in the phase 3 studies but the results to date support the importance of this new class of antibiotics with a novel mechanism of action that does not target native gut bacteria."
Robert J. DeLuccia, Executive Chairman of Acurx, stated: "These new comparative data are very important and timely to enhance our data package for an end of Phase 2 FDA Meeting which is targeted for second quarter this year. He further stated: "Parallel preparations continue on schedule for Phase 3 clinical trials start up later this year, including timely availability of clinical trial supply.
David P. Luci, President & CEO of Acurx, stated: "Ibezapolstat continues to demonstrate success compared to a standard of care, oral vancomycin, to treat patients with CDI. We anticipate that favorable differentiation between the two therapeutic options will continue to be shown in Q1 2024 including extended clinical cure and additional microbiome comparison data. We expect to leverage this success in a $1 billion plus US CDI global market as we move forward with an international Phase 3 clinical trial mandate." He added: "The Company also anticipates its price point for ibezapolstat, if approved, could meet or beat other antibiotics recommended for use in treating patients with CDI, thereby providing the whole package of clinical comparability with microbiome health, safety and cost for patients with this life-threatening disease."
About the Ibezapolstat Phase 2 Clinical Trial
The completed multicenter, open-label single-arm segment (Phase 2a) study was followed by a double-blind, randomized, active-controlled, non-inferiority, segment (Phase 2b) at 28 US clinical trial sites which together comprise the Phase 2 clinical trial. (see https://clinicaltrials.gov/ct2/show/NCT04247542). This Phase 2 clinical trial was designed to evaluate the clinical efficacy of ibezapolstat in the treatment of CDI including pharmacokinetics and microbiome changes from baseline and continue to test for anti- recurrence microbiome properties seen in the Phase 2a trial, including the treatment- related changes in alpha diversity and bacterial abundance and effects on bile acid metabolism.
The completed Phase 2a segment of this trial was an open label cohort of up to 20 subjects from study centers in the United States. In this cohort, 10 patients with diarrhea caused by C. difficile were treated with ibezapolstat 450 mg orally, twice daily for 10 days. All patients were followed for recurrence for 28± 2 days. Per protocol, after 10 patients of the projected 20 Phase 2a patients completed treatment (100% cured infection at End of Treatment), the Trial Oversight Committee assessed the safety and tolerability and made its recommendation regarding early termination of the Phase 2a study and advancement to the Ph2b segment. The Company's Scientific Advisory Board concurred with this recommendation.
In the now completed Phase 2b trial segment, 32 patients with CDI were enrolled and randomized in a 1:1 ratio to either ibezapolstat 450 mg every 12 hours or vancomycin 125 mg orally every 6 hours, in each case, for 10 days and followed for 28 ± 2 days following the end of treatment for recurrence of CDI. The two treatments were identical in appearance, dosing times, and number of capsules administered to maintain the blind. The overall observed Clinical Cure rate in the combined Phase 2 trials in patients with CDI was 96% (25 out of 26 patients), based on 10 out of 10 patients (100%) in Phase 2a in the Modified Intent to Treat Population, plus 15 out of 16 (94%) patients in Phase 2b in the Per Protocol Population, who experienced Clinical Cure during treatment with ibezapolstat. Ibezapolstat was well-tolerated, with three patients each experiencing one mild adverse event assessed by the blinded investigator to be drug-related. All three events were gastrointestinal in nature and resolved without treatment. There were no drug-related treatment withdrawals or no drug-related serious adverse events, or other safety findings of concern. In the Phase 2b vancomycin control arm, 14 out of 14 patients experienced Clinical Cure. The Company is confident that based on the pooled Phase 2 ibezapolstat Clinical Cure rate of 96% and the historical vancomycin cure rate of approximately 81% (Vancocin® Prescribing Information, January 2021), we will demonstrate non-inferiority of ibezapolstat to vancomycin in Phase 3 trials in accordance with the applicable FDA Guidance for Industry (October, 2022).
The Phase 2b clinical trial segment was discontinued due to success. The Company made this decision in consultation with its medical and scientific advisors and statisticians based on observed aggregate blinded data and other factors, including the cost to maintain clinical trial sites and slow enrollment due to COVID-19 and its aftermath. The Company had determined that the trial performed as anticipated for both treatments, ibezapolstat and the control antibiotic vancomycin (a standard of care to treat patients with CDI), with high rates of clinical cure observed across the trial without any emerging safety concerns. Accordingly, an Independent Data Monitoring Committee was not required to perform an interim analysis of this Phase 2b trial data as originally planned. The Company anticipated that this decision would allow the Company to advance this first-in-class, FDA QIDP/Fast Track-designated antibiotic product candidate to Phase 3 clinical trials more expeditiously.
The Phase 2b trial was originally designed to be a non-inferiority (NI) trial and later amended to include an interim efficacy analysis with review by an Independent Data Monitoring Committee (IDMC). The decision to end the trial early based on blinded clinical observations obviated the need for an interim analysis, IDMC review, and NI assessment. The Company determined, in consultation with its clinical and statistical experts, that presenting clinical cure rates for the primary efficacy endpoint is the most appropriate representation for the clinical activity of ibezapolstat in treating CDI.
In the Phase 2 clinical trial, the Company will also evaluate pharmacokinetics (PK) and microbiome changes and test for anti-recurrence microbiome properties, including the change from baseline in alpha diversity and bacterial abundance, especially overgrowth of healthy gut microbiota Actinobacteria and Firmicute phylum species during and after therapy. Phase 2a data demonstrated complete eradication of colonic C. difficile by day three of treatment with ibezapolstat as well as the observed overgrowth of healthy gut microbiota, Actinobacteria and Firmicute phyla species, during and after therapy. Very importantly, emerging data show an increased concentration of secondary bile acids during and following ibezapolstat therapy which is known to correlate with colonization resistance against C. difficile. A decrease in primary bile acids and the favorable increase in the ratio of secondary-to-primary bile acids suggest that ibezapolstat may reduce the likelihood of CDI recurrence when compared to vancomycin.
About Ibezapolstat
Ibezapolstat is a novel, orally administered antibiotic being developed as a Gram-Positive Selective Spectrum (GPSS™) antibacterial. It is the first of a new class of DNA polymerase IIIC inhibitors under development by Acurx to treat bacterial infections. Ibezapolstat's unique spectrum of activity, which includes C. difficile but spares other Firmicutes and the important Actinobacteria phyla, appears to contribute to the maintenance of a healthy gut microbiome.
In June 2018, ibezapolstat was designated by the U.S. Food and Drug Administration (FDA) as a Qualified Infectious Disease Product (QIDP) for the treatment of patients with CDI and will be eligible to benefit from the incentives for the development of new antibiotics established under the Generating New Antibiotic Incentives Now (GAIN) Act. In January 2019, FDA granted "Fast Track" designation to ibezapolstat for the treatment of patients with CDI. The CDC has designated C. difficile as an urgent threat highlighting the need for new antibiotics to treat CDI.
About Clostridioides difficile Infection (CDI). According to the 2017 Update (published February 2018) of the Clinical Practice Guidelines for C. difficile Infection by the Infectious Diseases Society of America (IDSA) and Society or Healthcare Epidemiology of America (SHEA), CDI remains a significant medical problem in hospitals, in long-term care facilities and in the community. C. difficile is one of the most common causes of health care- associated infections in U.S. hospitals (Lessa, et al, 2015, New England Journal of Medicine). Recent estimates suggest C. difficile approaches 500,000 infections annually in the U.S. and is associated with approximately 20,000 deaths annually. (Guh, 2020, New England Journal of Medicine). Based on internal estimates, the recurrence rate for the antibiotics currently used to treat CDI is between 20% and 40% among approximately 150,000 patients treated. We believe the annual incidence of CDI in the U.S. approaches 600,000 infections and a mortality rate of approximately 9.3%.
About the Microbiome in C. difficile Infection (CDI) and Bile Acid Metabolism
C. difficile can be a normal component of the healthy gut microbiome, but when the microbiome is thrown out of balance, the C. difficile can thrive and cause an infection. After colonization with C. difficile, the organism produces and releases the main virulence factors, the two large clostridial toxins A (TcdA) and B (TcdB). (Kachrimanidou, Microorganisms 2020, 8, 200; doi:10.3390/microorganisms8020200.) TcdA and TcdB are exotoxins that bind to human intestinal epithelial cells and are responsible for inflammation, fluid and mucous secretion, as well as damage to the intestinal mucosa.
Bile acids perform many functional roles in the GI tract, with one of the most important being maintenance of a healthy microbiome by inhibiting C. difficile growth. Primary bile acids, which are secreted by the liver into the intestines, promote germination of C. difficile spores and thereby increase the risk of recurrent CDI after successful treatment of an initial episode. On the other hand, secondary bile acids, which are produced by normal gut microbiota through metabolism of primary bile acids, do not induce C. difficile sporulation and therefore protect against recurrent disease. Since ibezapolstat treatment leads to minimal disruption of the gut microbiome, bacterial production of secondary bile acids continues which may contribute to an anti-recurrence effect. Beneficial effects of bile acids include a decrease in primary bile acids and an increase in secondary bile acids in patients with CDI, which was observed in the Company's Ph2a trial results and previously reported (CID, 2022).
About Acurx Pharmaceuticals, Inc.
