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>>> Innovation Pharma Completes Interim Safety Data Review—DMC Approves Increased Dosing Frequency in Phase 2 Clinical Trial of Brilacidin in Hospitalized COVID-19 Patients
Innovation Pharmaceuticals Inc.
April 5, 2021
https://finance.yahoo.com/news/innovation-pharma-completes-interim-safety-113000256.html
Study drug dosing regimen to increase to 5 days (from 3 days) based on DMC recommendation
Enrollment has reached 25 percent in the Company’s ~120-patient Phase 2 COVID-19 clinical trial
WAKEFIELD, Mass., April 05, 2021 (GLOBE NEWSWIRE) -- Innovation Pharmaceuticals (OTCQB:IPIX) (“the Company”), a clinical stage biopharmaceutical company, today announced that an independent Data Monitoring Committee (DMC) completed its scheduled review of interim safety data in the Company’s randomized, double-blind, placebo-controlled Phase 2 clinical trial of Brilacidin for treatment of moderate-to-severe COVID-19 in hospitalized patients (see NCT04784897).
Upon reaching 25 percent enrollment (30 subjects), recruitment was paused and a pre-specified unblinded safety data review and evaluation was conducted by the DMC. Following their review, the DMC recommended increasing the dosing regimen of Brilacidin from 3 days to 5 days of treatment, as intended per the protocol, which the Company has since implemented. Recruitment for the 5-day treatment regimen has begun. Efficacy data was not reviewed by the DMC and remains blinded.
“We are thrilled with the recommendation of the DMC pertaining to the safety profile of Brilacidin, a drug that has repeatedly been shown in lab studies by virology experts as a top candidate for fighting infectious and resilient coronaviruses, including SARS-CoV-2, the pathogen responsible for COVID-19,” said Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals. “There is a sense of urgency to develop novel pan-coronavirus therapeutics for COVID-19, particularly given the growing spread of variants worldwide, including P1 and B.1.1.7, which appear to be contributing to cases of infection cropping up in patients who have already been vaccinated. With its unique mechanism of action to directly disrupt the novel coronavirus, we believe Brilacidin possesses potential to address these worrisome reminders that SARS-CoV-2 isn’t going anywhere. More broadly, Brilacidin’s antiviral potency also appears to extend beyond coronaviruses to other types of viruses, based on independent ongoing laboratory research, further supporting Brilacidin’s broad spectrum antiviral potential.”
The clinical rationale for consideration of expanding treatment duration to 5 days, from the initial dosing regimen of 3 days, is to provide a longer duration of systemic Brilacidin exposure at a level that can strongly suppress SARS-CoV-2 virus replication, and associated symptoms, and thus maximize therapeutic benefits to hospitalized patients with moderate or severe COVID-19. The expanded dosing regimen has particular value in that a 5-day dosing is the recommended initial treatment duration for Gilead Sciences’ Veklury® (remdesivir), the only FDA-approved antiviral drug for treatment of COVID-19. Trial data for Veklury could serve as a benchmark in evaluating Brilacidin outcomes.
The Company aims to expedite enrollment in the coming weeks toward study completion and release of topline results. Two sites, in addition to the 9 currently active and recruiting patients, have been initiated, with a further two sites planned to initiate within the next week.
Innovation Pharma is developing Brilacidin, a defensin-mimetic, under Fast Track designation from the U.S. Food and Drug Administration (FDA).
Alerts
Sign-up for Innovation Pharmaceuticals email alerts is available at:
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About Innovation Pharmaceuticals
Innovation Pharmaceuticals Inc. (IPIX) is a clinical stage biopharmaceutical company developing a world-class portfolio of innovative therapies addressing multiple areas of unmet medical need, including inflammatory diseases, cancer, infectious diseases, and dermatologic diseases.
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BPTH announces successful completion of safety cohort in a Phase II in AML.
(I thought the early results in this small study were very impressive, although not emphasized in the PR header.)
Stock up 36% in pre-Market.
April 5, 2021 7:00 AM
BIO-PATH HOLDINGS ANNOUNCES SUCCESSFUL COMPLETION OF SAFETY COHORT OF TRIPLE COMBINATION OF PREXIGEBERSEN, DECITABINE AND VENETOCLAX IN STAGE 2 OF PHASE 2 CLINICAL TRIAL IN ACUTE MYELOID LEUKEMIA
HOUSTON, April 05, 2021 (GLOBE NEWSWIRE) -- Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize ® antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, today announced the successful completion of the safety run-in of the Stage 2 of the Phase 2 clinical study of prexigebersen (BP1001), a liposomal Grb2 antisense, for the treatment of acute myeloid leukemia (AML), in combination with frontline therapies, decitabine and venetoclax, in acute myeloid leukemia (AML) patients. The safety run-in of Stage 2 of the Phase 2 clinical trial was comprised of six evaluable patients who were treated with the triple combination of prexigebersen, decitabine and venetoclax.
“We are particularly pleased with the clean side effect profile and lack of toxicity shown in this segment of the study, as our Phase 2 efficacy segment will include de novo fragile AML patients for whom drug side effect profiles are particularly important. We are also very encouraged by the efficacy signals shown in this dataset, with five of six evaluable relapsed, refractory and newly diagnosed AML patients demonstrating clinical activity. These positive signals give us further confidence in the potential for this program in these late-stage and compromised patients,” stated Peter H. Nielsen, Chief Executive Officer of Bio-Path Holdings.
“We look forward to advancing this Phase 2 study, as we believe its unique design provides us with several definable registration pathways. We believe that prexigebersen, with its promising efficacy and safety profile, has the potential to be an ideal combination candidate with frontline therapies,” concluded Mr. Nielsen.
In the safety run-in, six evaluable patients were treated with the combination of prexigebersen, decitabine and venetoclax. These patients included four relapsed/refractory AML patients, and two newly diagnosed AML patients. In the preliminary safety data review, five of the patients (83%) responded to treatment, including four (67%) achieving complete response (CR) and one (17%) complete response with incomplete hematologic recovery (CRi). CR rates to combination treatment with decitabine and venetoclax for relapsed/refractory AML patients is 42-52% 1,2 and 0-39% 1,2 for relapsed/refractory secondary AML patients. Response rates to frontline treatment decitabine and venetoclax for newly diagnosed AML patients is 62-71% 3 ,4 . These preliminary data showed the treatment was well-tolerated and there were no dose limiting toxicities attributed to prexigebersen. Three patients remained on treatment for more than one cycle.
Stage 2 of the Phase 2 clinical trial has three treatment cohorts, which the Company believes provides for several potential regulatory pathways. The first two cohorts will treat patients with the triple combination of prexigebersen, decitabine and venetoclax. The first cohort includes newly diagnosed AML patients and the second cohort includes relapsed/refractory AML patients. Finally, the third cohort treats relapsed/refractory AML patients who are venetoclax resistant or intolerant with the two-drug combination of prexigebersen and decitabine.
The Phase 2 clinical trial continues with 21 patients currently enrolled across all three cohorts. Enrollment of 19 patients in each cohort should enable a data review to determine if there is a comparative increase in efficacy versus the decitabine and venetoclax combination therapy sufficient to support petitioning the FDA for approval to switch to breakthrough therapy for accelerated approval. The Phase 2 trial will be conducted at up to ten clinical sites in the U.S. For more information on the Phase 2 study, visit www.clinicaltrials.gov .
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>>> Humanigen Reports Positive Phase 3 Topline Results Demonstrating That Lenzilumab™ Improves Survival Without Need for Mechanical Ventilation in Hospitalized Patients With COVID-19
Yahoo Finance
March 29, 2021
https://finance.yahoo.com/news/humanigen-reports-positive-phase-3-110000526.html
Lenzilumab improved the relative likelihood of survival without need for invasive mechanical ventilation (IMV) by 54%, achieving the primary endpoint of the Phase 3 study
Clinical improvement was observed over and above other treatments, including steroids and/or remdesivir
Humanigen, Inc. (Nasdaq: HGEN) ("Humanigen"), a clinical-stage biopharmaceutical company focused on preventing and treating an immune hyper-response called ‘cytokine storm’ with its lead drug candidate, lenzilumab, today announced positive topline results from its Phase 3 clinical trial evaluating the efficacy and safety of lenzilumab in patients hospitalized with COVID-19. Trial results showed that patients who received lenzilumab and other treatments, including steroids and/or remdesivir, had a 54% greater relative likelihood of survival without the need for IMV compared with patients receiving placebo and other treatments. These results are statistically significant.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210329005301/en/
"The results from our Phase 3 clinical trial with lenzilumab treatment were associated with better outcomes in hospitalized hypoxic COVID-19 patients who had not yet progressed to the point of requiring IMV," said Cameron Durrant, MD, MBA, Chief Executive Officer, Humanigen. "Additionally, the trial incorporated a diverse population with various comorbidities, most commonly a body mass index above 30, which is representative of a real-world, high-risk population. Our next step is to submit an application for Emergency Use Authorization (EUA) to the Food and Drug Administration (FDA) as soon as possible. We are also sharing these results with US governmental agencies and other authorities worldwide."
"Mayo Clinic is pleased to have been part of the investigation of lenzilumab from the earliest days of the development program in COVID-19 and are excited by these data," said Andrew Badley, MD, Professor of Infectious Diseases, and Professor and Chair of the Department of Molecular Medicine at Mayo Clinic. "If lenzilumab is authorized for emergency use by FDA, and based on our clinical trial experience to date, it may then be considered a part of our treatment armamentarium for newly hospitalized patients with COVID-19."
Study results demonstrate that lenzilumab significantly improved patient outcomes. The study achieved its primary endpoint of ventilator-free survival measured through day 28 following treatment (HR: 1.54; 95%CI: 1.03-2.33, p=0.0365). Ventilator-free survival is a validated and reliable measure used in studies that evaluate respiratory distress.1 The Kaplan-Meier estimate for IMV and/or death was 15.6% (95%CI: 11.5-21.0) in the lenzilumab arm versus 22.1% (95%CI: 17.4-27.9) in the placebo arm, representing a 54% improvement in the relative likelihood of survival without the need for IMV. Although this study was not powered to demonstrate a difference in mortality, a favorable trend in mortality was observed: 9.6% (95%CI: 6.4-14.2) in the lenzilumab arm compared with 13.9% (95%CI: 10.1-19.0) in the placebo arm (HR: 1.39; 95%CI: 0.82-2.39; p=0.2287). Approximately 88% of patients received dexamethasone (or other steroids), 62% received remdesivir, and 57% received both, balanced across both arms of the study. Serious adverse events (SAEs) were balanced in both study arms and the SAE profile was similar to that previously documented in prior lenzilumab studies. In this study, lenzilumab appeared to be safe and well-tolerated; no new SAEs were identified, and none were attributed to lenzilumab.
"The data strongly suggest that lenzilumab improved outcomes for hospitalized patients with COVID-19 pneumonia," said Zelalem Temesgen, MD, Professor of Medicine at Mayo Clinic and Principal Investigator of the Phase 3 trial. "The dosing regimen used in this study was specifically designed for hospitalized patients with COVID-19 pneumonia as a potential foundational therapy. Lenzilumab could make the difference between going on a ventilator, which reduces one’s chance of survival, and leaving the hospital alive."
"It is impressive to see lenzilumab achieve this milestone. At Emory University, a key center in the National Institutes of Health (NIH) ACTIV-5 study, which is currently enrolling, we are hopeful that lenzilumab will emerge as a valuable therapy for newly hospitalized patients. We believe there may be future opportunities to study lenzilumab in even larger trials, and further explore lenzilumab’s impact on mortality rates," added Vincent Marconi, MD, Professor of Medicine at Emory University School of Medicine.
About the Lenzilumab Phase 3 Study
This study was a randomized, double-blind, placebo-controlled, multi-center Phase 3 trial for the treatment and prevention of serious and potentially fatal outcomes in patients who were hospitalized with COVID-19 pneumonia. The primary objective was to assess whether lenzilumab, in addition to other treatments, which included dexamethasone (or other steroids) and/or remdesivir, could alleviate the immune-mediated cytokine release syndrome (CRS) and improve ventilator-free survival. Ventilator-free survival is a composite endpoint of time to death and time to IMV, which is a robust measure that is less prone to favor a treatment with discordant effects on survival or days free of ventilation.1 The trial enrolled 520 patients in 29 sites in the US and Brazil who were at least 18 years of age; experienced blood oxygen saturation (SpO2) of less than or equal to 94%; or required low-flow supplemental oxygen, or high-flow oxygen support, or non-invasive positive pressure ventilation (NIPPV); and were hospitalized but did not require IMV. Following enrollment, subjects were randomized to receive three infusions of either lenzilumab or placebo, each infusion separated by eight hours over a 24-hour period with other treatments. The primary endpoint was the difference between lenzilumab treatment and placebo treatment in ventilator-free survival through 28 days following treatment. Key secondary endpoints, also measured through 28 days, included ventilator-free days, duration of ICU stay, incidence of invasive mechanical ventilation, extracorporeal membrane oxygenation (ECMO), and/or death, time to death, all-cause mortality, and time to recovery. Results of the trial are planned to be submitted for potential publication in a peer-reviewed journal.
About Humanigen, Inc.
Humanigen, Inc. is developing its portfolio of clinical and pre-clinical therapies for the treatment of cancers and infectious diseases via its novel, cutting-edge GM-CSF neutralization and gene-knockout platforms. Humanigen’s immediate focus is to prevent or minimize cytokine release syndrome that precedes severe lung dysfunction in hospitalized and hypoxic patients with COVID-19 pneumonia. Humanigen is also working to create next-generation combinatory gene-edited CAR-T therapies using strategies to improve efficacy while employing GM-CSF gene knockout technologies to control toxicity. In addition, Humanigen is developing its own portfolio of proprietary first-in-class EphA3-CAR-T for various solid cancers and EMR1-CAR-T for various eosinophilic disorders. Humanigen is also exploring the effectiveness of its GM-CSF neutralization technologies (either through the use of lenzilumab as a neutralizing antibody or through GM-CSF gene knockout) in combination with other CAR-T, bispecific or natural killer (NK) T-cell-engaging immunotherapy treatments to break the efficacy/toxicity linkage, including to prevent and/or treat Graft versus Host Disease (GvHD) in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). Additionally, Humanigen and Kite, a Gilead Company, are evaluating lenzilumab in combination with Yescarta® (axicabtagene ciloleucel) in patients with relapsed or refractory large B-cell lymphoma in a clinical collaboration. For more information, visit www.humanigen.com and follow Humanigen on LinkedIn, Twitter, and Facebook.
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KZIA licenses Paxalisib to Simcere for Greater China
https://kza.irmau.com/irm/PDF/4d768237-e0f5-4b62-8f07-96257d93c124/KazialicensespaxalisibtoSimcereinGreaterChina
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>>> Surface Oncology Reports Financial Results and Corporate Highlights for Fourth Quarter and Full Year 2020
Yahoo Finance
Surface Oncology, Inc.
March 9, 2021
https://finance.yahoo.com/news/surface-oncology-reports-financial-results-120100357.html
New Collaboration with Merck Will Evaluate SRF388, Targeting IL-27, in Combination with KEYTRUDA® (pembrolizumab) in Patients with Solid Tumors
CAMBRIDGE, Mass., March 09, 2021 (GLOBE NEWSWIRE) -- Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation therapies that target the tumor microenvironment, today reported financial results and corporate highlights for the fourth quarter and full year 2020, as well as anticipated 2021 corporate milestones.
“The fourth quarter of 2020 was transformational for Surface. During this quarter, we provided encouraging clinical data from our lead candidates and validated our preclinical discovery capabilities with a second major outlicense agreement that provided the company with substantial financial flexibility for several years,” said Rob Ross, M.D., incoming chief executive officer. “Today, we are also announcing another clinical trial collaboration further enabling rapid assessment of our lead product candidates (in this case SRF388) in combination with pembrolizumab, focusing on patients with liver and kidney cancers. As we look forward into 2021, we are targeting ASCO in June to share additional clinical data on SRF617 and SRF388, and we will work with our partner, GSK, to advance SRF813 into the clinic.”
Recent Corporate Highlights:
On March 9, 2021, Surface announced a clinical trial collaboration with Merck to evaluate the safety and efficacy of combining Surface’s SRF388, an investigational antibody therapy targeting IL-27, with Merck’s KEYTRUDA® (pembrolizumab), the first anti-PD-1 therapy approved in the United States. This combination will be studied as a component of the first-in-human Phase 1 study of SRF388 and will be evaluated in patients with solid tumors, with a focus on patients with liver cancer and kidney cancer.
In December, Surface announced an agreement for GlaxoSmithKline (GSK) to exclusively license worldwide development and commercial rights to Surface Oncology’s preclinical program SRF813, a fully human IgG1 antibody targeting PVRIG (also known as CD112R), an inhibitory protein expressed on natural killer cells (NK cells) and T cells. Under the terms of the agreement, GSK made an $85 million upfront payment in December 2020. In addition, Surface Oncology may receive up to an additional $730 million in future milestone payments, as well as be eligible to receive tiered royalties on global net sales.
