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Blade, Phenomenal move, and I see that analysts have price targets of $10 and $20, so could be lots more to go :o)
>>> Why Shares of Gracell Biotechnologies Jumped Tuesday
Motley Fool
By Jim Halley
Jun 13, 2023
https://www.fool.com/investing/2023/06/13/why-shares-of-gracell-biotechnologies-jumped-tuesd/?source=eptyholnk0000202&utm_source=yahoo-host&utm_medium=feed&utm_campaign=article
KEY POINTS
Two analysts upgraded their ratings for Gracell Biotechnologies' stock.
Over the weekend, Gracell announced positive follow-up trial data regarding its lead therapy.
The clinical-stage biotech has no revenue and cash of $186 million, as of the first quarter.
Gracell climbed higher for a second consecutive day.
What happened
Shares of Gracell Biotechnologies (GRCL 28.06%) were up 21.5% Tuesday afternoon. The healthcare stock rose for the second consecutive day, boosted by an analyst's upgrade, as well as by positive trial news. The stock is up more than 156% so far this year.
So what
Gracell is a clinical-stage biotech company that makes cell therapies to treat cancers and autoimmune diseases. Over the weekend, the company announced positive long-term follow-up trial data regarding its lead therapy, GC012F, to treat patients with relapsed/refractory B-cell non-Hodgkin's lymphoma. The CAR-T therapy is a CD19 and B-cell maturation antigen.
In the study, all nine patients showed an overall response rate nine months after the therapy, with 77.8% having a complete response (CR) rate at three months and 66.7% having a CR at six months. The study comes on the heels of an announcement on June 3 that said GC012F did well in long-term follow-ups as a therapy to treat relapsed/refractory multiple myeloma.
In likely responses to the trial data, BTIG analyst Justin Zelin reiterated his buy rating on the stock with a price target of $20, and analyst Joseph Catanzaro from Piper Sandler maintained his buy rating on Gracell, with a price target of $10.
Now what
The company's pipeline has 12 programs, including six potential indications for GC012F. That the therapy has shown a strong safety profile, as well as long-term efficacy, is a big deal, but it is still in early-stage trials.
It's also worth noting that the small patient population size -- only nine participants -- means it may be difficult replicating GC012F's results in a larger relapsed/refractory B-cell non-Hodgkin's lymphoma patient group.
The company had, as of the first quarter, $186 million in cash, enough to probably finance operations into 2026.
<<<
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GRCL sets new 52-week high at $6.37 obliterating its previous high of $5.94. Up almost 30%.
Bladerunner
GRCL up 25% today hitting it's 52-week high. Pulling back a bit as I write this.
Blade
Blade, Thanks. Looks great, and the stock has been flying :o) I see the company also has 3 proprietary platform technologies, so a good sign. Hopefully US-China relations don't deteriorate too far, but either way the ex-US market for GRCL should be plenty big.
Btw, looks like MRSN is also having a nice bounce, and JSPR is hanging in there after its big move :o)
---
GRCL present updated results at EHA. These rsults are outstanding, albeit in a small sample size.
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GlobeNewswire
Gracell Biotechnologies Presents Longer-Term Results for FasTCAR-T GC012F in B-Cell Non-Hodgkin’s Lymphoma at EHA2023, Highlighting 100% Overall Response Rate
Gracell Biotechnologies Inc.
Sat, June 10, 2023 at 7:30 AM PDT·7 min read
In this article:
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Gracell Biotechnologies Inc.
Gracell Biotechnologies Inc.
Data on CD19/BCMA dual-targeting FasTCAR-T GC012F showed 100% overall response rate (ORR) and 66.7% 6-month complete response (CR) rate among treated patients, all with diffuse large B-cell lymphoma (DLBCL) subtype
Data on GC012F for treatment of relapsed/refractory multiple myeloma (RRMM) and donor-derived CAR-T GC007g for treatment of relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) have been presented as posters on June 9 at EHA2023
SAN DIEGO, Calif., and SUZHOU and SHANGHAI, China, June 10, 2023 (GLOBE NEWSWIRE) -- Gracell Biotechnologies Inc. ("Gracell" or the "Company", NASDAQ: GRCL), a global clinical-stage biopharmaceutical company dedicated to developing innovative and highly efficacious cell therapies for the treatment of cancer and autoimmune disease, today presented longer-term follow-up data from a first-in-human study evaluating GC012F, a CD19 and B-cell maturation antigen (BCMA) dual-targeted autologous CAR-T therapeutic candidate, in patients with relapsed/refractory B-cell non-Hodgkin’s Lymphoma (r/r B-NHL) as an oral presentation (abstract #S234) at the European Hematology Association (EHA2023) Congress.
While CD19-directed CAR-T cell therapy has been demonstrated to be a valuable treatment option for r/r B-NHL, other studies have identified that 39% to 97% of clinical B-NHL samples express BCMA as well.1,2,3 To further improve safety and efficacy of NHL treatment, Gracell is exploring the clinical potential of GC012F, a CD19 and BCMA dual-targeting CAR-T cell therapy, for treatment of r/r B-NHL. GC012F is manufactured through a novel next-day FasTCAR process and demonstrated a younger phenotype of CAR-T cells and highly effective tumor killing activity in preclinical animal models.
In the single-arm, open label investigator-initiated trial (IIT), nine r/r B-NHL patients were enrolled and treated with GC012F, and completed at least three months of follow-up. Doses range between 3.7x104 to 3x105 CAR-T cells/kg. All nine patients are classified as relapsed/refractory DLBCL. All patients’ lymphoma samples expressed CD19, and samples from seven out of eight tested patients expressed BCMA.
As of the April 12, 2023 data cutoff date, with a median follow-up of 293 days (range: 131-546 days), patients treated with GC012F achieved a high response rate and outstanding durability of response:
100% (9/9) overall response rate (ORR) at 3 months among nine patients with r/r DLBCL;
77.8% (7/9) complete response (CR) rate at 3 months;
66.7% (6/9) CR rate at 6 months;
GC012F CAR-T cells were detectable in tumor biopsies from all tested patients, indicating the infiltration of CAR-T cells into the tumor lesions.
GC012F also continued to show a favorable safety profile:
Cytokine release syndrome (CRS) was mostly Grade 1 (56%; 5/9). Grade 3 CRS was observed in one patient (duration of 2 days) with quick recovery after standard of care treatment. No Grade 4/5 CRS events occurred;
No neurotoxicity or immune effector cell-associated toxicity (ICANS) of any grade were observed.
“One year after we reported initial data from the IIT evaluating FasTCAR-T GC012F in B-NHL at EHA2022, we are proud to be back at EHA presenting longer-term results from more patients as an oral presentation,” said Dr. Wendy Li, Chief Medical Officer of Gracell. “Durable responses, enhanced safety, and timely access to cell therapy remain significant unmet needs for NHL patients. With a 100% ORR at 3 months and CR rates of 78% at 3 months and 67% at 6 months, particularly among DLBCL patients, the updated data further support the clinical potential and wide applicability of GC012F, and the benefits of a CD19/BCMA dual-targeting approach combined with FasTCAR next-day manufacturing.”
Gracell is also evaluating GC012F in RRMM, newly-diagnosed multiple myeloma (NDMM), and systemic lupus erythematosus (SLE).
On June 9, Gracell also presented the following during poster sessions:
First results from a Phase 1 study of the donor-derived allogeneic CAR-T GC007g (abstract #P369), showing 100% ORR and a favorable safety profile for treatment of r/r B-ALL;
Updated results from the IIT evaluating GC012F for the treatment of RRMM (abstract #P869), which were also presented as an oral presentation at the 2023 ASCO Annual Meeting. The data demonstrated 100% minimal residual disease (MRD) negativity and 82.8% MRD negative stringent complete response (sCR) in a predominantly high-risk RRMM population.
Additional information about the presentations and the EHA2023 Hybrid Congress is available on the EHA website.
[1] Blood Cancer Journal (2020); 10:73.
[2] Blood (2017); 130:2755.
[3] Hum Gene Ther (2018); 29(5): 585.
About GC012F
GC012F is Gracell's FasTCAR-enabled BCMA/CD19 dual-targeting autologous CAR-T cell therapy, which aims to transform cancer and autoimmune disease treatment by driving fast, deep and durable responses with improved safety profile. GC102F is currently being evaluated in investigator-initiated trials in multiple hematological cancers as well as autoimmune disease, and has demonstrated a consistently strong efficacy and safety profile. In February 2023, Gracell announced regulatory clearance of Investigational New Drug applications in the United States and China to commence clinical trials evaluating GC012F for the treatment of relapsed/refractory multiple myeloma. Gracell has also initiated an investigator-initiated trial evaluating GC012F for the treatment of SLE.
About FasTCAR
Introduced in 2017, FasTCAR is Gracell's revolutionary next-day autologous CAR-T cell manufacturing platform. FasTCAR is designed to lead the next generation of therapy for cancer and autoimmune diseases, and improve outcomes for patients by enhancing effect, reducing costs, and enabling more patients to access critical CAR-T treatment. FasTCAR drastically shortens cell production from weeks to overnight, potentially reducing patient wait times and probability for their disease to progress. Furthermore, FasTCAR T-cells appear younger than traditional CAR-T cells, making them more proliferative and effective at killing cancer cells. In November 2022, FasTCAR was named the winner of the Biotech Innovation category of the 2022 Fierce Life Sciences Innovation Awards for its ability to address major industry obstacles.
About Gracell
Gracell Biotechnologies Inc. ("Gracell") is a global clinical-stage biopharmaceutical company dedicated to discovering and developing breakthrough cell therapies. Leveraging its pioneering FasTCAR and TruUCAR technology platforms and SMART CAR™ technology module, Gracell is developing a rich clinical-stage pipeline of multiple autologous and allogeneic product candidates with the potential to overcome major industry challenges that persist with conventional CAR-T therapies, including lengthy manufacturing time, suboptimal cell quality, high therapy cost, and lack of effective CAR-T therapies for solid tumors and autoimmune disease. For more information on Gracell, please visit www.gracellbio.com. Follow @GracellBio on LinkedIn.
Cautionary Noted Regarding Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the expected trading commencement and closing date of the offering. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including factors discussed in the section entitled "Risk Factors" in Gracell's most recent annual report on Form 20-F as well as discussions of potential risks, uncertainties, and other important factors in Gracell's subsequent filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Gracell specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Readers should not rely upon the information on this page as current or accurate after its publication date.
CONTACT: Media contacts Marvin Tang marvin.tang@gracellbio.com Jessica Laub jessica.laub@westwicke.com Investor contacts Gracie Tong gracie.tong@gracellbio.com Stephanie Carrington stephanie.carrington@westwicke.com
Bladerunner
>>> Acurx Announces Ibezapolstat Scientific Poster and Update on its Pol IIIC Pipeline Presented at ECCMID 2023 Scientific Conference
PR Newswire
Apr 19, 2023
https://finance.yahoo.com/news/acurx-announces-ibezapolstat-scientific-poster-110100500.html
A scientific poster highlighting a novel pharmacologic property of oral ibezapolstat for C. difficile Infection likely related to its unique mechanism of action was presented at ECCMID 2023
An update on the Company's pre-clinical antibiotic program in Lead Optimization stage for systemic gram-positive bacterial infections was also presented
Ibezapolstat has previously received FDA QIDP and Fast-Track Designation
STATEN ISLAND, N.Y., April 19, 2023 /PRNewswire/ -- Acurx Pharmaceuticals, Inc. (NASDAQ: ACXP) ("Acurx" or the "Company"), a clinical stage biopharmaceutical company developing a new class of antibiotics for difficult-to-treat bacterial infections, today announced two presentations were given at the 33rd Annual European Congress of Clinical Microbiology and Infectious Disease (ECCMID) in Copenhagen. The scientific poster entitled "Novel Pharmacology and Susceptibility of Ibezapolstat Against C. difficile Isolates with Reduced Susceptibility to C. difficile-directed Antibiotics" was co-presented on April 17 by Dr. Kevin Garey, Professor and Chair, University of Houston College of Pharmacy, and the Principal Investigator for microbiome aspects of our ibezapolstat clinical trial program, and by Dr. Eugénie Bassères, Research Scientist Faculty at the University of Houston. According to Dr. Garey: "Our work is based on the hypothesis that ibezapolstat's mechanism of action is not only bactericidal to C. difficile but also could inhibit some of its virulence mechanisms. C. difficile strains with reduced susceptibility to metronidazole, vancomycin, or fidaxomicin were susceptible to ibezapolstat, confirming its unique mechanism of action. To study anti-virulence effect, our group investigated an under-studied virulence property of C difficile, namely, flagellar movement of the organism. Using sub-MIC concentrations of ibezapolstat, we demonstrated decreased movement of C. difficile through a semi-solid agar in concert with reduced expression of the primary genes used to synthesize flagella. All of these positive and unexpected findings reflect the unique mode of action in inhibiting DNA pol IIIC and support the continued development of ibezapolstat to treat C. difficile infection."
Also, Acurx Executive Chairman, Robert J. DeLuccia, presented an update on April 15 on the Company's preclinical, systemic oral and IV program for treatment of other gram-positive infections caused by MRSA, VRE and DRSP at the "Pipeline Corner" featured session at ECCMID, organized by Dr. Ursula Theuretzbacher, a world-renowned microbiology expert involved in antibacterial drug research, discovery and development strategies and policies for clinical and public health needs. Mr. DeLuccia summarized the progress of the company's GPSS™ (Gram Positive Selective Spectrum) program stating: "Our potential lead compound meets Dr. Theurezbacher's criteria for innovation in that it is a new chemical class, has novel mechanism and bacterial target, and has not shown cross-resistance in early in vitro microbiology studies. He further stated: "Following clinical validation of the pol IIIC bacterial target in a Ph2a proof-of-principal trial showing 100% cure of C. difficile Infection, with no recurrence after 30 days' follow up, we have made substantial progress toward lead compound selection of our pan-active, gram-positive IV and oral compounds. We've made significant improvements in cytotoxicity, solubility and protein binding, in vitro and in vivo safety and have demonstrated oral and IV efficacy in a number of mouse infection models."
The poster and presentation are available on the Company's website www.acurxpharma.com.
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.
The Company currently is enrolling patients in a Ph2b clinical trial of ibezapolstat to treat patients with C. difficile infection (CDI). The Company successfully completed Phase 1 and Phase 2a clinical trials of ibezapolstat. The Phase 2a trial demonstrated 100% clinical cure and 100% sustained clinical cure in patients with CDI, along with beneficial microbiome changes during treatment including overgrowth of Actinobacteria and Firmicutes phylum species while on therapy and new findings which demonstrate potentially beneficial effects on bile acid metabolism. The Ph2b clinical trial is designed to enroll 64 patients and is a randomized (1:1), non-inferiority, double-blind trial of oral ibezapolstat compared to oral vancomycin, a standard of care to treat CDI.
The FDA has accepted the Company's plan to have an Independent Data Monitoring Committee (IDMC) conduct an interim review of clinical outcome from the ongoing Ph2b clinical trial of patients with C. difficile Infection (CDI). The interim review will be conducted upon reaching enrollment of 36 patients in total. FDA's acceptance was based on the Company's filing of a protocol amendment to its Investigational New Drug Application (IND) with FDA in January 2023. The Company's filing and intention for the IDMC to conduct an interim review of data was based on the observed blinded data to date from the ongoing Ph2b clinical trial at that time. Upon conducting the interim review, the IDMC will determine and recommend to the Company whether the most appropriate course of action is to terminate the Ph2b clinical trial early due to success, as the Company had done with the Ph2a clinical trial, or to continue patient enrollment. The Company intends to report available data promptly after the IDMC conducts this interim review. The IDMC initial organizational meeting was conducted in March 2023 and it has completed all organizational matters required to ensure readiness for data review.
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 ECCMID
ECCMID (European Congress of Clinical Microbiology and Infectious Diseases) is recognized as the largest international forum for presentations and discussions of research in the fields of clinical microbiology and infection for experts from academia, the clinical setting and the industry. ESCMID's (European Society of Clinical Microbiology and Infectious Diseases) yearly congress attracts over 14,000 participants. ECCMID offers a wide range of sessions including: keynotes, symposia, poster sessions, educational workshops, meet-the-expert sessions and more. The society's executive power is vested in ESCMID in Executive Committee elected by the ESCMID members. The administrative ESCMID office is in Basel, Switzerland.
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 of two of the three 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 Clostridioides 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 the Ibezapolstat Phase 2 Clinical Trial
The multicenter, open-label single-arm segment of this study (Phase 2a) is to be followed by a double- blind, randomized, active-controlled segment (Phase 2b) which, together, comprise the Phase 2 clinical trial. The Phase 2 clinical trial is designed to evaluate ibezapolstat in the treatment of CDI. Phase 2a of this trial is completed and 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, the Trial Oversight Committee assessed the safety and tolerability and made its recommendation regarding early termination of the Phase 2a study. Based on the recommendation of Acurx's Scientific Advisory Board (SAB) and Trial Oversight Committee, we terminated enrollment in Phase 2a early and are now advancing to Phase 2b. The SAB unanimously supported the early termination of the Phase 2a trial after 10 patients were enrolled in the trial instead of 20 patients as originally planned. The early termination was based on the evidence of meeting the primary and secondary endpoints of eliminating the infection (100%), with no recurrences of infection (100%), and with an acceptable adverse event profile. In the upcoming Phase 2b, approximately 64 additional patients with CDI will be enrolled and randomized in a 1:1 ratio to either ibezapolstat 450 mg every 12 hours or vancomycin 125mg 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 will be identical in appearance, dosing times, and number of capsules administered to maintain the blind. This Phase 2 clinical trial also will evaluate pharmacokinetics (PK) and microbiome changes and continue to 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.