Acurx Pharmaceuticals is a late-stage biopharmaceutical company focused on developing a new class of small molecule antibiotics for difficult-to-treat bacterial infections. The Company's approach is to develop antibiotic candidates with a Gram-positive selective spectrum (GPSS®) that blocks the active site of the Gram+ specific bacterial enzyme DNA polymerase IIIC (pol IIIC), inhibiting DNA replication and leading to Gram-positive bacterial cell death. Its R&D pipeline includes antibiotic product candidates that target Gram-positive bacteria, including Clostridioides difficile, methicillin-resistant Staphylococcus aureus (MRSA), vancomycin resistant Enterococcus (VRE) and drug-resistant Streptococcus pneumoniae (DRSP).
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Acurx - The replay of the Jan 11 JP Morgan presentation is up on Acurx's website (links below). Luci said all the remaining data should be out in Jan, and includes the extended durability (94 day), microbiome comparison data, and microbiology comparison data. So the next 2 weeks should be very interesting :o)
https://d3ua9t66dzwd8a.cloudfront.net/HC24/p_50099_Acurx.mp3
https://ir.acurxpharma.com/
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ATM (At The Market) Program -
https://www.investopedia.com/terms/a/atthemarket.asp#:~:text=An%20at%2Dthe%2Dmarket%20(,(and%20a%20better%20price).
>>> At-the-Market Offering FAQs
What Is an At-the-Market Offering?
An at-the-market offering (ATM) takes place after a company goes public, as a sort of follow-up. In an ATM, a company can offer secondary public shares on any given day, usually depending on the prevailing market price, to raise capital.
An at-the-market (ATM) offering gives the issuing company the ability to raise capital as needed. If the company is not satisfied with the available price of shares on a given day, it can refrain from offering them, saving its new shares for another day (and a better price).
ATM offerings are sometimes referred to as controlled equity distributions because of their ability to sell shares into the secondary trading market at the current prevailing price.
How Does an At-the-Market Offering Affect the Stock Price?
Shareholders often react negatively to secondary offerings because they dilute existing shares and many are introduced below market prices. However, unlike the typical 7% to 10% drop in stock price that follows the announcement of a traditional follow-on equity offering, the average stock price change following the announcement of an ATM is minimal—often, just 1% to 3%.
Where Can I Find At-the Market Offerings?
Issuing companies set up ATM programs to prepare prospectuses and issue shares—a streamlined version of a regular initial public offering. A sales agent—usually an investment bank—then circulates news of the ATM to investors and financial firms, announcing a launch date when shares will be available.
Why Do Companies Do At-the Market Offerings?
An ATM offering program may provide a company with a more attractive and less dilutive capital-raising option. The availability of an ATM program also allows a company to take advantage of a temporarily higher stock price, a good earnings report (typically, the best time to launch an offering is shortly after the filing of the issuer’s Form 10-K or 10-Q), or an upcoming milestone event to raise money.
ATMs also tend to be faster and cheaper than traditional IPOs or other follow-on equity offerings. There is no lock-up period, and the incremental sale of shares has a minimal impact on the prevailing stock price.
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Acurx - CEO interview -
>>> Coya Therapeutics, Inc. (COYA), a clinical-stage biotechnology company, engages in the development of proprietary medicinal products to modulate the function of regulatory T cells (Tregs). The company's product candidate pipeline is based on therapeutic modalities, such as Treg-enhancing biologics, Treg-derived exosomes, and autologous Treg cell therapy.
It is developing COYA 101, an autologous regulatory T-cell product candidate that has completed Phase 2a clinical trial for use in the treatment of Amyotrophic Lateral Sclerosis. The company's product candidates in IND-enabling studies include COYA 301, a low-dose interleukin 2 Treg-enhancing biologic for use in the treatment of Frontotemporal Dementia; and COYA 302, a biologic combination for subcutaneous and/or intravenous administration intended to enhance Treg function while depleting T effector function and activated macrophages for use in the treatment of neurodegenerative and autoimmune diseases. It is also developing COYA 201, an antigen directed Treg-derived exosome product candidate that is in preclinical stage for use in the treatment of neurodegenerative, autoimmune, and metabolic diseases; and COYA 206, an antigen directed Treg-derived exosome product candidate, which is in discovery stage. The company has a collaboration with Dr. Reddy's Laboratories SA for the development and commercialization of COYA 302, an investigational combination therapy for treatment of amyotrophic lateral sclerosis. The company was incorporated in 2020 and is headquartered in Houston, Texas.
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AbbVie, Axsome - >>> 2 Biotech Stocks You Can Buy and Hold for the Next Decade
Motley Fool
January 2, 2024
https://finance.yahoo.com/news/2-biotech-stocks-buy-hold-134500692.html
There are many ways to grow one's wealth over a decade, but few are as accessible as investing in stocks. Even with a relatively modest sum, picking the right investments and letting time and compounding work their magic will generate the kinds of returns most people would be proud to achieve. However, with scores of options to choose from on equity markets, it can sometimes be challenging to know which stocks are worth investing in.
Let's consider two biotech stocks that could generate steady returns in the next 10 years: AbbVie (NYSE: ABBV) and Axsome Therapeutics (NASDAQ: AXSM).
1. AbbVie
AbbVie has been a terrific investment over the past decade. Revenue and earnings have generally grown at a good clip, and it has raised its dividend by 288%. Now that its blockbuster medicine Humira has seen its patent expire in the U.S., things are more complicated -- but that's no reason to give up on the company.
As Humira's sales drop, those of Skyrizi and Rinvoq, a pair of immunology medicines whose indications substantially overlap with those of Humira, are rising. Management expects this pair of drugs to exceed Humira's annual sales by 2027.
AbbVie is looking for growth elsewhere, too. It recently announced the acquisition of Cerevel Therapeutics, a neuroscience-focused biotech, for $8.7 billion in cash. The transaction will improve AbbVie's pipeline in that particular therapeutic area.
Back in 2020, AbbVie closed its acquisition of Allergan, which gave it access to key products, including its Botox franchise. There is nothing wrong with drugmakers relying on acquiring smaller companies to improve their lineups or pipelines, and that's what AbbVie is doing again.
The company does have an extensive pipeline of its own, with several dozen ongoing programs, many of which should result in brand-new approvals or label expansions. For instance, it launched a brand-new cancer medicine this year called Epkinly. So, despite its poor performance in the market this year, AbbVie isn't dead in the water -- far from it.
Patient investors can still expect the company to deliver solid returns and consistent dividend increases. AbbVie is part of the elite group of Dividend Kings: It has raised its payouts for 52 consecutive years, an impressive feat. The company's current yield is 4% -- much higher than the S&P 500's average of 1.62%.
With an underlying business that can still support dividend growth and a reasonable cash payout ratio of 42%, AbbVie can afford many more dividend hikes. That's another great reason to stick with this stock in the next decade.
2. Axsome Therapeutics
Axsome Therapeutics is a mid-cap biotech with just two products on the market. The company's recent financial results aren't great. In the third quarter, it posted revenue of just about $58 million, which soared by 243% year over year. Its net loss per share of $1.32 was worse than the net loss per share of $1.07 recorded in the prior-year period.
However, things could soon change for Axsome Therapeutics given its pipeline. The company has a string of potential approvals, regulatory submissions, and late-stage clinical trials due to start or release top-line data in the next 12 months. For instance, one of its products, depression treatment Auvelity, is seeking a label expansion in treating Alzheimer's disease agitation (aggressive and restless symptoms). The biotech is running a phase 3 clinical trial whose data should be revealed next year.
Auvelity is also being developed to help with smoking cessation, with a phase 2/3 study planned for next year. Axsome Therapeutics' other approved product, narcolepsy treatment Sunosi, is undergoing a phase 3 study targeting ADHD, with top-line results due in the second half of 2024. There is much more to the biotech's pipeline.
Axsome's lineup should look very different in the next three years. Considering that even its oldest approved product, Sunosi, has been on the market only since 2019, the company should be able to deliver solid top-line growth through the next decade.
That could translate into superior stock market returns, too -- especially since, with the company's market capitalization of just $3.5 billion, investors don't appear to fully appreciate the potential of Axsome's late-stage pipeline. That makes it an attractive biotech stock to buy now and hold.
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>>> 11 Best Psychedelic Stocks to Buy in 2024
Insider Monkey
Sheryar Siddiq
December 29, 2023
https://finance.yahoo.com/news/11-best-psychedelic-stocks-buy-134746256.html
In this article, we discuss 11 of the best psychedelic stocks to buy in 2024. If you want to see more stocks in this selection, check out 5 Best Psychedelic Stocks to Buy.
Unlike substances like marijuana or cocaine, psychedelics are potent and serve as a form of gateway to an 'alternate reality' for those who use them. Psychedelics represent a category of hallucinogenic drugs capable of inducing non-ordinary states of consciousness. This class encompasses various chemical substances, including LSD and plant-derived compounds. Psychedelics have the potential to alter or intensify sensory perceptions, thought processes, and energy levels, often facilitating spiritual experiences. They are classified into two main groups: empathogens and dissociative drugs (e.g., PCP) and serotonergic substances (e.g., LSD). Beyond recreational use, these drugs are being explored for therapeutic purposes, showing promise in treating conditions such as major depressive disorders, treatment-resistant depression, panic disorder, post-traumatic stress disorder, and opiate addiction, among others.
Venture-capital investors have played a pivotal role in the resurgence of interest in psychedelics. In early 2020, startups in this field noticed a surge in investor appetite, marking the onset of a "psychedelic renaissance." Clara Burtenshaw, a partner at Neo Kuma Ventures, the largest venture capital fund in Europe focused on psychedelic investments, expressed her enthusiasm for psychedelic healthcare on February 16, 2023, stating:
“Psychedelic healthcare is a very exciting area because it’s really this apex of drugs, clinics, and experimental treatments.”