In November, Surface announced that both of its lead clinical programs, SRF617 (targeting CD39) and SRF388 (targeting IL-27), have achieved predefined criteria for advancement into combination and expansion stages of the ongoing Phase 1 trials. These criteria include acceptable safety profiles at biologically relevant doses, as well as demonstration of target engagement and meaningful pharmacodynamic activity in the ongoing Phase 1 trials.
Effective April 1, 2021, Rob Ross, M.D., who has served as chief medical officer at Surface Oncology since 2016, will become the company’s president and chief executive officer and will also be appointed to the board of directors. Rob will succeed current CEO Jeff Goater, who will assume the role of chairman of the Surface Oncology board of directors.
Selected Anticipated Near-term Corporate Milestones:
Preclinical data presentations for SRF617 and SRF388 at the American Association for Cancer Research (AACR) Virtual Annual Meeting in April.
Targeting the American Society of Clinical Oncology (ASCO) Virtual Annual Meeting in June for detailed clinical data presentations for SRF617 and SRF388.
Investigational new drug (IND) filing for SRF813 anticipated in 2021.
Financial Results:
As of December 31, 2020, cash, cash equivalents and marketable securities were $175.1 million, compared to $105.2 million on December 31, 2019.
Revenue recognized in the fourth quarter ended December 31, 2020 was $87.6 million, compared to revenue of less than $1.0 million for the same period in 2019. The increase was a result of the $85 million upfront payment received in the fourth quarter 2020 from GSK. Revenue recognized in the full year ended December 31, 2020 was $126.2 million, compared to $15.4 million for the same period in 2019. The increase was a result of the $85 million upfront payment received in the fourth quarter 2020 from GSK, as well as the expiration of the final Novartis option purchase period in January 2020 and the corresponding recognition of the remaining deferred revenue under the agreement.
Research and development (R&D) expenses were $10.7 million for the fourth quarter ended December 31, 2020, compared to $11.7 million for the same period in 2019. R&D expenses were $41.0 million for the full year ended December 31, 2020, compared to $52.1 million for the same period in 2019. This decrease was primarily driven by a reduction in expenses associated with contract manufacturing and other IND-enabling activities, as a result of the SRF617 and SRF388 IND filings in 2019, offset by an increase in spend on the SRF617 and SRF388 Phase 1 clinical trials, which began in 2020. R&D expenses included $2.8 million in stock-based compensation expense for the full year ended December 31, 2020.
General and administrative (G&A) expenses were $8.9 million for the fourth quarter ended December 31, 2020, compared to $5.1 million for the same period in 2019. G&A expenses were $23.6 million for the full year ended December 31, 2020, compared to $20.6 million for the same period in 2019. This increase was primarily due to increased consulting costs related to the GSK Agreement, as well as increased stock-based compensation expense and bonus achieved in 2020. G&A expenses included $4.9 million in stock-based compensation expense for the full year ended December 31, 2020.
For the fourth quarter ended December 31, 2020, net income was $67.3 million, or basic net income per share attributable to common stockholders of $1.66, and diluted net income per share attributable to common stockholders of $1.56. Net loss was $16.0 million for the same period in 2019, or basic and diluted net loss per share attributable to common stockholders of $0.57. For the full year ended December 31, 2020 net income was $59.3 million, or basic net income per share attributable to common stockholders of $1.67, and diluted net income per share attributable to common stockholders of $1.57. Net loss was $54.8 million for the same period in 2019, or basic and diluted net loss per share attributable to common stockholders of $1.97.
Financial Outlook:
Based upon our current operating plan, Surface continues to project cash runway sufficient through 2023.
About Surface Oncology:
Surface Oncology is an immuno-oncology company developing next-generation antibody therapies focused on the tumor microenvironment. Its pipeline includes two wholly-owned clinical-stage programs targeting CD39 (SRF617) and IL-27 (SRF388), as well as a preclinical program focused on depleting regulatory T cells via targeting CCR8 (SRF114). In addition, Surface has two partnerships with major pharmaceutical companies: a collaboration with Novartis targeting CD73 (NZV930; Phase 1) and a collaboration with GlaxoSmithKline targeting PVRIG (SRF813; preclinical). Surface’s novel cancer immunotherapies are designed to achieve a clinically meaningful and sustained anti-tumor response and may be used alone or in combination with other therapies. For more information, please visit www.surfaceoncology.com.
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Curis Reports Fourth Quarter and Year-End 2020 Financial Results
Tue March 16, 2021 4:01 PM|PR Newswire|About: CRIS
- Presented CA-4948 Phase 1 data showing broad clinical activity in patients with R/R AML and MDS and Phase 1 data in patients with R/R non-Hodgkin's lymphoma (NHL) showing durable and dose-dependent reductions in tumor burden -
- Raised $169.6M of gross proceeds in December 2020 public offering; extending cash runway into 2024 -
- Multiple data readouts expected in 2021 from CA-4948 and anti-VISTA antibody CI-8993 clinical trials -
- Management to host conference call today at 4:30 p.m. ET -
PR Newswire
LEXINGTON, Mass., March 16, 2021 /PRNewswire/ -- Curis, Inc. (CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, today reported its financial results for the fourth quarter and year ended December 31, 2020.
"2020 was a transformative year for Curis, as we made significant progress in our mission to develop the next generation of targeted cancer therapies that meaningfully improve and extend the lives of patients. Despite the difficulties and uncertainty brought about by the ongoing coronavirus pandemic, we significantly advanced and expanded each program in our clinical pipeline, headlined by the very encouraging data from our Phase 1 trials of lead asset, CA-4948, presented in December in conjunction with ASH," said James Dentzer, President and Chief Executive Officer of Curis. "We look forward to providing additional updates on our IRAK4 program throughout the year, with clinical data readouts from our ongoing studies, including the CA-4948 monotherapy study in AML/MDS and the CA-4948/ibrutinib combination study for patients with R/R hematologic malignancies that we initiated in early 2021. We also continue to enroll patients and bring additional trial sites online in our Phase 1a/1b trial of CI-8993, our first-in-class monoclonal anti-VISTA antibody for the treatment of patients with R/R solid tumors, and look forward to providing preliminary data from this exciting study later this year."
Mr. Dentzer continued, "2020 was also a pivotal year on the corporate side for Curis. Through the execution of several key financings and partnerships, we have the resources needed to advance our programs through their next data catalysts, while also providing us the ability to invest efficiently in our pipeline of first-in-class cancer therapeutics. We are excited about the opportunities stemming from our Q4 signing of the CRADA with the NCI in addition to the recently announced Phase 2 IST of CA-4948 for the treatment of anemia in patients with lower-risk MDS led by Dr. Uwe Platzbecker at Universität Leipzig. These new partnerships provide powerful validation of our IRAK4 platform and allow us to leverage the resources of premier research organizations to significantly expand the reach of our clinical and preclinical programs."
Fourth Quarter 2020 and Recent Operational Highlights
Precision oncology, CA-4948 (IRAK4 Inhibitor; Aurigene collaboration):
• In December 2020, Curis announced positive preliminary data from its ongoing Phase 1 study of CA-4948 monotherapy in patients with R/R AML and high-risk MDS, including marrow blast reductions observed in all evaluable patients and 2 of 6 evaluable patients experiencing a marrow complete response. Curis continues to enroll patients, is currently enrolling in the 500mg BID dose cohort of the study and expects to report additional data in mid-year 2021.
• In December 2020, Curis provided updated preliminary data from its ongoing Phase 1 study of CA-4948 showing durable and dose-dependent reductions in tumor burden in patients with R/R NHL and announced the recommended Phase 2 dose, in addition to the identification of two potentially predictive biomarkers demonstrating target engagement and potential for patient enrichment, in an oral presentation at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition.
• In February 2021, Curis announced the dosing of the first patient in its Phase 1 dose-escalation and expansion study of CA-4948, an IRAK4 kinase inhibitor, and the BTK inhibitor, ibrutinib, for the treatment of patients with relapsed or refractory hematologic malignancies. In preclinical models, CA-4948 demonstrated synergistic anti-cancer activity when combined with a potent BTK inhibitor such as ibrutinib.
• Approximately 18 patients will be enrolled in the dose-escalation portion and will receive starting dose and escalation doses that have been observed to be safe and effective, combined with ibrutinib doses appropriate for their respective NHL subtype.
• The primary endpoints of Part 1 will be determination of maximum tolerated dose (MTD), and the recommended Phase 2 dose (RP2D).
• Part 2 of the study will enroll patients across a basket of four cohorts:
• Marginal zone lymphoma (MZL)
• Activated B-cell subtype of Diffuse Large B-cell Lymphoma (ABC-DLBCL)
• Primary central nervous system lymphoma (PCNSL)
• NHL with adaptive ibrutinib resistance.
• An interim futility analysis will be conducted after approximately 15-20 patients are enrolled in each cohort.
• Primary endpoints of Part 2 will be complete response or objective response rate and duration of response.
• Curis expects to report initial data from the study in the fourth quarter of 2021.
• In February 2021, Curis announced the initiation of the investigator-sponsored Phase 2 LUCAS trial of CA-4948 for the treatment of anemia in patients with very low, low, or intermediate-risk MDS. The trial is expected to start recruitment in the second quarter of 2021 and is expected to enroll 84 patients across two cohorts:
• Cohort A: Erythropoiesis stimulating agent (ESA) refractory/intolerant patients
• Cohort B: ESA naïve patients with transfusion dependence (min. 20 patients) or transfusion independence (min. 20 patients)
Patients in both cohorts will receive 300mg CA-4948 twice-daily (BID) for 21 days in at least four repeating cycles lasting 28 days each.
• The primary endpoint of the study is to evaluate the proportion of patients that develop an erythroid response (HI-E) according to IWG 2018 criteria.
Immuno (IMGN)-oncology, CI-8993 (anti-VISTA antibody; ImmuNext collaboration):
• In November 2020, Curis published trial design details from its ongoing Phase 1a/1b dose-escalation study of its first-in-class monoclonal anti-VISTA antibody for the treatment of R/R solid tumors.
• Curis continues to enroll patients in the study and expects to report initial safety and efficacy data in the second half of 2021.
Corporate:
• In December 2020, Curis closed an underwritten public offering of 29,500,000 shares of its common stock, including the exercise in full by the underwriters of their option to purchase up to an additional 3,847,826 shares, raising gross proceeds of approximately $169.6 million before deducting underwriting discounts and commissions and offering expenses.
• In November 2020, Curis entered into a CRADA with the NCI. Under the CRADA, Curis will collaborate with the NCI Experimental Therapeutics Program (NExT) and the NCI Cancer Therapy Evaluation Program to conduct non-clinical and clinical studies of Curis' proprietary compound, CA-4948, an IRAK4 kinase inhibitor that acts as a Toll-like Receptor (TLR) suppressor, as an anti-cancer agent.
Upcoming 2021 Planned Milestones
• Report additional clinical data from the Phase 1 study of CA-4948 in patients with AML and high-risk MDS, including patients with spliceosome mutations that encode oncogenic IRAK4-L in mid-year 2021.
• Report additional clinical biomarker data gathered in the Phase 1 study of CA-4948 in patients with R/R NHL in mid-year 2021.
• Announce initial safety and efficacy data from the ongoing Phase 1 study of CA-4948 in combination with ibrutinib in patients with R/R NHL in the second half of 2021.
• Report initial safety and efficacy data from the ongoing Phase 1a/1b dose-escalation study of CI-8993 for the treatment of R/R solid tumors in the second half of 2021.
Full Year and Fourth Quarter 2020 Financial Results
For the year ended December 31, 2020, Curis reported a net loss of $29.9 million, or $0.61 per share on both a basic and diluted basis, as compared to a net loss of $32.1 million, or $0.97 per share on both a basic and diluted basis in 2019. For the fourth quarter of 2020, Curis reported a net loss of $7.5 million or $0.11 per share on both a basic and diluted basis, as compared to a net loss of $8.6 million, or $0.26 per share on both a basic and diluted basis for the same period in 2019.
Revenues for the year ended December 31, 2020, were $10.8 million as compared to $10.0 million for the same period in 2019. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche's net sales of Erivedge®. Revenues for the fourth quarters of 2020 and 2019 were $3.0 million and $3.3 million, respectively.
Operating expenses for the year ended December 31, 2020 were $35.7 million as compared to $34.4 million for the same period in 2019. Operating expenses for the fourth quarter of 2020 were $9.3 million, as compared to $10.6 million for the same period in 2019, and comprised the following:
Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche's Erivedge net sales, were $0.5 million for the years ended December 31, 2020 and 2019. Cost of royalty revenues were $0.2 million for the fourth quarter of 2020 and 2019.
Research and Development Expenses. Research and development expenses were $23.1 million for the year ended December 31, 2020, as compared to $22.3 million for the same period in 2019. Research and development expenses were $5.6 million for the fourth quarter of 2020 as compared to $7.5 million for the same period in 2019. The decrease was primarily due to a decrease in clinical and manufacturing costs related to CA-170 and fimepinostat.
General and Administrative Expenses. General and administrative expenses were $12.1 million for the year ended December 31, 2020, as compared to $11.6 million for the same period in 2019. General and administrative expenses were $3.5 million for the fourth quarter of 2020, as compared to $3.0 million for the same period in 2019. The increase was primarily due to an increase in personnel related costs.
Other Expense, Net. Net other expense was $5.0 million for the year ended December 31, 2020, as compared to $7.8 million for the same period in 2019. For the fourth quarter of 2020 and 2019, net other expense was $1.2 million and $1.3 million, respectively. Net other expense primarily consisted of imputed interest expense related to future royalty payments.
As of December 31, 2020, Curis's cash, cash equivalents and investments totaled $183.1 million, and there were approximately 91.5 million shares of common stock outstanding. Curis expects that its existing cash, cash equivalents and investments should enable it to maintain its planned operations into 2024.
Conference Call Information
Curis management will host a conference call today, March 16, 2021, at 4:30 p.m. ET, to discuss these financial results, as well as provide a corporate update.
To access the live conference call, please dial 1-888-346-6389 from the United States or 1-412-317-5252 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section.
About Curis, Inc.
Curis is a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer. In 2015, Curis entered into a collaboration with Aurigene in the areas of immuno-oncology and precision oncology. As part of this collaboration, Curis has exclusive licenses to oral small molecule antagonists of immune checkpoints including the VISTA/PDL1 antagonist CA-170, and the TIM3/PDL1 antagonist CA-327, as well as the IRAK4 kinase inhibitor, CA-4948. CA-4948 is currently undergoing testing in a Phase 1 in patients with non-Hodgkin's lymphoma both as a monotherapy and in combination the with BTK inhibitor ibrutinib. Curis is also evaluating CA-4948 in a Phase 1 trial in patients with acute myeloid leukemia and myelodysplastic syndromes. In addition, Curis is engaged in a collaboration with ImmuNext for development of CI-8993, a monoclonal anti-VISTA antibody, which is currently undergoing testing in a Phase 1a/1b trial in patients with solid tumors. Curis is also party to a collaboration with Genentech, a member of the Roche Group, under which Genentech and Roche are commercializing Erivedge® for the treatment of advanced basal cell carcinoma. For more information, visit Curis' website at www.curis.com.
Bladerunner
>>> Oncternal Therapeutics Provides Business Update and Announces Fourth Quarter and Full Year 2020 Financial Results
Yahoo Finance
Oncternal Therapeutics
March 11, 2021
https://finance.yahoo.com/news/oncternal-therapeutics-provides-business-announces-210100905.html
Interim Phase 1/2 results for cirmtuzumab with ibrutinib in MCL presented at ASH in December 2020 compare favorably to historical single-agent ibrutinib data (47% CR vs. 20% CR historical single-agent ibrutinib)
Accelerating development of ROR1-targeting CAR-T cell therapies
Two durable complete responses in patients with metastatic relapsed/refractory Ewing sarcoma treated with TK216 in ongoing Phase 1/2 clinical trial
Appointed Edwina Baskin-Bey, M.D., as Acting Chief Medical Officer
Management to host webcast today at 5:00 pm ET
SAN DIEGO, March 11, 2021 (GLOBE NEWSWIRE) -- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today provided a business update and reported fourth quarter and full year 2020 financial results.
“In 2021, we are advancing a deep pipeline of differentiated oncology assets. We have now initiated early-stage work in ROR1-targeting immunotherapies, including CAR-T and CAR-NK cell therapies, while moving forward the later-stage clinical development of cirmtuzumab, an antibody targeting ROR1, in MCL, which has generated encouraging data in this difficult to treat cancer. At the same time, we continue to evaluate TK216, an ETS inhibitor which has generated promising results in Ewing sarcoma,” said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. “We have also strengthened our balance sheet by raising $125 million in 2020, which provides us with the runway to advance our promising programs into 2023. We have several key data read-outs pending in the second quarter this year.”
Recent Highlights
In January 2021, we announced an agreement with Lentigen Technology, Inc., a wholly-owned subsidiary of Miltenyi Biotec B.V. & Co. KG, to manufacture lentiviral vectors to support Oncternal’s investigational ROR1-targeting CAR-T cell therapy program.