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 that target the DNA polymerase IIIC enzyme and 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).
To learn more about Acurx Pharmaceuticals and its product pipeline, please visit www.acurxpharma.com.
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>>> Gracell Biotechnologies Reports First Quarter 2023 Unaudited Financial Results, and Provides Corporate Update
Gracell Biotechnologies Inc.
May 15, 2023
https://www.globenewswire.com/news-release/2023/05/15/2668595/0/en/Gracell-Biotechnologies-Reports-First-Quarter-2023-Unaudited-Financial-Results-and-Provides-Corporate-Update.html
Presenting longer-term follow-up data from investigator-initiated trial (IIT) evaluating BCMA/CD19 dual-targeting FasTCAR-T GC012F in relapsed/refractory multiple myeloma (RRMM) at 2023 ASCO and EHA
On track to commence a Phase 1b/2 clinical trial in U.S. and a Phase 1/2 clinical trial in China evaluating GC012F for the treatment of RRMM in the second quarter and third quarter of 2023, respectively
Launched the new IIT evaluating GC012F in systemic lupus erythematosus (SLE)
Presenting updated data from the ongoing IIT evaluating GC012F in relapsed/refractory B-cell non-Hodgkin lymphoma (r/r B-NHL) at 2023 ASCO and EHA2023
Presenting the Phase 1 clinical data of donor-derived allogeneic CAR-T GC007g in relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) at EHA2023
Hosting KOL call on May 22 at 9:00 am ET to highlight unmet clinical need for RRMM in U.S.
Well-funded with cash runway to the end of 2024
Management to host conference call at 8:00 a.m. ET today
SAN DIEGO and SUZHOU, China and SHANGHAI, China, May 15, 2023 (GLOBE NEWSWIRE) -- Gracell Biotechnologies Inc. (NASDAQ: GRCL) (“Gracell” or the “Company”), a global clinical-stage biopharmaceutical company dedicated to discovering and developing highly efficacious and affordable cell therapies for the treatment of cancer and autoimmune diseases, today reported first quarter unaudited financial results for the period ended March 31, 2023, and provided corporate updates.
“We continue to focus our efforts on the advancement of our lead candidate GC012F, a FasTCAR-enabled autologous CAR-T therapy candidate dual-targeting B cell maturation antigen (BCMA) and CD19. We look forward to showcasing the updated clinical data of GC012F in two indications RRMM and r/r B-NHL at both 2023 ASCO and EHA2023, including in two oral presentations. We are on track to initiate the U.S. IND trial for GC012F in RRMM in the second quarter of 2023,” said Dr. William (Wei) Cao, founder, Chairman and CEO of Gracell. “We are also pleased to present the first clinical data from the Phase 1/2 clinical trial evaluating the donor-derived allogeneic CAR-T GC007g for the treatment of r/r B-ALL at EHA2023, demonstrating a 100% minimal residual disease negative complete response or complete response with incomplete count recovery (MRD- CR/CRi) in a challenging set of patients.”
Dr. Cao continued, “We are excited to announce our strategic decision to pursue clinical development in systemic lupus erythematosus (SLE) for GC012F. We believe the FasTCAR-T GC012F is well positioned as an ideal candidate for a wide range of autoimmune indications, given its CD19/BCMA dual-targeting capability, consistently favorable safety profile demonstrated in the over 50 patients treated across three IITs, and the proprietary FasTCAR manufacturing technology. We recently commenced an IIT in China in refractory SLE patients and plan to file IND in U.S. and China subsequently.”
Pipeline Updates
FasTCAR Platform: Next-day manufacturing for autologous CAR-T cell therapy with enhanced cell fitness.
GC012F: FasTCAR-enabled autologous CAR-T therapy candidate dual-targeting BCMA and CD19, currently being evaluated for the treatment of RRMM, newly-diagnosed multiple myeloma (NDMM) and B-NHL.
Longer term follow-up from a multicenter IIT evaluating GC012F for the treatment of RRMM in heavily pretreated, mostly high-risk patients will be presented as oral presentation at 2023 American Society of Clinical Oncology Annual Meeting (2023 ASCO) and as poster presentation at European Hematology Association 2023 Congress (EHA2023).
Company-sponsored Phase 1b/2 clinical trial in U.S. (NCT05850234) evaluating GC012F in RRMM on track to initiate in the second quarter of 2023.
Company-sponsored Phase 1/2 clinical trial in China evaluating GC012F in RRMM expected to initiate in the third quarter of 2023.
Follow-up continued in the ongoing IIT evaluating GC012F in NDMM.
The first clinical data presented at ASH 2022 demonstrated that one single infusion of GC012F achieved a 100% overall response rate (ORR) and 100% minimal residual disease (MRD) negativity in 16 newly-diagnosed, high risk, transplant eligible patients across all dose levels. 75% of the treated patients did not experience any cytokine release syndrome.
On track to share updated clinical data in 2023.
Enrollment and follow-up continued in the IIT in China evaluating GC012F in r/r B-NHL.
Updated data to be presented as oral presentation at EHA2023 and as poster presentation at 2023 ASCO.
Initiated a new IIT evaluating GC012F in refractory SLE.
GC007g for the treatment of B-ALL: Allogeneic CD19-targeted CAR-T cell therapy, derived from human leukocyte antigen (HLA) matched donor, for the treatment of r/r B-ALL patients who failed transplant and may not be eligible for autologous CAR-T therapy.
Phase 1 data to be presented at EHA2023 in a poster presentation.
Between March 2021 and May 2022, nine r/r B-ALL patients were enrolled and treated in the Phase 1 portion of the registrational Phase 1/2 clinical trial at two different dose levels.
At day 28 after infusion, 100% patients achieved MRD- CR/CRi.
With a median follow-up duration of 445 days (range 218-649 days), seven of nine (78%) patients remained in CR/CRi.
Phase 2 portion of the registration Phase 1/2 clinical trial in China is ongoing.
TruUCAR Platform: Novel designs enabling “off-the-shelf” allogeneic CAR-T therapy.
GC502: CD19/CD7 dual-directed allogeneic CAR-T cell therapy candidate being studied in a Phase 1 IIT in China for the treatment of B-cell malignancies. GC502 is manufactured from T cells of non-HLA-matched healthy donors.
Data from an IIT evaluating GC502 in r/r B-ALL patients were presented at EHA2022. Three of four patients achieved MRD- CR/CRi at day 28 post-infusion.
SMART CARTTM Technology Module: With unique construct to take advantage of the suppressive tumor microenvironment (TME) and effectively combat solid tumors, SMART CARTTM is designed to enhance CAR-T cell proliferation and duration of killing, and to resist exhaustion with improved persistence of CAR-T cells.
On track to commence a China IIT for GC506 in Claudin18.2-positive solid tumors in the second quarter of 2023.
Financial Results for the First Quarter Ended March 31, 2023
As of March 31, 2023, the Company had RMB1,277.3 million (US$186.0 million) in cash and cash equivalents and short-term investments. In addition, the Company had short-term borrowings and current portion of long-term borrowings of RMB122.4 million (US$17.8 million) and long-term borrowings of RMB41.7 million (US$6.1 million).
Net loss attributable to ordinary shareholders for the three months ended March 31, 2023 was RMB151.7 million (US$22.1 million), compared to RMB158.6 million for the corresponding prior year period.
Research and Development Expenses
Research and development expenses for the three months ended March 31, 2023 were RMB137.5 million (US$20.0 million), compared to RMB121.8 million in the corresponding prior year period. The increase was primarily due to the increased spending on research, development, and clinical trials, including license expenses with Seagen Inc.
Administrative Expenses
Administrative expenses for the three months ended March 31, 2023 were RMB29.1 million (US$4.2 million), compared to RMB37.9 million for the corresponding prior year period. The decrease was primarily driven by a decrease in professional service as well as a decrease in share-based compensation expenses.
Interest income for the three months ended March 31, 2023 was RMB14.6 million (US$2.1 million), compared to RMB2.5 million for the corresponding prior year period. Interest expense for the three months ended March 31, 2023 was RMB1.7 million (US$0.2 million), compared to RMB1.4 million for the corresponding prior year period.
As of March 31, 2023, 338,573,189 ordinary shares (excluding 24,817,479 ordinary shares issued to depositary bank as of March 31, 2023, for bulk issuance of American depository shares (ADSs) reserved for future issuances upon the exercise or vesting of awards granted under our share incentive plans), par value of US$0.0001 per share, were issued and outstanding. As of March 31, 2023, 18,927,261 options were granted and 15,049,943 options were outstanding, and 1,974,391 restricted share units (“RSUs”) were granted under our employee stock option plan. Each of our ADS represents five ordinary shares.
Conference Call and Webcast Details:
Monday, May 15, 2023 @ 8:00am ET
Investor domestic dial-in: (800) 715-9871
Investor international dial-in: (646) 307-1963
Conference ID: 2756776
Live webcast link: https://ir.gracellbio.com/news-events/events-and-presentations
A replay of the webcast will be available on ir.gracellbio.com shortly after the conclusion of the event for 90 days.
About FasTCAR
Introduced in 2017, FasTCAR is Gracell’s revolutionary next-day autologous CAR-T cell manufacturing platform. FasTCAR is designed to lead the next generation of cancer and autoimmune disease therapy and improve outcomes for patients by enhancing efficacy, reducing costs, and enabling more patients to access critical CAR-T treatment. FasTCAR drastically shortens cell production from weeks to overnight, potentially reducing patient wait times and probability for their disease to progress. Furthermore, FasTCAR T-cells appear younger and are more robust than traditional CAR-T cells, making them more proliferative and effective at killing cancer cells. In November 2022, FasTCAR was named the winner of the Biotech Innovation category of the 2022 Fierce Life Sciences Innovation Awards for its ability to address major industry obstacles.
About TruUCAR
TruUCAR is Gracell’s proprietary technology platform and is designed to generate high-quality allogeneic CAR-T cell therapies that can be administered “off-the-shelf” at lower cost and with greater convenience. With differentiated design enabled by gene editing of unique genes, TruUCAR is designed to control host vs graft rejection (HvG) as well as graft vs host disease (GvHD) without the need of being co-administered with additional immunosuppressive drugs.
About SMART CARTTM
SMART CARTTM is Gracell’s proprietary technology module designed to strengthen the functionality of CAR-T cells further, and aims to overcome tumor microenvironment (TME). SMART CARTTM includes altered expression of the receptor and signaling mechanism of an inhibitory TME molecule to enhance expansion and persistence and to reduce the exhaustion of CAR T cells. This design reverses and turns immunosuppressive signals of TME into stimulatory reactions of CAR-T cells. SMART CARTTM technology can be applied to many targets for the treatment of solid tumors.
About Gracell
Gracell Biotechnologies Inc. (“Gracell”) is a global clinical-stage biopharmaceutical company dedicated to discovering and developing breakthrough cell therapies. Leveraging its pioneering FasTCAR and TruUCAR technology platforms and SMART CARTTM technology module, Gracell is developing a rich clinical-stage pipeline of multiple autologous and allogeneic product candidates with the potential to overcome major industry challenges that persist with conventional CAR-T therapies, including lengthy manufacturing time, suboptimal cell quality, high therapy cost and lack of effective CAR-T therapies for solid tumors. For more information on Gracell, please visit www.gracellbio.com and follow @GracellBio on LinkedIn.
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GRCL reports earning on the 15th. IOVA should know by the end of May whether the FDA has accepted their BLA for review. That will be a big deal for IOVA. IOVA is also hoping for accelerated approval.
Blade
Blade, Thanks, I'll check them out :o)
I see GRCL is a Chinese company, early stage, but looks like they have almost 1.5 Bil in cash (per Yahoo) which is unusual. Btw, nice to see IMGN having success :o)
One fairly conservative one I found is Simulations Plus (SLP). Not a biotech per se, but their technology is used by biopharma companies to accelerate development. A nice 10 year chart, and I'm hoping it resumes its upward trajectory -
>>> Simulations Plus (SLP) is a premier developer of groundbreaking drug discovery and development simulation software, which is licensed and used for drug research by major pharmaceutical and biotechnology companies worldwide. <<<
https://investorshub.advfn.com/boards/read_msg.aspx?message_id=171750833
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Take a look at: CRDF, JSPR, IOVA, MRSN, GRCL
Bladerunner
Dew - Apellis Pharma - >>> APLS’ Syfovre has_impressive_first_month—contrary to my skepticism about the drug’s efficacy:
https://www.globenewswire.com/news-release/2023/05/04/2662038/0/en/Apellis-Pharmaceuticals-Reports-First-Quarter-2023-Financial-Results.html
APLS sold $18.4M of Syfovre during March (the commercial launch was 3/1/23). Some of this may be a stocking bolus by medical practices.
https://investorshub.advfn.com/boards/read_msg.aspx?message_id=171843665
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>>> Apellis Pharmaceuticals, Inc. (APLS), a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapeutic compounds through the inhibition of the complement system for autoimmune and inflammatory diseases.
It offers EMPAVELI for the treatment of paroxysmal nocturnal hemoglobinuria, cold agglutinin disease, C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis, amyotrophic lateral sclerosis, and hematopoietic stem cell transplantation-associated thrombotic microangiopathy; and -
SYFOVRE for treating geographic atrophy (GA). The company also develops -
APL-2006, a bispecific C3 and VEGF inhibitor for treating wet age-related macular degeneration and GA;
APL-1030, a C3 inhibitor for the treatment of various neurodegenerative diseases;
and the combination of EMPAVELI and a small interfering RNA for reducing the production of C3 proteins by the liver.
It has a collaboration and license agreement with Swedish Orphan Biovitrum AB (publ) for development and commercialization of pegcetacoplan; and a collaboration with Beam Therapeutics Inc. focused on the use of Beam's base editing technology to discover new treatments for complement-driven diseases. The company was incorporated in 2009 and is based in Waltham, Massachusetts.
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https://finance.yahoo.com/quote/APLS/profile?p=APLS
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>>> Apellis Pharma's Newly Approved Drug Exceeds Sales Projections, Bullish Analyst Raises Expectations
Benzinga
Vandana Singh
May 5, 2023
https://finance.yahoo.com/news/apellis-pharmas-newly-approved-drug-195711302.html
Apellis Pharmaceuticals Inc (NASDAQ: APLS) reported Q1 revenues of $44.8 million, surpassing the consensus of $26.10 million and $14.38 million a year ago.
The company reported an EPS loss of $(1.56), higher than $(1.42) a year ago and missing the consensus of $(1.46).
Needham writes that Syfovre's sales of $18.4 million were well above their estimate of $5 million and the Street's $1.5 million estimates. The analyst has raised the price target to $110 from $80, with a Buy rating.
The management has tempered expectations for 2023 sales given the relatively short launch time and noted that demand and sales might be lumpy in 2023.
In February, the FDA approved Apellis' Syfovre (pegcetacoplan) for geographic atrophy (GA) secondary to age-related macular degeneration. Syfovre is the first and only FDA-approved treatment for GA.
The analyst has raised the 2023 Syfovre sales estimate to $113 million from $100 million, and it anticipates the pre-1Q23 Street 2023 estimate of $61.5 million to go up after the strong 1Q23 showing.
Empaveli sales of $20.4 million exceeded Needham's $22.0 million estimate.
Empaveli self-injector PDUFA was initially set for March 15, 2023, but FDA has extended its review of the sNDA for an undetermined amount of time. The company is awaiting further detail on timing.
Price Action: APLS shares are up 10.63% at $93.29 on the last check Friday.
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>>> Eli Lilly Surges On Promising Data From Late-Stage Alzheimer's Drug Trial
"This is the first Phase 3 trial of any investigational medicine for Alzheimer's disease to deliver 35% slowing of clinical and functional decline," Eli Lilly said.
The Street
by MARTIN BACCARDAX
MAY 3, 2023
https://www.thestreet.com/investing/stocks/eli-lilly-surges-on-promising-data-from-alzheimers-drug-trial
Eli Lilly (LLY) - shares moved firmly higher Wednesday after the drugmaker posted promising data from a late-stage trial of its developing Alzheimer's treatment.
Eli Lilly said the treatment, known as donanemab, slowed the progression of Alzheimer's in Phase 3 trial patients by 35%, when compared to placebo, with a 40% decline on the "ability to perform activities of daily living."
The results improve on Lilly's Phase 2 trial, which was published in late January, and suggest donanemab -- which targets amyloid plaque -- proteins found in spaces between nerve cells that can disrupt communication to the brain -- could find favor with the U.S. Food & Drug Administration.
The FDA rejected Eli Lilly's request for an accelerated review of donanemab earlier this year, citing "the limited number of patients with at least 12 months of drug exposure data provided in the submission." Eli Lilly said the speed at which donanemab reduced amyloid plaque meant that some patients left the Phase 2 study, known as Trailblazer, after six months, reducing its overall same size. (?)