Numerous pharmaceutical companies specializing in psychedelics are pursuing regulatory pathways for the legal approval of their drugs. Hallucinogens, notably psilocybin, are demonstrating significant potential in addressing conditions like depression and schizophrenia. Some of these pharmaceutical firms are publicly listed, including the likes of Johnson & Johnson (NYSE:JNJ), Merck & Co., Inc. (NYSE:MRK), and AbbVie Inc. (NYSE:ABBV), which have garnered heightened interest from investors. This trend mirrors the trajectory observed with marijuana stocks in recent years, where cannabis, once on the periphery, has evolved into a substantial and expanding industry.
Our Methodology
We selected the following psychedelic stocks based on overall hedge fund sentiment toward each stock. We have assessed the hedge fund sentiment from Insider Monkey’s database of 910 elite hedge funds tracked as of the end of the third quarter of 2023. The list is arranged in ascending order of the number of hedge fund holders in each firm.
11. Cybin Inc. (NYSE:CYBN)
Number of Hedge Fund Holders: 1
Based in Toronto, Canada, Cybin Inc. (NYSE:CYBN) is a biopharmaceutical company with a primary focus on the development of therapeutic solutions using psychedelics. The company is engaged in the creation of medications for major depressive disorders, anxiety, and alcohol use disorders, along with addressing neuroinflammation. Additionally, Cybin Inc. (NYSE:CYBN) has introduced EMBARK, a program centered around psychedelic-assisted psychotherapy.
In late November, Cybin Inc. (NYSE:CYBN) disclosed encouraging results from the Phase 2 trial of CYB003, its exclusive deuterated psilocybin analog designed for treating major depressive disorder ("MDD"). The company reported that CYB003 met the primary efficacy endpoint, demonstrating swift and statistically significant enhancements in depression symptoms following a single dose. Additionally, a second dose conferred clear incremental benefits, leading to remission in 4 out of 5 patients from their depression within 6 weeks.
As of the end of Q3, Steve Cohen's Point72 Asset Management emerged as the primary and sole stakeholder, based on Insider Monkey's third-quarter database.
In addition to Johnson & Johnson (NYSE:JNJ), Merck & Co., Inc. (NYSE:MRK), and AbbVie Inc. (NYSE:ABBV), Cybin Inc. (NYSE:CYBN) ranks as one of the best psychedelic stocks to invest in.
10. Mind Medicine (MindMed) Inc. (NASDAQ:MNMD)
Number of Hedge Fund Holders: 3
Mind Medicine (MindMed) Inc. (NASDAQ:MNMD) is a biotechnology company based in New York that specializes in psychedelic medicine. The company is dedicated to the development of psychoplastogens and therapeutic solutions inspired by psychedelics to tackle mental illnesses and addiction.
Mind Medicine (MindMed) Inc. (NASDAQ:MNMD) recently reported that it is nearing the launch of its LSD-based treatment for anxiety patients. The company anticipates more phase two trial data by the fourth quarter of this year and proof-of-concept data by the end of Q1 next year. In its Q3 financial report released on Thursday, the psychedelic firm disclosed a net loss of $17.9 million for the period ending Sept. 30, up from the $16.5 million loss in the same quarter last year. However, MindMed also stated having $117.7 million in funds, expected to cover ongoing trials and research activities "into 2026, if certain milestones are achieved that unlock additional capital."
In 2023, Mind Medicine (MindMed) Inc. (NASDAQ:MNMD) also expended $43.8 million in operating expenses, including $6.4 million for MM-120 studies, their LSD version for treating generalized anxiety disorder. Results from a 198-patient study of MM-120 are anticipated before year-end, with "safety and efficacy results" expected in Q1 next year. The proof-of-concept study, involving 53 patients, is also projected to reveal results in Q1 2024.
Among the hedge funds being tracked by Insider Monkey, Israel Englander’s Citadel Investment Group is a leading shareholder in Mind Medicine (MindMed) Inc. (NASDAQ:MNMD) with 237,475 shares worth more than $743,297. Overall, 3 hedge funds reported holding stakes in Mind Medicine (MindMed) Inc. (NASDAQ:MNMD) as of the third quarter of 2023.
9. Atai Life Sciences N.V. (NASDAQ:ATAI)
Number of Hedge Fund Holders: 5
Founded in 2018 and headquartered in Berlin, Germany, Atai Life Sciences N.V. (NASDAQ:ATAI) is a clinical-stage biopharmaceutical company specializing in the development of treatments for mental health disorders. These include conditions such as treatment-resistant depression, schizophrenia, opioid use disorder, anxiety disorder, and mild traumatic brain injuries.
In its third quarter 2023 financial results, Atai Life Sciences (NASDAQ:ATAI) reported limited revenue as the company continues to develop its drug portfolio. The net income of $44.2 million was primarily attributed to a $69 million non-cash change in the fair value of other investments related to an accounting method change for its CCOMPASS Pathways plc (NASDAQ:CMPS) investment. Additionally, Atai Life Sciences (NASDAQ:ATAI) recorded an $8.3 million non-cash share-based compensation expense. Despite this, the company maintains a robust cash position of $209 million, anticipating that it, along with committed term loan funding, will be sufficient to sustain operations into the first half of 2026.
According to Insider Monkey’s third quarter database, 5 hedge funds were bullish on Atai Life Sciences N.V. (NASDAQ:ATAI), compared to 7 funds in the earlier quarter. John Overdeck And David Siegel’s Two Sigma Advisors is the largest stakeholder of the company, with 107,500 million shares worth $138,675.
8. Seelos Therapeutics, Inc. (NASDAQ:SEEL)
Number of Hedge Fund Holders: 5
Seelos Therapeutics, Inc. (NASDAQ:SEEL) is a clinical-stage biopharmaceutical company headquartered in New York. The company is dedicated to advancing the development and commercialization of therapeutics targeting central nervous system, respiratory, and other disorders.
Seelos Therapeutics, Inc. (NASDAQ:SEEL) has two programs currently undergoing clinical testing. In September 2023, the company disclosed outcomes from a phase 2 study evaluating the intranasal ketamine drug SLS-002 for the treatment of acute suicidal ideation and behavior in patients with major depressive disorder. Additionally, SLS-005 (intravenous trehalose, a sugar containing two glucose molecules) is in phase 2b/3 trials for addressing the neurological disease amyotrophic lateral sclerosis (ALS) and is in phase 2 testing for managing the neurodegenerative disorder spinocerebellar ataxia type 3.
According to Insider Monkey’s third quarter database, 5 hedge funds were bullish on Seelos Therapeutics, Inc. (NASDAQ:SEEL), compared to 3 funds in the earlier quarter.
7. GH Research PLC (NASDAQ:GHRS)
Number of Hedge Fund Holders: 10
GH Research PLC (NASDAQ:GHRS) is a clinical-stage biopharmaceutical company that was established in 2018 and is headquartered in Dublin, Ireland. The company is focused on the development of therapies for the treatment of psychiatric and neurological disorders.
According to Insider Monkey’s third quarter database, 10 hedge funds were long GH Research PLC (NASDAQ:GHRS), compared to 13 funds in the earlier quarter. Mark Lampert’s Biotechnology Value Fund / BVF Inc is the biggest position holder in the company, with 9.27 million shares worth approximately $93.2 million.
Much like Johnson & Johnson (NYSE:JNJ), Merck & Co., Inc. (NYSE:MRK), and AbbVie Inc. (NYSE:ABBV), GH Research PLC (NASDAQ:GHRS) is one of the best psychedelic stocks that investors should pay attention to.
6. Relmada Therapeutics, Inc. (NASDAQ:RLMD)
Number of Hedge Fund Holders: 13
Relmada Therapeutics, Inc. (NASDAQ:RLMD) is a biotechnology company based in Florida, specializing in the clinical-stage development of diverse products for the treatment of central nervous system diseases and other disorders. Analysts consider it one of the promising psychedelic stocks in the market.
In September of this year, Relmada Therapeutics, Inc. (NASDAQ:RLMD) announced encouraging safety and efficacy outcomes from the extended Phase III REL-1017-310 trial, which assessed REL-1017 in individuals diagnosed with major depressive disorder (MDD). Over the course of up to one year, patients receiving daily doses of REL-1017 demonstrated enduring enhancements in depressive symptoms and related functional challenges. The extended administration of REL-1017 exhibited favorable tolerability in patients, featuring low rates of adverse events and the absence of any newly identified safety concerns.
According to Insider Monkey’s third quarter database, 13 hedge funds were long Relmada Therapeutics, Inc. (NASDAQ:RLMD), compared to 11 funds in the prior quarter. David Kroin’s Deep Track Capital held the biggest position in the company.
5. COMPASS Pathways plc (NASDAQ:CMPS)
Number of Hedge Fund Holders: 21
COMPASS Pathways plc (NASDAQ:CMPS) is a mental healthcare company based in the United Kingdom. The company is actively engaged in the development of psilocybin therapy, progressing through late-stage clinical trials in both Europe and North America. The focus is on patients experiencing treatment-resistant depression (TRD). COMPASS Pathways plc (NASDAQ:CMPS) has formulated COMP360, a psilocybin formulation incorporating pharmaceutical-grade polymorphic crystalline psilocybin. A Phase IIb clinical trial for psilocybin therapy in TRD has been completed across 22 sites in Europe and North America, evaluating the safety and efficacy of COMP360 in three doses: 1mg, 10mg, and 25mg.
During 2023’s September quarter, 21 out of the 910 hedge funds profiled by Insider Monkey had held a stake in the company. COMPASS Pathways plc (NASDAQ:CMPS)’s biggest hedge fund investor is Catherine D. Wood’s ARK Investment Management due to its $18 million investment.