In January 2021, we announced a research and development collaboration with Karolinska Institutet in Stockholm, Sweden, to advance novel ROR1-targeting CAR-T and CAR-NK cell therapies from the laboratory into the clinic.
In December 2020, we announced an interim clinical data update from the ongoing Phase 1/2 clinical trial of cirmtuzumab, an investigational anti-ROR1 monoclonal antibody, in combination with ibrutinib in patients with mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL) at the American Society of Hematology 2020 Virtual Annual Meeting. Best objective response rate of 87%, including complete response (CR) rate of 47%, was reported for 15 evaluable patients with relapsed/refractory (r/r) MCL. Median progression-free survival (PFS) was not reached for patients with MCL, with the 95% confidence interval above 17.5 months, after a median follow-up of 12.1 months. The median PFS was not reached for patients with treatment-naïve CLL (n=19) after a median follow-up of 16.6 months, and median PFS was 29.5 months for patients with r/r CLL (n=30) after a median follow-up of 17.1 months. The combination of cirmtuzumab and ibrutinib was well tolerated in this trial.
In November 2020, we announced an interim clinical data update from the ongoing Phase 1/2 clinical trial evaluating TK216, an investigational, potentially first-in-class, targeted small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins, in patients with r/r Ewing sarcoma at the Connective Tissue Oncology Society 2020 Virtual Annual Meeting. The reported disease control rate (CR, partial response or stable disease) was 43%, including two patients with durable complete responses that were ongoing at over 1.5 years and 8 months on treatment.
In October 2020, we announced that the U.S. Food and Drug Administration granted Rare Pediatric Disease designation for TK216 for the treatment of Ewing sarcoma.
In November and December 2020, we raised an aggregate of approximately $109 million in gross proceeds from two underwritten offerings.
Expected Upcoming Milestones
Cirmtuzumab (ROR1 antibody) programs
Clinical data update for patients with MCL and CLL treated with cirmtuzumab plus ibrutinib in the ongoing Phase 1/2 study in Q2 2021
Clinical data update for patients with HER2-negative breast cancer in the ongoing Phase 1b study in Q2 2021
Preclinical data in additional ROR1 expressing tumors in Q2 2021
ROR1 CAR-T program
First-in-human dosing in China in the second half of 2021
TK216 (ETS inhibitor) program
Clinical data for patients with Ewing sarcoma treated in the ongoing Phase 1/2 expansion cohort in Q2 2021
Preclinical data in additional ETS-driven tumors in Q2 2021
Fourth Quarter and Full Year 2020 Financial Results
Our grant revenue was $1.6 million for the fourth quarter ended December 31, 2020. Our grant revenue is derived from a subaward under a grant from the California Institute for Regenerative Medicine (CIRM) to UC San Diego, which was awarded to advance our Phase 1/2 clinical trial evaluating cirmtuzumab in combination with ibrutinib for the treatment of patients with MCL or CLL. For the full year 2020, grant revenue was $3.4 million.
Our total operating expenses for the fourth quarter ended December 31, 2020 were $4.4 million. Research and development expenses for the quarter totaled $3.0 million, and general and administrative expenses for the quarter totaled $1.5 million. Net loss for the fourth quarter was $2.6 million, or a loss of $0.09 per share, basic and diluted. For the full year 2020, total operating expenses were $20.9 million. Net loss for the full year 2020 was $17.2 million, or a loss of $0.85 per share, basic and diluted.
As of December 31, 2020, we had $116.7 million in cash and cash equivalents. We believe these funds will be sufficient to fund our operations into 2023. As of December 31, 2020, we had approximately 48.8 million shares of common stock outstanding.
Management Webcast
As previously announced, Oncternal will host a webcast today, March 11, 2020, at 5:00 p.m. ET. The live webcast will be available online and may be accessed from the “Investors” page of the company website at http://investor.oncternal.com/. A replay of the webcast will be available beginning approximately one hour after the conclusion of the call and will remain available for at least 30 days thereafter.
About Oncternal Therapeutics
Oncternal Therapeutics is a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies for the treatment of cancers with critical unmet medical need. Oncternal focuses drug development on promising yet untapped biological pathways implicated in cancer generation or progression. The clinical pipeline includes cirmtuzumab, an investigational monoclonal antibody designed to inhibit the ROR1 pathway, a type I tyrosine kinase-like orphan receptor, that is being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of patients with mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL) and in an investigator-sponsored, Phase 1b clinical trial in combination with paclitaxel for the treatment of women with HER2-negative metastatic or locally advanced, unresectable breast cancer. The clinical pipeline also includes TK216, an investigational targeted small-molecule inhibitor of the ETS family of oncoproteins, that is being evaluated in a Phase 1 clinical trial for patients with Ewing sarcoma alone and in combination with vincristine chemotherapy. In addition, Oncternal has a program utilizing the cirmtuzumab antibody backbone to develop a CAR-T therapy that targets ROR1, which is currently in preclinical development as a potential treatment for hematologic cancers and solid tumors. More information is available at www.oncternal.com.
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Bladerunner, Midastouch, Just wondering if you have any opinions on Enlivex Therapeutics (ENLV)? Thanks
>>> Has Israel just found the cure for Covid?
Experimental Covid drug cures 30 out of 30 moderate to severe cases in Phase I clinical trial at Israeli hospital. Second new drug also shows promise.
Israel 21c
By Abigail Klein Leichman
FEBRUARY 7, 2021
https://www.israel21c.org/has-israel-just-found-the-cure-for-covid/
Even with Israel’s world-leading rollout of Covid-19 vaccinations, drugs to treat Covid patients are in desperate need across the world.
Two such drugs developed in Israel show great promise in clinical trials: EXO-CD24 and Allocetra.
EXO-CD24, an experimental inhaled medication developed at Tel Aviv Sourasky Medical Center, cured all 30 moderate-to-severe cases in a Phase I clinical trial.
Developed over the past six months at the hospital, EXOCD24 stops the “cytokine storm” – where the immune system goes out of control and starts attacking healthy cells – that occurs in the lungs of 5-7% of Covid-19 patients.
“To date, the preparation has been tried with great success on 30 severe patients, in 29 of whom the medical condition improved within two to three days and most of them were discharged home within three to five days. The 30th patient also recovered but after a longer time,” the hospital reports.
“The drug is based on exosomes, [vesicles] that are released from the cell membrane and used for intercellular communication. We enrich the exosomes with 24CD protein. This protein is expressed on the surface of the cell and has a known and important role in regulating the immune system,” explained Dr. Shiran Shapira, director of the laboratory of Prof. Nadir Arber, who has been researching the CD24 protein for over two decades.
“The preparation is given by inhalation, once a day, for only a few minutes, for five days,” Shapira said.
She said the experimental treatment has two unique characteristics. The first is that it inhibits the over-secretion of cytokines. The second is that it is delivered directly to the lungs and therefore has no systemic side effects that injected or oral drugs can cause.
“Even if the vaccines perform their function, and even if no new mutations are produced then still in one way or another the corona will remain with us,” said Arber, director of the medical center’s Integrated Cancer Prevention Center. “To this end, we have developed a unique drug, EXO-CD24.”
Arber added that this advanced preparation “can be produced quickly and efficiently and at a very low cost in every pharmaceutical facility in the country, and in a short time globally.”
Prof. Ronni Gamzu, CEO of the medical center, said, “Prof. Arber’s results for first-phase research were excellent and gave us all confidence in the method he has been researching [here] for many years. I personally assisted him in further obtaining the approvals from the Ministry of Health for further research.”
Allocetra
Meanwhile, Enlivex Therapeutics last week reported positive results from a multi-center Phase II clinical trial of its experimental Covid-19 immunotherapy drug Allocetra in severe and critical Covid-19 patients.
We reported in October that five Covid-19 intensive care patients were discharged from Hadassah University Medical Center in Jerusalem after treatment with Allocetra.
Nine severe and seven critical Covid-19 patients were treated with Allocetra in the Phase II clinical trial. Fourteen of them recovered and were discharged from the hospital after an average of 5.3 days.
The Phase II trial originally was expected to enroll 24 patients but was “completed early in support of anticipated accelerated regulatory filings of the trial’s positive safety and efficacy data,” Enlivex reported.
Altogether, 19 out of 21 Phase II and Phase Ib Allocetra trial patients recovered and were discharged from the hospital after an average of 5.6 days. Most of the patients in both studies had pre-existing risk factors such as male gender, obesity and hypertension.
“The results we have seen from the 12 Covid-19 patients treated to date with Allocetra are exciting,” said Prof. Vernon van Heerden, head of the General Intensive Care Unit at Hadassah and the lead investigator of both clinical trials.
“The Phase II patients who have been discharged from the hospital are currently healthy. We believe that these compelling results have demonstrated the safety and efficacy of Allocetra in these complicated patients, highlighting the potential of Enlivex’s product candidate to benefit severe and critical Covid-19 patients as well as others suffering from cytokine storms and organ dysfunctions across various clinical indications.”
Allocetra is based on the research of Enlivex chief scientific and medical officer Dr. Dror Mevorach, head of internal medicine and of one of Hadassah’s coronavirus wards. It works by restoring balance to the immune system.
Mevorach said Allocetra “may have utility as a safe and efficacious treatment … regardless of the specific coronavirus mutation that afflicted the patients, and across different life-threatening, high mortality clinical indications with high unmet medical needs.”
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A bullish case for MGTA (from Motley Fool).
Magenta Is Ready to Paint Your Portfolio Green
This clinical-stage biotech is looking to make stem cell transplantation faster, easier, and safer for both donors and patients.
Patrick Bafuma
(Fuma102)
Feb 27, 2021 at 8:08AM
For patients with certain blood cancers, a stem cell transplant can often be a curative treatment. However, it is also a complex process riddled with severe toxicities and high rates of relapse or failure. These issues are at least part of the reason why only about 60% of cancer patients, a third of genetic disease patients, and 5% of autoimmune patients who are eligible to receive a stem cell transplant actually receive one. It's not hard to imagine that should a safer, more efficient process exist, the market could potentially expand. Enter Magenta Therapeutics (NASDAQ:MGTA).
The clinical-stage biotech's lead compound, MGTA-145, has the potential to revolutionize the current standard-of-care transplant process for both patients and donors, making it faster and safer for all parties.
Focusing on donors
When you donate stem cells, on average it takes five or more days to collect them. This is an inefficient process for a donor because it requires daily injections of medication called G-CSF, which boosts the release of stem cells from bone marrow into the bloodstream for collection. These daily injections cause significant bone pain and a handful of other side effects. To make matters worse, about 15% of donors will need multiple collection attempts, which mean further daily G-CSF injections. Perhaps it's not surprising that about 50% of registered donors decline to donate when they are called to do so.
Patients receiving a stem cell transplant, meanwhile, first have to undergo G-CSF treatment to boost their stem cells' release from bone marrow, then undergo chemo and radiation to try to get rid of any bad or cancerous cells originating in the bone marrow before they get their transplant. That's a lot of steps, a lot of time, and a lot of potential side effects.
Magenta is working to streamline this process, and MGTA-145 has the potential to become the first medicine approved for this mobilization of stem cells in both donors as well as transplant recipients. (While G-CSF is commonly utilized by clinicians off-label for the mobilization of donor stem cells, the drug is actually not explicitly approved for that usage.) Phase 1 studies demonstrated a threefold increase in the number of stem cells collected from healthy donors when treated with MGTA-145 compared with G-CSF, and amazingly, 88% of donors infused with MGTA-145 were able to have their stem cells collected that same day, compared with zero for the G-CSF group. Lastly, there was only a 1% rate of grade 2 to grade 4 side effects with MGTA-145; that rate is 38% for G-CSF.
Data from two phase 2 trials are expected in the second half of 2021. One trial is looking at MGTA-145's effect on stem cell mobilization and collection, as well as the ever-important progression-free survival rate, in cases of multiple myeloma. The other is examining MGTA-145's effect on stem cell mobilization, collection, and disease outcomes for acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and myelodysplastic syndrome (MDS).
How green can Magenta get?
So far, MGTA-145 appears faster, safer, and requires fewer collections from donors than the current standard of care, with no competition on the horizon. So what's the addressable market look like?
One place to start measuring is with Sanofi's (NASDAQ:SNY) plerixafor, which is currently given to approximately half of patients who undergo stem cell treatment for certain blood cancers. That drug had sales of $200 million in 2019.
However, because MGTA-145 makes donation less of a hassle, it's no stretch to imagine that the drug could work as a catalyst to increase the number of stem cell transplants performed annually. Management estimates that they can grow the market from today's about 90,000 worldwide to more than 175,000. Magenta has also begun to explore MGTA-145's utility in the gene therapy market, having already partnered with Beam therapeutics (NASDAQ:BEAM), bluebird bio (NASDAQ:BLUE), and AVROBIO (NASDAQ:AVRO) to more efficiently mobilize stem cells for their gene therapies. Partnered phase 2 trials are already under way.
Should future MGTA-145 trials continue to be positive, which I think is likely, $200 million would likely be just the beginning of the addressable market. It could easily be more than double that when you account for the potential increase in donors and procedures, as well as the gene therapy market. With a current market cap of about $500 million, and other promising candidates in the pipeline, Magenta is well positioned to add some green to healthcare investors' portfolios over the coming years -- not to mention making a difference for tens of thousands of patients and donors.
This article represents the opinion of the writer, who may disagree with the “official” recommendation position of a Motley Fool premium advisory service. We’re motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.
Patrick Bafuma owns shares of Bluebird Bio. The Motley Fool owns shares of and recommends Bluebird Bio. The Motley Fool has a disclosure policy.
Bladerunner
gfp, I'm watching GMTX, but haven't pulled the trigger. Sorry to hear about your parents.
Bladerunner
Blade, I saw your post on GMTX and wondered if you decided to invest? Also saw the bio newsletter post over on Dew's board (link below), and the author mentions 4 stocks involved in the dry macular space. Things looked great until seeing that 20% of the dry macular patients had flipped to 'wet' during treatment (compared to 1% in the placebo group).
Not sure if GMTX's drug will do better, but 20% of patients going wet is no laughing matter since the wet form is devastating. My mom had it, and after several years of Avastin and then Lucentis injections, she ultimately lost most of her vison over a 3 day period. She went from 20/80 to basically blind, what a nightmare. My dad had the dry form and luckily it stayed dry.
With the total lack of current treatments (other than vitamins/minerals), the GMTX drug could be a blockbuster if it can improve on that 20% risk aspect. I think that other drug was from Apellis Pharma -
https://mattbiotech.substack.com/p/4-the-geographic-atrophy-issue-apls
https://investorshub.advfn.com/boards/read_msg.aspx?message_id=161872601
Blade, >> BPTH <<
Thanks, sounds interesting -
>>> Bio-Path Quadruples on Receipt of Cancer-Drug Tech Patent
Bio-Path shares as much as quadrupled. The company received a patent that builds on earlier ones protecting the platform tech for its nanoparticle drugs.
DAN WEIL
FEB 10, 2021
https://www.thestreet.com/investing/bio-path-shares-up-on-patent-for-cancer-drug-technology?puc=yahoo&cm_ven=YAHOO
Bio-Path Holdings (BPTH) - shares as much as quadrupled on Wednesday after the biotechnology company said it received a patent for its cancer-treatment technology.
Bio-Path recently traded at $16.75, compared with its Tuesday close at $5.20. It has traded on Wednesday at as much as a 52-week high $24.34. It was trading below $3 in mid-March.
The technology receiving the patent is DNAbilize liposomal delivery and antisense technology, used to develop a portfolio of targeted nucleic acid cancer drugs.
The patent is titled "P-ethoxy nucleic acids for liposomal formulation," Bio-Path said.
“The new patent builds on earlier patents granted that protect the platform technology for DNAbilize, the company’s novel RNAi nanoparticle drugs.”
The U.S. Patent and Trademark Office also has sent notification for a patent related to the company’s lead product candidate, prexigebersen, Bio-Path said.
“Our innovative DNAbilize platform improves upon the drawbacks of traditional approaches, which are limited by the toxicity induced by either the DNA backbone or the lipid delivery,” Chief Executive Peter Nielsen said in a statement.
“DNAbilize overcomes these challenges by combining a neutral charge P-ethoxy DNA backbone with a neutral charge liposome.”
Further, “The result is a high payload liposome with DNA safely delivered inside non-toxic cell membrane-like molecules, allowing us to deliver antisense DNA in high doses to target cells through the blood and lymphatic system with no evidence of toxicity in patients in clinical trials to date, in contrast to other lipid delivery technologies with dose limiting toxicities,” he said.
The P-ethoxy nucleic acids patent “is the third patent in our family of platform intellectual property and offers expanded defense of our DNAbilize platform technology,” Nielsen said.
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gfp, I sold all my HGEN (nice profit) way too early.
I have a small position in TRIL.
I've established a decent-sized position in BPTH.
Bladerunner
Blade, Yes, nice moves for KZIA and MGTA, and ONCT is also looking good.