Alzheimer's disease is a progressive brain disorder that affects more than 50 million people around the world. To date, no drug has been found to address the disease, which can accelerate into dementia and other more serious cognitive conditions.
"Over the last 20 years, Lilly scientists have blazed new trails in the fight against Alzheimer's disease by elucidating basic mechanisms of AD pathology and discovering imaging and blood biomarker tools to track the pathology," said Daniel Skovronsky Lilly's chief scientific and medical officer.
"We are extremely pleased that donanemab yielded positive clinical results with compelling statistical significance for people with Alzheimer's disease in this trial," he added. "This is the first Phase 3 trial of any investigational medicine for Alzheimer's disease to deliver 35% slowing of clinical and functional decline."
Eli Lilly shares were marked 3.7% higher in early Wednesday trading immediately following news of the trial data to change hands at $419.08 each.
Late last year, European drugmaker Roche AG said that trials of its gantenerumab, a drug used to treat patients with early-stage Alzheimer's, failed to reach the primary endpoint of preserving cognitive functions affected by the disease.
The results leave Biogen (BIIB) - which unveiled better-than-expected results from a late-stage study of its Alzheimer's treatment developed with Japan-based partner Eisai Co Ltd., as well as Eli Lilly, as potential market leaders in the multi-billion market.
Treatment pricing, however, has been a controversial issue and the U.S. Centers of Medicare and Medicaid Services noted in September that its decision to only cover treatment with Aduhelm -- Biogen's FDA-approved Alzheimer's drug -- if patients are enrolled in a clinical trial as a major factor in lowering overall premiums for Medicare Part B.
Biogen was forced to slash the price of Aduhelm by around 50%, to $28,200 per year, in order to help expand its potential reach following the CMS decision late last year.
Aduhelm, Biogen noted in earlier studies, has been show to erode amyloid beta, a plaque which builds up around the brain and can lead to neuron damage.
The FDA approved Aduhelm for the treatment of Alzheimer’s through its accelerated approval pathway, which can be used for a drug for a serious or life-threatening illness that provides a meaningful therapeutic advantage over existing treatments, last year.
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>>> Why long COVID could be a ticking time bomb for public health
Salon
by Philip Finkelstein
4-24-23
https://www.msn.com/en-us/health/medical/why-long-covid-could-be-a-ticking-time-bomb-for-public-health/ar-AA1ahhqo?ocid=hpmsn&cvid=9d045fab09154240833b24ee52f26fbe&ei=28
The 1918 influenza pandemic, commonly referred to as the Spanish Flu, infected approximately one-third (500 million) of the world's population (then 1.8 billion) and killed an estimated 50 million. With such a high mortality rate, even among young and healthy individuals, this acute infectious disease took its toll, erasing from existence nearly 3% of all people on Earth. But the damage did not stop there: across the globe, survivors of the initial viral infection reported "long flu" symptoms — profound fatigue, brain fog, depression, tremors, sleeplessness, and a litany of neurological disorders.
While the initial pandemic forced governments to organize a response to the sudden crisis, an epidemic of chronic illness may not raise alarms that spur us into immediate action.
This "long flu," an echo of sorts of the Spanish Flu epidemic itself, has its parallel in long COVID today — a similar cluster of symptoms that persist in those who were previously infected with COVID-19. And the similarities suggest that what we think of as long COVID is not necessarily a novel condition, but merely one more instance of the medical aftermath that accompanies certain infections.
The medical establishment calls this condition post-acute infection syndrome (PAIS). Back in 1918, these mysteriously persistent long flu symptoms wreaked havoc on human health and local economies. For example, many claim that debilitating lethargy caused by this post-viral syndrome led to the "famine of corms" in the region that is Tanzania today, as farmers lacked the energy to plant, harvest, and shear months after getting sick.
Around the same time, cases of a new brain-attacking disease called encephalitis lethargica started to emerge, affecting up to one million people worldwide. The cause of encephalitis lethargica remains one of the largest medical mysteries of the 20th century, though some scientists contend that the Spanish Flu may have been the trigger. The condition was colloquially known as "sleeping sickness," as those infected developed extreme fatigue, neurocognitive impairments, psychiatric illness, and movement disorders. A subset of these individuals fell into a semi-comatose state that lasted for decades. About one-third of encephalitis lethargica patients eventually died from respiratory failure caused by neurological dysfunction, while many survivors continued to suffer from ongoing Parkinson's-disease-like (neurocognitive) symptoms.
In 1969, as chronicled in his book "Awakenings," the neurologist Oliver Sacks discovered that temporary remission of these chronic symptoms, coined post-encephalitic parkinsonism, could be achieved through the use of the Parkinson's drug L-DOPA. Like with Parkinson's disease itself, the benefits of the drug wore off over time, but the finding indicated that encephalitis lethargica impacted the substantia nigra (the part of the brain that helps control movement).
We now have a plethora of information suggesting that COVID-19 is the latest addition to the list of infections spawning post-acute infection syndrome.
Although the medical community has long known that acute infectious diseases are not always entirely self-limiting, chronic sequelae (meaning the secondary symptoms that appear after an infection) receive little attention, remain under-researched, and continue to be misdiagnosed and overlooked by doctors. According to a study published in the scientific journal Nature, post-acute infection syndrome is associated with a number of infections, including Epstein Barr virus, cytomegalovirus, Lyme disease, Q fever, West Nile virus, Dengue fever, and the aforementioned influenza. Often presenting well after the initial infection, post-acute infection syndrome manifests as a complex and variable disorder, typically entailing severe fatigue, gastrointestinal issues, confused sensory perception, and neurocognitive abnormalities.
Despite the growing pool of data from patients suffering from post-acute infection syndrome, a comprehensive explanation of the biological mechanisms by which the syndrome's symptoms arise has yet to be established. This lack of scientific understanding creates an untold degree of hardship for those dealing with severe and chronic sequelae of infections. Worse, when doctors cannot find a biological explanation for reported symptoms, patients are often left with little recourse and the feeling that their doctor believes the cause of their suffering is rooted in mental illness.
Years into our current pandemic, we now have a plethora of information suggesting that COVID-19 is the latest addition to the list of infections spawning post-acute infection syndrome; that is, "long COVID." Multinational surveys have been conducted, with thousands upon thousands of adult participants reporting that recovery from an initial COVID infection took more than 35 weeks. Some of these studies highlight the fact that new ailments are reported 6-12 months after an initial COVID infection, which most commonly include fatigue, post-exertional malaise, and cognitive dysfunction.
According to the CDC, in June 2022, almost one in five American adults who had COVID-19 still had long COVID. This statistic seems to be borne out by my anecdotal experience; I have met with and spoken to many people around the world who have lost their sense of smell, had to take medical leave, been fired from work, seen a drop in their focus during school, experienced overwhelming exhaustion and migraines, or become depressed after being infected with COVID. My home state's newspapers recently shared the sad medical saga of a man, Charlie Vallee, whom I grew up with in Vermont. After only mild respiratory symptoms during his initial bout of COVID-19, Vallee went on to develop such severe long COVID symptoms, including brain fog, that he left his job as an intelligence officer in D.C. and tragically took his own life. His family has set up a foundation to fund long COVID research in the hopes of one day understanding how this pernicious form of post-acute infection syndrome can cause an otherwise happy and healthy individual to die by suicide.
In other words, long COVID is affecting more people than we likely know. And it eerily parallels other post-acute infection syndrome scenarios throughout history, including those potentially linked to epidemics of parkinsonism. In other words, the threat of long COVID could lead to a future public health catastrophe, much as the "long" effects of the Spanish Flu did a hundred years ago. Unfortunately, the pharmaceutical and medical community are not approaching long COVID with the same fervor that they had for COVID-19. As a result, there is a real danger that a broad-scale investigation into the origin of long COVID is postponed or neglected by funding agencies and the medical establishment.
While the initial pandemic forced governments to organize a response to the sudden crisis, an epidemic of chronic illness may not raise alarms that spur us into immediate action. Like climate change, a gradually-evolving threat, especially one perceived to be far away, is much harder to address. But the threat here is not that far off, as emerging science reveals — which is why it is of grave importance that we push for an explanatory theory of long COVID (and post-acute infection syndrome) that can fully account for the totality of symptoms observed after an initial infection with SARS-CoV-2 despite no clinical findings of active infection.
Multiple studies published in the journal Nature (one published last year and one published in February of this year) explain how COVID-19 has the ability to trigger the aggregation of proteins within the human body. The research suggests that SARS-CoV-2 can cause normal proteins to abnormally misfold. These misfolded proteins are known as "amyloids," which are toxic to cells when they build up.
Specifically, amyloids occur when proteins misfold into twisted clumps and form long fibers, hindering cellular function. These so-called clumps can start stacking excessively, creating harmful deposits in the body — sort of like cholesterol in the bloodstream but at the cellular level. When misfolding of a protein named "alpha-synuclein" in the nervous system occurs, the amyloid buildup this causes in a neuron can lead to the formation of what is known as a "Lewy body," which is resistant to breakdown and clearance. Think of it as plaque buildup in the nervous system. Lewy bodies spread as pieces of these amyloids break away and seed the formation of new Lewy bodies in neighboring neurons.
The scariest thing about this? Misfolded alpha-synuclein is a hallmark of Parkinson's disease, Lewy body dementia, multiple system atrophy, and pure autonomic failure — all neurodegenerative diseases collectively known as synucleinopathies. And what can cause alpha-synuclein misfolding? Genetic mutations, exposure to certain toxins, and infections. COVID-19 may be one such infection — and that means long COVID symptoms may be a reflection of a developing neurological disorder.
Alarmingly, two studies published by the Mayo Clinic and the Medical University Innsbruck corroborate the findings in the Nature articles, recording signs of dream-enactment sleep disorder among one-third of patients after being infected with COVID-19. Over 80% of patients with dream-enactment sleep disorder go on to develop a Parkinson's-like disease within two decades.
So we need to ask the question: is the recent rise of dream-enactment sleep disorder after COVID related to neurodegeneration? Preliminary research from Stanford University and Beth Israel Deaconess Medical Center suggests that this may be the case, as disease-causing clumps of alpha-synuclein have been discovered in some long COVID patients.
So how does all of this connect? Basically, if dream-enactment sleep disorder is more common in those who have had COVID, and the vast majority of those who suffer from this kind of sleep disorder ultimately develop neurological diseases like Parkinson's, then COVID-19 could lead to an explosion of these diseases in the coming years.
This is not mere speculation; animal models further substantiate these claims. For example, a study of macaques demonstrated that SARS-CoV-2 induces Lewy body formation (a feature of Parkinson's disease), even after an asymptomatic infection. And, whether or not COVID is determined to be a direct cause of Parkinson's, it could also accelerate the disease course in patients who are predisposed. This was exemplified by a study performed by infecting mice with COVID-19, which found that the virus made the brain more susceptible to toxic compounds known to cause Parkinson's disease. The lead researcher on this study, Richard Smeyne, PhD, who serves as Chair of the Department of Neuroscience at Thomas Jefferson University and Director of the Jefferson Comprehensive Movement Disorder Center reviewed this article before publication, affirming what has been outlined and reiterating his study's findings: "Should the predicted risk from SARS-CoV-2 manifest, the diverse consequences would represent a substantial burden on patients, families, and society."
Dr. Smeyne elaborated on the seriousness of these findings, telling Salon, "Our studies in mice predict a 30-50% increase in Parkinson's risk for those moderately to severely infected with the Alpha variant. While on an individual basis this only changes a person's risk from 2% to 3% for developing Parkinson's, over the whole of the population we would expect to see millions more develop Parkinon's disease than would have if not for their COVID infection."
"We still have to examine if the newer strains of SARS-CoV-2 also have the potential to increase the risk for Parkinson's disease."
A prominent theory for explaining Parkinson's disease, put forth by Heiko Braak, a German doctor who studies Parkinson's, aligns well with all these long COVID findings. It states that Parkinson's is caused by a pathogen affecting either the nasal cavity or digestive system, thereby first initiating protein misfolding in the peripheral nervous system before spreading into the brain later on (sometimes decades later). This is why the onset of Parkinson's often entails autonomic dysfunction — which means involuntary processes like heart rate, blood pressure, respiration, etc. are compromised. As autonomic dysfunction is a common symptom of long COVID, it is thus possible that the post-acute infection syndrome mechanism responsible for long COVID progresses to the central nervous system over time and could eventually present as Parkinson's disease or a similar disorder.
In other words, while long COVID is not caused by the lingering viral remnants of COVID-19 per se, the initial infection could be precipitating amyloid buildup and Lewy body formation. If this is so, long COVID would mimic a chronic or slowly-evolving infection caused by the virus, similar to other post-acute infection syndrome cases, with the symptoms fluctuating and emerging unpredictably as the amyloids slowly spread throughout the nervous system.
Braak's hypothesis was based on autopsy data, which indicated a distinct pattern of aggregated alpha-synuclein in those who died from/with Parkinson's disease. However, according to Dr. Smeyne, "As of yet, there is no good non-invasive marker for alpha-synuclein aggregation in living patients, which is why The Michael J. Fox Foundation is offering a $2 million prize to any person or group that successfully develops such a marker."
The way forward
To investigate these claims, larger studies need to evaluate patients with long COVID for markers of Parkinson's-like diseases, such as misfolded alpha-synuclein. A clinical trial is currently underway to do just that — so that the history of post-encephalitic parkinsonism in the years following the Spanish Flu does not repeat itself. Considering the mounting evidence, it is crucial that we address the long COVID public health emergency promptly, to provide answers to those suffering from long COVID and prevent a potential increase in "post-COVID parkinsonism."
When asked about his outlook for the future, Dr. Smeyne said, "We are entering a period where we will have to learn to live with COVID being present as a fact of life. This means we still have to examine if the newer strains of SARS-CoV-2 also have the potential to increase the risk for Parkinson's disease and whether vaccination against this virus can reduce the increased Parkinson's risk, as has been shown following vaccination against influenza. Once we determine the answers to these questions, we can begin to look at other ways to interfere with the process."
Salon then asked what it will take to definitively prove whether COVID-19 can trigger a Parkinson's-like disease and whether long COVID is in fact the early stages of such a disease. Dr. Smeyne responded, "My best guess is we will need anywhere from five to ten years from the initial outbreak to see any statistically measurable effect."
Encouragingly, Dr. Smeyne went on to say, "One bright spot in these observations is that there is a considerable period, often about a decade, between viral exposure and the development of a neurological disease like Parkinson's. And there are currently scientists devoting their lives' efforts to find ways to solve this problem — the lag between exposure and disease gives me hope that we will find a way to stop the progression from infection to disease in its tracks."
There have been more than 760 million globally documented cases of COVID-19, with the real number of cases, including asymptomatic cases, presumably much higher. More than 750 million have survived, but, as reported, long COVID is occurring in 20-30% of these cases, meaning that hundreds of millions of people could be at higher risk of developing Parkinson's disease or other neurodegenerative issues later in life. If it comes to pass, the public health resources required to help will be astronomical. It behooves us to study long COVID now, lest we end up in such a crisis.
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>>> Simulations Plus (SLP) is a premier developer of groundbreaking drug discovery and development simulation software, which is licensed and used for drug research by major pharmaceutical and biotechnology companies worldwide.
https://finance.yahoo.com/news/4-low-beta-tech-stocks-143202937.html
Currently, Simulations Plus has a Zacks Rank #2. It has a beta of 0.54. Shares of SLP have climbed 15.9% YTD.
Lancaster, CA-headquartered SLP’s performance is being driven by strength in its Services revenues, which increased 4% to $5.3 million in second-quarter fiscal 2023. In March, the company partnered with the Sino-American Cancer Foundation to boost the development of anticancer therapies. Earlier in the same month, it collaborated with the Institute of Medical Biology of the Polish Academy of Sciences to design new compounds for the ROR?/ROR?T nuclear receptors. As part of the agreement, the company is leveraging artificial intelligence/machine learning technologies in the ADMET Predictor platform for the development of new compounds.
The Zacks Consensus Estimate for SLP’s fiscal 2023 earnings is pegged at 66 cents, unchanged over the past seven days. The consensus mark for fiscal 2024 earnings has also remained steady for the past seven days at 71 cents, suggesting an 8.3% year-on-year rise.
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>>> Skin cancer vaccine (mRNA) an 'exciting' advance in reducing risk of melanoma relapse, trial shows
NBC News
by Linda Carroll and Reynolds Lewis
April 17, 2023
https://news.yahoo.com/mrna-cancer-vaccine-exciting-advance-170100554.html
In 2019, Gary Keblish got a diagnosis from his surgeon that left him stunned. The flat, dark-brown mole he’d had on his back for as long as he could remember had turned into a melanoma that was already advanced, putting him at risk of dying.
“I felt numb,” the Brooklyn teacher, 61, said in an interview.
Fortunately, Keblish was able sign on for a small clinical trial testing a preventive vaccine that might possibly keep the disease from coming back.
The trial focused on a personalized vaccine using mRNA technology that used mutations to target mutations unique to a patient’s cancer but not the healthy cells in the body. All participants in the trial would receive the immunotherapy drug Keytruda (pembrolizumab), the standard of care for high-risk melanoma patients like Keblish. Two-thirds of the participants would also receive the vaccine.