4. Intra-Cellular Therapies, Inc. (NASDAQ:ITCI)
Number of Hedge Fund Holders: 37
Intra-Cellular Therapies, Inc. (NASDAQ:ITCI) is a biopharmaceutical company specializing in the discovery, development, and commercialization of small molecule drugs targeting unmet medical needs, particularly in neuropsychiatric and neurological disorders. Caplyta, the sole approved drug in Intra-Cellular Therapies, Inc. (NASDAQ:ITCI)’s portfolio, received FDA approval in December 2019 for the treatment of schizophrenia in adults. Subsequently, in December 2021, it gained FDA approval for treating bipolar depression. Following these approvals, Caplyta’s sales have experienced substantial growth, and the company anticipates maintaining this positive momentum.
In the third quarter of 2023, Intra-Cellular Therapies, Inc. (NASDAQ:ITCI) reported a loss of $0.25 per share. The incurred loss was narrower than the $0.57 per share loss in the same quarter of the previous year, attributed to increased product sales. The total revenues, which include product sales and grant revenues, reached $126.2 million, marking a significant increase from $71.9 million in the corresponding period of the prior year.
By the end of this year’s third quarter, 37 out of the 910 hedge funds part of Insider Monkey’s research had invested in the company. Intra-Cellular Therapies, Inc. (NASDAQ:ITCI)’s biggest hedge fund investor is David Kroin’s Deep Track Capital through its $135 million stake.
3. AbbVie Inc. (NYSE:ABBV)
Number of Hedge Fund Holders: 73
AbbVie Inc. (NYSE:ABBV) operates as a specialized biopharmaceutical company dedicated to researching, developing, manufacturing, and distributing medications tailored for chronic and intricate illnesses. The company is renowned for its flagship drug, Humira, a crucial treatment for conditions like moderate-to-severe rheumatoid arthritis and Crohn’s disease. In May 2020, AbbVie Inc. (NYSE:ABBV) entered the psychedelic drugs market by acquiring a stake in Allergan PLC.
With an impressive 50-year streak of continuous dividend growth, the American pharmaceutical giant currently boasts a dividend yield of 4.48% as of December 13.
As of the close of the third quarter in 2023, Insider Monkey’s database, monitoring 910 hedge funds, indicated 73 holdings in AbbVie Inc. (NYSE:ABBV), marking a slight decrease from the 74 hedge funds in the previous quarter. The collective value of these holdings surpasses $3.27 billion.
2. Merck & Co., Inc. (NYSE:MRK)
Number of Hedge Fund Holders: 85
Merck & Co., Inc. (NYSE:MRK) is a distinguished American multinational pharmaceutical company with its headquarters in Rahway, New Jersey. Originally part of the Merck Group established in Germany in 1668, it retains the name of its former parent company. Operating as Merck Sharp & Dohme or MSD outside the United States and Canada, the company holds a prominent position in the pharmaceutical industry, specializing in the development of medicines, vaccines, biologic therapies, and animal health products.
Showcasing a consistent pattern of dividend growth for 11 consecutive years, the company currently provides a quarterly dividend of $0.77 per share, resulting in a dividend yield of 2.91% as of December 13.
Insider Monkey delved into the investment activities of 910 hedge funds in the third quarter of 2023, revealing that 85 had invested in Merck & Co., Inc. (NYSE:MRK), marking an increase from 78 in the previous quarter.
1. Johnson & Johnson (NYSE:JNJ)
Number of Hedge Fund Holders: 88
Established in 1886, Johnson & Johnson (NYSE:JNJ) is a prominent American multinational corporation known for its groundbreaking contributions to medical devices, pharmaceuticals, and consumer packaged goods. The company’s pharmaceutical division has consistently demonstrated impressive dividend growth for over 62 years. As of November 19, Johnson & Johnson (NYSE:JNJ) offers a quarterly dividend of $1.19 per share, reflecting a dividend yield of 3.18%.
On October 17, Johnson & Johnson (NYSE:JNJ) reported adjusted earnings and revenue that exceeded Wall Street expectations, prompting an upward revision of its full-year guidance. The robust sales performance in both pharmaceuticals and medical devices contributed to the positive results. The company posted a net income of $4.31 billion, equivalent to $1.69 per share, maintaining consistency with the previous year’s net income of $4.31 billion, or $1.62 per share, for the same period.
In the third quarter of 2023, the number of hedge funds tracked by Insider Monkey with holdings in Johnson & Johnson (NYSE:JNJ) decreased to 84, down from 88 in the previous quarter. The collective investments by these hedge funds surpass a total value of $4.15 billion. Bridgewater Associates, led by Ray Dalio, stands out as a prominent hedge fund investor in Johnson & Johnson (NYSE:JNJ) with a substantial stake valued at approximately $424.3 million.
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>>> A potent antibiotic has emerged in the battle against deadly, drug-resistant superbugs
LA Times
by Corinne Purtill
January 3, 2024
https://www.yahoo.com/news/potent-antibiotic-emerged-battle-against-160058569.html
Under a microscope, this drug-resistant superbug looks as benign as a handful of pebbles. Yet carbapenem-resistant Acinetobacter baumannii, or CRAB, is a nightmare for hospitals worldwide, as it kills roughly half of all patients who acquire it.
Identified as a top-priority pathogen by both the World Health Organization and the U.S. Centers for Disease Control and Prevention, CRAB is the most common form of a group of bacteria that are resistant to nearly all available antibiotics. Victims are typically hospitalized patients who are already sick with blood infections or pneumonia. In the U.S. alone, the bug sickens thousands and kills hundreds every year.
But 2024 is starting with some encouraging news on the global health front: For the first time in half a century, researchers have identified a new antibiotic that appears to effectively kill A. baumannii.
The compound, zosurabalpin, attacks bacteria from a novel angle, disrupting the route that a key toxin takes on its journey from inside the bacterial cell to the outer membrane that shields the bug from the immune system’s defensive onslaughts.
No other antibiotic approved by the U.S. Food and Drug Administration takes this approach, and the element of surprise is an important advantage against even microscopic foes. A. baumannii has had no opportunity to develop resistance against the drug, which means that, for at least a little while, zosurabalpin could ward off severe illness and death.
“As far as I can tell, the scientific approach is brilliant,” said Dr. Oladele A. Ogunseitan, a professor of population health and disease prevention at UC Irvine who was not involved with the study.
The drug was developed jointly by scientists at the Swiss pharmaceutical company Roche and at Harvard University. Their findings were published Wednesday in the journal Nature.
Carbapenem-resistant A. baumannii is a type of gram-negative bacteria, a vexing category of superbugs. Encased in both an inner and outer membrane that antibiotics struggle to cross, gram-negative bacteria are resistant to most currently available therapies. They are also astonishingly canny for unicellular organisms, with the ability to rapidly develop new defenses against antibiotics and then pass them along to other bacteria through genetic material.
Antibiotic-resistant superbugs claim the lives of more than 1 million people globally each year. The rise of drug resistance is due in part to human folly — we have long over-prescribed and misused antibiotics — but it is also because bacteria are continually finding ways to evade threats. Over the last 50 years, these pathogens have evolved defenses faster than we can produce new drugs.
In their search for a new weapon, the Roche and Harvard scientists turned their attention to a group of compounds called tethered macrocyclic peptides. After testing a library of 45,000 MCPs, the researchers came across one that seemed especially lethal against A. baumannii. After some chemical tinkering, that compound became zosurabalpin.
“This is a very promising advance,” said Paul J. Hergenrother, a chemistry professor at the University of Illinois who was not involved in the research but wrote of the findings for Nature. “Zosurabalpin kills bacteria in a way that is different from all other approved antibiotics.”
The drug kicks into gear only in the presence of lipopolysaccharide, a bacterial toxin. LPS is made inside the bacterial cell and is carried by a dedicated transport system to the bug’s outer defenses.
“The bacterial outer membrane is important for bacteria because it helps them to live in harsh conditions and to survive attacks by our immune system,” said Kenneth Bradley, Roche’s global chief of discovery for infectious diseases.
Zosurabalpin essentially cuts off the LPS transport route. Without a way to get to the outer membrane of the cell, where it can get to work fighting off drugs and immune attacks, the toxin builds up inside the bacteria and eventually kills the cell.
In mice studies, the drug effectively killed off CRAB infections in the blood, lungs and thighs, a selection that mirrors the ways the bug infects humans.
It’s currently in Phase I trials in humans, where researchers are looking at the drug’s safety, tolerability and the amount of the chemical that remains in patients’ bodies over time, said Michael Lobritz, Roche’s infectious diseases chief.
“It has been more than 50 years since the last distinct class of antibiotic was launched that is capable of treating infections by Gram-negative bacteria,” Lobritz said in an email. “Any new antibiotic class that has the ability to treat infections caused by multidrug resistant (MDR) bacteria such as carbapenem-resistant Acinetobacter baumannii (CRAB) would be a significant breakthrough.”
Encouraging as the early results are, scientists stressed that it would be foolish to get cocky in the fight against a bug that, time and time again, has found ways to evade our most advanced pharmaceutical weaponry.
“Resistance has emerged to every antibiotic ever created, and it is likely that resistance will emerge to zosurabalpin in the future too, if it successfully becomes a clinical antibiotic,” Bradley said.
In their findings, the authors noted a few gene mutations in the lab that significantly decreased the drug’s success against A. baumannii. These were rare but worrying; one freak mutation reduced the drug’s effectiveness 256-fold.
“Although the rates of appearance of these resistant organisms is low, and comparable to standard-of-care antibiotics, the observation affirms the principle that we can never rest on our laurels with the chemical and biochemical warfare that we are waging on bacterial pathogens,” Ogunseitan said.
