Annexon Interim Mid-Stage Results On Huntington's Candidate Fails To Cheer

 Annexon Inc (NASDAQ: ANNX) posted interim data from its ongoing Phase 2 trial of ANX005 in patients with Huntington's disease (HD) who completed the 24-week treatment period.

• Annexon is developing ANX005, its lead monoclonal antibody candidate, for a range of complement-mediated disorders, including HD.

• Results showed that relative to baseline after six months of treatment, improvement in mean Composite Unified Huntington's Disease Rating Scale (cUHDRS) was observed in more than half of all evaluable patients.

• Additionally, as of the cutoff date, 56% of patients showed improvement from baseline in cUHDRS and several subdomains of cUHDRS over six months of treatment.

• Also, 75% of evaluable patients demonstrated excess complement activity at baseline.

• The open-label trial of ANX005 enrolled 28 participants. The most common adverse events reported were infusion-related reactions.

• The Company anticipates reporting complete data from all patients enrolled, including data from the three-month follow-up period, in Q2 of 2022.

• Pending results from the entire dataset, Annexon plans to evaluate the opportunity for a Phase 3 trial of ANX005 in HD patients.

https://finance.yahoo.com/news/annexons-interim-mid-stage-results-113307348.html

Edgewise: Positive Topline Results in Phase 1b Trial in Becker Muscular Dystrophy

 DG-5506 was well tolerated with no serious adverse events observed –

– Pharmacokinetic (PK) data demonstrated robust target engagement with achievement of muscle concentrations beyond levels predicted to provide meaningful clinical benefit –

– EDG-5506 treatment resulted in significant lowering of muscle damage biomarkers in adults with BMD after only two weeks of dosing –

– Management hosting webcast to discuss findings today at 9:00 a.m. Eastern Time –

Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the treatment of rare muscle disorders, announced today positive topline results from the BMD, or Phase 1b, portion of a first-in-human Phase 1 clinical trial assessing the safety, tolerability, PK and pharmacodynamics (PD) of EDG-5506, an orally administered small molecule myosin inhibitor designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies such as Duchenne muscular dystrophy (DMD) and BMD.

"BMD is a rare, serious, progressive, permanently debilitating, and potentially fatal inherited neuromuscular disorder, which causes great hardship to individuals with BMD and their families and caregivers," said Craig McDonald, M.D., Professor and Chair of the Department of Physical Medicine & Rehabilitation at UC Davis. "I am encouraged by the EDG-5506 Phase 1b clinical data in individuals with BMD, for which there are no approved treatments. I look forward to working with fellow investigators and the BMD and DMD communities in the upcoming Phase 2 trials of EDG-5506."

The seven adults with BMD enrolled in the Phase 1b clinical trial were administered 20 mg oral doses of EDG-5506 (n=5) or placebo (n=2). EDG-5506 was shown to be well-tolerated with no discontinuations or dose reductions. The most common adverse event, observed in all Phase 1b participants, was dizziness which was mild, transient and self-resolving.

https://finance.yahoo.com/news/edgewise-therapeutics-announces-positive-topline-130000837.html

Mainz Biomed Acquires Exclusive Rights to Novel mRNA Biomarkers

 Potential for ColoAlert to Emerge as the Most Robust and Accurate At-home Screening Test for Colorectal Cancer

Biomarkers Demonstrated Unique Ability to Identify Curable Precancerous Colonic Polyps as well as Curable Early-Stage Colorectal Cancer

 Mainz Biomed N.V. (NASDAQ:MYNZ) (“Mainz Biomed” or the “Company”), a molecular genetics diagnostic company specializing in the early detection of cancer, announced today it has entered into a Technology Rights Agreement with Socpra Sciences Santé Et Humaines S.E.C. (“TTS”) to access a portfolio of novel mRNA biomarkers for potential future integration into ColoAlert, the Company’s highly efficacious, and easy-to-use detection test for colorectal cancer (“CRC”). Mainz is currently marketing ColoAlert in Europe through its unique business model of partnering with third-party laboratories for test kit processing versus the traditional methodology of operating a single facility. The Company is also preparing to initiate ColoAlert’s regulatory pathway for approval in the United States.