Keblish was one of those who received the vaccine — which teaches the body’s immune system to recognize cancer cells as different from normal cells so that, working along with the immunotheraoy drug, it can attack them.
After two years, Keblish's cancer hasn't returned.
Personalized cancer vaccine shows benefit
On Sunday, the results of the phase 2 trial, which showed that the combination of the vaccine and immunotherapy reduced the risk of recurrence by nearly half, were presented at the annual meeting of the American Association for Cancer Research.
It's the first randomized, controlled trial to show a benefit from this type of cancer vaccine, said senior investigator Dr. Jeffrey Weber, a deputy director of NYU Langone’s Perlmutter Cancer Center and a professor of medicine at the NYU Grossman School of Medicine.
To test the effectiveness of the vaccine, the international team of researchers recruited 157 melanoma patients whose tumors had been surgically removed and who were at high risk of experiencing a recurrence of their cancers. Fifty patients received only the immunotherapy medication and 107 also got the personalized vaccination.
One of the ways cancer evades the immune system is to fool the body into thinking the threat is over, at which point the natural braking system that prevents the immune system from staying constantly on kicks in. Weber compares the way pembrolizumab works to cutting a stuck brake cable on a car so it can go forward.
Once the system’s braking system has been partially disabled, “the immune system works really well,” Weber said, adding that the downside of “cutting brake cable” is that the immune system stays turned up and some people end up with inflammation and something that resembles an autoimmune disease.
One other way cancer can avoid being destroyed is through mutations, so the immune system’s soldiers cease to recognize it as a threat.
That’s where the personalized mRNA vaccine comes in. After a patient’s tumor is removed, doctors identify proteins that are specific to that person’s tumor and no other cells in the body. As many as 34 proteins from a patient’s tumor were then the target of the vaccine.
In the trial, 40% of the patients who received only the immunotherapy drug had a recurrence of their cancer during the two-year follow-up. In comparison, 22.4% of patients who got the drug plus the vaccine had a recurrence, leading to a difference of 44% between the two groups.
The new findings are significant, said Dr. Antoni Ribas, a professor of medicine at the University of California, Los Angeles, and director of Tumor Immunology Program at UCLA’s Jonsson Comprehensive Cancer Center.
It’s the first time a cancer vaccine has been shown to have this level of benefit, close to a 50% decrease in the risk of relapse,” said Ribas. “It tells us these vaccines actually work and can turn on an immune response against the patient’s own cancer."
The trial’s results are “very exciting,” said Dr. Thomas Marron, director of the Early Phase Trials Unit at the Tisch Cancer Institute and an associate professor of medicine at the Icahn School of Medicine at Mount Sinai in New York.
“Once the tumor is removed, we know it can come back because tiny microscopic bits have traveled elsewhere in the body and set up shop there,” Marron said. The recurrence often appears quickly, between six months and two years, he said.
The beauty of the vaccine in this study is that it targets up to 34 mutations, Marron said. “That’s like taking 34 shots on goal," he said. You’re teaching the immune system to recognize 34 different things that are unique to that cancer.”
The researchers anticipate the phase 3 trial, scheduled to launch this summer, to show similar results. With follow-up and monitoring, it could be at least two years before data is registered with the Food and Drug Administration and up to three years before the vaccine combination would be approved for use in patients, Weber said.
Still, it's an exciting advance in the field of cancer vaccines, particularly for stopping melanoma, the deadliest form of skin cancer, experts said.
The study is “important because it’s the first randomized study of a cancer vaccine with a clinically meaningful endpoint: stopping tumors from coming back,” said Dr. Margaret Callahan, research director of the Memorial Sloan Kettering Immunotherapeutics Program, who expressed cautious optimism about the findings.
“This is an exciting advance in the field of cancer vaccines, an area notoriously tough to make progress in,” Callahan said.
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>>> Merck to buy Prometheus Biosciences for about $11 billion
Reuters
4-16-23
https://www.msn.com/en-us/money/companies/merck-to-buy-prometheus-biosciences-for-about-11-billion/ar-AA19VksJ?OCID=ansmsnnews11
(Reuters) - Merck & Co has agreed to acquire Prometheus Biosciences Inc for about $10.8 billion to bolster the company's presence in immunology, the companies said on Sunday.
The joint statement said that Merck, through one of its subsidiaries, will pay $200 per share for the biotechnology company that specializes in products for treatment of immunological diseases. That represents a 75% premium to the $114.01 closing price for Prometheus shares on Friday.
Prometheus had a market capitalization of $5.42 billion at Friday's close.
Merck has been looking for deals to protect itself from eventual revenue loss as patents on its cancer immunotherapy Keytruda begin to expire towards the end of the decade.
"The agreement with Prometheus will accelerate our growing presence in immunology where there remains substantial unmet patient need. This transaction adds diversity to our overall portfolio," said Merck Chairman and Chief Executive Robert Davis.
The deal, which was first reported by the Wall Street Journal, is expected to close in the third quarter of the year, the companies said.
Merck in February forecast 2023 earnings below Wall Street estimates and an expected steep decline in sales of its COVID-19 antiviral treatment.
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>>> As superbug infections grow more common, the world is running out of drugs to treat them
NBC News
by Caroline Hopkins
March 29, 2023
https://investorshub.advfn.com/secure/post_new.aspx?board_id=37829
Late last year, one of Dr. Vance Fowler’s patients — a man in his 60s who’d returned to North Carolina from visiting his family in Nepal — died of a bacterial infection. He’d been treated at a top U.S. hospital with access to the strongest antibiotics. But the infection, a drug-resistant strain of E. coli, surged on.
“Antibiotic resistance is a real problem that, with little or no warning, can affect the lives of any of us at any time,” said Fowler, an infectious disease specialist at Duke Health. “We don’t have enough drugs.”
Health officials have warned the public about antibiotic resistance for decades. That’s become more urgent in light of an upcoming World Health Organization report tallying only a handful of new antibiotics in development.
Preliminary data from the report, released by the WHO this month, paint a dire picture: Just 27 new antibiotics for the most threatening infections are in the clinical trial stage of drug development. In contrast, there were more than 1,300 cancer drugs in clinical trials in 2020, according to a report from the trade organization Pharmaceutical Research and Manufacturers of America.
Of the antibiotics in trials, the WHO considers only six of them innovative enough to overcome antibiotic resistance, and just two capable of targeting the most resistant bacteria. Agency officials will present the full report during the European Congress of Clinical Microbiology and Infectious Diseases next month.
There’s no telling whether this handful of new drugs in clinical trials will work, either. From 2017 to 2021, just one new antibiotic, cefiderocol, was approved that could treat the superbugs on WHO’s most critical list, including strains of Acinetobacter baumannii, Pseudomonas aeruginosa and Enterobacteriaceae. These pathogens can cause a range of potentially severe infections in the lungs, urinary tract, ears, blood, open wounds, or even the brain and the spinal cord.
Resistance on the rise
Drug-resistant bacteria are growing more common. In the United States alone, the Centers for Disease Control and Prevention estimates more than 2.8 million people develop drug-resistant infections each year, and more than 35,000 people die as a result. Certain drug-resistant strains of gonorrhea are on its list of urgent superbugs, as is Clostridioides difficile, or C. diff, which can cause life-threatening diarrhea and colon inflammation. The CDC estimates 12,800 people die of C. diff each year.
One of the most common superbugs in the U.S., methicillin-resistant Staphylococcus aureus, or MRSA, kills 9,800 people each year. MRSA can spread rapidly in long-term care facilities and hospital settings, where cases spiked 13% during the first year of the Covid pandemic.
Infections from the drug-resistant Shigella bacteria, often resulting in severe diarrhea and stomach pain, have been climbing at an alarming rate, too. In February, the CDC warned that last year, 5% of Shigella infections were “extensively drug-resistant” — meaning they didn’t respond to a number of antibiotics — up from zero percent in 2015. Shigella can spread through sexual contact, particularly among men who have sex with men.
Bacteria aren’t the only culprit. In the U.S., antifungal drug-resistant infections from the fungus Candida auris increased 60% in 2020, according to the CDC.
The WHO has identified 12 resistant superbugs deemed “priority pathogens,” and the CDC tracks a list of 18 drug-resistant bacteria and fungi.
Globally, more than 5 million people die from antibiotic resistance, outnumbering deaths from HIV, tuberculosis and malaria combined, said Valeria Gigante, a team lead within the WHO’s antimicrobial resistance division.
“Antimicrobial resistance is one of the top global health threats facing humanity,” she said. “We shouldn’t call it a silent pandemic anymore. We should be loud and clear: it is indeed a pandemic.”
Bacteria and fungi are more likely to develop resistance the more they’re exposed to antibiotics or antifungals. In the case of Fowler’s patient, the specific E. coli bacteria strain had a gene that makes a protein called NDM-1, which can break down even the strongest, last-resort antibiotics, called carbapenems. Right now, most E. coli strains — there are more than 700 — aren’t deadly. But beyond E. coli, several strains of Klebsiella, Enterobacter and Acinetobacter already have this gene, and according to Fowler, more may soon acquire it.
“They don’t need a passport to travel,” Fowler said. And bacteria can swap DNA “kind of like baseball cards.”
“Maybe five or 10 years down the road, the U.S. will be facing resistance patterns reasonably similar to what we’re facing in India or Greece,” said Dr. Venkatasubramanian Ramasubramanian, president of the Clinical Infectious Diseases Society of India. Both countries have seen outbreaks of bacteria resistant to the strongest, last-resort antibiotics. “It’s a matter of time.”
The WHO says the rate of antibiotic resistance is accelerating. From 1970 to 2000, the average time it took for resistance to develop to new antibiotics was just two to three years, down from the 11-year average from 1930 to 1950. Resistance skyrocketed during the early days of the pandemic when many health systems overused antibiotics, which have no impact on Covid because it is caused by a virus, not a bacterium.
Where are all the new drugs?
The economic model for new drugs — pharmaceutical companies invest large sums upfront to test a drug’s safety and efficacy, then earn that money back in sales once it’s approved — “doesn’t work for antibiotics,” Ramasubramanian said.
Developing one new antibiotic can take up to two decades and usually costs $568 million to $700 million according to PhRMA. And just 1 in 30 of these drugs are ultimately approved to treat patients. But unlike medicines meant for widespread use, there’s an international push to use fewer antibiotics. Excessive or unnecessary antibiotic use ups the odds of a pathogen developing resistance.
“With a new antibiotic, we say, ‘don’t use it,’ or ‘use it sparingly so it lasts longer,’” Ramasubramanian said. “It is not an attractive proposition for anyone in the industry.”
Some countries have rolled out what they call “antibiotic stewardship programs.” These encourage doctors to prescribe the drugs only when there’s a clear need. In the U.S., for instance, the CDC offers training courses and guidelines to help curb antibiotic use and stave off resistance.
Even in cases where antibiotics are truly necessary, they’re often prescribed for just days or weeks, making them far less lucrative than long-term, daily-use drugs for chronic conditions such as blood pressure or diabetes.
“At the end of the day, the ‘supply and demand’ model is not tenable for antibiotics,” Fowler said.
No easy answer
If drugmakers don’t start developing new antibiotics soon, the world may be facing a “doomsday scenario,” WHO officials warned.
More people could die from once-treatable infections, such as bacterial pneumonia, gonorrhea or salmonella. Those who need antibiotics most, like immunocompromised people and those undergoing cancer treatment, will be most vulnerable.
“We have arrived in the post-antibiotic era,” Ramasubramanian warned in a statement March 15. “The current antibacterial pipeline is woefully insufficient to make a difference in tackling the ongoing threat of antibiotic resistance.”
Fowler, who wasn’t involved in the WHO report, agreed with the organization’s use of bold “doomsday” language. “The WHO is 100% spot-on,” he said. “I was thrilled to see them make such a strong statement because I think it’s true.”
While there’s no single fix for catalyzing new antibiotic development, Gigante said, government funding and policy could help move the needle. For instance, some countries have devised new economic models for incentivizing antibiotic development.
In the U.S., lawmakers are debating a legislation called the PASTEUR Act that would pay pharmaceutical companies contractually to make these critical new drugs available.
“It would fundamentally be a Netflix-like subscription model,” Fowler said. Drug companies wouldn’t have to rely on the minimal revenue they’d get selling their antibiotics on the commercial market. The proposed model has been controversial and hasn’t become law, but it’s an example of the type of “new economic model” WHO officials like Gigante want policymakers to explore.
U.S. taxpayer dollars do support some new antibiotics research already. For instance, Fowler leads a program called the Antibacterial Resistance Leadership Group, which funds new trials with grants from the National Institutes of Health.
“It’s an enormous amount of money, but there’s a great deal more that’s needed,” he said.
Better tests could help
Beyond calling for new antibiotics, the WHO officials want to see better, faster, ways of diagnosing bacterial infections. Right now, for the first 48 hours or so after a patient comes in with an infection, “you don’t know what germ you’re treating,” Fowler said.
The more quickly and accurately doctors zero in on the specific bug infecting their patients, the less likely they’ll be to prescribe antibiotics that won’t work, which could cause more resistance.
The diagnostic process involves collecting a swab, sending it to a lab, growing the bacteria from that swab until there’s enough to test, then, while still in the lab, trying out a bunch of different antibiotics to see which will work. It can take days or weeks, and the sickest patients can’t wait that long.
“It’s not through any lack of good clinical practice, but the state-of-the-art in treating infection in 2023 is an educated guess,” Fowler said. “It’s frightening when you think about it.”
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>>> DNA from Beethoven's hair reveals new details into his cause of death over a century later: study
Researchers discover Beethoven was genetically predisposed to liver disease and had hepatitis B in the months before his death
By Elizabeth Pritchett
Fox News
https://www.foxnews.com/health/dna-beethovens-hair-reveals-new-details-cause-death-centuries-later-study
A genetic study conducted on locks of Ludwig van Beethoven's hair revealed more details about the composer's death at a relatively young age in March 1827.
The University of Cambridge biological anthropologist Tristan Begg, the lead researcher in the study "Genomic analyses of hair from Ludwig van Beethoven," which was published in Current Biology on Wednesday, said eight strands of hair attributed to the German musician were tested in hopes of explaining potential underlying genetic and infectious causes of his illnesses.
It was already known that Beethoven, who died at the age of 56 from a protracted illness, began losing his hearing in his 20s and was functionally deaf by his mid-40s. He was also known to have experienced severe abdominal pains and chronic bouts of diarrhea since he was 22.
Though the study did not find a primary cause for his hearing loss or gastrointestinal problems, it shed light on other health issues the composer experienced during his lifetime.
Five of the eight strands of hair tested were found to be "perfect genetic matches" and were deemed "almost certainly authentic," the study said, allowing researchers to determine Beethoven had a genetic predisposition for liver disease – something that was thought to have contributed to his death.
The testing also discovered that the famous composer had a hepatitis B infection during the months prior to his death, at the least.
Researchers concluded that his genetic predisposition and heavy alcohol consumption presented "plausible explanations" for Beethoven's severe liver disease.
During the study, researchers also came across an unexpected result as an analysis of Y chromosomes from five living members of the Van Beethoven patrilineage compared with the DNA from Beethoven's hair revealed a mismatch in paternal ancestry generations before his birth.
"This finding suggests an extrapair paternity event in his paternal line between the conception of Hendrik van Beethoven in Kampenhout, Belgium in c.1572 and the conception of Ludwig van Beethoven seven generations later in 1770, in Bonn, Germany," Begg wrote.
The study also debunked a forensic investigation completed in 2007 that suggested lead poisoning could have sped up his death, if not the primary cause for the symptoms that ultimately claimed his life.
Though lead poisoning was probable due to drinking from lead vessels and medical treatments of the time that used lead, the hair used to complete that study nearly 16 years ago was found to have come from an unknown woman, not the composer.
Researchers said the hairs used in the study were gathered from public and private collections during the last six years of Beethoven's life.
Following Beethoven's death on March 26, 1827, various researchers and medical professionals studied the cause of his hearing loss – something the composer had requested in writing years prior.
He initially wanted his favorite physician, Dr. Johann Adam Schmidt, to reveal his health struggles to the public, but Schmidt died before Beethoven.
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Vertex Pharmaceuticals - >>> Vertex Pharmaceuticals (VRTX 1.23%) has remained the premier presence in the cystic fibrosis treatment space for over a decade now since its first therapy to treat the underlying factors that cause the genetic disease was given the green light by the U.S. Food and Drug Administration.
https://www.fool.com/investing/2023/03/09/2-top-stocks-to-buy-in-march-and-hold-forever/?source=eptyholnk0000202&utm_source=yahoo-host&utm_medium=feed&utm_campaign=article
Since that time, Vertex has seen its portfolio of CFTR modulators -- the class of drugs that work to correct the root cause of cystic fibrosis -- expand to four approved therapies, and it remains the only company with approved CFTR modulators on the market at the time of this writing.
While its portfolio of top-selling therapies faces consistent and growing demand -- management estimates that as many as 20,000 cystic fibrosis patients could take its CFTR modulators but aren't yet doing so -- Vertex is looking toward future sources of growth to spur its business and financials forward in the years ahead.