Zosurabalpin is essentially unknown to bacteria. If it proves safe and effective in humans, there’s probably a limited window in which it could effectively spare lives and suffering. But no matter how sophisticated our tools, scientists said, these potentially deadly cells will always have a major advantage against us.
“Bacteria have a big numbers advantage — billions can be in a flask,” said Hergenrother. “Bacteria will eventually evolve resistance to virtually every antibiotic, which is why we need a steady supply of new antibiotic candidates.”
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>>> Acurx to present at the 42nd Annual J.P. Morgan Healthcare Conference
PR Newswire
January 3, 2024
https://finance.yahoo.com/news/acurx-present-42nd-annual-j-130000189.html
STATEN ISLAND, N.Y., Jan. 3, 2024 /PRNewswire/ -- Acurx Pharmaceuticals, Inc. (NASDAQ: ACXP) ("Acurx" or the "Company") today announced that President and Chief Executive Officer David P. Luci will present at the 42nd Annual J.P. Morgan Healthcare Conference on Thursday, January 11, 2024 at 12:00 pm PST. An audio webcast of the presentation will be available live at https://jpmorgan.metameetings.net/events/healthcare24/sessions/50099-acurx-pharmaceuticals-inc/webcast?gpu_only=true&kiosk=true and will be available upon completion of the conference on the Company's website at www.acurxpharma.com under the Investors section.
About Acurx Pharmaceuticals, Inc.
Acurx Pharmaceuticals is a clinical stage biopharmaceutical company focused on developing new antibiotics for difficult to treat infections. The Company's approach is to develop antibiotic candidates with a Gram-positive selective spectrum (GPSS®) that blocks the active site of the Gram+ specific bacterial enzyme DNA polymerase IIIC (pol IIIC), inhibiting DNA replication and leading to Gram-positive bacterial cell death. Its R&D pipeline includes antibiotic product candidates that target Gram-positive bacteria, including Clostridioides difficile, methicillin-resistant Staphylococcus aureus (MRSA), vancomycin resistant Enterococcus (VRE) and drug-resistant Streptococcus pneumoniae (DRSP).
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>>> Anixa Biosciences, Inc. (ANIX) is developing therapies and vaccines that are focused on critical unmet needs in oncology. Anixa’s programs are using the body’s immune system to take multiple approaches in fighting cancer—CAR-T cell therapy to treat cancer and vaccines to prevent cancer.
https://www.anixa.com/
Therapeutics
CAR-T
We are developing chimeric antigen receptor T-cell (CAR-T) based immuno-therapy drugs which genetically engineer a patient’s own immune cells to fight cancer.
Vaccines
Breast Cancer Vaccine
We are developing a revolutionary vaccine technology focused on preventing breast cancer, specifically triple negative breast cancer, the most lethal form of the disease.
Ovarian Cancer Vaccine
Using a similar mechanism of action as our breast cancer vaccine technology, we are developing a vaccine to prevent the occurrence of ovarian cancer.
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>>> Acurx Announces Positive Phase 2b Results Showing 100% of Patients Who Had Clinical Cure with Ibezapolstat Also Had Sustained Clinical Cure
PR Newswire
December 11, 2023
https://finance.yahoo.com/news/acurx-announces-positive-phase-2b-120100375.html
All 15 ibezapolstat-treated patients in Phase 2b who achieved Clinical Cure (CC) at end of treatment (EOT) remained free of C. difficile Infection (CDI) recurrence through one month after EOT, for a Sustained Clinical Cure (SCC) rate of 100%
2 of 14 patients treated with standard of care, oral vancomycin, experienced recurrent infection within one month after EOT for a SCC of 86%
100% of the 25 ibezapolstat-treated patients in Phase 2 (Phase 2a and 2b) who had CC at EOT remained cured through one month after EOT
Further analyses will be forthcoming Q1 2024, as data become available, regarding other endpoints, from the Phase 2b trial, including Extended Clinical Cure (ECC) data up to 94 days and comparative effects vs vancomycin on the gut microbiome
Preparation underway for meetings with FDA, European Medicines Agency and other global regulatory agencies and advancement to international Phase 3 clinical trials
STATEN ISLAND, N.Y., Dec. 11, 2023 /PRNewswire/ -- Acurx Pharmaceuticals, Inc. (NASDAQ: ACXP) ("Acurx" or the "Company"), a late-stage biopharmaceutical company developing a new class of small molecule antibiotics for difficult-to-treat bacterial infections, today announced positive Phase 2b results showing 100% of CDI patients who had CC with ibezapolstat, the company's late-stage antibiotic candidate, also had SCC.
The efficacy results from the Phase 2 trial (Phase 2a and Phase 2b) are summarized in the table below:
Clinical Cure
(CC)
at EOT
Sustained Clinical Cure
(SCC)
One Month After EOT for
all evaluable patients
Sustained
Clinical Cure* (SCC) One
Month After EOT
ibezapolstat Phase 2a
10/10 (100%)
10/10 (100%)
10/10 (100%)
ibezapolstat Phase 2b
15/16 (94%)
15/16 (94%)
15/15 (100%)
ibezapolstat Phase 2a +
Phase 2b Combined
25/26 (96%)
25/26 (96%)
25/25 (100%)
vancomycin
14/14 (100%)
12/14 (86%)
12/14 (86%)
*Sustained Clinical Cure was evaluated only for patients who were CC at EOT.
Kevin Garey, PharmD, MS, Professor and Chair, University of Houston College of Pharmacy, the Principal Investigator for microbiome aspects of the ibezapolstat clinical trial program and Acurx Scientific Advisory Board member stated: "These results help validate our ongoing scientific investigations into the anti-CDI recurrence properties of ibezapolstat including maintenance and regrowth of healthy gut microbes and bile acid homeostasis. I'm excited about our ongoing investigations into a new scientific paradigm optimizing C. difficile antibiotic development to effectively cure CDI and prevent recurrence."
According to Stuart Johnson, MD, Professor of Medicine, Loyola University (Infectious Disease) and Acurx Scientific Advisory Board member: "Treatment of CDI remains an important unmet medical need, for 2 reasons. First, the potential for development of resistance in C. difficile to currently available drugs like vancomycin threatens our standard therapeutic approach. Second, recurrent disease is a very serious problem with limited available treatment options. Although vancomycin is still an effective treatment, CDI patients treated with oral vancomycin experience a recurrence rate of 18-23%. Ibezapolstat, by virtue of its novel mechanism of action, lack of cross-resistance with any marketed antibiotics, narrow antibacterial spectrum, and selective effects on the gut microbiome, appears to be a promising potential new addition to our therapeutic armamentarium. I continue to be encouraged by the accumulating data showing that ibezapolstat is clinically comparable to vancomycin in treating CDI and preventing recurrence."
Robert J. DeLuccia, Executive Chairman of Acurx, stated: "The overall Phase 2 data demonstrate a high clinical cure rate of 96% together with this 100% recurrence-free rate is a promising one-two punch to C. difficile infection for a potential front-line treatment option for patients with CDI." He further stated: "These two clinical trial endpoints, together with the Phase 1 and Phase 2a clinical trial data and with additional data analyses to come, will form the basis for a comprehensive, solid data package to present to global regulatory authorities to support advancement to Phase 3 clinical trials during the second half next year and move one step closer on its pathway to commercialization."
David P. Luci, President & CEO of Acurx, stated: "Ibezapolstat continues to demonstrate success compared to a standard of care, oral vancomycin, to treat patients with CDI. We anticipate favorable separation between the two therapeutic options will continue in Q1 2024 with extended clinical cure and microbiome comparison data. We expect to leverage this success in a $1 billion plus US CDI market internationally as we move forward with an international Phase 3 clinical trial mandate." He added: "The Company also announced its "Made in America" policy initiative for manufacture of ibezapolstat capsules for Phase 3 clinical trials and commercial supply to ensure patients have uninterrupted access to this potentially life-saving antibiotic mitigating potential supply chain disruptions."
About the Ibezapolstat Phase 2 Clinical Trial
The completed multicenter, open-label single-arm segment (Phase 2a) study was followed by a double-blind, randomized, active-controlled, non-inferiority, segment (Phase 2b) at 28 US clinical trial sites which together comprise the Phase 2 clinical trial (see https://clinicaltrials.gov/ct2/show/NCT04247542). This Phase 2 clinical trial was designed to evaluate the clinical efficacy of ibezapolstat in the treatment of CDI
including pharmacokinetics and microbiome changes from baseline and continue to test for anti- recurrence microbiome properties seen in the Phase 2a trial, including the treatment- related changes in alpha diversity and bacterial abundance and effects on bile acid metabolism.
The completed Phase 2a segment of this trial was an open label cohort of up to 20 subjects from study centers in the United States. In this cohort, 10 patients with diarrhea caused by C. difficile were treated with ibezapolstat 450 mg orally, twice daily for 10 days. All patients were followed for recurrence for 28± 2 days. Per protocol, after 10 patients of the projected 20 Phase 2a patients completed treatment (100% cured infection at End of Treatment), the Trial Oversight Committee assessed the safety and tolerability and made its recommendation regarding early termination of the Phase 2a study and advancement to the Ph2b segment. The Company's Scientific Advisory Board concurred with this recommendation.