Btw, are you still following HGEN? It looks interesting for a possible run-up ahead of their Phase 3 topline data in March. CYDY is their main competitor in the Covid 'cytokine storm' space, and the CYDY Phase 3 data announcement is now long overdue (trial ended mid-Jan). CYDY has shady management, and in addition to HGEN's own Phase 3 data, HGEN could get an added boost if the CYDY trial disappoints.
TA/charts are not generally as useful with bio stocks, but the HGEN chart looks like an impending breakout.
Also, just curious if you are still in TRIL? Thanks.
KZIA was up 20% today on 4X average daily volume.
MGTA was up 17% today on 3X average daily volume.
Two of my largest positions.
No news on either one that I'm aware of.
Bladerunner
P.S. ONCT has been moving up nicely.
ONCT up 9% hits 52-week high
Bladerunner
Blade, >> CRVS <<
It figures I would buy right before the financing announcement, lol. But promising stocks often bounce back quickly after a financing. Better to have the company adequately funded than to worry about a temporary dip in the share price.
Btw, that 'Psychedelic Medicine' sector idea is working out pretty well so far, and my basket of stocks already up 4-5%. Since the ETF is not available in the US yet, I put $500 each into 8 of the ETF's main holdings, for a total investment of $4000. So not huge either way, but I wanted to participate in the emerging sector. These stocks have started to move up with the recent introduction of the ETF. Personally I'm not thrilled with the idea of legalizing recreational use of cannabis and psychedelics, but investment-wise it has become a bonafide sector -
>>> The First Psychedelic's ETF is Here, Should You Invest?
February 11, 2021
by Neal Farmer
https://www.investorsobserver.com/news/featured/the-first-psychedelics-etf-is-here-should-you-invest
The world’s very first psychedelic exchange-traded fund began trading on Wednesday on the NEO Exchange in Toronto. The Horizons Psychedelic Stock Index ETF (TSX:PSYK) tracks the North American Psychedelics Index. The development marks a major milestone for the growing interest in the use of these drugs as potential mental health treatment options. The investment world has taken notice and has led to a few major companies attempting to capitalize on the fast-growing industry.
What’s in the ETF?
The Kevin O’Leary backed MindMed (MMEDF) is one of the 17 companies included in the ETF. MindMed has seen its shares soar since September as many are expecting it to follow the outlook for cannabis legalization. MindMed currently trades on the NEO Exchange and the over-the-counter market in the United States but has applied to be uplisted on the NASDAQ. Others such as Seelos Therapeutics (SEEL) and Nova Mentis Life Science (NOVA) have seen share prices jump in this time as well.
The 17 companies included in the Horizons Psychedelic Stock Index ETF are focused on using psychedelics to treat mental-health problems such as depression, anxiety, post-traumatic stress disorder (PTSD), and others. The introduction of many companies to such a hot corner of the investing market was bound to lead to at least one ETF eventually. The evolution of people’s opinions on cannabis leading it to be legalized for recreational use in many states and legal for medicinal purposes in the vast majority has partly led to bullish analysis on psychedelics for mental health treatment.
Case for Psychedelic Investment
The progression mindset about cannabis and its potentially beneficial attributes has led to some enthusiasm bleeding over to psychedelics but the biggest recent factor that led to share prices rising was the legalization of MDMA (i.e. ecstasy) in Oregon and Washington D.C. at licensed facilities. Other psychedelic substances were approved at licensed facilities for mental health purposes in these states with similar proposals in California, Vermont, and Iowa. This development along with some expectations that President Biden will advocate for the medicinal legalization due to mental health struggles in the U.S. along with an opioid crisis, has many wanting to invest now while its just taking off. Early legalization efforts and a massive mental health market are promising signs for this industry, but if cannabis is its most comparable product, it may take longer than most expect.
Case Against Psychedelic Investment
While a large majority of states have voted to legalize cannabis for medicinal use, only 11 have legalized for recreational use. The bigger problem for psychedelic medicinal use is that it took a long time for cannabis to get where it is now and it's still not fully legalized. Even the states that have approved it have many towns and cities that have banned retail stores. Additionally, cannabis stocks experienced a nice surge once they began trading but have been struggling since early 2019. Investors looking to jump on before this rocket takes off might want to consider that Oregon legalizing something isn't exactly a guarantee that the rest of the country jumping on board any time soon.
There were a lot of social factors that kept cannabis from being legalized even just for medicinal purposes for a long time. It seems arrogant to think psychedelics won't have similar problems. Getting over the preconception that these drugs aren't just for use at rave parties will be particularly difficult for a large segment of the population. Even still, some believe these drugs will disrupt a $70 billion market for mental health treatment that has only gotten more attention in recent years due to the nation’s various struggles especially now coming out of the coronavirus pandemic.
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CRVS announces pricing of public offering
Close this content, you can also use the Escape key at anytime
GlobeNewswire
Corvus Pharmaceuticals Announces Pricing of Public Offering of Common Stock
Corvus Pharmaceuticals, Inc.
Fri, February 12, 2021, 6:00 AM
More content below
CRVS
-10.4036%
BURLINGAME, Calif., Feb. 12, 2021 (GLOBE NEWSWIRE) -- Corvus Pharmaceuticals, Inc. (NASDAQ:CRVS), a clinical-stage biopharmaceutical company, today announced the pricing of an underwritten public offering of 8,571,429 shares of its common stock at a price to the public of $3.50 per share, for gross proceeds of $30.0 million before deducting estimated underwriting discounts and commissions and estimated offering expenses payable by Corvus. All of the shares are being offered by Corvus. In addition, Corvus has granted the underwriters of the offering a 30-day option to purchase up to an additional 1,285,714 shares of common stock at the public offering price, less underwriting discounts and commissions.
Corvus currently expects to use the net proceeds from this offering to fund its Phase 3 clinical trial of CPI-006 and development of its other product candidates, with any remaining proceeds for working capital and general corporate purposes.
The offering is expected to close on or about February 17, 2021, subject to satisfaction of customary closing conditions.
Cantor Fitzgerald & Co. and H.C. Wainwright & Co. are acting as joint book-running managers for the offering.
A shelf registration statement on Form S-3 relating to the securities being sold in this offering was declared effective by the Securities and Exchange Commission on March 19, 2020. The offering of these securities is being made only by means of a prospectus forming a part of the effective registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the offering have been filed with the SEC and a final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. A copy of the final prospectus supplement and accompanying prospectus relating to the offering, when available, may be obtained from: Cantor Fitzgerald & Co., Attention: Capital Markets, 499 Park Avenue, 6th Floor, New York, NY 10022 or by email at prospectus@cantor.com or H.C. Wainwright & Co., LLC, 430 Park Avenue, 3rd Floor, New York, NY 10022, by telephone at (646) 975-6996 or by email at placements@hcwco.com.
Bladerunner
Gfp,
I sold KURA quite a while ago. I should have held longer, Another screw-up.
Bladerunner
Blade, Thanks. Also, looks like KURA has been doing well. Any thoughts on them? TIA.
Btw, I recently set up a board to follow the emerging 'Psychedelic Medicine' sector (link below). Back in 2019 I followed the saga of Spravato / Esketamine (the mirror image isomer of Ketamine), which received FDA approval for treatment resistant depression. It's become obvious that these substances are to be made legal (for better or worse), so the search by investors for early stage companies is on. An ETF just came out covering the sector (Horizons Psychedelic Stock Index ETF), but so far is only available in Canada -
https://investorshub.advfn.com/Psychedelic-Medicine-Sector-37972/
MGTA is one of my largest positions. If everything works out, MGTA wull transform the way transplant medicine is done.
Bladerunner
Blade, Any thoughts on MGTA? The stock has had a good move in the last several months. I'm not up on the stem cell area, but looks like some advanced science. I remember you had mentioned MGTA a while back -
>>> Magenta Therapeutics, Inc. (MGTA), a clinical-stage biotechnology company, develops novel medicines to extend the curative power of stem cell transplant, gene therapy, genome editing, and cell therapy to patients. The company is developing C100, C200, and C300 targeted antibody-drug conjugates for transplant conditioning; MGTA-145, a novel stem cell mobilization product candidate to control stem cell mobilization; MGTA-456, an allogeneic stem cell therapy to control stem cell growth; E478, a small molecule aryl hydrocarbon receptor antagonist for the expansion of gene-modified stem cells; and G100, an antibody-drug conjugate program to prevent acute graft and host diseases. It has a has a research and clinical collaboration agreement with AVROBIO, Inc.to evaluate targeted antibody-drug conjugate as a conditioning regimen for lentiviral gene therapies; and clinical trial collaboration with bluebird bio, Inc. to evaluate the utility of MGTA-145, in combination with plerixafor, for mobilization and collection of stem cells in adults and adolescents with sickle cell disease. The company was formerly known as HSCTCo Therapeutics, Inc. and changed its name to Magenta Therapeutics, Inc. in February 2016. Magenta Therapeutics, Inc. was founded in 2015 and is headquartered in Cambridge, Massachusetts. <<<
Blade, >> CRDF <<
Thanks. I replaced LIFE with CRDF on the list. It's great having a knowledgeable guy like you around :o)
It's going to take a while to get even moderately up to speed on these stocks. Bio is the hardest sector there is, and no matter how much research one does, it never seems to be enough.
For stock exposure, I mostly am using broad index ETFs, and sector ETFs have largely replaced the role that individual stocks once played in the portfolio. Still, it's nice to have some promising stocks to follow. I use strict position limits, so you don't have to worry about me misusing your stock ideas to 'bet the farm' :o)
gfp,
LIFE is only on the list because of the Redditors. Fundamentally, I don't really like it.
CRDF has results out today and my cancer docs really like the results. I don't own any right now, but may get back in, if I have some cash.
Cardiff Oncology Presents Phase 2 mCRPC Trial Data Showing a Two-Fold Increase in Efficacy with an Optimized Onvansertib Dosing Schedule
Thu, February 11, 2021, 5:00 A
CRDF
+9.85%
- Two-fold increase (29% to 63%) in disease control rate at 12 weeks (the trial's primary efficacy endpoint) seen in patients receiving onvansertib for 14 days vs. 5 days in a 21-day cycle
- 75% (6/8) of evaluable patients in the optimized dosing cohort had stable disease upon radiographic scan at 12 weeks
- All patients in the optimized dosing cohort achieving the primary efficacy endpoint remain on treatment
- Trial on track to meet prespecified criteria for success on its primary efficacy endpoint, with a 35% disease control rate at 12 weeks in evaluable patients across all three cohorts. Patients eligible for the trial have two consecutive rises in PSA levels, indicating initial resistance to Zytiga® (abiraterone).
SAN DIEGO, Feb. 11, 2021 /PRNewswire/ -- Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage biotechnology company developing drugs to treat cancers with the greatest medical need for new treatment options, including KRAS-mutated colorectal cancer, pancreatic cancer, castrate-resistant prostate cancer and leukemias, today announced that updated data from its Phase 2 metastatic castrate-resistant prostate cancer (mCRPC) trial were featured in a virtual oral poster presentation at the American Society of Clinical Oncology Genitourinary Cancers Symposium (ASCO-GU). The ongoing Phase 2 trial evaluates the all-oral combination of onvansertib, abiraterone and prednisone in patients showing initial abiraterone resistance, as defined by two consecutive rises in prostate-specific antigen (PSA) levels.
Cardiff Oncology is a clinical-stage biotechnology company and our mission is to develop new treatment options for cancer patients in indications with the greatest medical need. Our goal is to overcome resistance, improve response to treatment and increase overall survival. Our investigational drug, onvansertib, a first-in-class, third-generation Polo-like Kinase 1 (PLK1) inhibitor, is being evaluated in combination with standard-of-care chemotherapy and targeted therapeutics.
We are assessing tumor genomics and using our expertise in biomarker technology to rapidly evaluate patient response to treatment.
Cardiff Oncology is a clinical-stage biotechnology company and our mission is to develop new treatment options for cancer patients in indications with the greatest medical need. Our goal is to overcome resistance, improve response to treatment and increase overall survival.
Our investigational drug, onvansertib, a first-in-class, third-generation Polo-like Kinase 1 (PLK1) inhibitor, is being evaluated in combination with standard-of-care chemotherapy and targeted therapeutics. We are assessing tumor genomics and using our expertise in biomarker technology to rapidly evaluate patient response to treatment.
Newly presented data from the Phase 2 mCRPC trial showed that increasing the number of days of treatment with onvansertib from 5 to 14 in a 21-day cycle was associated with a greater than two-fold increase (29% to 63%) in disease control rate (DCR; defined by lack of PSA progression) at 12 weeks, the trial's primary efficacy endpoint. Six of eight (75%) evaluable patients receiving onvansertib for 14 of 21 days per cycle had stable disease upon radiographic scan at 12 weeks and five of these patients remain on treatment to-date. Across all cohorts, the DCR at 12 weeks is 35% (13/37), indicating the trial is on track to meet the stated criteria for success on its primary efficacy endpoint (30% DCR at 12 weeks).
"The preliminary data presented at ASCO-GU support a clinically meaningful onvansertib exposure effect," said David Einstein, M.D., attending physician at Beth Israel Deaconess Medical Center and principal investigator of the onvansertib mCRPC Phase 2 trial. "In the first eight patients treated to-date on Arm C, we are excited to see an increase in DCR with greater time on onvansertib, without excessive toxicity. Together with the clinically meaningful rates of disease control, and duration of disease control, we are seeing across all cohorts, these data demonstrate onvansertib's potential to address a critical unmet need for patients with abiraterone-resistant mCRPC."
"As data from this trial continue to emerge, we are very pleased to see increased efficacy with an optimized dosing schedule that is both well tolerated and increases the number of days a patient receives onvansertib in combination with abiraterone by nearly 3-fold" said Mark Erlander, Ph.D., chief executive officer of Cardiff Oncology. "These data suggest that the synergy demonstrated between onvansertib and abiraterone in pre-clinical models is being observed clinically. The trial's biomarker analyses are also promising, as the identification of mutations associated with response to the combination of onvansertib and abiraterone may enable more efficient design of future clinical studies and the identification of patients most likely to benefit from this combination."
Key data and conclusions from the ASCO-GU presentation include:
Efficacy:
The optimized dosing schedule of cohort C shows a greater than two-fold improvement in disease control rate compared to cohorts A and B
75% (6/8) of evaluable patients in cohort C had radiographic SD at 12 weeks, compared to 53% (9/17) in cohort A, 42% (5/12) in cohort B and 54% (20/37) across all cohorts
All cohort C patients achieving the primary efficacy endpoint remain on treatment
35% (13/37) of evaluable patients across all cohorts (A-C) achieved the primary efficacy endpoint of disease control at 12 weeks
Efficacy was observed in patients harboring androgen receptor (AR) alterations associated with abiraterone resistance across all 3 arms
Biomarker:
Circulating tumor DNA (ctDNA) analysis revealed differences in baseline genomic profiles of patients achieving SD at 12 weeks vs. those progressing at or before 12 weeks
Mutations present exclusively in patients with SD at 12 weeks were associated with cell cycle and DNA repair pathways that may result in increased efficacy of the onvansertib-abiraterone combination
Safety:
Data show that the combination of onvansertib and abiraterone is well tolerated across the three different dosing schedules of cohorts A-C:
The virtual poster, A Phase 2 Study of the Polo-like Kinase 1 (PLK1) Inhibitor Onvansertib in Combination with Abiraterone and Prednisone in Patients with Metastatic Castration-Resistant Prostate Cancer (mCRPC), is available on the "Scientific Presentations" section of the Cardiff Oncology website at https://cardiffoncology.com/scientific-presentations/.
About the Phase 2 Trial of Onvansertib in Metastatic Castration-Resistant Prostate Cancer
This trial is a Phase 2 open-label study of onvansertib in combination with abiraterone and prednisone, all administered orally, in patients with metastatic castration-resistant prostate cancer showing signs of early progressive disease (demonstrated by two rising prostate-specific antigen values separated by at least one week with no or minimal symptoms) while on Zytiga®/prednisone therapy. The primary efficacy endpoint is the proportion of patients achieving disease control after 12 weeks of study treatment, as defined by a lack of prostate-specific antigen (PSA), radiographic, or symptomatic progression. The trial is being conducted by Beth Israel Deaconess Medical Center (BIDMC), Dana-Farber Cancer Institute (Dana-Farber), and Massachusetts General Hospital Cancer Center (MGH). David Einstein, M.D., Genitourinary Oncology Program at BIDMC, is the principal investigator for the trial. For more information on the trial, please visit https://www.clinicaltrials.gov/ct2/show/NCT03414034.
About Cardiff Oncology, Inc.
Cardiff Oncology is a clinical-stage biotechnology company with the singular mission of developing new treatment options for cancer patients in indications with the greatest medical need. Our goal is to overcome resistance, improve response to treatment and increase overall survival. We are developing onvansertib, a first-in-class, third-generation Polo-like Kinase 1 (PLK1) inhibitor, in combination with standard-of-care chemotherapy and targeted therapeutics. Our clinical development programs incorporate tumor genomics and biomarker technology to enable assessment of patient response to treatment. We have three clinical programs that have demonstrated the safety and efficacy of onvansertib: a Phase 1b/2 study of onvansertib in combination with FOLFIRI/Avastin® (bevacizumab) in KRAS-mutated metastatic colorectal cancer (mCRC); a Phase 2 study of onvansertib in combination with Zytiga® (abiraterone)/prednisone in metastatic castration-resistant prostate cancer (mCRPC); and a Phase 2 study of onvansertib in combination with decitabine in relapsed or refractory acute myeloid leukemia (AML). A new Phase 2 trial of onvansertib in combination with nanoliposomal irinotecan, leucovorin and fluorouracil for the second-line treatment of patients with metastatic pancreatic ductal adenocarcinoma (PDAC) is planned for initiation in the first half of 2021. For more information, please visit https://www.cardiffoncology.com.