Its current pipeline includes a non-opioid drug candidate for acute pain, a cystic fibrosis drug for patients who can't take CFTR modulators (management estimates that more than 5,000 patients fit this category), and a potential one-time functional care for two rare blood disorders, for which it's in the process of undergoing regulatory submissions.
The company is also wading into the lucrative diabetes care market. Last year, Vertex finished its acquisition of ViaCyte, a company that is working on stem cell therapies to treat type 1 and type 2 diabetes. The cystic fibrosis treatment market is set to reach a valuation of $32 billion by the year 2027, achieving a compound annual growth rate of more than 24% from its 2019 valuation of about $5 billion.
However, the broad opportunities within this space are just the tip of the iceberg for the long-term potential that Vertex appears to have as it continues to explore lucrative yet underpenetrated segments of the rare-disease drug market. For investors searching for a healthcare stock in which to retain a multi-year, buy-and-hold investment, Vertex looks like a compelling choice to consider.
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>>> Could future computers run on human brain cells?
Johns Hopkins researchers tout the promise of 'organoid intelligence', which could one-day yield computers that are faster, more efficient, and more powerful than silicon-based computing and AI
JOHNS HOPKINS UNIVERSITY
By Roberto Molar Candanosa
Feb 28, 2023
https://hub.jhu.edu/2023/02/28/organoid-intelligence-biocomputers/
A "biocomputer" powered by human brain cells could be developed within our lifetime, according to Johns Hopkins University researchers who expect such technology to exponentially expand the capabilities of modern computing and create novel fields of study.
The team outlines their plan for "organoid intelligence" today in the journal Frontiers in Science.
"Computing and artificial intelligence have been driving the technology revolution, but they are reaching a ceiling," said Thomas Hartung, a professor of environmental health sciences at the Johns Hopkins Bloomberg School of Public Health and Whiting School of Engineering who is spearheading the work. "Biocomputing is an enormous effort of compacting computational power and increasing its efficiency to push past our current technological limits."
For nearly two decades scientists have used tiny organoids, lab-grown tissue resembling fully grown organs, to experiment on kidneys, lungs, and other organs without resorting to human or animal testing. More recently Hartung and colleagues at Johns Hopkins have been working with brain organoids, orbs the size of a pen dot with neurons and other features that promise to sustain basic functions like learning and remembering.
"This opens up research on how the human brain works," Hartung said. "Because you can start manipulating the system, doing things you cannot ethically do with human brains."
Hartung began to grow and assemble brain cells into functional organoids in 2012 using cells from human skin samples reprogrammed into an embryonic stem cell-like state. Each organoid contains about 50,000 cells, about the size of a fruit fly's nervous system. He now envisions building a futuristic computer with such brain organoids.
Computers that run on this "biological hardware" could in the next decade begin to alleviate energy-consumption demands of supercomputing that are becoming increasingly unsustainable, Hartung said. Even though computers process calculations involving numbers and data faster than humans, brains are much smarter in making complex logical decisions, like telling a dog from a cat.
"The brain is still unmatched by modern computers," Hartung said. "Frontier, the latest supercomputer in Kentucky, is a $600 million, 6,800-square-feet installation. Only in June of last year, it exceeded for the first time the computational capacity of a single human brain—but using a million times more energy."
It might take decades before organoid intelligence can power a system as smart as a mouse, Hartung said. But by scaling up production of brain organoids and training them with artificial intelligence, he foresees a future where biocomputers support superior computing speed, processing power, data efficiency, and storage capabilities.
"It will take decades before we achieve the goal of something comparable to any type of computer," Hartung said. "But if we don't start creating funding programs for this, it will be much more difficult."
Organoid intelligence could also revolutionize drug testing research for neurodevelopmental disorders and neurodegeneration, said Lena Smirnova, a Johns Hopkins assistant professor of environmental health and engineering who co-leads the investigations.
"We want to compare brain organoids from typically developed donors versus brain organoids from donors with autism," Smirnova said. "The tools we are developing toward biological computing are the same tools that will allow us to understand changes in neuronal networks specific for autism, without having to use animals or to access patients, so we can understand the underlying mechanisms of why patients have these cognition issues and impairments."
To assess the ethical implications of working with organoid intelligence, a diverse consortium of scientists, bioethicists, and members of the public have been embedded within the team.
Johns Hopkins authors included: Brian S. Caffo, David H. Gracias, Qi Huang, Itzy E. Morales Pantoja, Bohao Tang, Donald J. Zack, Cynthia A. Berlinicke, J. Lomax Boyd, Timothy DHarris, Erik C. Johnson, Jeffrey Kahn, Barton L. Paulhamus, Jesse Plotkin, Alexander S. Szalay, Joshua T. Vogelstein, and Paul F. Worley.
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The Baker Bros portfolio has some great ideas -
https://investorshub.advfn.com/boards/read_msg.aspx?message_id=171262104
Being a 'reformed' bio investor, I only own a few large caps. The poster Bladerunner follows the emerging small caps.
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>>> All 109 Baker Brothers Portfolio Stocks
Current 2022 Holdings
Updated on November 23rd, 2022
by Nikolaos Sismanis
https://www.suredividend.com/baker-brothers/
Founded in 2000 by Julian and Felix Baker, Baker Bros. Advisors is a private hedge fund based out of New York City. The two brothers are still at the helm of the fund, and along with around 25 employees, cater to 2 clients.
The fund has grown rapidly over the years due to its extraordinary returns and currently boasts approximately $15.2 billion of assets under management (AUM). Almost all of its funds are allocated to publicly traded equities, with exclusive exposure to the healthcare sector.
Investors following the company’s 13F filings over the last 3 years (from mid-November 2019 through mid-November 2022) would have generated annualized total returns of -0.82%. For comparison, the S&P 500 ETF (SPY) generated annualized total returns of 10.2% over the same time period.
Baker Brothers’ Philosophy and Strategy
Brothers Julian and Felix Baker have earned their guru status on Wall Street, having delivered an exceptional track record of annualized returns over the years. Julian has a business background from Harvard, while Felix has a Ph.D. in Immunology from Stanford.
Together, they have combined their individual expertise to generate superior returns by focusing solely on the biotech industry. Assets under management grew from $250 million in 2003, to $15.2 billion as of November 15th, 2022.
The fund’s strategy includes utilizing a fundamentally-driven way of investing to come up with its investment decisions, also known as “bottom-up investing”. Unlike top-down investing, which suggests studying the bigger picture of economic factors to make investment decisions, bottom-up investing involves looking at the company-specific fundamentals.
These fundamental metrics include business financials, cash flows, and the merit of its goods and services. This is crucial when investing in the biotech industry, as each company is very unique, requiring niche knowledge to understand its business model.
The fund’s philosophy stands in holding its investments ordinarily for three years, though its higher-conviction investments can be seen held for longer. Additionally, Baker Bros. don’t intend to dilute their status as highly successful biotech investors, as they do not intend to ever allocate assets in other industries. Still, some minor stakes in the industrial sector had been reported in the past.
Finally, the two brothers don’t believe in diversifying the fund’s portfolio. Instead, they emphasize that focusing on specific companies, which they can analyze and understand deeply and place concentrated positions in their securities, can generate superior returns over the long term.
Baker Brothers Investments’ Portfolio & 5 Largest Public-Equity Investments
Upon looking at Baker Bros’ portfolio, one can see that it holds 109 individual stocks, questioning the fund’s disbelief in diversification. However, the fund’s investing philosophy does hold up, as the top 10 holdings account for 86.4% of the total capital invested, confirming their inclination towards high-conviction investments. Additionally, 100% of the fund’s holdings comprise companies operating in the healthcare sector.
Source: 13F filing
Seagen Inc. (SGEN)
Seagen is a biotechnology company that focuses on the development and commercialization of therapies for the treatment of cancer. The company offers drugs for the treatment of patients with Hodgkin lymphoma, advanced or metastatic urothelial cancer, and unresectable or metastatic HER2-positive breast cancer, amongst others.
Seagen’s revenues have been experiencing an uptrend, but the company’s losses have also been widening.
Baker Bros owns around 25.5% of the company, the stock occupies around 38.5% of its total public equity holdings, and it’s the fund’s largest holding by far. The position was held stable during the quarter.
Incyte Corporation (INCY):
Incyte Corporation focuses on the discovery, development, and commercialization of various therapeutics. Its flagship products include JAKAFI, which is a drug for the treatment of myelofibrosis and polycythemia, and Iclusig, a kinase inhibitor to treat chronic myeloid leukemia.
Unlike many biotech companies, which are pre-revenue, Incyte has been growing its top and bottom line for years. Revenues have expanded from around $169 million in 2010 to $3.33 billion over the past four quarters. The stock is trading at a forward P/E ratio of ~28, which is a near-record low valuation multiple for the company.
EPS over the medium-term is expected to grow by around 30% since Incyte is an industry leader, having essentially monopolized its areas of treatment. In that regard, the valuation seems compressed. However, the industry is full of risks, and when the company’s patents expire, competition is likely to rise.
The fund owns around 16.3% of the company, with a market cap of $23 billion. The position was boosted by less than 1% in the previous quarter.
BeiGene, Ltd. (BGNE):
BeiGene is an early commercial-stage biopharmaceutical firm working on developing and commercializing innovative molecularly-targeted and immune-oncology drugs for the treatment of cancer. It is the fund’s second-largest holding, occupying 14.9% of its total portfolio.
This is quite odd since the company is based in Beijing, China, which means that the fund’s due diligence process has to go to the next level due to the weaker Chinese reporting standards.
Despite the uncertainty surrounding BeiGene, the company has developed into a fully integrated global biotechnology company with operations in China, the United States, Europe, and Australia. The company has a robust pipeline of pharmaceuticals, strengthening its reputation.
Nonetheless, BeiGene produces miniature revenues against its $18.7 billion market cap, indicating that investors are betting heavily on the company’s long-term prospects. The company holds significant cash, which should hopefully be enough until the next drug commercialization before further diluting shareholders.
Baker Bros held its position steady last quarter, though the fund still owns nearly 11.4% of the company.
ACADIA Pharmaceuticals Inc. (ACAD):
ACADIA Pharmaceuticals focuses on the development and commercialization of small molecule drugs aimed at unmet medical needs in central nervous system disorders. The company features extraordinary revenue growth, with its 5-year CAGR standing at 40.6%. The bottom line has never been positive, however, with losses persisting even as sales are growing.
In March of 2021, Acadia had announced deficiencies identified by the FDA regarding its marketing application for Pimavanserin in hallucinations and delusions associated with dementia-related psychosis. Shares plunged by a massive 45%, and they have yet to recover since then. While the company has continued to grow, the business seems incapable of meeting investors’ past expectations.
This is one of the fund’s highest conviction picks, as Baker Bros still owns nearly 26% of the company’s shares, which have been held since 2010. While the fund has made great gains since, the recent plunge has definitely compressed its unrealized gains, as the position was held stable once again.
BioMarin Pharmaceutical Inc. (BMRN):
BioMarin Pharmaceutical formulates and markets therapies for people with severe and life-threatening rare diseases and medical ailments. While the company’s revenue growth has seemingly stagnated over the past couple of years, it appears that the company’s development pipeline remains rather strong. Most recently, BioMarin got a positive CHMP opinion in Europe for ValRox for the treatment of Hemophilia type A.
BioMarin has had a place in Baker Bros’ portfolio since Q2 2012. The position was left unchanged during the previous quarter. BioMarin is now Baker Bros’ fifth largest holding, with the fund owning 4.14% of the company’s outstanding shares.
Final Thoughts
The Baker brothers have built a truly special hedge fund. Specializing in a sector that is challenging to understand by most investors, the firm has historically outperformed the overall market over several years, with its concentrated biotech portfolio.
Performance over the past three years has lagged, but it could be a temporary phase for the fund, which, after all, focuses on long-term returns. Investors that are familiar with biotech companies are likely to find some hidden gems amongst their holdings.
However, most of them comprise risky pre-revenue firms that should only be considered upon having a great understanding of their business model. Retail investors should be wary of just “copying” the fund’s portfolio.
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>>> Axsome’s stock falls after disclosing Teva’s plans to market generic Auvelity
Market Watch
Feb. 13, 2023
By Jaimy Lee
https://www.marketwatch.com/story/axsomes-stock-falls-after-disclosing-tevas-plans-to-market-generic-auvelity-12d75af9?siteid=yhoof2
Shares of Axsome Therapeutics Inc. AXSM, -0.97% tumbled about 10.0% in premarket trading on Monday after the company said in a securities filing that Teva Pharmaceutical Industries Ltd. TEVA, +0.39% is seeking approval from the Food and Drug Administration to market a generic version of Auvelity, Axsome’s depression treatment. Axsome’s stock has gained 95.8% over the past year, while the S&P 500 SPX, +1.14% declined 7.6%.
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>>> Axsome Therapeutics -
https://www.fool.com/investing/2023/01/23/2-promising-new-growth-stocks-to-watch-in-2023/?source=eptyholnk0000202&utm_source=yahoo-host&utm_medium=feed&utm_campaign=article
Unlike InMode, Axsome Therapeutics actually beat the bear market last year. The stock soared more than 100%. But there's still room for this growth stock to run. That's because the company is just at the start of its revenue story with two new products. And others may follow in the near future.
Last year, Axsome started selling Sunosi, a sleep disorder drug it bought from Jazz Pharmaceuticals. And it also launched an antidepressant it developed in-house. Though there's a lot of competition in the antidepressant space, Auvelity may stand out because it's fast acting. Auvelity could even reach blockbuster status by the end of the decade, analysts predict.
Now a look at the pipeline. And what's great here is that all of Axsome's programs are phase 2 or farther along. That means they've passed many of the drug development hurdles. And it also means that, if all continues to go well, product launches aren't too far away.
Two to be optimistic about in the near future are candidates for migraine and Alzheimer's disease agitation. Axsome aims to submit the migraine candidate for regulatory approval during the third quarter of this year. And the company recently reported positive date from a phase 3 trial of the Alzheimer's candidate.
All of this means it isn't too late to buy Axsome stock. The company's growth is just revving up -- we should see true acceleration once Axsome starts reporting a few quarters of earnings with its new treatments. And then potentially more growth as other products launch. So Axsome is definitely a stock to watch in 2023 – and beyond.
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>>> Scientists Are Reincarnating the Woolly Mammoth to Return in 4 Years
Popular Mechanics
by Tim Newcomb
January 30, 2023
https://finance.yahoo.com/news/scientists-reincarnating-woolly-mammoth-return-193800409.html
Colossal recently added $60 million in funding to move toward a 2027 de-extinction of the woolly mammoth.
The Dallas-based company is now working to edit the genes for the reincarnation of the mammal.
Colossal planned to reintroduce the woolly mammoth into Russia, but that may shift.
The long-dead woolly mammoth will make its return from extinction by 2027, says Colossal, the biotech company actively working to reincarnate the ancient beast.
Last year, the Dallas-based firm scored an additional $60 million in funding to continue the, well, mammoth gene-editing work it started in 2021. If successful, not only will Colossal bring back an extinct species—one the company dubs a cold-resistant elephant—but it will also reintroduce the woolly mammoth to the same ecosystem in which it once lived in an effort to fight climate change, according to a recent Medium post.
Colossal calls the woolly mammoth’s vast migration patterns an active part of preserving the health of the Arctic, and so bringing the animal back to life can have a beneficial impact on the health of the world’s ecosystem. While Colossal originally hoped to reintroduce the woolly mammoth into Siberia, the company may explore other options based on the current political framework of the world.
The woolly mammoth’s DNA is a 99.6 percent match of the Asian elephant, which leads Colossal to believe it’s well on its way toward achieving its goal. “In the minds of many, this creature is gone forever,” the company says. “But not in the minds of our scientists, nor the labs of our company. We’re already in the process of the de-extinction of the Woolly Mammoth. Our teams have collected viable DNA samples and are editing the genes that will allow this wonderful megafauna to once again thunder through the Arctic.”
Through gene editing, Colossal scientists will eventually create an embryo of a woolly mammoth. They will place the embryo in an African elephant to take advantage of its size and allow it to give birth to the new woolly mammoth. The eventual goal is to then repopulate parts of the Arctic with the new woolly mammoth and strengthen local plant life with the migration patterns and dietary habits of the beast.
If Colossal proves successful on reincarnating the woolly mammoth—ditto the thylacine, also known as the Tasmanian tiger—expect a variety of new ethical questions to arise on how to handle the creature and potential reintroduction issues.
<<<
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>>> Why Amgen Stock Wilted on Wednesday
Motley Fool
By Eric Volkman
Feb 1, 2023
https://www.fool.com/investing/2023/02/01/why-amgen-stock-wilted-on-wednesday/?source=eptyholnk0000202&utm_source=yahoo-host&utm_medium=feed&utm_campaign=article
KEY POINTS
The biotech reported earnings for its final quarter of 2022.
It beat on the top line and met analyst expectations for profitability.
The company's latest quarter wasn't its most impressive.
What happened
The stock of veteran biotech Amgen was looking a little tired on Wednesday. The company's shares fell more than 2% in price, "thanks" to fourth-quarter results that didn't put smiles on investors' faces. In contrast to gloomy Amgen, the S&P 500 index ticked up by slightly more than 1%.