In the now completed Phase 2b trial segment, 32 patients with CDI were enrolled and randomized in a 1:1 ratio to either ibezapolstat 450 mg every 12 hours or vancomycin 125 mg orally every 6 hours, in each case, for 10 days and followed for 28 ± 2 days following the end of treatment for recurrence of CDI. The two treatments were identical in appearance, dosing times, and number of capsules administered to maintain the blind. The overall observed Clinical Cure rate in the combined Phase 2 trials in patients with CDI was 96% (25 out of 26 patients), based on 10 out of 10 patients (100%) in Phase 2a in the Modified Intent to Treat Population, plus 15 out of 16 (94%) patients in Phase 2b in the Per Protocol Population, who experienced Clinical Cure during treatment with ibezapolstat. Ibezapolstat was well-tolerated, with three patients each experiencing one mild adverse event assessed by the blinded investigator to be drug-related. All three events were gastrointestinal in nature and resolved without treatment. There were no drug-related treatment withdrawals or no drug-related serious adverse events, or other safety findings of concern. In the Phase 2b vancomycin control arm, 14 out of 14 patients experienced Clinical Cure. The Company is confident that based on the pooled Phase 2 ibezapolstat Clinical Cure rate of 96% and the historical vancomycin cure rate of approximately 81% (Vancocin® Prescribing Information, January 2021), we will demonstrate non-inferiority of ibezapolstat to vancomycin in Phase 3 trials in accordance with the applicable FDA Guidance for Industry (October, 2022).
The Phase 2b clinical trial segment was discontinued due to success. The Company made this decision in consultation with its medical and scientific advisors and statisticians based on observed aggregate blinded data and other factors, including the cost to maintain clinical trial sites and slow enrollment due to COVID-19 and its aftermath. The Company had determined that the trial performed as anticipated for both treatments, ibezapolstat and the control antibiotic vancomycin (a standard of care to treat patients with CDI), with high rates of clinical cure observed across the trial without any emerging safety concerns. Accordingly, an Independent Data Monitoring Committee was not required to perform an interim analysis of this Phase 2b trial data as originally planned. The Company anticipated that this decision would allow the Company to advance this first-in-class, FDA QIDP/Fast Track-designated antibiotic product candidate to Phase 3 clinical trials more expeditiously.
The Phase 2b trial was originally designed to be a non-inferiority (NI) trial and later amended to include an interim efficacy analysis with review by an Independent Data Monitoring Committee (IDMC). The decision to end the trial early based on blinded clinical observations obviated the need for an interim analysis, IDMC review, and NI assessment. The Company determined, in consultation with its clinical and statistical experts, that presenting clinical cure rates for the primary efficacy endpoint is the most appropriate representation for the clinical activity of ibezapolstat in treating CDI.
In the Phase 2 clinical trial, the Company will also evaluate pharmacokinetics (PK) and microbiome changes and test for anti-recurrence microbiome properties, including the change from baseline in alpha diversity and bacterial abundance, especially overgrowth of healthy gut microbiota Actinobacteria and Firmicute phylum species during and after therapy. Phase 2a data demonstrated complete eradication of colonic C. difficile by day three of treatment with ibezapolstat as well as the observed overgrowth of healthy gut microbiota, Actinobacteria and Firmicute phyla species, during and after therapy. Very importantly, emerging data show an increased concentration of secondary bile acids during and following ibezapolstat therapy which is known to correlate with colonization resistance against C. difficile. A decrease in primary bile acids and the favorable increase in the ratio of secondary-to-primary bile acids suggest that ibezapolstat may reduce the likelihood of CDI recurrence when compared to vancomycin.
About Ibezapolstat
Ibezapolstat is a novel, orally administered antibiotic being developed as a Gram-Positive Selective Spectrum (GPSS™) antibacterial. It is the first of a new class of DNA polymerase IIIC inhibitors under development by Acurx to treat bacterial infections. Ibezapolstat's unique spectrum of activity, which includes C. difficile but spares other Firmicutes and the important Actinobacteria phyla, appears to contribute to the maintenance of a healthy gut microbiome.
In June 2018, ibezapolstat was designated by the U.S. Food and Drug Administration (FDA) as a Qualified Infectious Disease Product (QIDP) for the treatment of patients with CDI and will be eligible to benefit from the incentives for the development of new antibiotics established under the Generating New Antibiotic Incentives Now (GAIN) Act. In January 2019, FDA granted "Fast Track" designation to ibezapolstat for the treatment of patients with CDI. The CDC has designated C. difficile as an urgent threat highlighting the need for new antibiotics to treat CDI.
About Clostridioides difficile Infection (CDI). According to the 2017 Update (published February 2018) of the Clinical Practice Guidelines for C. difficile Infection by the Infectious Diseases Society of America (IDSA) and Society or Healthcare Epidemiology of America (SHEA), CDI remains a significant medical problem in hospitals, in long-term care facilities and in the community. C. difficile is one of the most common causes of health care- associated infections in U.S. hospitals (Lessa, et al, 2015, New England Journal of Medicine). Recent estimates suggest C. difficile approaches 500,000 infections annually in the U.S. and is associated with approximately 20,000 deaths annually. (Guh, 2020, New England Journal of Medicine). Based on internal estimates, the recurrence rate for the antibiotics currently used to treat CDI is between 20% and 40% among approximately 150,000 patients treated. We believe the annual incidence of CDI in the U.S. approaches 600,000 infections and a mortality rate of approximately 9.3%.
About the Microbiome in C. difficile Infection (CDI) and Bile Acid Metabolism
C. difficile can be a normal component of the healthy gut microbiome, but when the microbiome is thrown out of balance, the C. difficile can thrive and cause an infection. After colonization with C. difficile, the organism produces and releases the main virulence factors, the two large clostridial toxins A (TcdA) and B (TcdB). (Kachrimanidou, Microorganisms 2020, 8, 200; doi:10.3390/microorganisms8020200.) TcdA and TcdB are exotoxins that bind to human intestinal epithelial cells and are responsible for inflammation, fluid and mucous secretion, as well as damage to the intestinal mucosa.
Bile acids perform many functional roles in the GI tract, with one of the most important being maintenance of a healthy microbiome by inhibiting C. difficile growth. Primary bile acids, which are secreted by the liver into the intestines, promote germination of C. difficile spores and thereby increase the risk of recurrent CDI after successful treatment of an initial episode. On the other hand, secondary bile acids, which are produced by normal gut microbiota through metabolism of primary bile acids, do not induce C. difficile sporulation and therefore protect against recurrent disease. Since ibezapolstat treatment leads to minimal disruption of the gut microbiome, bacterial production of secondary bile acids continues which may contribute to an anti-recurrence effect. Beneficial effects of bile acids include a decrease in primary bile acids and an increase in secondary bile acids in patients with CDI, which was observed in the Company's Ph2a trial results and previously reported (CID, 2022).
About Acurx Pharmaceuticals, Inc.
Acurx Pharmaceuticals is a clinical stage biopharmaceutical company focused on developing new antibiotics for difficult to treat infections. The Company's approach is to develop antibiotic candidates with a Gram-positive selective spectrum (GPSS®) that blocks the active site of the Gram+ specific bacterial enzyme DNA polymerase IIIC (pol IIIC), inhibiting DNA replication and leading to Gram-positive bacterial cell death. Its R&D pipeline includes antibiotic product candidates that target Gram-positive bacteria, including Clostridioides difficile, methicillin-resistant Staphylococcus aureus (MRSA), vancomycin resistant Enterococcus (VRE) and drug-resistant Streptococcus pneumoniae (DRSP).
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>>> Anixa Biosciences and Cleveland Clinic Present Positive New Data from Phase 1 Study of Breast Cancer Vaccine
DECEMBER 06, 2023
https://ir.anixa.com/press-releases/detail/1030/anixa-biosciences-and-cleveland-clinic-present-positive-new?ftag=YHF4eb9d17
– Antigen-specific T cell responses were observed at all dose levels –
– IFN? and IL-17, immune-mediated biomarkers of T cell activation, increased over time from baseline –
– Vaccine was safe and well tolerated –
– Conference call to commence today at 6:30 p.m. ET –
SAN JOSE, Calif., Dec. 6, 2023 /PRNewswire/ -- Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, today announced new and updated positive results from the Phase 1 clinical trial of its breast cancer vaccine. The trial is being conducted in collaboration with Cleveland Clinic with funding by a grant from the U.S. Department of Defense.
The data were presented at the 2023 San Antonio Breast Cancer Symposium by G. Thomas Budd, M.D., staff physician at Cleveland Clinic Cancer Institute and principal investigator of the study, in a poster entitled "Phase I Trial of alpha-lactalbumin vaccine in high-risk operable triple negative breast cancer (TNBC) and patients at high genetic risk for TNBC."
Patients who had been curatively treated for TNBC received three vaccinations given once every two weeks. IFN? and IL-17, which are T cell immune response indicators (cellular immunity), and antibody production (B cell humoral immunity) were measured to evaluate the vaccination effect. Data from the 16 patients treated to date showed that:
The majority of patients developed ELISpot (T-cell) responses that met the rigorous protocol-specified definition of an immune response, with a measurable but lesser magnitude of response noted in the remaining patients.
12 (75%) of the women had antigen-specific IFN? and/or IL-17 ELISpot responses that were observed at all dose levels, while ELISA antibody responses were observed at Dose Level 2 and higher.
A statistically significant (P = 0.03) increase in IFN? over baseline (Day 0) was observed by Day 56; while a significant (P = 0.0001) increase in IL-17 over baseline was observed by Day 14.
Among the doses studied, Dose Level 1 (10 mcg a-lactalbumin/10 mcg zymosan) was determined to be a usable immunologic dose as well as the maximum tolerated dose (MTD).
No significant side effects were observed, at the MTD, besides irritation at the sites of injection. No myalgias, flu-like symptoms, or aberrant laboratory values were noted.
Anixa and Cleveland Clinic plan to investigate additional intermediate dose levels and continue studying the vaccine's safety and immunologic effects in two additional patient cohorts.