Bladerunner
Blade, >> MYOV <<
I see MYOV has a relatively huge $745 mil cash position, so a good sign. I noticed you have 3 UK based biotechs (and one Australian) among your current favorites, so you must be an 'Anglophile' :o) I like the UK also, and have been there several times on vacation.
Btw, I decided to put my recent IPIX profits into your current bio picks. It was only $10 K, but it's a start, and I spread it evenly among -
CRVS
CRIS
FSTX
LIFE - waiting for a pullback
MYOV
ONCT
PRVB
I already had similar size positions in HGEN and KZIA, so counting IPIX that makes 10 stocks. So a lot to get up to speed on. These bios take forever to research, but I'll start with the basic info and go from there. I also have a modest investment in the ARK Genomic Revolution ETF (ARKG), which is the easy path to take :o)
Let me know when you have some additions/deletions to your fav lists,
Thanks
MYOV presents 3rd quarter fiscal year 2020
(How this stock is not trading at least three times higher than where it is is baffling to me. It's trading for about 3X cash.)
Myovant Sciences Announces Corporate Updates and Financial Results for Third Quarter of Fiscal Year 2020
Myovant Sciences, Inc.
Thu, February 11, 2021, 3:55 AM
MYOV
-0.13%
ORGOVYX™ (relugolix) approved by the U.S. Food and Drug Administration (FDA) in December 2020 as the first and only oral GnRH receptor antagonist for adult patients with advanced prostate cancer; ORGOVYX launched in the U.S. in early January 2021
Announced collaboration with Pfizer in December 2020 to jointly develop and commercialize relugolix in oncology and relugolix combination tablet in women’s health in the U.S. and Canada
Reported positive one-year efficacy and safety data from Phase 3 SPIRIT program for relugolix combination therapy in women with endometriosis-associated pain, including bone mineral density data
FDA review of New Drug Application for relugolix combination tablet for uterine fibroids remains on track for a decision by June 1, 2021 target action date; European Commission decision on uterine fibroids Marketing Authorization Application expected in mid-calendar year 2021
Announced Phase 3 SERENE study to assess the contraceptive efficacy of relugolix combination tablet; study designed to support potential benefit of prevention of pregnancy for women taking relugolix combination tablet for the treatment of uterine fibroids and endometriosis, if approved for those indications
Cash, cash equivalents, and marketable securities of $745.8 million as of December 31, 2020
BASEL, Switzerland, Feb. 11, 2021 (GLOBE NEWSWIRE) -- Myovant Sciences (NYSE: MYOV), a healthcare company focused on redefining care for women and for men, today announced corporate updates and financial results for the third quarter of fiscal year 2020.
“The FDA approval of ORGOVYX and the landmark collaboration with Pfizer represent pivotal catalysts that have advanced Myovant’s purpose of redefining care and its transformation into a commercial-stage company with compelling near-term opportunities in oncology and women’s health,” said David Marek, Chief Executive Officer of Myovant Sciences, Inc. “We are encouraged by the early launch progress for ORGOVYX and expect this momentum to continue to build with the Pfizer uro-oncology sales team recently joining the Myovant sales team in the field. ORGOVYX has the potential to become the new androgen deprivation therapy standard of care in advanced prostate cancer. Combined with relugolix combination tablet, if approved in uterine fibroids and endometriosis, these products are expected to drive significant revenue growth for Myovant in the years to come.”
Third Quarter Fiscal Year 2020 and Recent Corporate Updates
Pfizer Collaboration
In December 2020, Myovant entered into a collaboration agreement with Pfizer under which Myovant and Pfizer will jointly develop and commercialize relugolix in oncology and women’s health and equally share profits and certain expenses, in the U.S. and Canada (the Co-Promotion Territory).
In December 2020, Myovant received a $650.0 million upfront payment and is eligible to receive additional regulatory and sales milestones, for total net payments of up to $4.2 billion.
Myovant granted Pfizer an exclusive option to acquire development and commercialization rights to relugolix in oncology outside of the Co-Promotion Territory (excluding certain Asian markets). If Pfizer exercises this option, Myovant will receive an additional $50.0 million payment and will be eligible to receive double-digit royalties on net sales. Pfizer’s decision is expected in the first half of calendar year 2021.
ORGOVYX
On December 18, 2020, the U.S. Food and Drug Administration (FDA) approved ORGOVYX for the treatment of adult patients with advanced prostate cancer. ORGOVYX, which was granted Priority Review by the FDA, is the first and only oral GnRH receptor antagonist for men with advanced prostate cancer.
ORGOVYX was launched in the U.S. and authorized specialty distribution channels were fully stocked in early January 2021.
The ORGOVYX patient support program launched in early January, including access to free trial for new commercial and government insured patients and co-pay support for eligible commercial patients.
Myovant completed the hiring of its 100-person oncology sales force during December 2020. All sales professionals completed training and began actively promoting ORGOVYX to target prescribers in early January 2021.
Pfizer’s uro-oncology sales force was trained in January 2021 and began actively promoting ORGOVYX to target prescribers in early February 2021.
Myovant is engaging in coverage negotiations with key commercial and Medicare Part D payors, with some coverage anticipated by the middle of calendar year 2021 and broad coverage anticipated by the end of calendar year 2021.
Relugolix Combination Tablet
• Uterine Fibroids
In October 2020, Myovant presented the following data for relugolix combination therapy in women with uterine fibroids at the American Society for Reproductive Medicine (ASRM) 2020 Virtual Congress:
- One-year efficacy and safety data from the LIBERTY long-term extension study (Scientific Congress Prize Paper Session 1).
- A validated exposure-response model simulating long-term effects of relugolix combination therapy on bone mineral density at the lumbar spine.
- A poster describing the improvement of pain associated with uterine fibroids in the LIBERTY Phase 3 program (1st Place in Poster Competition).
• Endometriosis
In October 2020, data from the replicate Phase 3 SPIRIT 1 and SPIRIT 2 studies were presented at the ASRM 2020 Virtual Congress in an oral presentation named the Prize Paper by the Endometriosis Special Interest Group.
In January 2021, Myovant and Pfizer announced positive one-year data from the SPIRIT extension study of once-daily relugolix combination therapy in women with endometriosis. 84.8% and 73.3% of women reported clinically meaningful reductions in dysmenorrhea (menstrual pain) and non-menstrual pelvic pain over one year (52 weeks) with stable bone mineral density after initial minimal loss. The data are consistent with the efficacy and safety profile observed through 24 weeks in the Phase 3 SPIRIT 1 and SPIRIT 2 studies.
The results of the SPIRIT clinical program will support a New Drug Application (NDA) for relugolix combination tablet for the treatment of women with endometriosis, anticipated to be submitted to the FDA in the first half of calendar year 2021.
CEO Appointment
In January 2021, Myovant announced the appointment of David Marek as Chief Executive Officer of Myovant Sciences, Inc. Concurrent with this appointment, Mr. Marek was also appointed as Principal Executive Officer of Myovant Sciences Ltd. and as a member of its Board of Directors.
COVID-19 Pandemic Environment
Myovant’s priorities during the COVID-19 pandemic are protecting the health and safety of its employees and patients while continuing its mission to redefine care for women and for men. To date, the impact of the COVID-19 pandemic on Myovant’s ability to advance its clinical studies, regulatory activities, commercial launch activities for ORGOVYX, and preparations for the potential commercialization of relugolix combination tablet has been limited, and all of Myovant’s publicly announced milestones remain on track. At this time, Myovant does not believe that the COVID-19 pandemic has disproportionately impacted it relative to other companies in Myovant’s industry and the medical community appears to be highly engaged with Myovant’s field team. To date, Myovant has not experienced supply constraints, and believes it has procured sufficient quantities of relugolix drug substance to meet its U.S. ORGOVYX launch plans and U.S. launch plans for relugolix combination tablet, if approved. However, if the COVID-19 pandemic persists, and depending on the further evolution of the pandemic and its effects on Myovant’s activities, Myovant may experience more significant impacts on its business operations.
Expected Upcoming Milestones
Data from the LIBERTY randomized withdrawal study, including efficacy and safety data of relugolix combination therapy in women with uterine fibroids for up to two years, is expected in the first quarter of calendar year 2021.
Marketing Authorization Application (MAA) submission to the European Medicines Agency (EMA) for relugolix monotherapy for advanced prostate cancer expected in the first quarter of calendar year 2021.
Myovant and Pfizer plan to initiate in the first half of calendar year 2021 a Phase 3 open-label clinical study in the U.S. to assess the contraceptive efficacy of relugolix combination tablet. The SERENE study will enroll sexually active, healthy premenopausal women ages 18-35 years with presumed normal fertility. Women will receive once-daily relugolix combination tablet for 13 28-day cycles. The primary efficacy endpoint will be the Pearl Index, defined as the number of on-treatment pregnancies per 100 women-years of treatment. Positive data from the SERENE study would further differentiate relugolix combination tablet by potentially adding the benefit of prevention of pregnancy for women taking relugolix combination tablet for the treatment of uterine fibroids and endometriosis, if approved for these indications.
FDA decision for relugolix combination tablet for the treatment of uterine fibroids expected by June 1, 2021 target action date.
NDA submission to the FDA for relugolix combination tablet for the treatment of women with endometriosis-associated pain expected in the first half of calendar year 2021.
European Commission decision on the uterine fibroids MAA expected in mid-calendar year 2021. If approved, this launch will be executed by Gedeon Richter, Myovant’s commercialization partner for relugolix combination tablet for the uterine fibroids and endometriosis indications in Europe and certain other international markets.
MAA submission to EMA for relugolix combination tablet for the treatment of women with endometriosis-associated pain expected in calendar year 2021. Gedeon Richter will be the MAA sponsor.
Third Quarter Fiscal Year 2020 Financial Summary
Collaboration revenue in the three months ended December 31, 2020, was $1.4 million and represents partial amortization of the upfront payment received from Pfizer.
Research and development (R&D) expenses in the three months ended December 31, 2020, were $30.5 million compared to $48.9 million for the comparable prior year period. The decrease in R&D expenses reflects a reduction in clinical study costs as a result of the completion and continued wind down of Myovant’s Phase 3 LIBERTY, HERO, and SPIRIT studies, cost reimbursements from Pfizer for certain R&D expenses, and a reduction in share-based compensation expenses. This decrease was partially offset by an increase in personnel expenses, mainly driven by the continued expansion of Myovant’s medical affairs organization, in preparation for Myovant’s U.S. commercial launch of ORGOVYX for adult patients with advanced prostate cancer and the potential U.S. commercial launches of relugolix combination tablet for the women’s health indications, if approved.
Selling, general, and administrative (SG&A) expenses in the three months ended December 31, 2020, were $49.2 million compared to $29.1 million for the comparable prior year period. The increase was primarily due to higher expenses related to commercial readiness activities to support the ORGOVYX U.S. commercial launch and the potential U.S. commercial launches of relugolix combination tablet as well as higher personnel-related costs primarily due to the hiring of Myovant’s commercial operations, marketing, market access teams, and the oncology sales force. The increase in SG&A expenses was also driven by general overhead expenses to support Myovant’s organizational growth. These items were partially offset by lower share-based compensation expenses in the three months ended December 31, 2020, as the prior year period included incremental expense related to the accelerated vesting of certain equity awards as a result of the change in control of Myovant.
Interest expense was $2.6 million in the three months ended December 31, 2020, compared to $3.7 million in the comparable prior year period. The decrease in interest expense, despite higher outstanding loan balances, was driven by the significantly lower interest rates associated with the Sumitomo Dainippon Pharma Loan Agreement as compared to Myovant’s previously outstanding debt obligations, which were repaid in December 2019.
Loss on extinguishment of debt in the three months ended December 31, 2019 was $4.9 million which resulted from the early retirement of Myovant’s outstanding obligations to NovaQuest and Hercules. There were no such losses in the three months ended December 31, 2020.
Interest income in the three months ended December 31, 2020, was less than $0.1 million compared to $0.6 million for the comparable prior year period. The decrease was primarily due to decreases in interest rates and lower balances in cash equivalents and marketable securities during the current year period.
Other income, net in the three months ended December 31, 2020, was $5.9 million compared to $0.6 million for the comparable prior year period. This was primarily the result of a larger foreign currency exchange gain on Myovant’s outstanding balance under the Sumitomo Dainippon Pharma Loan Agreement during the three months ended December 31, 2020.
Net loss for the three months ended December 31, 2020, was $73.8 million compared to $85.6 million for the comparable prior year period. On a per common share basis, net loss was $0.82 and $0.96 for the three months ended December 31, 2020, and 2019, respectively.
Capital resources: Cash, cash equivalents, marketable securities, and committed amounts available under the Sumitomo Dainippon Pharma Loan Agreement totaled $832.1 million as of December 31, 2020, and consisted of $745.8 million of cash, cash equivalents, and marketable securities and $86.3 million of available borrowing capacity under the Sumitomo Dainippon Pharma Loan Agreement.
Conference Call
As previously announced, Myovant will hold a webcast and conference call at 8:30 a.m. Eastern Time (5:30 a.m. Pacific Time) today, February 11, 2021, to discuss corporate updates and financial results for its third fiscal quarter ended December 31, 2020. Investors and the general public may access a live webcast of the call by visiting the investor relations page of Myovant’s website at investors.myovant.com. Institutional investors and analysts may also participate in the conference call by dialing 1-800-532-3746 in the U.S. or +1-470-495-9166 from outside the U.S.
The webcast will be archived on Myovant’s Investor Relations website following the call.
About Relugolix
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist that reduces testicular testosterone, a hormone known to stimulate the growth of prostate cancer, and ovarian estradiol, a hormone known to stimulate the growth of uterine fibroids and endometriosis. Relugolix (120 mg) is FDA-approved as ORGOVYX™ for adult patients with advanced prostate cancer. Relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) is under regulatory review in Europe and the U.S. for women with uterine fibroids and is under development for women with endometriosis.
About Myovant Sciences
Myovant Sciences aspires to redefine care for women and for men through purpose-driven science, empowering medicines, and transformative advocacy. We have one FDA-approved medicine, ORGOVYX™ (relugolix), for adult patients with advanced prostate cancer. Our lead product candidate, relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg), is under regulatory review in Europe and the U.S. for women with uterine fibroids and is under development for women with endometriosis. Myovant is also developing MVT-602, an oligopeptide kisspeptin-1 receptor agonist, which has completed a Phase 2a study for female infertility as part of assisted reproduction. Sumitovant Biopharma, Ltd., a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd., is Myovant’s majority shareholder. For more information, please visit our website at www.myovant.com. Follow @Myovant on Twitter and LinkedIn.
About Sumitomo Dainippon Pharma Co., Ltd.
Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including the U.S., Japan, China, and the European Union. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com.
About Sumitovant Biopharma Ltd.
Sumitovant is a global biopharmaceutical company with offices in New York City and London. Sumitovant is the majority shareholder of Myovant and Urovant, and wholly owns Enzyvant, Spirovant and Altavant. Sumitovant's promising pipeline is comprised of early-through late-stage investigational medicines across a range of disease areas targeting high unmet needs. Sumitovant is a wholly owned subsidiary of Sumitomo Dainippon Pharma. For further information about Sumitovant, please visit https://www.sumitovant.com.
Bladerunner
Blade, CRVS starting to show signs of life, both it and KZIA were up nice today. Still early, but perhaps CRVS' 9% short position and the low float (14 mil shares) are attracting the Reddit crowd. Kazia has an even lower float (6 mil shares), though the short position is negligible.
HGEN also looks interesting, and the short position is 15% of the float. They reportedly have top line Phase 3 Covid data coming in March. The good thing with these stocks (KZIA, CRVS, HGEN) is they have plenty of merit beyond the Reddit angle. The odds of picking a Reddit target are slim at best, so much better to own good promising stocks.
With IPIX I managed to get a 90% profit in a week, a rare occurrence, but welcome :o)
$ITRM 13g/a by RA capital 10.6% added 3.7 mill shares.
re: CNSP & LIFE
gfp,
CNSP's drug is a chemotherapeutic, while LIFE's drug is a targeted therapy.
LIFE & CCXI both hit 52-week highs today.
Bladerunner
Blade, The other stock mentioned in that Tip Ranks article (in addition to LIFE) was CNSP, and it also took off huge as soon as the article came out (article below). CNSP also looks like competition for KZIA -
CNS Pharmaceuticals (CNSP) - >>> 2 “Strong Buy” Penny Stocks With Over 200% Upside on the Horizon
Tip Ranks
February 4, 2021
https://finance.yahoo.com/news/2-strong-buy-penny-stocks-165357551.html
...Building on this, Eduardo Lecubarr, chief of the Small/Mid-Cap Strategy team, sees opportunity for investors now, especially in the smaller value stocks.