So what
For its fourth quarter of 2022, Amgen's revenue amounted to just under $6.84 billion, which was down marginally from the same period the previous year. There was a greater difference in non-GAAP (adjusted) net income -- this fell by 11% across the one-year stretch to slightly more than $2.2 billion, or $4.09 per share.
On average, analysts tracking Amgen stock were estimating that the company would book $6.74 billion on the top line. Their collective estimate for adjusted net income was $4.09, matching reality.
Amgen is a sprawling biotech, with many approved products and quite a wide pipeline. Standout commercialized drugs for the biotech during the quarter included osteoporosis treatment Prolia, which saw its sales climb 14% year over year to set an all-time quarterly record of $992 million. Other encouraging increases were notched by cardiovascular drug Repatha (up 22% to $333 million) and another osteoporosis treatment, Evenity (57% higher to $225 million, a new record).
Now what
But investors want overall growth for Amgen, and that didn't happen during the quarter. Compounding that, the company proffered full-year 2023 guidance of $26 billion to $27 billion in revenue, and per-share, adjusted net profit of $17.40 to $18.60. The former fell short of the average analyst projection of $27.3 billion for revenue, while the latter barely contains the prognosticators' $18.33 estimate.
Amgen's guidance does not include the results of Horizon Therapeutics, which it agreed to acquire last December in a nearly $28 billion deal.
<<<
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The Dealreporter is reporting that the FSTX deal is not going through due to CIFUS delaying action, citing sources close to InvoX, the company buying FSTX. The stock is down well below $4/share. (This will be the final nail in my coffin.) FSTX has four drugs in clinical trials, so maybe some company will step up to the table. But FSTX is very short on cash.
If, by chance, the report is wrong and allows certain investors to get in cheap before the buyout at $7.11 on the 31st, then this will be one of biggest (coolest?) scams in all time.
I’m trying to get in touch with management. Stock down 40%.
Bladerunner
JSPR announces pricing of public offering: 60 million shares @ $1.50/share. JSPR now has approximately 100 million shares outstanding. The offering gets them another two years to advance Briquilimab. Perhaps, they now have a little leverage to negotiate ex-U.S. rights in MDS/AML.
GlobeNewswire
Jasper Therapeutics Announces Pricing of $90 Million Public Offering of Common Stock
Jasper Therapeutics
Wed, January 25, 2023 at 5:00 AM PST
In this article:
JSPR
+1.4793%
Jasper Therapeutics
Jasper Therapeutics
REDWOOD CITY, Calif., Jan. 25, 2023 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (“Jasper”), a biotechnology company focused on developing novel antibody therapies targeting c-Kit (CD117) to address diseases such as chronic spontaneous urticaria and lower to intermediate risk myelodysplastic syndromes (MDS) as well as novel stem cell transplant conditioning regimes, announced today the pricing of its underwritten public offering of 60,000,000 shares of its common stock. Each share of common stock will be sold at a public offering price of $1.50 per share, for gross proceeds of approximately $90 million, before deducting underwriting discounts and commissions and offering expenses. Jasper intends to use the net proceeds from the offering for general corporate purposes, which may include capital expenditures, working capital and general and administrative expenses. In addition, Jasper has granted the underwriters a 30-day option to purchase up to 9,000,000 additional shares of its common stock, at the public offering price, less underwriting discounts and commissions. All of the shares of common stock are being offered by Jasper. The offering is expected to close on or about January 27, 2023, subject to the satisfaction of customary closing conditions.
Credit Suisse Securities (USA) LLC and William Blair & Company, L.L.C. are acting as the active book-running managers for the proposed offering. Oppenheimer & Co. Inc. is acting as a passive book-running manager for the proposed offering.
The securities described above were offered by Jasper pursuant to an effective “shelf” registration statement on Form S-3 (File No. 333-267777) previously filed with the Securities and Exchange Commission (the “SEC”) on October 7, 2022 and declared effective by the SEC on October 18, 2022. The securities may be offered only by means of a prospectus. A preliminary prospectus supplement and the accompanying prospectus relating to and describing the offering has been filed with the SEC. Electronic copies of the preliminary prospectus and, when available, copies of the final prospectus supplement and the accompanying prospectus relating to the offering may be obtained by visiting the SEC’s website at www.sec.gov or by contacting Credit Suisse Securities (USA) LLC, Attention: Prospectus Department, One Madison Avenue, New York, NY 10010, by calling toll-free (800) 221-1037 or by emailing newyork.prospectus@credit-suisse.com, William Blair & Company, L.L.C., Attention: Prospectus Department, 150 North Riverside Plaza, Chicago, IL 60606, by calling toll-free (800) 621-0687, or by emailing prospectus@williamblair.com or Oppenheimer & Co. Inc., Attention: Syndicate Prospectus Department, 85 Broad Street, 26th Floor, New York, NY 10004, or by telephone at (212) 667-8055, or by email at EquityProspectus@opco.com.
About Jasper
Jasper is a clinical-stage biotechnology company developing briquilimab, a monoclonal antibody targeting c-Kit (CD117) as a therapeutic for chronic mast and stem cell diseases such as chronic urticaria and lower to intermediate risk myelodysplastic syndromes (MDS) and as a conditioning agent for stem cell transplants for rare diseases such as sickle cell disease (SCD), Fanconi anemia (FA) and severe combined immunodeficiency (SCID). To date, briquilimab has a demonstrated efficacy and safety profile in over 130 dosed subjects and healthy volunteers, with clinical outcomes as a conditioning agent in SCID, acute myeloid leukemia (AML), MDS, FA, and SCD. In addition, briquilimab is being advanced as a transformational non-genotoxic conditioning agent for gene therapy.
Bladerunner
MRSN initiated at Citi with a $12 price target.
With Upcoming Ovarian Cancer Data, Analyst Sees 100% Upside For Mersana Therapeutics
Vandana Singh
Fri, January 20, 2023 at 12:16 PM PST
In this article:
MRSN
+9.97%
Citi initiated coverage on Mersana Therapeutics Inc (NASDAQ: MRSN) with a Buy rating and a price target of $12.
The analyst says the topline registrational data (expected in mid-2023) from the UPLIFT Ph2 study evaluating upifitamab rilsodotin (UpRi; NaPi2b ADC) in platinum-resistant ovarian cancer (PROC) will be a catalyst of significant debate.
Citi analyst says that while readout will be binary, investors are underappreciating UPLIFT’s probability of success in NaPi2b-high patients, given the promising efficacy observed in Phase 1.
If the UPLIFT study results are positive, it will lead to a clear pathway to an accelerated approval filing by YE23.
The analyst writes that UpRi’s durability in Phase 1 was in-line or better than single-agent chemo SOC and the recently approved ADC Elahere.
On toxicity, reduced dosing and patient selection strategies should mitigate the risk of Gr4/5 pneumonitis seen earlier in Phase 1.
In December, Mersana announced its third significant collaboration agreement in 2022, reflecting its increasing role as an ADC partner.
The company announced a research collaboration and commercial license agreement with a subsidiary of Merck KGaA (OTC: MKKGY) to discover novel Immunosynthen ADCs directed against up to two targets.
Mersana received an upfront payment of $30 million and is eligible to receive reimbursement of certain costs, up to $800 million in potential milestone payments.
Price Action: MRSN shares are up 10.48% at $6.43 on the last check Friday. (Up more in AH.)
Bladerunner
Axsome - >>> 2 Stocks That Could Double Your Money in 2023
Motley Fool
By Prosper Junior Bakiny
Jan 7, 2023
https://www.fool.com/investing/2023/01/07/2-stocks-that-could-double-your-money-in-2023/?source=eptyholnk0000202&utm_source=yahoo-host&utm_medium=feed&utm_campaign=article
KEY POINTS
Progress on Axsome's depression therapy efforts could help it deliver solid returns ahead.
Bluebird Bio recently earned approval for innovative treatments for two rare illnesses.
Earning outsize returns in the stock market over a short period can be exciting, but doing so over the long run is even better. Can some companies provide both? Yes, especially those relatively small corporations that have yet to become household names. After all, many currently well-established companies were once small- or mid-cap stocks.
That's why it's worth keeping an eye on promising corporations on the smaller side. With that as a backdrop, let's look at two stocks along those lines: Axsome Therapeutics (AXSM 1.16%) and Bluebird Bio (BLUE -14.35%). These biotechs could deliver excellent returns this year, but it's essential to look carefully at their risk-reward profiles before pulling the trigger.
1. Axsome Therapeutics
Axsome Therapeutics is a mid-cap drugmaker coming off a solid performance in 2022, during which its shares more than doubled. The company could carry this momentum into this year. Axsome is launching Auvelity, a fast-acting treatment for depression that earned approval in August. And there are other catalysts on the way for the biotech.
The company is currently running a phase 3 study for AXS-12 as a potential treatment for narcolepsy (a sleep disorder); it expects data from this trial in the first half of this year. Positive results could help move the company's shares in the right direction.
Elsewhere, Axsome is planning a regulatory submission for another candidate, AXS-14, in treating fibromyalgia. This disorder is characterized by symptoms such as pain, fatigue, and sleep problems. Axsome will also resubmit its potential treatment for acute migraines, AXS-07, to U.S. regulators.
In May, the Food and Drug Administration (FDA) declined to approve AXS-07 due to manufacturing issues. But after discussing the matter with the agency, Axsome Therapeutics plans on having a second go at this therapy, with the resubmission planned for the third quarter.
There is still more news from the company's pipeline. In November, Axsome released positive results from a phase 3 study for Auvelity in treating Alzheimer's disease (AD) agitation, which is characterized by anxiety, aggressive behavior, and emotional distress.
Patients treated with the medicine showed statistically significant improvement in AD agitation compared to those treated with a placebo. Axsome Therapeutics started another late-stage study for Auvelity in AD agitation in September. The potential here is vast. There are roughly 6.5 million AD patients in the U.S., up to 70% of whom display symptoms of agitation.
Yet, no treatment is approved for it. Auvelity still has a long way to go before earning this indication as Axsome Therapeutics is planning a data readout of the second late-stage study in 2025. Still, it helps highlight the biotech's long-term potential. The company has a newly approved therapy, and multiple exciting candidates that could expand its lineup within a couple of years, some of which, including Auvelity, could earn various label expansions.
Axsome Therapeutics looks like a solid pick this year as it could again deliver excellent returns thanks to several potential catalysts. And even for patient long-term investors, the biotech is worth serious consideration.
2. Bluebird Bio
Late last year, small-cap biotech Bluebird Bio earned U.S. approval for Zynteglo and Skysona, which are gene-editing treatments for transfusion-dependent beta-thalassemia (TDT, a rare blood disease) and cerebral adrenoleukodystrophy (CALD, a progressive neurological disease), respectively. The biotech is in the process of launching these therapies.
Also, the company is eyeing another regulatory submission this year. Bluebird plans to request FDA approval for lovo-cel in the first quarter. Lovo-cel is a potential treatment for sickle cell disease, a blood-related disorder that causes red blood cells to turn crescent-shaped, eventually leading to a lower count of red blood cells, which can cause serious health complications.
So far, all of the illnesses Bluebird has targeted have very few safe and effective treatment options. There is no question that the company has been an innovator. And if money starts rolling in from Zynteglo and Skysona -- and the company moves forward with lovo-cel -- investors could reward the biotech this year, especially considering its tiny market capitalization of $575 million. At these levels, Bluebird's stock could indeed soar if the stars align.
But Bluebird's prospects remain uncertain. The rollout of its two approved gene-editing treatments will likely be slow, given they can only be administered in qualified treatment centers with the equipment and trained staff to work with gene editing therapies. The process of manufacturing Zynteglo is done for each specific patient using the person's collected cells sent to a specialized lab.
Once manufactured, the therapy is inserted back into the patient via intravenous infusion. That's the short version. And even then, it sounds like a long and complicated process. The complexity of its treatments could be a significant roadblock for Bluebird. As could the price tags: Zynteglo and Skysona go for $2.8 million and $3 million, respectively.
If third-party payers are willing to pay these exorbitant prices, enough qualified treatment centers get ready to administer Skysona and Zynteglo, and the company treats a reasonable number of patients while lovo-cel gets approved, its shares will perform very well from here on out. But that's a lot of "ifs," and the slightest misstep could sink the company's stock, making it a risky bet.
Investors should keep that in mind before even considering initiating a small position in this biotech company.
<<<
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Blade, Thanks. I hope they don't mind if I post the list here (below). It should be fun to check these out :o)
https://www.siliconinvestor.com/readmsg.aspx?msgid=34141890
A.J. MULLEN
MRTX
NBIX
ONT.L
PACB
SCLP.L
TDOC
TWST
XENE
-----------------------------
ALONER
AFMD
CDTX
CLSD
CRDF
CRIS
CRVS
KZIA
MGTA
MRNA
PRVB
-----------------------------------
ARTHUR RADLEY
ABCM
AKYA
ALVR
ASRT
EYEN
IDIA.SW
ISEE
LQDA
---------------------------------
BLADERUNNER
CRDF --- (20%)
CRIS --- (15%)
ENTA --- (5%)
IOVA --- (10%)
JSPR --- (20%)
MBIO --- (10%)
MGTA --- (5%)
MRSN --- (15%)
----------------------------
BMAZ001
AADI
ACRS
AVTX
BCAB
ELYM
GTHX
LIAN
LRMR
PDSB
PIRS
PRQR
-----------------------------
BULBAMAN
ATXS
BFLY
FDMT
IMTX
OMER
PTE
RFL
TCON
VOR
----------------------------------
DEW DILIGENCE
ALGS
ASMB
ENTA --- (20%)
IMGO
ORIC
PMGM
PRDS
RVNC --- (20%)
---------------------------
ERIKOTTO
CABA
DAWN
FULC
ICVX
INZY
LIFE
OCUL
PDSB
PRQR
URGN
---------------------------------
GENEGURU
AXSM
CMRX
DCTH
GLYC
INVA
MODD
PRQR
XAIR
-------------------------------------
JACK HARTMANN
AMRN
GMDA
HUMA
ICPT
IOVA
LYEL
NGN
PDSB
RBOT
STTK
-------------------------------
RAJU_BIJLEE
ALLO
ALT
ATRA
BCDA
BLCM
FATE
IMTX
IOVA
MBIO
TGTX
----------------------------
RKRW
ETON
HROW
KALV
KPTI
MOR
OCUL
PHAT
TCON
TELA
URGN
---------------------------------
STEVE LOKNESS
AKYA
CRBU
GRPH
MGTA
MRUS
NAUT
PACB
PSNL
QSI
-----------------------
TECHNETIUM
APTA
DAWN
ENTA
KRYS
LLY
MCRB
MDGL
QURE
RVNC
VKTX
------------------------
TOMATO
ETNB
ETON
HROW
OCUL
PTE
TARA
TCON
TELA
<<<
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SI Charity Biotech Stock Picking Contest. See the stocks chosen.
https://www.siliconinvestor.com/readmsg.aspx?msgid=34141890
Bladerunner
re: KZIA
gfp, I think you're right; the Australian exchanges seem a little more liberal with their use of the halt than their U.S. counterparts.
Bladerunner
Blade, With Kazia, just curious if halting of stock trading is typical for Australian stocks when they do a financing? Seems unusual, though I haven't followed many Aussie stocks in the past.
The company only has ~7 mil in cash (per Yahoo), so a financing would make sense, especially since the stock has doubled off of the Dec bottom, and they've had a burn rate ~ 24 mil/yr. But without the halt, it may have reached 1.50 or more, based on the chart anyway.
Guess we'll find out more on the 16th..
---
Full article - >>> Jasper Therapeutics Announces New Positive Briquilimab Data to be Presented at the 2023 Tandem Meetings: Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR
Jasper Therapeutics
January 13, 2023
https://finance.yahoo.com/news/jasper-therapeutics-announces-positive-briquilimab-130000761.html
Three abstracts highlight the safety and efficacy of briquilimab combined with low-toxicity radiation conditioning to achieve full donor engraftment and leukemia disease eradication
Data demonstrates that briquilimab is safe and well-tolerated as a conditioning agent in older patients, median age 70, with AML/MDS undergoing allogeneic hematopoietic cell transplantation
Durable remissions achieved in 8 of the first 12 AML patients treated at one-year follow up
Lower than expected rates of severe acute and chronic graft-vs-host disease in 29 AML and MDS patients
Report of 12 study patients treated at a single center who underwent outpatient conditioning and donor cell transplant, which is associated with lower hospital resource use
REDWOOD CITY, Calif., Jan. 13, 2023 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (“Jasper” or the “Company”), a biotechnology company developing novel antibody therapies addressing chronic diseases such as urticaria, lower-risk myelodysplastic syndromes (MDS) and stem cell transplant conditioning agents targeting c-Kit, today announced that new positive data for briquilimab (formerly known as JSP191), will be presented at the 2023 Tandem Meetings: Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, taking place on February 15-19, 2023 in Orlando, Florida.
Three abstracts, covering data related to the Phase 1 study of briquilimab in combination with fludarabine and low-dose irradiation (Flu/TBI) conditioning in older adults (62 to 79 years) with acute myeloid leukemia (AML) or MDS undergoing allogeneic hematopoietic cell transplant (HCT), will be presented. The studies demonstrate that a regimen of briquilimab plus Flu/TBI leads to successful engraftment of donor blood stem cell without the usual short and long-term toxicities that accompany alternative busulfan-based regimens commonly used in transplant of donor or gene-corrected cells. Based on its mechanism of action, briquilimab is known to potently synergize with radiation, amplifying its stem cell depleting effects without increasing off-target toxicity.