The first cohort, which opened for enrollment in August 2023, is evaluating the combination of the Company's breast cancer vaccine with Keytruda® (pembrolizumab) in post-operative patients found to have residual disease following neoadjuvant chemo-immunotherapy.
The second cohort will investigate the safety and immunologic effects of the vaccine in patients who are BRCA1, BRCA2, or PALB2 mutation positive and are planning prophylactic risk-reducing mastectomies.
"The data from our Phase 1 trial to date has exceeded our expectations, and we are pleased with our progress. This vaccine is designed to direct the immune system to destroy TNBC cancer cells through a mechanism that has never previously been utilized for cancer vaccine development," stated Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences. "We look forward to reviewing additional data as the trial continues to completion, and we are in the planning stages of the Phase 2/3 studies of this vaccine. Our goal is to initially evaluate the vaccine's ability to prevent recurrence of cancer in survivors, and continue with extension studies to eventually determine its effectiveness in preventing the initial onset of TNBC."
"There is a large unmet need for preventing TNBC, an aggressive form of breast cancer with few targeted treatment options available," said Dr. Budd, Cleveland Clinic. "We are encouraged by the data gathered to date and look forward to determining the optimal vaccine dose in additional patient cohorts. Our hope is that future studies will demonstrate that the antigen-specific T cell responses we observed translate to the prevention of breast cancer recurrence."
Anixa is the exclusive worldwide licensee to the novel breast cancer vaccine technology invented at Cleveland Clinic, the site of the Phase 1 trial. The grant from the U.S. Department of Defense was made directly to Cleveland Clinic.
Conference Call Information
Anixa is pleased to invite all interested parties to participate in a conference call, during which this new data will be discussed.
Conference Call Details:
Presentation host:
Anixa management, with special guest speakers
Date and time:
Today, December 6, 2023, at 6:30 p.m. ET
Phone access:
Registration Link to receive your dial-in number and unique PIN
Webcast:
Available at www.anixa.com under "Events & Presentations"
About Triple-Negative Breast Cancer
One in eight women in the U.S. will be diagnosed with an invasive breast cancer at some point in their lives. Approximately 10-15% of those diagnoses are TNBC, however TNBC accounts for a disproportionately higher percentage of breast cancer deaths and has a higher rate of recurrence. This form of breast cancer is twice as likely to occur in African-American women, and approximately 70% to 80% of the breast tumors that occur in women with mutations in the BRCA1 genes are triple-negative breast cancer.
About Anixa Bioscience's Breast Cancer Vaccine
Anixa's breast cancer vaccine takes advantage of endogenously produced proteins that have a function at certain times in life, but then become "retired" and disappear from the body. One such protein is a breast-specific lactation protein, a-lactalbumin, which is no longer found post-lactation in normal, aging tissues, but is present in the majority of triple-negative breast cancers. Activating the immune system against this "retired" protein provides preemptive immune protection against emerging breast tumors that express a-lactalbumin. The vaccine also contains an adjuvant that activates an innate immune response, which allows the immune system to mount a response against emerging tumors to prevent them from growing. This vaccine technology was invented by the late Dr. Vincent Tuohy, who was the Mort and Iris November Distinguished Chair in Innovative Breast Cancer Research in the Department of Inflammation and Immunity at Cleveland Clinic's Lerner Research Institute. Dr. Tuohy was inventor of the technology, which Cleveland Clinic exclusively licensed to Anixa Biosciences. He was entitled to a portion of the commercialization revenues received by Cleveland Clinic and also held equity in Anixa.
About Anixa Biosciences, Inc.
Anixa is a clinical-stage biotechnology company focused on the treatment and prevention of cancer. Anixa's therapeutic portfolio consists of an ovarian cancer immunotherapy program being developed in collaboration with Moffitt Cancer Center, which uses a novel type of CAR- T, known as chimeric endocrine receptor T-cell (CER-T) technology. The Company's vaccine portfolio includes a novel vaccine being developed in collaboration with Cleveland Clinic to prevent breast cancer – specifically triple negative breast cancer (TNBC), the most lethal form of the disease – as well as a vaccine to prevent ovarian cancer. These vaccine technologies focus on immunizing against "retired" proteins that have been found to be expressed in certain forms of cancer. Anixa's unique business model of partnering with world-renowned research institutions on clinical development allows the Company to continually examine emerging technologies in complementary fields for further development and commercialization. To learn more, visit www.anixa.com or follow Anixa on Twitter, LinkedIn, Facebook and YouTube.
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Anixa Biosciences - >>> An end to breast cancer? California company develops groundbreaking vaccine with promising future
CBS News
by Ashley Sharp
December 8, 2023
SACRAMENTO -- An estimated one in eight women will develop breast cancer in their lifetime, killing on average 42,000 women a year in the United States.
What if there was a vaccine that would significantly lower each woman's chance of ever getting it in the first place? In that question lies what could be the answer to one day eliminating the deadly disease.
A groundbreaking vaccine created through decades of research at the Cleveland Clinic and developed by Anixa Biosciences in San Jose, California is driving innovation by targeting triple-negative breast cancer, the disease's deadliest and most aggressive form.
"This vaccine could potentially eliminate breast cancer," said Dr. Amit Kumar, Anixa CEO.
The vaccine's findings from its first trial with 16 women were published Wednesday, with each participant reporting no bad side effects and no resurgence of their cancer so far.
Jennifer Davis, a brave woman from small-town Ohio, was the first woman in the world to get the vaccine in October 2021.
"This is how we advance medicine. It's important to be a part of those things," Davis said. "I am just beyond grateful."
When Davis heard the dreaded words "you have cancer" in September 2018, it came six months after her first alert to an abnormality on a routine mammogram and ultrasound.
At the time, her biopsy turned up negative for cancer.
"I really wanted to believe everything was OK, but I knew something wasn't right," Davis said.
At 41 years old, she had no history of cancer. Still, she could feel a lump growing and decided to go for a second opinion and a second biopsy.
She was diagnosed with triple-negative breast cancer. Her mind instantly went to her family and three children.
"It was very hard to tell them and try and be strong for them," Davis said. "With triple negative, there is nothing for us to take — no pill or anything to prevent recurrence. The rate is high and outcomes are poor if it does come back."
After chemotherapy, a double mastectomy and radiation, Davis was finally free of cancer, but she was not free of the fear that lingers.
"I was always nervous and afraid of it coming back," she said.
So when she learned of an experimental vaccine trial while receiving her cancer care at the Cleveland Clinic, she thought, "What do I have to lose?"
"It was something that was going to give me peace of mind," Davis said. "If this could work for me, then I wouldn't have to worry about a recurrence."
She became the first woman in the world to take the breast cancer vaccine.
A registered nurse herself, what eased her mind was the fact that in years of trials in animals, there had been no cancer recurrence and no anaphylactic reaction.
"That was all I needed to hear," said Davis, who reports that in the two years since taking the vaccine, she has never felt better.
The vaccine has been studied for more than two decades at the Cleveland Clinic, pioneered by pre-clinical research led by the late Dr. Vincent Tuohy.
Inspired by this and what it could mean for the future of cancer diagnosis, Dr. Kumar approached the clinic about developing the vaccine.
"I looked at it and I saw the vision," Kumar said.
So how does it work?
"Is it, in essence, teaching your body not to grow a tumor?" CBS13 reporter Ashley Sharp asked.
"That's exactly right. It's teaching your body to destroy the cells that can grow a tumor," Kumar said.
If a virus shows up in the body, the immune system teaches itself how to destroy it, knowing, easily, which cells are bad.
In cancer, it is more difficult, Dr. Kumar explained.
"All of the cells that become cancerous in your body came from normal, healthy cells," Kumar said. "The difference is not big, so the immune system has a harder time recognizing a cancer cell and distinguishing it from a healthy cell."
According to the Cleveland Clinic, the vaccine works by targeting a lactation protein called a-lactalbumin, which is no longer found after lactation in normal, aging tissues. It is, however, present in most triple-negative breast cancer patients. If breast cancer develops, the vaccine is designed to instruct the immune system to attack the tumor and keep it from growing entirely.
"The results are incredibly promising," Kumar said. "The vision is one day to be able to give this to any woman who wants to prevent cancer from ever occurring in her body. It's a small step and we have many more steps to go, but it's incredible if we can make this happen."
It's a promising find for the future of fighting cancer that started with one woman but hopefully ends with every woman.
"The bigger picture of this is overwhelming for me," Davis said.
The second vaccine trial is set to start in 2024, this time with 600 women instead of 16. This study will be on a much larger scale, where half the women will get the vaccine and the other half will get a placebo.
The hope is that within five years, they can get FDA approval to distribute the vaccine to the public.
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>>> Anixa Biosciences, Inc. (ANIX), a biotechnology company, develops therapies and vaccines focusing on critical unmet needs in oncology and infectious diseases. The company's therapeutics programs include the development of a chimeric endocrine receptor T-cell therapy, a novel form of chimeric antigen receptor T-cell (CAR-T) technology focusing on the treatment of ovarian cancer; and the development of anti-viral drug candidates for the treatment of COVID-19 focused on inhibiting certain protein functions of the virus. Its vaccine programs comprise the development of a vaccine against triple negative breast cancer; and a preventative vaccine against ovarian cancer. The company is also developing immuno-therapy drugs against cancer. The company was formerly known as ITUS Corporation and changed its name to Anixa Biosciences, Inc. in October 2018. Anixa Biosciences, Inc. was incorporated in 1982 and is based in San Jose, California.
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An Acurx interview tonight with Bloomberg TV (see below), so this could be part of the catalyst for today's jump in the stock.