Bearing this in mind, we used TipRanks’ database to find two compelling penny stocks, according to Wall Street analysts. Both tickers boast a Strong Buy consensus rating and could climb over 200% higher in the year ahead.
CNS Pharmaceuticals (CNSP)
We will start with CNS Pharmaceuticals, a biotechnology company with a focus on the treatment of glioblastomas, a class of aggressive tumors that attack the braid and spinal cord. These cancers, while rare, are almost always terminal, and CNS is working a new therapy designed to more effectively cross the blood-brain barrier to attack glioblastoma.
Berubicin, CNS’s flagship drug candidate, is an anthracycline, a potent class of chemotherapy drugs derived from the Streptomyces bacteria strains, and used in the treatment of a wide variety of cancers. Berubicin is the first drug in this class to show promise against glioblastoma cancers.
The drug candidate has completed its Phase 1 clinical trial, in which 44% of patients showed a clinical response. This number included one patient who showed a ‘Durable Complete Response’, defined as a demonstrated lack of detectable cancer.
Following the success of the Phase 1 study, CNS applied for, and received, FDA approval of its Investigational New Drug application. This gives the company the go-ahead to conduct a Phase 2 study on adult patients, an important next step in the development of the drug. CNS plans to start the mid-stage trial in 1Q21.
Based on the potential of the company’s asset in glioblastoma, and with its share price at $2.22, several analysts believe that now is the time to buy.
Among the bulls is Brookline’s 5-star analyst Kumaraguru Raja who takes a bullish stance on CNSP shares.
“Until now, the inability of anthracyclines to cross the blood brain barrier prevented its use for treatment of brain cancers. Berubicin is the first anthracycline to cross the blood-brain barrier in adults and access brain tumors… Berubicin has promising clinical data in a Phase 1 trial in recurrent glioblastoma (rGBM) and has Orphan drug designation for treatment of malignant gliomas from the FDA. We model approval of Berubicin for treatment of recurrent glioblastoma in 2025 based on the Phase 2 data with 55% probability of success for approval. We model peak sales of $533 million in 2032,” Raja opined.
“CNS pipeline also includes WP1244 (novel DNA binding agent) that is 500x more potent than daunorubicin in inhibiting tumor cell proliferation is expected to enter the clinic in 2021… In vivo testing in orthotopic models of brain cancer showed high uptake of WP1244 by brain and subsequent antitumor activity,” the analyst added.
To this end, Raja rates CNSP a Buy, and his $10 price target implies room for a stunning 350% upside potential in the next 12 months.
What does the rest of the Street have to say? 3 Buys and 1 Hold add up to a Strong Buy consensus rating. Given the $8.33 average price target, shares could climb ~275% in the year ahead. (See CNSP stock analysis )
<<<
The actual float for LIFE is a little over 11 million shares and that's straight from the CEO's mouth.
Bladerunner
P.S. MEIP is running up on high volume.
Blade, FSTX also has an extremely small float of ~ 1.6 mil shares, though no significant short position (under 1%). But ANVS was like that also, tiny float (4 mil) but only 1% short position, and they ran it up from 5 to 35.
LIFE is another one with a small float (8 mil) and no significant short position (under 1%). This article (below) could explain the recent spike up, and coincides exactly with when the move started (Feb 4th). That suggests the Reddit group isn't involved, unless they are working with Tip Ranks in a coordinated pump, which seems unlikely, but who knows -
>>> 2 “Strong Buy” Penny Stocks With Over 200% Upside on the Horizon
Tip Ranks
February 4, 2021
https://finance.yahoo.com/news/2-strong-buy-penny-stocks-165357551.html
aTyr Pharma (LIFE)
The next stock we’re looking at, aTyr Pharma, has a focus on inflammatory disease. Its leading drug candidate, ATYR1923, is a Neuropilin-2 (NRP2) agonist, working through the receptor proteins expressed by the NRP2 gene. These pathways are important for cardiovascular development and disease, and play a role in the inflammatory lung disease pulmonary sarcoidosis.
In December, the company reported that the drug candidate had completed enrollment of 36 patients in a Phase 1b/2a clinical trial, testing the drug in the treatment of pulmonary sarcoidosis. Results of the current study are expected in 3Q21, and will inform further trials of ATYR1923, including against other forms of inflammatory lung disease.
On a more immediate note, in early January the company announced top-line results of another Phase 2 clinical involving ATRY1923 – this time in the treatment of patients hospitalized with severe respiratory complications from COVID-19. The results were positive, showing that a single dose of ATYR1923 (at 3 mg/kg) resulted in a 5.5-day median recovery time. Overall, of the patients dosed in this manner, 83% saw recovery in less than one week.
Covering LIFE for Roth Capital, 5-star analyst Zegbeh Jallah noted, “We like the risk profile here, with two shots on goal, and updated data details from the COVID study is expected in the coming months. Also announced recently, is that data from aTyr's Pulmonary Sarcoidosis program, will be reported in 3Q21… the success of either of these studies could result in a doubling or more of the market cap as these opportunities appear to barely be accounted for by investors.”
In line with his optimistic approach, Jallah gives LIFE shares a Buy rating and his $15 price target suggests an impressive 277% potential upside for the coming year. (To watch Jallah’s track record, click here)
Other analysts are on the same page. With 2 additional Buy ratings, the word on the Street is that LIFE is a Strong Buy. On top of this, the average price target is $13.33, suggesting robust growth of ~236% from the current price of $3.97. (See LIFE stock analysis on TipRanks)
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I forgot to mention FSTX. That's one to really keep an eye on.
Bladerunner
I've thought about the Reddit issue and "wallstreetbets." There was an article I read that said that the Redditors were moving into small-cap biotech.
If the Redditors figure out that small-cap biotechs with low-floats are a good way to drive up share prices, then shares of many companies could benefit.
I bought 100 shares of LIFE on Friday, because I thought this was one company that could benefit and the share volume had been moving up considerably. (I also spoke to the CEO, Dr. Shukla, for thirty minutes on Friday.) I never expected the kind of move we're seeing today. Of course, I wish I had bought thousands more shares.
I think CRVS and MGTA could benefit from Redditor interest, KZIA as well.
ATOS has already seen a spike in volume and share price. CVM as well. I don't trust the CEO's of either company, but you can still make money on them, if you're nimble enough.
Bladerunner
Blade, I was wondering if you have any ideas concerning possible targets for the Reddit Army? I see they may have pivoted into the bio sector, with ANVS, AVXL, and SAVA, but any ideas on what could be next? Those 3 stocks happened to have CNS programs, but I figure that particular aspect might not be repeated.
It appears they don't go after OTC stocks, preferring instead stocks traded on the Nasdaq GM, GS, and CM, or the NYSE. Usually there is an existing short position, usually over 10%, but several times they have gone after stocks based on their extremely low floats. With ANVS the short position was only 1%, but the float was extremely tiny at 4 mil, so the low float made the big spike up possible even without inducing a short squeeze.
CRVS looks like a possibility, and has the main criteria -- trades on the Nasdaq GM, float only 14 mil, short position 9% of float, and the chart is way down and flat, and the share price only 4. So looks like a possibility. But there must be hundreds of suitable Reddit targets, and the odds of picking one of them is fairly remote.
In a way you have to admire these Reddit guys, they found an efficient way to achieve fast riches using their social media army. Very resourceful..
Thanks Blade, I'll add FSTX and LIFE to the watchlist. I assume you still like KZIA? That research report was excellent, and the former Genentech drug's ability to cross the BBB barrier could mean a major breakthrough not only for various brain cancers, but to treat/prevent brain metastases with other cancers. Definitely some big potential there, and the chart looks promising also.
Wish I had more time to research these bio stocks. Other sectors have been going gangbusters, so biotech has taken a back seat, but it's still extremely interesting to follow.
I've been concentrating on IPIX at the moment, and the drug Brilacidin, which I followed many years ago. A lot of potential there, and the stock is starting to move, doubling in the past week.
Hope you are doing well, and thanks for the stock updates :o)
Hi gfp, happy Superbowl Weekend.
I sold all my CRDF--probably a mistake--but the recent data readout didn't impress me nor the Street. It's come back after I sold it. Great timing, as always for me.
I've made good money recently on FSTX, a stock unknown on The Street. It's woth keeping an eye on.
I finally sold my last tranche of CCXI at $64.50. It's my biggest winner ever and has secured my retirement.
ONCT has been performing nicely.
CRVS is a disappointment so far, but I'm holding.
I'm looking at LIFE. The CEO called me and we had a nice 30-minute chat. Very nice guy. I'm not sure about the target indication.
I think (hope) that CRIS will move up later in the year with EHA and ASH meetings late in the year. They may have something at ASCO.
Bladerunner
Blade, I saw your comments on CRIS, and have added it to my watchlist, which now includes -
CCXI
CRDF
CRIS
CRVS
KZIA
MYOV
ONCT
PRVB
Just curious if this looks up to date with your current favorites? Any additional names you are considering? Thanks.
I have some KZIA as a long term hold, and have been trying to get up to speed on the others, though it takes considerable time/effort for even one bio stock. A compound I followed years ago called Brilacidin (see recent post) has been 'reborn' and is entering a Covid Phase 2, so I've been concentrating on that, in addition to following numerous other sectors, so my time is being spread a little thin. Thanks for your stock ideas, they are appreciated :o)
GameStop Unraveling Quickens as Redditors Pivot to Biotech
Bailey Lipschultz and Cristin Flanagan
Thu, February 4, 2021, 11:04 AM·3 min read
GME
-35.20%
(Bloomberg) -- GameStop Corp. shares tumbled for a third day in four as retail traders flocked to other corners of the stock market such as small drug developers.
The video-game retailer extended losses to 36% and was trading a couple dollars above this week’s low of $57.02 at 2 p.m. in New York. Trading volume picked up after a slow start, with 47 million shares exchanged, which was about one-third lower than the volume seen over the past two weeks.
Meanwhile, demand shrunk for companies such as headphone maker Koss Corp. and apparel retailer Express Inc., which had also skyrocketed over recent weeks.
The volatility in GameStop’s shares have resulted in market value swings of over $30 billion for the company this year and Wednesday’s session showed selling pressures were ramping. But the euphoria surrounding the stock, which was born in day-trader chat rooms, seems to be turning now to groups of smaller names, such as drug developers.
Retail traders using Reddit to communicate about their bullish bets and outlooks have helped fuel rallies in Anavex Life Sciences Corp., Cassava Sciences Inc., and Annovis Bio Inc.
The gains show that Reddit is continuing to “play a major role in trading action” for single stocks, wrote Jared Holz, a Jefferies health-care equity strategist.
A trio of biotechs chasing after Alzheimer’s cures added more than $1 billion into their market value on Thursday after Cassava reported results from a small study in 100 patients earlier this week.
Reddit and Twitter users extolled upon the virtues of biotechs chasing after new medicines for Alzheimer’s as well as Covid-19 and cancer, though new drugs in development face long odds on making it to the market.
Read more: Three Biotech Stocks Surge on Alzheimer’s Drug Optimism
Other companies favored by retail investors, including Naked Brand Group Limited and Sundial Growers Inc. erased gains. The retailer and cannabis firm Sundial reversed rallies to drop about 2% each.
Short interest as a percentage of GameStop’s free float slipped again to 51% from a peak last month above 140%, according to data from S3 Partners. That plunge in short interest could signal the squeeze has played out, according Amy Kong, chief investment officer of Barrett Asset Management.
“It is also important to consider market psychology,” she said. “If one sees the stock plunge the way it did over the past two days, it may prompt investors to sell ‘before it’s too late.”’
GameStop shares are down 82% this week and have lost momentum even after news of a trio of executive hires, including the appointment of a chief technology officer.
While the executive additions are “all great hires on paper” it’s hard to attribute recent share movement to anything in particular, Wedbush Securities analyst Michael Pachter said in an email. The analyst, who rates the shares at neutral, said he looks forward to hearing details about the company’s strategy.
GameStop grabbed headlines this month after a coordinated effort by retail investors to squeeze short sellers caused the stock to surge multiple times. It has lost more than 85% of those gains in recent days, but despite that has still added about $2.8 billion in value this year.
Bladerunner
Blade - Here's an interesting bio - Innovation Pharma (IPIX) -
Their drug Brilacidin was originally developed at the Univ of Penn as a broad spectrum antibiotic with a totally unique mechanism. The approach is called HDP-mimetics, and it mimics the oldest biological defense system - host defense proteins. This has phenomenal potential against both bacteria and viruses.
Years ago their Phase 2 against MRSA was amazingly successful, but mismanagement of the company led it to being taken over by IPIX (then called Cellceutix), who had other clinical priorities. But with the appearance of Covid, the drug (Brilacidin) underwent quasi government testing (U.S. Regional Biocontainment Laboratory) and had phenomenal in-vitro activity against Covid. Brilacidn subsequently received Fast Track Designation from the FDA, and last week the company announced the Phase 2 (120 patients) would start this week. The stock has started to take off, but market cap still only $140 mil.
Anyway, this is definitely one to watch (I have 50,000 shares, avg cost of .23 cents). I followed Brilacidin in the early days (~2012), and kept it on my radar, figuring the vast potential might some day be realized.
One big plus with the defensin mimetic approach is the extreme unlikelihood of resistance ever developing to the drug. The mechanism works directly at the cell wall, instead of via inhibiting the internal cellular machinery or DNA. Pores/holes form on the cell wall which kills the virus or bacteria, which is the same mechanism used by the body's own innate defense protein system. It should also work on the various mutated forms of Covid, and potentially be a broad spectrum 'pan-coronavirus' therapeutic. Check it out -
https://en.wikipedia.org/wiki/Brilacidin#The_HDP-mimetic_pipeline
http://www.ipharminc.com/
CRVS initiates a Phase III with drug used to treat COVID-19
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GlobeNewswire
Corvus Pharmaceuticals Initiates Phase 3 Clinical Trial of CPI-006 for Patients with COVID-19
Corvus Pharmaceuticals, Inc.
Thu, February 4, 2021, 4:30 AM
Phase 1 study results, along with pre-clinical research, shows that CPI-006 activates antigen specific B cells, triggering the body’s inherent immune response to generate a robust and durable humoral response to the SARS-CoV-2 virus. This includes stimulating the generation of high titers of durable, polyclonal IgG and IgM antibody responses to SARS-CoV-2 and increased levels of memory B cells. This novel mechanism of action has several advantages over passively administered monoclonal antibodies and could potentially be used to treat other infectious diseases.
Phase 1 study results, along with pre-clinical research, shows that CPI-006 activates antigen specific B cells, triggering the body’s inherent immune response to generate a robust and durable humoral response to the SARS-CoV-2 virus. This includes stimulating the generation of high titers of durable, polyclonal IgG and IgM antibody responses to SARS-CoV-2 and increased levels of memory B cells. This novel mechanism of action has several advantages over passively administered monoclonal antibodies and could potentially be used to treat other infectious diseases.
Phase 1 study results, along with pre-clinical research, shows that CPI-006 activates antigen specific B cells, triggering the body’s inherent immune response to generate a robust and durable humoral response to the SARS-CoV-2 virus. This includes stimulating the generation of high titers of durable, polyclonal IgG and IgM antibody responses to SARS-CoV-2 and increased levels of memory B cells. This novel mechanism of action has several advantages over passively administered monoclonal antibodies and could potentially be used to treat other infectious diseases.
Novel immunotherapy approach designed to stimulate immune response by activating B cells to enhance polyclonal antibody production and potentially reduce hospitalization and progression to respiratory failure
Randomized, double-blind, placebo-controlled study expected to enroll ~1,000 patients across global sites with results expected in Q4 2021
Company to host conference call and webcast today at 8:30 a.m. ET / 5:30 a.m. PT
BURLINGAME, Calif., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, today announced that it has initiated a Phase 3 clinical trial of CPI-006 for the treatment of hospitalized patients with COVID-19. The study is expected to enroll approximately 1,000 patients at sites in North America, Europe, South Africa and Latin America.
“The COVID-19 pandemic has been devastating to patients and the healthcare system, and it is critical that we develop new therapies that can improve patient outcomes,” said Gerard J. Criner, MD, FACP, FACCP, Chair and Professor of Thoracic Medicine and Surgery at the Lewis Katz School of Medicine at Temple University and Director of the Temple Lung Center and one of the lead investigators of the trial. “CPI-006 has already shown promising results in COVID-19 patients and its proposed mechanism of stimulating the immune system to specifically target the SARS-CoV-2 virus may have several potential advantages over existing therapies, including the potential to address recently emerging variants of the virus. We look forward to offering this therapy to our patients as part of the Phase 3 study.”