The first abstract demonstrates that briquilimab was safe, well-tolerated, and achieved durable remissions in 8 of 12 of the first treated AML patients. All 8 patients were relapse-free at one-year follow up. Six of 9 patients who entered transplant with detectable AML, a group known to have a poor prognosis with high relapse rates, showed long-term eradication of the AML clones at one-year. In a companion abstract, the total group of 29 AML and MDS patients treated with briquilimab and Flu/TBI demonstrated lower than expected rates of acute and chronic graft-versus-host disease (GVHD). The third abstract, to be presented in the Best Abstract session, evaluated the costs and healthcare utilization of 12 briquilimab plus Flu/TBI study patients who received outpatient conditioning and donor cell transplant at a single study center. During the first 100 days post-procedure there were a total of 7 hospitalizations in the 12 patients, with an overall mean stay of 4 days. These results demonstrate the feasibility and potential significant cost savings of outpatient briquilimab plus Flu/TBI conditioning followed by outpatient donor cell transplant in older patients with AML or MDS.
“Our data presentations at the ASTCT meeting add to the significant body of clinical evidence supporting the safety and clinical potential of briquilimab in a variety of indications and patient types,” said Ronald Martell, President and Chief Executive Officer of Jasper. “While we are focusing our near-term resources on the development of briquilimab for chronic diseases and as a conditioning agent for stem cell transplants addressing rare diseases, we believe these data demonstrate that briquilimab is an agent that can markedly improve the safety and efficacy of stem cell transplants for a wide range of malignant and rare diseases.”
Abstract details:
Title:
Subanalysis from Phase 1 Study of JSP191, an Anti-CD117 Monoclonal Antibody, in Combination with Low Dose Irradiation and Fludarabine Conditioning, Shows Durable Remissions in Older Adults with Acute Myeloid Leukemia in Complete Remission Undergoing Allogeneic Hematopoietic Cell Transplantation
Author:
Lori Muffly, MD, MS, Stanford University School of Medicine
Abstract #:
21934 (oral presentation)
Title:
Immune Biomarkers Associated with Chronic GVHD in Phase 1 Study of JSP191, an AntiCD117 Monoclonal Antibody, in Combination with Low Dose Irradiation and Fludarabine Conditioning in Older Adults with MDS/AML Undergoing Allogeneic HCT
Author:
Minyoung Youn, PhD, Jasper Therapeutics, Inc.
Abstract #:
21949 (poster presentation)
Title:
Evaluation of Clinical Outcomes and Healthcare Resource Use of Outpatient Allogeneic Stem Cell Transplant in Older Adults with AML/MDS, Using JSP191, an AntiCD117 Monoclonal Antibody, in Combination with Low Dose Irradiation and Fludarabine Conditioning – a Single Center Analysis
Author:
Lori Muffly, MD, MS, Stanford University School of Medicine
Abstract #:
22152 (oral presentation)
About Briquilimab (formerly known as JSP191)
Briquilimab is a targeted, monoclonal antibody that inhibits the cell-surface receptor c-Kit, also known as CD117. It is currently being evaluated as a primary therapeutic for mast cell diseases such as chronic spontaneous urticaria (CSU), chronic inducible urticaria (CIndU), and allergic asthma, and for lower-risk MDS patients. It is also being studied as a conditioning agent for cell and gene therapies for rare diseases. To date, briquilimab has a demonstrated efficacy and safety profile in 130 dosed subjects and healthy volunteers, with clinical outcomes as a conditioning agent in severe combined immunodeficiency (SCID), acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), Fanconi anemia (FA), and sickle cell disease (SCD). In addition, briquilimab is being advanced as a transformational non-genotoxic conditioning agent for gene therapy.
About Jasper
Jasper is a clinical-stage biotechnology company developing novel antibody therapies and stem cell transplant conditioning agents targeting c-Kit (CD117), an important receptor found on stem cells and mast cells. The Company’s lead program is briquilimab, a first-in-class monoclonal antibody being developed as a therapeutic for chronic diseases and as a conditioning agent for stem cell transplants for rare diseases. For more information, please visit us at jaspertherapeutics.com.
<<<
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JSPR news out https://finance.yahoo.com/news/jasper-therapeutics-announces-positive-briquilimab-130000761.html
Stock up 44%.
Bladerunner
KZIA has been halted until January 16th, 2023. Possibly a financing of some sort.
Bladerunner
KZIA getting a nice rebound :o)
10 consecutive days above $1 and they will regain compliance with Nasdaq-CM
https://finance.yahoo.com/news/kazia-announces-receipt-nasdaq-minimum-120100697.html
---
Nice - >>> Revance Provides an Update on DAXXIFY® Commercial Launch and Preliminary Fourth Quarter and Full Year 2022 Financial Results
BusinessWire
January 9, 2023
https://finance.yahoo.com/news/revance-provides-daxxify-commercial-launch-130000788.html
DAXXIFY® PrevU program off to strong start with ~400 select practice partners and thousands of patients treated, generating positive feedback and strong, early uptake
Preliminary unaudited Q4 DAXXIFY® revenue from PrevU of between $10.5 million and $11.5 million
Preliminary unaudited Q4 RHA® Collection revenue of between $34.0 million and $35.0 million, a YoY increase of approximately 45%
Aesthetic accounts across products and services totaled over 5,000 at the end of the fourth quarter 2022
NASHVILLE, Tenn., January 09, 2023--(BUSINESS WIRE)--Revance Therapeutics, Inc. (RVNC) today provided an update on its early experience program for DAXXIFY® and its preliminary unaudited fourth quarter and full year 2022 financial results.
Financial Update:
Preliminary Unaudited Fourth Quarter and Full Year 2022 RHA® Collection Revenue. Revance expects preliminary unaudited fourth quarter 2022 RHA® Collection revenue to be between $34.0 million and $35.0 million, representing an approximately 45% increase from the same period last year, and full year 2022 preliminary unaudited RHA® Collection revenue of between $106.5 million and $107.5 million, representing an approximately 50% increase from full year 2021.
Preliminary Unaudited Fourth Quarter 2022 DAXXIFY® Revenue. Revance expects preliminary unaudited DAXXIFY® revenue from PrevU, its early experience program, to be between $10.5 million and $11.5 million in the fourth quarter 2022, the first quarter of limited commercial availability.
Preliminary Unaudited Fourth Quarter and Full Year 2022 Service Revenue. Revance expects preliminary unaudited fourth quarter 2022 service revenue from OPUL® and the legacy HintMD fintech platform to be between $2.5 million and $3.5 million and full year 2022 preliminary unaudited service revenue of between $6.5 million and $7.5 million.
Preliminary Unaudited Full Year 2022 Operating Expenses. Revance expects to be within its previously announced generally accepted accounting principles (GAAP) operating expense guidance range of between $375 million to $400 million and to be on the upper end of its previously announced Non-GAAP operating expense guidance range of between $260 million to $280 million. The company expects to provide its 2023 GAAP and Non-GAAP operating expense guidance in its fourth quarter 2022 earnings announcement.
Cash Update. Preliminary unaudited cash, cash equivalents and short-term investments as of December 31, 2022 were approximately $340.0 million.
"We are very pleased to end 2022 on a strong note, highlighted by continued growth and adoption of the RHA® Collection along with an excellent start to our early experience program for DAXXIFY®," said Mark J. Foley, Chief Executive Officer of Revance. "In particular, we are very encouraged to see the early traction of our DAXXIFY® PrevU program, which was initiated in Q4 and further benefited from the impact of traditional seasonality. We have been very pleased with the initial response to, and the positive feedback on, the product’s performance and ease of practice integration. While we are still in the beginning phase of the PrevU program, our progress thus far gives us confidence in our commercial strategy and in DAXXIFY’s full-scale commercial launch, which is expected to begin in late Q1/early Q2 of 2023. Looking ahead, our strategic priorities in the new year will center on delivering a successful commercial launch for DAXXIFY®, continuing the growth of our aesthetics portfolio, and unlocking our therapeutics opportunity by obtaining the FDA’s approval of DAXXIFY® for cervical dystonia."
"Building on the success of our launch of the RHA® Collection of dermal fillers, we initiated a strategic, phased roll out of DAXXIFY® with our PrevU program to approximately 400 select practices," said Dustin S. Sjuts, President of Revance. "With our core objectives of ensuring outstanding aesthetic outcomes and smooth practice integration of DAXXIFY® while also leveraging its unique value proposition, we are very pleased with the comprehensive training and education that we have provided to a group of early adopters at our Nashville headquarters. Further, as we executed this program during the busiest time of the year for aesthetic procedures, and through planned injection events held by practices, we generated real-world clinical insights from hundreds of injectors and thousands of patients, which will be invaluable to our commercial launch. We are very pleased with the results of our strategy and execution and look forward to completing PrevU while also preparing for our sales force expansion which we anticipate will happen by mid-year"
DAXXIFY® PrevU Program:
PrevU is an early experience program that focuses on product education, practice integration, and real-world clinical insights for optimizing aesthetic outcomes. Following the U.S. Food and Drug Administration’s approval of DAXXIFY® in September 2022, Revance trained a group of 20 U.S.-based faculty members who had access to the product prior to the initiation of the PrevU program. In December, Revance launched PrevU with approximately 400 select practices. These practices were invited to attend training sessions held over the first two weeks of December at the company’s Nashville headquarters and experience center that was led by the faculty trainers. Following onsite training, practices held planned patient injection days and events to drive product utilization and gain real-world clinical insights. PrevU training and education was also expanded to the additional injectors and staff of these practices through virtual sessions. These practices will continue to provide additional clinical feedback on the product through late Q1/early Q2 2023, after which the commercial launch of DAXXIFY® is expected to begin. Revance expects to launch the product first to its existing base of over 5,000 aesthetic accounts.
INDICATION
DAXXIFY® (DaxibotulinumtoxinA-lanm) for injection is an acetylcholine release inhibitor and neuromuscular blocking agent indicated for the temporary improvement in the appearance of moderate to severe glabellar lines associated with corrugator and/or procerus muscle activity in adult patients.
WARNING: DISTANT SPREAD OF TOXIN EFFECT
The effects of DAXXIFY® and all botulinum toxin products may spread from the area of injection to produce symptoms consistent with botulinum toxin effects. These symptoms have been reported hours to weeks after injection. Swallowing and breathing difficulties can be life threatening and there have been reports of death. DAXXIFY® is not approved for the treatment of spasticity or any conditions other than glabellar lines.
IMPORTANT SAFETY INFORMATION
Contraindications
DAXXIFY® contraindications include hypersensitivity to any botulinum toxin preparation or any of the components in the formulation and infection at the injection site(s).
Warnings and Precautions
Please refer to Boxed Warning for Distant Spread of Toxin Effect.
The potency units of DAXXIFY® are not interchangeable with other preparations of other botulinum toxin products. Recommended dose and frequency of administration should not be exceeded. Patients should seek immediate medical attention if respiratory, speech or swallowing difficulties occur. Use caution when administering to patients with pre-existing cardiovascular disease. Concomitant neuromuscular disorders may exacerbate clinical effects of treatment.
Adverse Reactions
The most commonly observed adverse reactions (≥1%) were headache (6%), eyelid ptosis (2%) and facial paresis (1%).
Drug Interactions
Co-administration of DAXXIFY® and aminoglycoside antibiotics, anticholinergic agents or any other agents interfering with neuromuscular transmission or muscle relaxants should only be performed with caution as the effect of DAXXIFY® may be potentiated. The effect of administering different botulinum neurotoxins during course of treatment with DAXXIFY® is unknown.
Use in Specific Populations
DAXXIFY® is not recommended for use in children or pregnant women.
Please see DAXXIFY® full Prescribing Information, including Boxed Warning and Medication Guide.
About DAXXIFY®
DAXXIFY® (DaxibotulinumtoxinA-lanm) for injection is the first and only FDA approved long-lasting peptide-formulated neuromodulator product for use in adults for the temporary improvement of moderate to severe frown lines (glabellar lines).1-2,7-11 DAXXIFY® has the ability to deliver year-long results for patients with potentially only two treatments per year and has been proven to be effective, and generally safe and well tolerated.2-5* DAXXIFY® is powered by a cell-penetrating peptide technology (Peptide Exchange Technology™), Revance's proprietary, synthetic, 35-amino-acid stabilizing excipient with a highly positive charge, and is free of human serum albumin or animal-based components.1,2,11 Manufactured exclusively in the U.S., DAXXIFY® is the first true innovation in neuromodulator product formulation in over 30 years. Revance has evaluated this neuromodulator formulation in other Phase 2 clinical studies in aesthetics, including the full upper face, forehead lines and crow’s feet as well as in therapeutic indications, including cervical dystonia and upper limb spasticity. Learn more at RevanceAesthetics.com.
About Revance
Revance is a biotechnology company setting the new standard in healthcare with innovative aesthetic and therapeutic offerings that elevate patient and physician experiences. Revance’s aesthetics portfolio of expertly created products and services, including DAXXIFY® (DaxibotulinumtoxinA-lanm) for injection, the RHA® Collection of dermal fillers, and OPUL®, the first-of-its-kind Relational Commerce platform for aesthetic practices, deliver a differentiated and exclusive offering for the company’s elite practice partners and their consumers. Revance has also partnered with Viatris Inc. to develop a biosimilar to BOTOX®, which will compete in the existing short-acting neuromodulator marketplace. Revance’s therapeutics pipeline is currently focused on muscle movement disorders including evaluating DAXXIFY® in two debilitating conditions, cervical dystonia and upper limb spasticity.
Revance is headquartered in Nashville, Tennessee, with additional office locations in Newark, Pleasanton and Irvine, California. Learn more at www.Revance.com or connect with us on LinkedIn.
"Revance" and the Revance logo, DAXXIFY®, and OPUL® are registered trademarks of Revance Therapeutics, Inc.
Resilient Hyaluronic Acid® and RHA® are trademarks of TEOXANE SA.
BOTOX® is a registered trademark of Allergan, Inc.
Data on File. DAXXIFY® Package Insert. Newark, CA: Revance Therapeutics, Inc, 2022.
Fabi SG, Cohen JL, et al. DaxibotulinumtoxinA for Injection for the treatment of glabellar lines: efficacy results from SAKURA 3, a large, open-label, phase 3 safety study. Dermatol Surg. 2020. doi:10.1097/DSS.0000000000002531.
Carruthers JD, Fagien S, et al. DaxibotulinumtoxinA for Injection for the treatment of glabellar lines: results from each of two multicenter, randomized, double-blind, placebo-controlled, phase 3 studies (SAKURA 1 and SAKURA 2). Plast Reconstr Surg. 2020;145(1):45-58.
Bertucci V, Solish N, et al. DaxibotulinumtoxinA for Injection has a prolonged duration of response in the treatment of glabellar lines: pooled data from two multicenter, randomized, double-blind, placebo-controlled, phase 3 studies (SAKURA 1 and SAKURA 2). J Am Acad Dermatol. 2020;82(4):838-845.
Green JB, Mariwalla K, Coleman K, et al. A large, open-label, phase 3 safety study of daxibotulinumtoxinA for injection in glabellar lines: a focus on safety from the SAKURA 3 study. Dermatol Surg. 2020. doi:10.1097/DSS.0000000000002463.
Data on File. Protocols 1620301-303. Post Hoc Analysis. Newark, CA: Revance Therapeutics, Inc, 2021.
Xeomin. Prescribing Information. Merz Pharmaceuticals GmbH; 2020.
Dysport. Prescribing Information. Ipsen Biopharmaceuticals, Ltd; 2020.
Jeuveau. Prescribing Information. Evolus, Inc; 2020.
Botox Cosmetic. Prescribing Information. Allergan, Inc; 2020.
Waugh JM, Lee J, Dake MD, Browne D. Nonclinical and clinical experiences with CPP-based self-assembling peptide systems in topical drug development. Methods Mol Biol. 2011; 683:553-572.
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>>> Apexigen Inc. (APGN), a clinical-stage biopharmaceutical company, discovers and develops antibody therapeutics for oncology. It develops a pipeline of product candidates, including APX005M, a humanized agonist antibody that is in Phase II clinical development for the treatment of solid tumors, such as melanoma, esophageal and gastroesophageal junction, sarcoma, and rectal and ovarian cancers in combination with immunotherapy, chemotherapy, radiation therapy, and cancer vaccines; and APX601, a humanized antagonist antibody that is in Phase I/II clinical trial for the treatment of multiple tumor indications, as well as APX801, an NK cell engager to activate natural killer cells to killing of tumor cells. The company was founded in 2010 and is based in San Carlos, California.
https://finance.yahoo.com/quote/APGN/profile?p=APGN
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re: APGN
Looks interesting. Up 80% in pre-Market, but still only $19 million MC
EF Hutton analyst Tony Butler initiated coverage of Apexigen with a Buy rating and $8 price target. The "recently de-SPACed" biotech is developing sotigalimab as an orthogonal immunoactivating therapy in combination with immune checkpoint inhibitors and chemotherapy against a number of cancer indications, Butler tells investors.