As we know, the first batch of durability data should be released in Dec, per CEO Luci's previous guidance. Then the next batch (day 94 data) should be ~ Jan, and then the microbiome data. So a 3 stage release of data over the next few months to make things interesting :o)
I figure the 94 day durability data in Jan is the most important, with the Dec data being the lead up. If the Dec data is clearly positive the stock should zoom. If it's ambiguous, then we wait for the 94 day data in Jan, and the microbiome data. As a bio 'binary event' this is unusual, since a disappointment in Dec can be made moot by good news in Jan. So this de-risks the stock considerably. We already know that Ibex leaves the microbiome relatively intact, compared to Vancomycin, so Ibez's 'recurrence advantage' should increase over time, which makes for excellent odds with the 94 day data.
All IMHO :o)
>>> NEW YORK, Dec. 07, 2023 (GLOBE NEWSWIRE) -- FMW Media's New to The Street announces its corporate interview lineup for its business show airing as a sponsored program on Bloomberg TV tonight, Thursday, December 7, 2023, at 9:30 PM PT.
... 3) Biopharmaceutical - Acurx Pharmaceuticals, Inc.'s (NASDAQ: ACXP) ($ACXP) interview with David Luci, President/CEO.
https://finance.yahoo.com/news/street-tv-announces-episode-536-142000722.html
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Repost from Dew's Revance board -- >>> RVNC 3Q23 financial summary:
https://investorshub.advfn.com/boards/read_msg.aspx?message_id=173182593
https://www.businesswire.com/news/home/20231108710804/en
• 3Q23 Daxxify sales were $22.0M, -3% QoQ. (There is no meaningful YoY growth figure because Daxxify was not yet on the market during 3Q22.) Note: The slight QoQ decline during the seasonally slow third calendar quarter is effectively an increase in the projected annualized rate.
• 3Q23 dermal-filler sales were $32.1M, +1% QoQ and +23% YoY.
• 3Q23 non-GAAP operating expenses (excluding revenue and COGS) were $69.0. (RVNC’s guidance for full-year 2023 non-GAAP operating expenses is $315-335M.)
• Cash at 9/30/23 was $300.2M, a $19.5M decrease relative to 6/30/23. (RVNC raised $50M in Aug 2023 from the third tranche of the Athyrium Capital loan.)
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Repost from Dew's ENTA board -- >>> ENTA reports FY4Q23* results and pipeline update:
https://investorshub.advfn.com/boards/read_msg.aspx?message_id=173266700
https://ir.enanta.com/news-releases/news-release-details/enanta-pharmaceuticals-reports-financial-results-its-fiscal-37
FY4Q23 royalty revenue was $18.9M, unchanged from the prior quarter. 54.5% of ENTA’s Mavyret royalties are payable to OMERS†, and this amount is treated for accounting purposes as an amortization of debt (see discussion in #msg-172603887).
9/30/23 cash = $370M, a decrease of $23M relative to 6/30/23.
Today, ENTA issued FY2024* guidance as follows:
• R&D expense of $100-120M, down sharply from $163.5M in FY2023.
• SG&A expense of $45-50M, down modestly from $52M in FY2023.
Based on the reduced operating expenses described above, ENTA says its cash and expected royalty revenue are sufficient to fund operations for the next four years—i.e. through the end of FY2027.
Pipeline
• EDP-938 (RSV N-protein inhibitor): ENTA expects to report data from the phase-2b RSVPEDs (pediatric) and the phase-2b RSVHR (high-risk adults) trials in calendar 3Q23, assuming there is a normal RSV season in the Northern Hemisphere.
• EDP-323 (RSV L-protein inhibitor): ENTA just started a phase-2a “challenge” study; data expected in calendar 3Q23.
• The preclinical program for single-agent dual inhibition of RSV and hMPV has been terminated.
• EDP-235 (COVID protease inhibitor): If a partnership is secured for a phase-3 trial, the population tested will be standard-risk patients with a primary endpoint based on symptom relief.
• Non-virology: “The company will announce new therapeutic programs beginning in early 2024.” An analyst on today’s CC asked if one of these programs was GLP-1, but ENTA declined to answer.
*ENTA’s fiscal years end on September 30
†Ontario Municipal Employees Retirements System (Canada’s largest pension fund)
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Acurx -- a summary slide of the various CDI trial results of the various antibiotics (below). Two are Phase 3 results, and one is from a Phase 2. In the Phase 2 (33 patients), the Vancomycin initial cure rate was 70%. Compare that to Acurx's Phase 2, where Vancomycin had a 100% initial cure rate. So it demonstrates just how limited the validity of all data is from a small Phase 2 -
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>> MEIP <<
Looks like they decided to not pursue that Phase 3 program, after further talks with the FDA, though their partner will continue it in Japan. And the $41 mil in 'net income' turns out to be from "recognition of deferred revenue associated primarily with the termination of the Kyowa Kirin Commercialization Agreement in July 2023" (link below), so not real net income derived from actual product sales. The cash level is $81 mil, but looks like the burn rate is just under $20 mil per quarter, so they project having sufficient cash for approx 1 year. Clinical programs look like Phase 1 and 1b, so early stage. So I guess MEIP's low $39 mil market cap is appropriate, all things considered -
https://finance.yahoo.com/news/mei-pharma-reports-first-quarter-210500033.html
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Blade, Just curious if you are still following MEIP? I noticed that MEIP's chart setup looks interesting for a possible upside breakout move, but I haven't kept up with the company's fundamentals. I remember last year the FDA wouldn't allow accelerated approval for MEIP's cancer drug based on the Phase 2, and would require the full Phase 3 to be completed for approval. But just curious if you might still be following the company? Yahoo Finance has their cash level at $82 mil, and revenues of $105 mil (!) and net income of $41 mil (!), which if accurate could make MEIP the bargain of the century, considering the current market cap is only $37 mil.
Thanks for any insights, and hope you are doing well :o)
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>>> MEI Pharma, Inc. (MEIP), a clinical-stage pharmaceutical company, focuses on the development and commercialization of various therapies for the treatment of cancer. The company develops Zandelisib, an oral phosphatidylinositol 3-kinase delta inhibitor for the treatment of patients with relapsed/refractory follicular lymphoma; and Voruciclib, an oral cyclin-dependent kinase 9 inhibitor, which is in Phase I clinical trial for acute myeloid leukemia and B-cell malignancies. It also develops ME-344, a mitochondrial inhibitor targeting the oxidative phosphorylation complex which has completed Phase I clinical trial for the treatment of human epidermal growth factor receptor 2 negative breast cancer. MEI Pharma, Inc. has a license agreement with Presage Biosciences, Inc. The company was formerly known as Marshall Edwards, Inc. and changed its name to MEI Pharma, Inc. in July 2012. MEI Pharma, Inc. was incorporated in 2000 and is headquartered in San Diego, California.
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>>> Halozyme Therapeutics, Inc. (HALO), a biopharma technology platform company, researches, develops, and commercializes proprietary enzymes and devices in the United States, Switzerland, Ireland, Belgium, Japan, and internationally. The company's products are based on the patented recombinant human hyaluronidase enzyme (rHuPH20) that enables delivery of injectable biologics, such as monoclonal antibodies and other therapeutic molecules, as well as small molecules and fluids. It offers Hylenex recombinant, a formulation of rHuPH20 to facilitate subcutaneous (SC) fluid administration for achieving hydration to enhance the dispersion and absorption of other injected drugs in SC urography and to enhance resorption of radiopaque agents; XYOSTED, an injection for SC administration of testosterone replacement therapy; NOCDURNA, a sublingual tablet to treat nocturia due to nocturnal polyuria; TLANDO, an oral formulation for testosterone replacement therapy; and ATRS-1902, a proprietary drug device combination product. The company also provides Herceptin (trastuzumab), Herceptin Hylecta, and Phesgo to treat breast cancer; Mabthera SC for the treatment of multiple blood cancer; HYQVIA to treat primary immunodeficiency disorders; and DARZALEX for patients with amyloidosis, smoldering myeloma, and multiple myeloma. In addition, it offers Epinephrine Injection to treat allergy and immunology; Sumatriptan injection for migraines; exenatide and teriparatide injections; Makena, a progestin drug to reduce the risk of preterm birth; and OTREXUP, a SC methotrexate injection for adults with severe active rheumatoid arthritis and severe recalcitrant psoriasis, as well as children with active polyarticular juvenile idiopathic arthritis. Further, the company provides ATRS-1902 for adrenal crisis rescue; ARGX-113; and ARGX-117 to treat severe autoimmune diseases in multifocal motor neuropathy. Halozyme Therapeutics, Inc. was founded in 1998 and is headquartered in San Diego, California.
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>>> Corcept Therapeutics Incorporated (CORT) engages in discovery and development of drugs for the treatment of severe metabolic, oncologic, endocrine, and neurological disorders in the United States. It offers Korlym (mifepristone) tablets as a once-daily oral medication for the treatment of hyperglycemia secondary to hypercortisolism in adult patients with endogenous Cushing's syndrome, who have type 2 diabetes mellitus or glucose intolerance, and have failed surgery or are not candidates for surgery. The company is developing relacorilant to treat patients with Cushing's syndrome; and nab-paclitaxel in combination with relacorilant, which has completed Phase II clinical trial to treat patients with advanced ovarian tumors, as well as for the treatment of cortisol excess. It is also developing selective cortisol modulator to treat patients with metastatic castration-resistant prostate cancer; selective cortisol modulator for the treatment of antipsychotic-induced weight gain and other disorders; and FKBP5 gene expression assays. The company was incorporated in 1998 and is headquartered in Menlo Park, California.
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