CPI-006 is a humanized monoclonal antibody that is designed to bind to and activate B cells that Corvus believes has the potential to provide a unique immunotherapy approach for the treatment of infectious diseases, including COVID-19. In a Phase 1 study involving 28 hospitalized, high-risk patients with moderate COVID-19 treated with CPI-006, no patients progressed to requiring mechanical ventilation and the median time to discharge from the hospital was 3.5 days. This compares favorably to published reports showing that, on average, approximately 20% of similarly affected patients will progress to requiring invasive mechanical ventilation. Patients in the study generated high titers of polyclonal antibodies against a diverse range of targets on the SARS-CoV-2 virus that were sustained over several months. They also had increased levels of circulating memory B cells, which could lead to long-term immunity.
Serum from 2 of 2 patients treated with CPI-006 early in the pandemic (July 2020), and before variants were discovered, were tested in neutralization assays against both the UK variant and wild type receptor binding domain of SARS-CoV-2 (N501Y mutation). Day 28 post CPI-006 treatment serum showed increases in neutralization titers of 4.5 fold and 1.7 fold to the UK variant for the two patients respectively, compared to the wild type virus. These data will require further confirmation with many more additional patients, but suggest that CPI-006 elicited a broad anti-viral response that may address the problem of immune escape.
The Phase 3 double-blind study, which was designed with guidance from the Food and Drug Administration (FDA), will evaluate the efficacy and safety of CPI-006 compared to placebo in hospitalized patients with mild-to-moderate COVID-19. Patients will be randomized in a 1:1:1 ratio to receive a single intravenous CPI-006 dose of either 2.0 mg/kg or 1.0 mg/kg or placebo; all patients will receive standard of care treatments for COVID-19. The primary endpoint is the proportion of patients progressing to respiratory failure or death during the 28 days after dosing. Respiratory failure is defined as requiring non-invasive or invasive mechanical ventilation. Additional secondary endpoints include time to recovery, time to resolution of COVID-19 symptoms, anti-viral antibody responses, etc. An interim futility and efficacy analysis will be conducted by an independent data monitoring committee when approximately 60% of subjects complete the 28-day post-treatment visit. Results from the study are expected to be available in the fourth quarter 2021.
“The devastating nature of the pandemic and emerging understanding of this disease compels us to rapidly develop CPI-006 for the treatment of COVID-19,” said Richard A. Miller, M.D., president and chief executive officer of Corvus. “We believe CPI-006’s novel mechanism of action, supported by Phase 1 results obtained to date, indicate that this agent may be well suited to combat this virus, which is now demonstrating a propensity to mutate, become more transmissible and potentially escape from current vaccination and therapeutic approaches such as the administration of passive anti-viral monoclonal antibodies. The advancement of this study is the top priority for the Company given the growth in new COVID-19 cases and could provide the foundation for its potential use in other infectious diseases. If successful and ultimately approved for treatment, we believe CPI-006 could be an important therapy if it is able to shorten recovery times and keep COVID-19 patients out of the intensive care unit and off of mechanical ventilators, while also providing durable, longer-term immunity and potential protection against re-infection.”
Corvus also announced that in collaboration with its Chinese partner, Angel Pharmaceuticals, it plans to initiate a global Phase 2 trial with CPI-818, an ITK inhibitor, in refractory peripheral T cell lymphoma by the end of 2021. The Company also announced its plans to collaborate with an academic consortium to evaluate ciforadenant, its A2A receptor antagonist, for the first line treatment of metastatic renal cell carcinoma in combination with pembrolizumab and a tyrosine kinase inhibitor.
Conference Call, Webcast and Slide Presentation Details
Corvus will host a conference call and webcast today, February 4, 2021, at 8:30 a.m. ET (5:30 a.m. PT), to discuss the Phase 3 CPI-006 clinical trial for COVID-19 and other topics. The conference call can be accessed by dialing 1-877-407-0784 (toll-free domestic) or 1-201-689-8560 (international) and using the conference ID 13715838. The live webcast, which will include presentation slides, may be accessed via the investor relations section of the Corvus website. A replay of the webcast will be available on Corvus' website for 90 days.
About Corvus Pharmaceuticals
Corvus Pharmaceuticals is a clinical-stage biopharmaceutical company. Corvus’ lead product candidates are
CPI-006, a humanized monoclonal antibody directed against CD73 that has exhibited immunomodulatory activity and activation of immune cells in preclinical studies, and ciforadenant (CPI-444), a small molecule inhibitor of the A2A receptor. CPI-006 is being evaluated in a Phase 3 clinical trial for the treatment of hospitalized patients with COVID-19 and in a multicenter Phase 1/1b oncology clinical trial as a single agent, in combination with ciforadenant and pembrolizumab. The Company’s third cancer clinical program, CPI-818, is an investigational, oral, small molecule drug that selectively inhibited ITK in preclinical studies, is in a multicenter Phase 1/1b clinical trial in patients with several types of T-cell lymphomas. For more information, visit www.corvuspharma.com.
About CPI-006
CPI-006 is an investigational, potent humanized monoclonal antibody that is designed to react with a specific site on CD73. In preclinical studies, it has demonstrated immunomodulatory activity resulting in activation of lymphocytes, induction of antibody production from B cells and effects on lymphocyte trafficking. While there are other anti-CD73 antibodies and small molecules in development for treatment of cancer, such agents react with a different region of CD73 and are designed to block production of adenosine, which is not involved in the immunomodulatory processes seen with CPI-006.
About CPI-818
CPI-818 is an investigational small molecule drug given orally that has selectively inhibited ITK (interleukin-2-inducible T-cell kinase) in preclinical studies. It was designed to possess dual properties: to block malignant T-cell growth and to modulate immune responses. ITK, an enzyme, is expressed predominantly in T-cells and plays a role in T-cell and natural killer (NK) cell lymphomas and leukemias, as well as in normal immune function. Interference with ITK signaling can modulate immune responses to various antigens. The Company believes the inhibition of specific molecular targets in T-cells may be of therapeutic benefit for patients with T-cell lymphomas and in patients with autoimmune diseases. The Company is conducting a Phase 1/1b trial in patients with refractory T-cell lymphomas.
About Ciforadenant
Ciforadenant (CPI-444) is an investigational small molecule, oral, checkpoint inhibitor designed to disable a tumor’s ability to subvert attack by the immune system by blocking the binding of adenosine in the tumor microenvironment to the A2A receptor. Adenosine, a metabolite of ATP (adenosine tri-phosphate), is produced within the tumor microenvironment where it may bind to the adenosine A2A receptor present on immune cells and block their activity. CD39 and CD73 are enzymes on the surface of tumor cells and immune cells. These enzymes work in concert to convert ATP to adenosine.
About Angel Pharmaceuticals
Angel Pharmaceuticals is a privately held biopharmaceutical company developing a pipeline of precisely targeted investigational medicines for cancer, autoimmune, infectious and other serious diseases in China. Angel Pharmaceuticals was launched through a collaboration with U.S.-based Corvus Pharmaceuticals and investments from investors in China. Angel Pharmaceuticals licensed the rights to develop and commercialize Corvus’ three clinical-stage candidates – ciforadenant, CPI-006 and CPI-818 – in greater China and obtained global rights to Corvus’ BTK inhibitor preclinical programs. Under the collaboration, Corvus initially retained a 49.7% equity stake in Angel Pharmaceuticals and designated three individuals on Angel’s five-person Board of Directors.
One of the main strategic rationales for Corvus’ collaboration with Angel is to gain clinical study synergies and accelerate development timelines, whereby data from patients enrolled in China studies could potentially be used as part of U.S. regulatory submissions as part of a global pivotal study protocol. Based on the tumor responses observed in the CPI-818 Phase 1/1b clinical trial, the companies are planning to collaborate on the next clinical study for CPI-818 for the treatment of T cell lymphomas.
Bladerunner
MEIP gives a clinical pipeline update
January 4, 2021 7:00 AM
MEI PHARMA ANNOUNCES EXPANSION OF PHASE 1B STUDY EVALUATING ZANDELISIB AND CLINICAL PIPELINE UPDATE
- Safety Review Committee approved Phase 1b expansion cohorts in study arm evaluating zandelisib in combination with zanubrutinib in various B-cell maligniancies -
- MEI PROVIDES CLINICAL PIPELINE OUTLOOK FOR 2021 -
- MEI PHARMA TO PRESENT AT THE H.C. WAINWRIGHT BIOCONNECT 2021 VIRTUAL CONFERENCE -
PR Newswire
SAN DIEGO , Jan. 4, 2021 /PRNewswire/ -- MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, today announced that the Phase 1b trial arm exploring zandelisib, an investigational selective phosphatidylinositol 3-kinase ("PI3K") delta inhibitor, in combination with zanubrutinib (marketed as BRUKINSA®), an inhibitor of Bruton's tyrosine kinase ("BTK") developed by BeiGene, Ltd. ("BeiGene"), completed the safety evaluation stage in patients with B-cell malignancies and is expanding into disease specific B-cell malignancy cohorts. The Safety Review Committee recommended moving forward with a dosing regimen found to be generally well tolerated and active following a planned safety analysis.
"Inhibition of the PI3K and BTK pathways with combination therapy is believed to present a promising opportunity for inducing a robust and durable response against various B-cell malignancies," said Richard Ghalie, M.D., senior vice president, clinical development, of MEI Pharma. "The intermittent dosing schedule and unique therapeutic profile of zandelisib makes it an attractive option for combination therapy. We are encouraged that the data to date supported the recommendation of the Safety Review Committee to move forward with the expansion cohorts, initially in follicular and mantle cell lymphomas, and we look forward to further exploring the potential of this and other combinations of zandelisib in patients with various B-cell malignancies with our global development partner, Kyowa Kirin."
A Series of Important Pipeline Updates in 2021
MEI Pharma also provided an update on pipeline programs and anticipated milestones for 2021 for zandelisib, voruciclib and ME-344.
Zandelisib – PI3K delta inhibitor for the treatment of various B-cell malignancies
Completion of enrollment in the Phase 2 TIDAL study evaluating zandelisib as a monotherapy for patients with relapsed or refractory follicular lymphoma anticipated around the end of first quarter of 2021, subject to the continuing impact of the COVID-19 pandemic
Initiation of a second arm in the Phase 2 TIDAL study enrolling patients with relapsed or refractory marginal zone lymphoma anticipated in the first quarter of 2021
Initiation of the Phase 3 study of zandelisib in combination with rituximab evaluating follicular and marginal zone lymphoma patients who received one or more prior lines of treatment anticipated in mid-2021; this study is intended to act as the required confirmatory study for the potential accelerated approval of zandelisib in patients with relapsed or refractory follicular lymphoma
Clinical updates for the Phase 1b study of zandelisib, including the combination with zanubrutinib, anticipated in mid-2021
Top line clinical data from the Phase 2 TIDAL study anticipated in the fourth quarter of 2021
Voruciclib – CDK9 inhibitor for the treatment of B-cell malignancies and acute myeloid leukemia
Program updates, including data from the Phase 1 program evaluating voruciclib in combination with venetoclax, anticipated in 2021
ME-344 – tumor selective mitochondrial inhibitor
Update plans for a potential Phase 2 pilot study of ME-344 in solid tumors anticipated by year-end 2021
MEI Pharma also confirmed today that it has a cash runway to fund operations through calendar year 2023.
H.C. Wainwright BIOCONNECT 2021 Virtual Conference Presentation
MEI Pharma will present at the H.C. Wainwright BIOCONNECT 2021 Virtual Conference. The presentation will provide a company overview and business update and will be available for on-demand listening beginning Monday January 11, 2020 at 6:00 a.m. Eastern Time . The presentation can be accessed on the Events & Presentations page of the Investors section of MEI Pharma's website at http://www.meipharma.com .
About Zandelisib
Zandelisib (formerly called ME-401) is an investigational oral phosphatidylinositol 3-kinase ("PI3K") delta inhibitor in clinical development for the treatment of patients with B-cell malignancies. In March 2020 the U.S. FDA granted zandelisib Fast Track designation.
PI3K delta is often overexpressed in cancer cells and plays a key role in the proliferation and survival of hematologic cancers. Zandelisib displays high selectivity for the PI3K delta isoform and has distinct pharmaceutical properties from other PI3K delta inhibitors.
Ongoing clinical studies of zandelisib include TIDAL ( T rials of P I 3K D elt A in Non-Hodgkin's L ymphoma), a global Phase 2 clinical trial evaluating the candidate as a monotherapy for the treatment of adults with relapsed or refractory follicular lymphoma after failure of at least two prior systemic therapies including chemotherapy and an anti-CD20 antibody. Subject to the results, upon completion of the Phase 2 clinical trial, MEI is planning a submission with the FDA to support an accelerated approval of a marketing application under 21 CFR Part 314.500, Subpart H.
Also ongoing is the multicenter, open-label, single-arm pivotal Phase 2 study of zandelisib in patients with indolent B-cell non-Hodgkin's lymphoma (iNHL) without Small lymphocytic lymphoma, lymphoplasmacytic lymphoma (LPL), and Waldenström's macroglobulinemia (WM) in Japan to evaluate zandelisib as a monotherapy for the treatment of Japanese patients with relapsed or refractory iNHL with at least two prior systemic therapies.
In October 2018 , MEI entered a clinical collaboration with BeiGene to evaluate the safety and efficacy of zandelisib in combination with BeiGene's zanubrutinib for the treatment of patients with various relapsed or refractory B-cell malignancies. Clinical study costs in the Phase 1b study evaluating the combination are equally shared, and each company is supplying its own investigational agent. MEI retains all commercial rights to zandelisib and BeiGene retains all commercial rights to zanubrutinib.
In April 2020, MEI and Kyowa Kirin Co., Ltd. (Kyowa Kirin) entered a global license, development, and commercialization agreement to further develop and commercialize zandelisib. MEI and Kyowa Kirin are co-developing and co-promoting zandelisib in the U.S., with MEI booking all revenue from the U.S. sales. Kyowa Kirin has exclusive commercialization rights outside of the U.S.
In May 2020 , updated data from the Phase 1b study of zandelisib was presented at the American Society of Clinical Oncology (ASCO) 2020 Virtual Scientific Program. These data evaluating patients on an intermittent dosing schedule showed that treatment was generally well tolerated and demonstrated an 83% overall response rate in patients with relapsed or refractory follicular lymphoma. Responses appeared durable: median duration of response was not yet reached after median follow-up of 13.2 months (range: 3.0-27.6).
About MEI Pharma
MEI Pharma, Inc. (Nasdaq: MEIP) is a late-stage pharmaceutical company focused on developing potential new therapies for cancer. MEI Pharma's portfolio of drug candidates contains four clinical-stage assets, including zandelisib, currently in an ongoing Phase 2 clinical trial which may support an accelerated approval marketing application with the U.S. Food and Drug Administration. Each of MEI Pharma's pipeline candidates leverages a different mechanism of action with the objective of developing therapeutic options that are: (1) differentiated, (2) address unmet medical needs and (3) deliver improved benefit to patients either as standalone treatments or in combination with other therapeutic options. For more information, please visit www.meipharma.com and follow us on Twitter and LinkedIn .
Bladerunner
PRVB files BLA for diabetes drug. Stock up 5% in pre-Market
January 4, 2021 7:00 AM
PROVENTION BIO ANNOUNCES U.S. FDA FILING OF A BIOLOGICS LICENSE APPLICATION (BLA) AND PRIORITY REVIEW FOR TEPLIZUMAB FOR THE DELAY OR PREVENTION OF CLINICAL TYPE 1 DIABETES IN AT-RISK INDIVIDUALS
-If approved, teplizumab will be the first disease-modifying therapy for type 1 diabetes-
-FDA sets PDUFA goal date of July 2, 2021-
-IF APPROVED, TEPLIZUMAB WILL BE THE FIRST DISEASE-MODIFYING THERAPY FOR TYPE 1 DIABETES-
PR Newswire
RED BANK, N.J. , Jan. 4, 2021 /PRNewswire/ -- Provention Bio, Inc. (Nasdaq: PRVB), a biopharmaceutical company dedicated to intercepting and preventing immune-mediated disease, today announced that the Biologics License Application (BLA) for teplizumab for the delay or prevention of clinical type 1 diabetes (T1D) in at-risk individuals has been filed by the U.S. Food and Drug Administration (FDA). The FDA also granted Provention's request for Priority Review and assigned a user fee goal date of July 2, 2021 , under the Prescription Drug User-Fee Act ( PDUFA ).
(PRNewsfoto/Provention Bio, Inc.)
"The FDA's acceptance of our BLA represents a significant achievement for Provention Bio in our mission to deliver the first potential disease-modifying T1D therapy and drive a paradigm shift in how individuals at risk of developing the disease are treated," stated Ashleigh Palmer , CEO and Co-Founder, Provention Bio. "We intend to work closely with the FDA to support their review while also preparing for a potential product launch in the third quarter of 2021."
In its acceptance letter, the FDA stated that it is currently planning to hold an advisory committee meeting, tentatively scheduled for May 27, 2021 .
Priority Review is afforded to drugs that, if approved, would represent a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious condition. Under the PDUFA, a Priority Review targets a review time of six months compared to a standard review time of ten months. The FDA previously granted teplizumab Breakthrough Therapy Designation.