Bladerunner
Blade, Looks like the armed MAB technologies have progressed a lot since the IMGN days :o)
Mersana - >>> DolaLock Technology
https://www.mersana.com/our-technology-platforms/dolalock-technology/
Once ADCs release their cytotoxic payload into targeted cells, the drug is often able to cross cell membranes, entering and potentially killing neighboring cells whether those cells are cancerous or not. This effect is called bystander-killing, which is advantageous when bystander cells are cancerous but toxic if the cytotoxic drug is able to enter adjacent healthy cells.
The DolaLock payload controls the bystander effect by locking the cytotoxic drug inside cells after allowing a short period of diffusion throughout the tumor. As the drug diffuses through neighboring tissue, the DolaLock payload is metabolized to a form that is still highly potent but no longer able to cross the cell membrane. DolaLock effectively locks the drug inside cells, controlling the bystander effect for a safer and more effective cancer therapy.
Our DolaLock payload is a proprietary auristatin cytotoxic drug and is a highly potent anti-tubulin agent selectively toxic to rapidly dividing cells.
A common mechanism of resistance in cancer is the up-regulation of multi-drug resistance (MDR) pumps, such as PgPs, which actively pump drugs out of cancer cells to help them survive. Our DolaLock payload cannot be pumped out by PgPs, thereby avoiding this resistance mechanism. In addition, our proprietary auristatin payload has also been shown in preclinical tests to cause immunogenic cell death and to stimulate the immune system through dendritic cell activation. Because of this, synergy with immuno-oncology agents such as PD-1 inhibitors has been observed in preclinical models. Our DolaLock payload with controlled bystander effect allows us to create ADCs that produce a highly potent, well-tolerated, and specifically-targeted cancer therapy.<<<
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MRSN provides a business update and milestones
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• MRSN
+4.17%
Mersana Therapeutics, Inc.
• Report top-line data from UPLIFT registrational trial in mid-2023, submit potential BLA around year end 2023, and prepare for potential U.S. commercial launch in 2024
• Advance Phase 3 UP-NEXT and Phase 1 UPGRADE-A trials in platinum-sensitive ovarian cancer
• Advance XMT-1660 and XMT-2056 Phase 1 trials
• Continue pursuing collaborations to maximize platform and pipeline potential
• Capital resources expected to support operating plan commitments into the second half of 2024
CAMBRIDGE, Mass., Jan. 06, 2023 (GLOBE NEWSWIRE) -- Mersana Therapeutics, Inc. (NASDAQ: MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, today provided a business update and announced strategic objectives and expected milestones for 2023.
“Following a year of tremendous accomplishment in 2022, we are now approaching top-line data from our first registrational trial of UpRi, which we believe will provide an opportunity to further demonstrate Mersana’s increasing role as an ADC leader,” said Anna Protopapas, President and Chief Executive Officer of Mersana Therapeutics. “The data we expect to report from our UPLIFT clinical trial in mid-2023 will represent the most significant milestone to date in our effort to establish UpRi as a foundational medicine for patients with ovarian cancer. Assuming positive data, we plan to target the submission of a BLA around the end of 2023 and prepare for a potential U.S. commercial launch in 2024. We will also continue to advance our UP-NEXT and UPGRADE-A trials of UpRi in earlier lines of treatment.”
“Beyond UpRi, our efforts to expand the reach of all three of our fully-scaled ADC platforms, each supported by substantial data, will remain a central theme in 2023,” continued Ms. Protopapas. “We will work aggressively to progress our two next-generation ADCs, XMT-1660 and XMT-2056, in Phase 1 trials and establish proof-of concept. Additionally, exploring new collaborations will remain a core component of our strategy as we seek to build upon Mersana’s recent business development successes.”
Strategic Objective: Establish UpRi as a Foundational Medicine in Ovarian Cancer
2022 Accomplishments
• Completed enrollment in UPLIFT, the company’s single-arm registrational trial of UpRi in platinum-resistant ovarian cancer
• Initiated Phase 3 UP-NEXT clinical trial of UpRi as a maintenance monotherapy in recurrent platinum-sensitive ovarian cancer
• Neared completion of dose escalation in Phase 1 UPGRADE-A trial of UpRi in combination with carboplatin in platinum-sensitive ovarian cancer
• Announced that the European Commission has designated UpRi as an orphan medicinal product for the treatment of ovarian cancer
Expected Milestones
• Report top-line data from UPLIFT in mid-2023
• Assuming positive data, submit a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) around the end of 2023
• Prepare for potential U.S. accelerated approval and commercial launch in 2024
• Significantly advance enrollment of UP-NEXT in 2023
• Initiate dose expansion portion of UPGRADE-A in the first quarter of 2023 and report interim data from UPGRADE-A in the second half of 2023
Strategic Objective: Advance Clinical-Stage Pipeline
2022 Accomplishments
• XMT-1660: Initiated multicenter Phase 1 clinical trial in patients with previously treated breast, endometrial and ovarian cancers
• XMT-1660: Announced Fast Track designation for the treatment of adult patients with advanced or metastatic triple-negative breast cancer
• XMT-2056: Announced FDA orphan drug designation for the treatment of gastric cancer
Expected Milestones
• XMT-1660: Complete dose escalation portion of Phase 1 clinical trial in 2023
• XMT-2056: Initiate Phase 1 clinical trial in the first quarter of 2023
Strategic Objective: Position Mersana as the ADC Partner-of-Choice
2022 Accomplishments
• Entered into the following agreements that collectively provided Mersana with $170 million in upfront payments and an opportunity for more than $3 billion in milestones, plus royalties:
• An Immunosynthen research collaboration and license agreement with Merck KGaA, Darmstadt, Germany for two targets, which includes a $30 million upfront payment to Mersana and the potential for up to $800 million in total potential milestones, plus tiered royalties up to the low double-digits on net sales
• A collaboration, option and license agreement with GlaxoSmithKline plc (GSK) for the co-development and commercialization of XMT-2056, which provided Mersana with a $100 million upfront option purchase fee and the potential to receive up to $1.36 billion in the form of an additional option exercise fee and milestone payments, plus an option for Mersana to retain a U.S. profit share and tiered royalties on net sales outside of the United States or to receive tiered royalties up to the mid-twenties on global net sales
• A Dolasynthen research collaboration and license agreement with Janssen Biotech, Inc. for three targets, which provided Mersana with a $40 million upfront payment and the potential to receive over $1 billion in total potential milestones, plus tiered royalties up to the low double-digits on net sales
Expected Milestones
• Pursue impactful new collaborations
• Execute against existing collaboration agreements
Financial Update
Mersana estimates that its cash, cash equivalents and marketable securities as of December 31, 2022 were approximately $280 million. This figure is preliminary and unaudited. The company expects to report its audited cash, cash equivalents and marketable securities, as well as other information necessary for a complete understanding of its financial position, in its Annual Report on Form 10-K for the year ended December 31, 2022. The company expects that its available funds, together with the $30 million upfront payment due from Merck KGaA, Darmstadt, Germany under the collaboration and license agreement referenced above, will be sufficient to support its operating plan commitments into the second half of 2024.
About Mersana Therapeutics
Mersana Therapeutics is a clinical-stage biopharmaceutical company using its differentiated and proprietary ADC platforms to rapidly develop novel ADCs with optimal efficacy, safety and tolerability to meaningfully improve the lives of people fighting cancer. Mersana’s lead product candidate, upifitamab rilsodotin (UpRi), is a Dolaflexin ADC targeting NaPi2b that is being studied in UPLIFT, a single-arm registrational trial in patients with platinum-resistant ovarian cancer; UPGRADE-A, a Phase 1 clinical trial evaluating UpRi in combination with carboplatin; and UP-NEXT, a Phase 3 clinical trial of UpRi as monotherapy maintenance following treatment with platinum doublets in recurrent platinum-sensitive ovarian cancer. Mersana is also advancing XMT-1660, a Dolasynthen ADC targeting B7-H4, and XMT-2056, an Immunosynthen ADC targeting a novel epitope of human epidermal growth factor receptor 2 (HER2), in addition to other earlier-stage assets. In addition, multiple partners are using Mersana’s platforms to advance their ADC pipelines. Mersana routinely posts information that may be useful to investors on the “Investors & Media” section of its website at www.mersana.com.
>>> UCSF researchers find cellular 'glue' holds promise for regenerative medicine
MSN.com
by Natalia Gurevich
Dec 12, 2022
https://www.msn.com/en-us/health/medical/ucsf-researchers-find-cellular-glue-holds-promise-for-regenerative-medicine/ar-AA15cHcF?OCID=ansmsnnews11
SAN FRANCISCO (KCBS RADIO) – Researchers at UCSF have developed a new 'cellular glue' that can help heal wounds and regenerate tissue, according to a study published Monday in the journal Nature.
The study was published Monday, Dec. 12, 2022 in Nature.
The discovery is a major step in building tissue and organs, a major part of regenerative medicine.
"We've found a way to control and engineer how cells are able to stick together and form very specific structures," said Dr. Adam Stevens, Hartz Fellow in the Cell Design Institute at UCSF and first author of the paper on KCBS Radio on Monday.
Our bodies consist of trillions of cells, which stick together in very particular ways to form tissue, like with skin or even neurons in the brain. Adhesive molecules help keep these structures together.
Now, these researchers have been able to program these cells to stick together in an easily customizable manner.
This is important in disease or injuries that damage cells. Cells suffering damage often lose the connection that they once had with one another and can’t be repaired.
"These could potentially be applied, in the long term, to engineer therapeutic cells that then traffic to these sites of disease and restore connection," said Stevens.
Heart disease is a major disease that these types of of cells could help repair.
But it is still early days, and more research is needed to better understand the scope of how this could be used as treatment.
"Being able to reconnect cells is one step that could be a useful tool within the process," he said.
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>>> Scientists discovered an organism that hunts and eats viruses
BGR
by Joshua Hawkins
https://www.msn.com/en-us/news/technology/scientists-discovered-an-organism-that-hunts-and-eats-viruses/ar-AA15PgRi?cvid=bb910864709a40adb19df70968b36dfd
Scientists have discovered something intriguing in a new type of organism that actually eats viruses. The organisms, which are known as virovores, don’t just eat viruses accidentally, either. These organisms actually go out of their way to support a diet that snacks on viruses.
The discovery of the organisms that eat viruses came when researcher John DeLong from the University of Nebraska-Lincoln set out to see if any microbes actively ate viruses. To do this, DeLong and his team collected samples of pond water and then isolated different microbes from the water. Then, they added chlorovirus, a freshwater virus that infects green algae.
From there, DeLong and his team watched as one particular microbe called Halteria began to snack on the virus. Further, in samples of the water that did not offer other food sources for Halteria, the microbe grew about 15 times within two days just by eating the chlorovirus. More intriguingly, samples that didn’t have the virus saw no growth in Halteria at all.
To further test the hypothesis that the organism was eating the virus, the researchers added dye to the chlorovirus DNA. They later found that the Halteria cells began to glow, confirming that it was indeed eating the virus to sustain itself. These experiments then allowed the group to coin a new term for these organisms – virovory.
Virovory essentially is exactly the same as herbivory and carnivory, thereby determining that these organisms eat viruses to survive and sustain themselves. Of course, Halteria is also unlikely to be the only virovore out there. As such, the researchers plan to continue experimenting and looking for other organisms that can eat viruses in the same way.
The researchers published their findings in the Proceedings of the National Academy of Sciences. Further research into this topic could assist with finding organisms that can eat viruses found in the human body, perhaps even giving us a new way to fight certain infections. With scientists finding ancient viruses, having organisms that can snack on them could be very helpful for keeping them under control.
We’re already using viruses to fight cancer, so it isn’t entirely impossible. Of course, that’s a long way away, but it is nice to think about the options this could unlock.
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>>> First human patient injected with revolutionary cancer-killing virus
BGR
By Joshua Hawkins
May 22nd, 2022
https://bgr.com/science/first-human-patient-injected-with-revolutionary-cancer-killing-virus/
Scientists have injected the first human patient with a new cancer-killing virus. The virus, known as Vaxinia, has seen successful tests in animals. However, the true test of its efficacy begins with this new clinical trial.
It’s easy to hear the word virus and instantly think of something bad. After all, there are a lot of deadly viruses out there. However, scientists are using a new cancer-killing virus known as Vaxinia in an experimental cancer treatment.
The hope here is that the virus will amplify the body’s immune response against cancer. The virus itself has been engineered specifically to kill cancer cells. And, in previous animal trials, scientists have seen very promising results. These kinds of viruses have been a “smoking gun” in the fight against cancer for over a century.
However, the success of these viruses has been very limited, to say the least. This time around, though, the scientists have engineered the cancer-killing virus to not only harm cancer cells, but also to make them more recognizable to the body’s immune system. Researchers hope that this will help make the body’s response stronger, allowing it to fight back better.
Previous clinical trials of cancer drugs have shown promising results, too. But, Vaxinia could help open new doors, too.
Early testing was full of promise
Of course, before moving on to the human tests, the scientists tested the virus on animals. In many cases, they saw huge success at shrinking tumors in early animal and lab experiments. The cancer-killing virus has shown that it can reduce the size of lung, breast, ovarian, pancreatic, and colon cancer tumors.
With that success, the scientists decided to move on to human testing. Results seen in animals do not always directly translate to human patients. There are a lot of reasons for this, obviously, but the researchers are hopeful this virus could improve patients’ chance to fight back against cancer.
Currently, Vaxinia will be tested in a Phase 1 trial of just 100 cancer patients. These patients have metastatic or advanced solid tumors, and each has tried at least two other treatments. The researchers plan to administer the drug in two different groups. The first will receive just Vaxinia. The second group will get the cancer-killing virus plus an immunotherapy drug.
Of course, Phase 1 trials are mostly about safety and finding the optimal dose. As such, it might not prove the efficacy of the virus as a whole. However, it is an important next step in possibly finding an alternative method to fighting cancer. The trial is currently expected to complete by early 2025. So, it’ll be a while before we see any final results.
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>>> Mustang Bio, Inc. (MBIO), a clinical-stage biopharmaceutical company, focuses on translating medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors, and rare genetic diseases. Its pipeline focuses on gene therapy programs for rare genetic disorders, chimeric antigen receptor (CAR) engineered T cell (CAR T) therapies for hematologic malignancies, and CAR T therapies for solid tumors. The company develops MB-107 and MB-207, a gene therapy program for X-linked severe combined immunodeficiency, a rare genetic immune system condition in which affected patients do not live beyond infancy without treatment. The company also develops MB-102 CAR T therapies for blastic plasmacytoid dendritic cell neoplasm, acute myeloid leukemia, and myelodysplastic syndrome; MB-106 CAR T cell program for B cell non-hodgkin lymphoma and chronic lymphocytic leukemia; MB-104 CAR T for multiple myeloma and light chain amyloidosis; MB-101 CAR T cell program for glioblastoma; MB-103 CAR T for glioblastoma multiforme (GBM) and metastatic breast cancer to brain; MB-105 CAR T for prostate and pancreatic cancers; and MB-108, a next-generation oncolytic herpes simplex virus. It has license agreements with Nationwide Children's Hospital, CSL Behring; Mayo Clin Mayo Clinic; Leiden University Medical Centre; SIRION Biotech GmbH, and Minaris Regenerative Medicine GmbH. The company was incorporated in 2015 and is headquartered in Worcester, Massachusetts.
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Name | Symbol | % Assets |
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Regeneron Pharmaceuticals Inc | REGN | 2.77% |
Moderna Inc | MRNA | 2.65% |
Gilead Sciences Inc | GILD | 2.43% |
Vertex Pharmaceuticals Inc | VRTX | 2.32% |
Biogen Inc | BIIB | 2.28% |
United Therapeutics Corp | UTHR | 2.26% |
Biomarin Pharmaceutical Inc | BMRN | 2.25% |
Seattle Genetics Inc | SGEN | 2.16% |
Exelixis Inc | EXEL | 2.06% |
ACADIA Pharmaceuticals Inc | ACAD | 2.04% |
Name | Symbol | % Assets |
---|---|---|
Regeneron Pharmaceuticals Inc | REGN | 4.88% |
Gilead Sciences Inc | GILD | 4.58% |
Qiagen NV | QGEN | 4.48% |
Biogen Inc | BIIB | 4.28% |
Seattle Genetics Inc | SGEN | 4.08% |
United Therapeutics Corp | UTHR | 3.93% |
Vertex Pharmaceuticals Inc | VRTX | 3.89% |
ACADIA Pharmaceuticals Inc | ACAD | 3.75% |
Biomarin Pharmaceutical Inc | BMRN | 3.69% |
Alnylam Pharmaceuticals Inc | ALNY | 3.53% |
Name | Symbol | % Assets |
---|---|---|
Pacific Biosciences of California Inc | PACB | 6.85% |
Teladoc Health Inc | TDOC | 5.94% |
CRISPR Therapeutics AG | CRSP | 5.77% |
Twist Bioscience Corp | TWST | 5.72% |
CareDx Inc | CDNA | 3.87% |
Iovance Biotherapeutics Inc | IOVA | 3.59% |
Exact Sciences Corp | EXAS | 3.58% |
Fate Therapeutics Inc | FATE | 3.47% |
Invitae Corp | NVTA | 3.42% |
Personalis Inc | PSNL | 3.26% |
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