Under the terms of the Technology Rights Agreement, the Company has the unilateral option to license the exclusive global rights to five gene expression biomarkers which have demonstrated a high degree of effectiveness in detecting CRC lesions including advanced adenomas (“AA”), a type of pre-cancerous polyp often attributed to this deadly disease. In a study evaluating these biomarkers published in the online peer review journal platform MDPI (March 11, 2021), study results achieved overall sensitivities of 75% for AA and 95% for CRC, respectively, for a 96% specificity outcome. If these statistical results are duplicated when the biomarkers are integrated into ColoAlert, we believe that it will ultimately position the Company’s CRC test to be the most robust and accurate at-home diagnostic screening test on the market. It will not only detect cancerous polyps with a high degree of accuracy but has the potential to prevent CRC through early detection of precancerous adenomas.

https://finance.yahoo.com/news/mainz-biomed-acquires-exclusive-rights-080100033.html

Lixte Candidate Reported to Convert Immunologically Unresponsive (“Cold”) to Responsive (“Hot”) Tumors

 Outside research shows that in models of colorectal, triple-negative breast and pancreatic cancer, LB-100 induces molecular changes that render the cancers more vulnerable to immunotherapy

 Lixte Biotechnology Holdings, Inc. (Nasdaq: LIXT), a clinical-stage drug discovery company developing pharmacologically active drugs for use in cancer treatment, announced today that in preclinical studies its lead clinical compound, LB-100, a protein phosphatase (PP2A) inhibitor, was found to increase the responsiveness of diverse cancers to immunotherapy. In Nature Communications (15 Dec 2021), lead author Yu-Ting Yen and colleagues at the China Medical University and Hospital, Taichung, Taiwan reported that treatment with LB-100 is associated with new antigen production, tumor infiltration of cytotoxic T cells, and enhanced responsiveness to immune checkpoint blockade in mouse models of colorectal, triple-negative breast, and pancreatic cancer. The authors state that “these data indicate that PP2A inhibition can be used to change the intrinsic and extrinsic factors of tumors to improve the success rate of anti-cancer immunotherapy.”

Lixte’s founder and CEO, John S. Kovach M.D, commented that “recent advances in therapy with immune checkpoint inhibitors have been a true breakthrough in the development of more effective and less toxic treatment for many cancers. Unfortunately, most cancer patients do not respond to immunotherapy. There are widespread efforts to find pharmacologic and/or immunologic ways to turn unresponsive (‘cold’) tumors into responsive (‘hot’) tumors. Cancers with a molecular abnormality termed microsatellite-instability (MSI) stemming from a defect in a DNA repair enzyme are generally ‘hot’ tumors, that is, tumors responsive to immunotherapy. Yen and colleagues now report that pharmacologic inhibition of PP2A by LB-100 modifies two distinct molecular pathways resulting in conversion of microsatellite stable (MSS) tumors into MSI tumors sensitive immune checkpoint blockade therapy.”

https://finance.yahoo.com/news/lixte-biotechnology-lb-100-reported-133000354.html

Regeneron cut to Underperform from Neutral by B of A

 Target to $575 from $675

https://finviz.com/quote.ashx?t=regn&ty=c&ta=1&p=d

Pfizer and BioNTech to Develop First mRNA-based Shingles Vaccine

 

• Third mRNA vaccine collaboration between the companies aims to accelerate development of an improved vaccine for shingles, a debilitating, disfiguring and painful disease that affects people all over the world

• Product candidates will be based on BioNTech’s proprietary mRNA technology and on Pfizer’s antigen technology

• Clinical trials are expected to start in the second half of 2022

• BioNTech will receive $225 million upfront, including an upfront cash payment of $75 million and an equity investment of $150 million, and will be eligible to receive future approval and sales milestone payments totaling up to $200 million as well as a share of gross profits arising from future product sales

• Pfizer will receive an upfront payment of $25 million from BioNTech for its proprietary antigen sequences identified by Pfizer

https://finance.yahoo.com/news/pfizer-biontech-sign-global-collaboration-114500755.html

NRx Submits Emergency Use Authorization Application for ZYESAMI in Patients at RIsk of Death from COVID

 Requested indication focuses on patients whose respiratory failure has progressed despite treatment with Remdesivir or other approved therapies

- Patients treated with ZYESAMI vs. placebo demonstrated statistically significant (P=.03), 2.8-fold increased odds of being alive and free of respiratory failure at day 28 and day 60 and a 4-fold increased odds of surviving to 60 days (P=.006)

- Patients at highest risk - those on ventilators at time of randomization - demonstrated a 10-fold increased odds of survival (P=.03)

- US National Institutes of Health-sponsored trial comparing ZYESAMI and Remdesivir individually and in combination continues to demonstrate safety and has enrolled more than 350 patients

https://finance.yahoo.com/news/nrx-pharmaceuticals-submits-emergency-authorization-114800038.html