About Teplizumab (PRV-031):
Teplizumab is an investigational anti-CD3 monoclonal antibody (mAb) with a filed BLA under Priority Review by the FDA for the delay or prevention of clinical T1D in at-risk individuals. More than 800 patients have received teplizumab in multiple clinical studies involving more than 1,000 subjects. In previous studies of newly diagnosed patients, teplizumab consistently demonstrated the ability to preserve beta-cell function, a measure of endogenous insulin production, and correspondingly reduced the need for exogenous insulin use. Teplizumab has been granted Breakthrough Therapy Designation by the FDA and PRIME designation by the European Medicines Administration. Provention Bio has submitted a Biologic License Application (BLA) to the FDA for teplizumab for the delay or prevention of clinical type 1 diabetes in at-risk individuals. Provention is currently also evaluating teplizumab in patients with newly diagnosed insulin-dependent T1D (the Phase 3 PROTECT study).
About Provention Bio, Inc.:
Provention Bio, Inc. (Nasdaq: PRVB) is a biopharmaceutical company focused on advancing the development of investigational therapies that may intercept and prevent debilitating and life-threatening immune-mediated disease. The Company has submitted a Biologics License Application (BLA) to the FDA for its lead investigational drug candidate, teplizumab, for the delay or prevention of clinical type 1 diabetes in at-risk individuals. The Company's pipeline includes additional clinical-stage product candidates that have demonstrated in pre-clinical or clinical studies proof-of-mechanism and/or proof-of-concept in other autoimmune diseases, including celiac disease and lupus. Visit www.ProventionBio.com for more information and follow us on Twitter: @ProventionBio.
Internet Posting of Information:
Provention Bio, Inc. uses its website, www.proventionbio.com , as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included on the Company's website in the "News" section. Accordingly, investors should monitor this portion of the Company's website, in addition to following its press releases, SEC filings and public conference calls and webcasts.
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Nice - >>> Myovant Up on $4.2B Pfizer Pact to Develop Prostate-Cancer Drug
Myovant and Pfizer will develop Orgovyx for advanced prostate cancer and, if approved, a combination tablet that includes the drug in women’s health.
The Street
ROB LENIHAN
DEC 28, 2020
https://www.thestreet.com/investing/myovant-up-on-4-2-billion-on-pfizer-prostate-cancer-plan?puc=yahoo&cm_ven=YAHOO
Myovant Sciences (MYOV) was higher Monday after the biopharma and Pfizer (PFE) said they would collaborate on a prostate-cancer treatment, an agreement valued at as much as $4.2 billion.
Shares of the Switzerland-based company at last check were climbing 19% at $27.10. Pfizer, the New York health-care giant, was up 0.4% at $37.41.
Under the agreement, the companies will develop and commercialize Myovant’s Orgovyx in advanced prostate cancer and, if approved, a combination tablet that includes relugolix, the active ingredient in Orgovyx, in women’s health in the U.S. and Canada.
The U.S. Food and Drug Administration has approved Orgovyx, generically relugolix, to treat adults with advanced prostate cancer. The FDA is reviewing the combination tablet for women with uterine fibroids, with the agency targeted to take action on the matter June 1.
The combination tablet is also under development for women with endometriosis. A new-drug application is expected to be submitted in the first half.
The two companies said they would be copromoting the drug early next year. They'll equally share profit and certain expenses for Orgovyx and the combination tablet with Myovant recording revenue.
Myovant will remain responsible for regulatory interactions and drug supply and continue to lead clinical development for the combination tablet.
Myovant will receive up to $4.2 billion, including an up-front payment of $650 million and $200 million in potential regulatory milestones for FDA clearances for the combination tablet in women’s health.
The company will receive tiered sales milestones upon reaching certain thresholds up to $2.5 billion in net sales for prostate cancer and also for the combined women’s health indications.
Pfizer will receive an exclusive option to commercialize the drug in oncology outside the U.S. and Canada, excluding certain Asian countries.
Pfizer has a strong track record across oncology and women’s health, Myovant Sciences Chief Executive Lynn Seely said in a statement.
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Myovant Sciences and Pfizer Announce Collaboration to Develop and Commercialize Relugolix in Oncology and Women’s Health
By GlobeNewswire, December 28, 2020, 06:30:00 AM EDT
• Myovant and Pfizer to jointly develop and commercialize ORGOVYX™ (relugolix) and relugolix combination tablet and share profits and expenses in the U.S. and Canada
• Myovant to receive an upfront payment of $650 million in addition to potential regulatory and sales milestones for a total payment of up to $4.2 billion
• Myovant to host conference call and webcast today at 8:30 a.m. Eastern Time / 5:30 a.m. Pacific Time
BASEL, Switzerland and NEW YORK, Dec. 28, 2020 (GLOBE NEWSWIRE) -- Myovant Sciences (NYSE:MYOV) and Pfizer Inc. (NYSE:PFE) today announced a collaboration to develop and commercialize relugolix - a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist - in oncology and women's health in the U.S. and Canada. Pfizer will also receive an exclusive option to commercialize relugolix in oncology outside the U.S. and Canada, excluding certain Asian countries.
"We are thrilled to partner with Pfizer to unlock the full potential of ORGOVYX in advanced prostate cancer and relugolix combination tablet in uterine fibroids and endometriosis, advancing our mission to redefine care for women and for men," said Lynn Seely, M.D., Chief Executive Officer, Myovant Sciences, Inc. "Pfizer is the ideal partner for Myovant given its impressive capabilities and track record across both oncology and women's health. This transformative collaboration will significantly strengthen the upcoming launch of ORGOVYX and the potential launches of relugolix combination tablet in women's health, while substantially enhancing our financial position and enabling us to expand our pipeline of potential new medicines."
Under the terms of the agreement, Myovant and Pfizer will jointly develop and commercialize ORGOVYX™ (relugolix) in advanced prostate cancer and, if approved, relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) in women's health in the U.S. and Canada. Myovant and Pfizer will begin co-promoting ORGOVYX for advanced prostate cancer in early 2021. Myovant and Pfizer will equally share profits and certain expenses for ORGOVYX and relugolix combination tablet with Myovant recording revenues.
Myovant will remain responsible for regulatory interactions and drug supply and continue to lead clinical development for relugolix combination tablet. Myovant will receive up to $4.2 billion, including an upfront payment of $650 million, $200 million in potential regulatory milestones for U.S. Food and Drug Administration (FDA) approvals for relugolix combination tablet in women's health, and tiered sales milestones upon reaching certain thresholds up to $2.5 billion in net sales for prostate cancer and also for the combined women's health indications. If Pfizer exercises the option to commercialize relugolix in oncology outside of the U.S. and Canada, excluding certain Asian countries, Myovant will receive $50 million and be entitled to receive double-digit royalties on sales.
"We are excited to join forces with Myovant and combine our capabilities to bring ORGOVYX to patients with advanced prostate cancer," said Andy Schmeltz, Global President, Pfizer Oncology. "This strategic collaboration builds on our leadership in serving prostate cancer patients in the U.S. and aligns with our goal to deliver more breakthroughs across the prostate cancer treatment paradigm."
"There continues to be a high unmet need among the millions of women who experience the common and debilitating symptoms associated with uterine fibroids and endometriosis," said Nick Lagunowich, Global President, Pfizer Internal Medicine. "We believe our deep heritage and leadership in women's health combined with our experienced women's health field force will enable us to maximize these opportunities with Myovant, potentially bringing valuable new treatment options to these women."
The FDA approved ORGOVYX on December 18, 2020 for the treatment of adult patients with advanced prostate cancer. ORGOVYX is the first and only oral GnRH antagonist for men with advanced prostate cancer. Relugolix combination tablet is currently under regulatory review by the FDA for women with uterine fibroids, with a target action date of June 1, 2021. Relugolix combination tablet is also under development for women with endometriosis, with a New Drug Application submission anticipated in the first half of 2021.
Myovant Sciences Conference Call
Myovant will hold a webcast and conference call at 8:30 a.m. Eastern Time / 5:30 a.m. Pacific Time today, December 28, 2020. Investors and the general public may access a live webcast of the call by visiting the investor relations page of Myovant's website at investors.myovant.com. Institutional investors and analysts may also participate in the conference call by dialing 1-800-532-3746 in the U.S. or +1-470-495-9166 from outside the U.S.
The webcast will be archived on Myovant's Investor Relations website following the call.
About Relugolix
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist that reduces testicular testosterone, a hormone known to stimulate the growth of prostate cancer, and ovarian estradiol, a hormone known to stimulate the growth of uterine fibroids and endometriosis. Relugolix (120 mg) is FDA-approved as ORGOVYX™ for adult patients with advanced prostate cancer. Relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) is under regulatory review in Europe and the U.S. for women with uterine fibroids and is under development for women with endometriosis.
About ORGOVYX™ (relugolix)
ORGOVYX (relugolix) is the first and only oral gonadotropin-releasing hormone (GnRH) receptor antagonist approved by the FDA for the treatment of adult patients with advanced prostate cancer. As a GnRH antagonist, ORGOVYX blocks the GnRH receptor and reduces production of testicular testosterone, a hormone known to stimulate the growth of prostate cancer.
For full prescribing information, including patient information, please click here.
Indication
ORGOVYX is approved for the treatment of adult patients with advanced prostate cancer.
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>>> AstraZeneca to Buy Alexion for $39 Billion in Rare-Disease Push
Bloomberg
by John Lauerman and James Paton
12-12-20
https://www.msn.com/en-us/money/companies/astrazeneca-to-buy-alexion-for-2439-billion-in-rare-disease-push/ar-BB1bRU5O?ocid=uxbndlbing
AstraZeneca Plc, one of the drugmakers leading the fight against Covid-19, highlighted where its growth will come from after the pandemic with a $39 billion deal for rare-disease specialist Alexion Pharmaceuticals Inc.
The proposed cash-and-stock acquisition will add treatments for uncommon blood and immunological disorders to the portfolio of Cambridge, U.K.-based AstraZeneca, which had spent years paring away older and less profitable products to focus on cancer.
While the pandemic smothered economies and AstraZeneca entered a relatively low-return project to develop a Covid-19 vaccine with the University of Oxford, Chief Executive Officer Pascal Soriot kept his eyes downfield. After months of patients avoiding hospitals and clinics for fear of viral exposure, immunizations are coming online that promise to return society, and the drug industry, to some semblance of normal.
The purchase is “an important step in the history of the company,” Soriot said on a call with reporters. “It’s a tremendous opportunity for us to accelerate our development of immunological” therapies.
High-priced medicines for rare diseases can generate billions in sales from relatively few patients. Snapping up drugmakers that focus on them has been a popular way for larger pharmaceutical companies to stimulate sales growth in recent years.
The offer values Alexion at $175 a share, a 45% premium to the closing price on Friday. It’s the largest deal for AstraZeneca since it was founded in a 1999 combination of British and Swedish companies, and would entrench its position among the world’s 10 biggest drugmakers. It’s also the biggest pharmaceutical and biotechnology takeover this year, as well as the fourth-largest transaction globally across all sectors, according to data compiled by Bloomberg.
Covid Vaccine
The news gives Soriot a chance to focus on something other than the company’s experimental coronavirus vaccine. AstraZeneca has been wrestling with questions surrounding the efficacy of the potential shot and the way the late-stage trials were handled.
Before the pandemic, AstraZeneca was one of the hottest pharmaceutical companies, with some 70% growth in value over the past three years as it churned out cancer drugs like Lynparza, Imfinzi and Tagrisso, its biggest seller. It’s divested rights to older brands like Seroquel for schizophrenia while halting costly development programs that looked unlikely to pan out.
But a takeover attempt from Pfizer Inc. that Soriot fended off six years ago taught the importance of scale. AstraZeneca made a preliminary approach to Gilead Sciences Inc., Bloomberg reported in June.
While those talks went no further, adding Alexion could put the U.K. drugmaker out of an acquirer’s reach. Relatively cash-poor for the past few years, AstraZeneca will also see benefits from gaining Alexion’s lucrative business.
‘Bigger Base’
That will help AstraZeneca pay its dividend and “gives them a much bigger base from which to invest in R&D,” said Sam Fazeli, a Bloomberg Intelligence analyst. Chief Financial Officer Marc Dunoyer said this would be the last of AstraZeneca’s big deals for a while.
The new products will also allow Soriot to indulge more in one of his priorities: the China market, which now accounts for roughly a fifth of the company’s revenue. Alexion doesn’t have a footprint in the country, making it the most important market for expanding that company’s reach, he said.
Alexion has specialized in developing drugs that selectively inhibit immune factors to fight diseases that involve the body’s protective system. Soliris, the company’s biggest product with almost $4 billion in 2019 sales, is a monoclonal antibody used to treat rare conditions such as paroxysmal nocturnal hemoglobinuria.
Monoclonal antibodies have garnered attention because two such drugs made by Eli Lilly & Co. and Regeneron Pharmaceuticals Inc. have been given emergency authorization in the U.S. for the treatment of Covid. Alexion announced plans in April to conduct late-stage tests of another monoclonal antibody, Ultomiris, in severely ill patients with the disease.
Alexion has been pressured in the past to put itself on the block. Activist investor Elliott Management Corp. opposed the company’s deal earlier this year to acquire Portola Pharmaceuticals, saying the transaction didn’t make strategic sense and didn’t fit with Alexion’s focus on rare diseases.
Deal Financing
In the sale to AstraZeneca, holders of each Alexion share will receive $60 in cash and 2.1243 AstraZeneca American depositary shares, the U.K. company said in a press release Saturday. The drugmaker will fund the acquisition with a $17.5 billion financing facility from Morgan Stanley, JPMorgan Chase & Co. and Goldman Sachs Group Inc.
Despite the premium, the price is attractive for AstraZeneca, and its agreement with Alexion could draw out other offers, SVB Leerink analyst Geoffrey Porges said in a note. Alexion shareholders could hold out for $200 per share or seek a higher cash portion, he said.
“We believe that in the coming days and weeks the debate about this transaction will center on whether this is enough, and whether other bidders might emerge, rather than whether this was too much,” Porges wrote.
The current deal includes a fee of $1.2 billion if Alexion agrees to sell itself to another bidder, while AstraZeneca faces a $1.4 billion breakup penalty. The acquisition is expected to close in the third quarter of 2021 and Alexion shareholders would own 15% of the combined companies.
Alexion had $5.9 billion in sales for the 12 months ending with the third quarter of 2020, with a 24% growth rate, AstraZeneca said.
Rare-Disease Headquarters
The U.K. company said it intends to establish its headquarters for rare diseases in Boston. There are no plans for a major reduction in employment, Soriot said. The combined companies should have a dozen blockbuster drugs -- those with more than $1 billion in sales -- by 2023, up from nine as of 2019, CFO Dunoyer said on a call.
The deal will add to earnings, with annual synergies of about $500 million projected three years after completion, the company said.
Evercore Partners International LLP and Centerview Partners UK LLP are AstraZeneca’s lead financial advisers, while Ondra LLP also gave advice. Bank of America Corp. worked with Alexion.
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>>> Five key genes linked to severe COVID-19 found, suggesting drug targets
Reuters
December 11, 2020
By Kate Kelland
https://www.yahoo.com/news/five-key-severe-covid-19-161436410.html
LONDON (Reuters) - Five key genes are linked with the most severe form of COVID-19, scientists said on Friday, in research that also pointed to several existing drugs that could be repurposed to treat people who risk getting critically ill with the pandemic disease.
Researchers who studied the DNA of 2,700 COVID-19 patients in 208 intensive care units across Britain found that five genes involving in two molecular processes - antiviral immunity and lung inflammation - were central to many severe cases.
"Our results immediately highlight which drugs should be at the top of the list for clinical testing," said Kenneth Baillie, an academic consultant in critical care medicine at Edinburgh University who co-led the research.
The genes - called IFNAR2, TYK2, OAS1, DPP9 and CCR2 – partially explain why some people become desperately sick with COVID-19, while others are not affected, Baillie said.
The findings, published in the journal Nature, should help scientists speed up the search for potential drugs for COVID-19 by conducting clinical trials of medicines that target specific antiviral and anti-inflammatory pathways.
Among those with the most potential, he said, should be a class of anti-inflammatory drugs called JAK inhibitors, which includes the arthritis drug baricitinib, made by Eli Lilly.
Baillie's team also found that a boost in the activity of the INFAR2 gene could create protection against COVID-19, because it is likely to mimic the effect of treatment with interferon.
Various existing drugs are being explored in clinical trials for their potential against COVID-19 including interferon-beta-1a, interleukin-1 receptor antagonist and Sanofi's arthritis drug Kevzara.
So far, a steroid called dexamethasone and a newly developed antiviral called remdesivir, made by Gilead, are the only drugs authorised around the world to treat COVID-19 patients - although remdesivir is not recommended for severe cases of the disease and has had mixed results in trials.
Last month, the U.S. Food and Drug Administration approved Eli Lilly's antibody drug for COVID-19, bamlanivimab, for patients who are not hospitalized but are at risk of serious illness because of their age or other conditions.
<<<
KZIA market cap is still around $100 million. Not much, if the drug works. 12 million shares (ADR's) outstanding.
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