Shares of Akero Therapeutics Inc. (NASDAQ:AKRO) were trading at $32.55, up $4.74, or 17%, after the South San Francisco-based firm disclosed preliminary top-line week 96 results from Harmony, a phase IIb study evaluating the efficacy and safety of lead candidate efruxifermin (EFX) in patients with pre-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH), fibrosis stage 2 or 3. The study previously met its primary endpoint of ≥1 stage improvement in fibrosis with no worsening of MASH after 24 weeks of treatment for both the 50-mg EFX (41%) and 28-mg EFX (39%) dose groups, compared to 20% for the placebo arm. At week 96, the response rates on the same endpoint increased to 75% for 50-mg EFX and 46% for 28-mg EFX, compared to 24% for placebo. The drug is an FGF21 analogue like 89bio Inc.’s pegozafermin, also in the works for MASH. A phase III study is planned. Shares of the firm (NASDAQ:ETNB), based in San Francisco, were selling for $14.34, up $1.72, or 13.6%.

Vivoryon’s varoglutamstat misses goal in early Alzheimer’s trial

Shares of Vivoryon Therapeutics NV (Euronext Amsterdam:VVY) lost 90% of their value March 4 on disappointing results from the phase IIb European Viviad study testing oral glutaminyl cyclase inhibitor varoglutamstat in patients with early Alzheimer’s disease. Top-line data showed Viviad did not hit statistical significance on the primary endpoint, defined as a change on cognition, as measured using the Cogstate neuropsychological test battery evaluating attention and working memory. Vivoryon said it will conduct an in-depth analysis, including for additional prespecified and exploratory endpoints, to determine next steps. Meanwhile, a phase IIb study in the U.S. remains ongoing.

Biocardia’s phase III bolsters cell therapy for heart failure, but stock down

While running a follow-on phase III study of its Cardiamp cell therapy, Biocardia Inc. has mined positive interim data from its predecessor. Those with advanced chronic heart failure patients saw a 37% relative risk reduction in heart death equivalent, including death, heart transplant, left ventricular assist device implantation, and a 9% relative risk reduction in non-fatal major adverse cardiac and cerebrovascular events. A data safety monitoring board determined in August that the original phase III would not meet its composite primary endpoint. Biocardia received U.S. FDA breakthrough device designation for Cardiamp for treating heart failure in February 2022. The therapy delivers 200 million autologous bone marrow cells to the heart in a catheter-based procedure with the goal of stimulating healing. The company’s Helix intramyocardial delivery system, which creates a self-healing helical pathway in the heart, allows patients to leave the hospital the day after their procedure. Company shares (NASDAQ:BCDA) were down sharply by 20% midday when they were trading at 46 cents each.

US price negotiation update: Counteroffers in, another challenge dismissed

With two U.S. courts rejecting constitutional challenges to Medicare drug price negotiations, every company that had a drug selected for the first round of negotiations submitted a counteroffer to Medicare’s initial offer of what it considered a maximum fair price by the March 2 deadline, according to the White House. On the eve of that deadline, the U.S. District Court for the District of Delaware dismissed Astrazeneca plc’s challenge to both the Inflation Reduction Act provision mandating the negotiations and the Centers for Medicare & Medicaid Services’ guidance on implementing the price-cutting program. In explaining the decision, the court cited Astrazeneca’s lack of standing and its failure to identify “a property interest protected by the Constitution that is put in jeopardy by the program.”

Newco news: Onko-innate seeks targets thought to be undruggable

A new spinout from Monash University in Melbourne, Australia, is tackling biology to better understand immune cell function and to find targets that were thought to be undruggable. Onko-innate co-founders Jai Rautela and Nicholas Huntington first worked together at Huntington’s lab at the Walter and Eliza Hall Institute of Medical Research in Melbourne where they studied the role of natural killer (NK) cells in tumor immunology and discovered some interesting regulatory pathways for cytokine responses. NK cells help drive inflammation in solid tumors, and by improving the function of NK cells in solid tumors, immune function can be increased, Huntington, who is the chief scientific officer, told BioWorld.

Biocom partnering meeting: Pharma is looking for deals to be done

With valuations heading higher, pharmaceutical companies are looking to make deals. “The M&A environment has kind of woken up a little bit,” Jay Stamatis, VP and head of business development and acquisitions at Abbvie Inc., told the audience at Biocom California’s Global Life Science Partnering & Investor Conference. Whether a biotech is looking to be acquired or to make a licensing deal, business development professionals from a variety of pharmaceutical companies on three separate panels during the conference were happy to dish advice on how to approach them and get deals done.

Lumicell imaging drug in the spotlight for adcom

Tomorrow’s meeting of the U.S. FDA’s Medical Imaging Drugs Advisory Committee could be the gateway to the first approved intraoperative technology for use in breast cancer that directly examines the lumpectomy cavity for residual cancer. If approved, Lumicell Inc.’s imaging drug Lumisight (pegulicianine), which would be used with its Direct Visualization System, could eliminate the need for a second surgery to address positive margins. The main concern the FDA has revolves around hypersensitivity, namely anaphylactic reactions, to the drug. In its briefing document for the meeting, the agency proposes several ways to mitigate that risk.

EMA validates two filings for Daiichi-Astrazeneca’s ADC

The EMA validated two marketing approval applications of Daiichi Sankyo Co. Ltd. and Astrazeneca plc’s antibody-drug conjugate (ADC) datopotamab deruxtecan (dato-dxd) on Mar. 4, for two types of lung and breast cancer. Datopotamab deruxtecan, listed on Clarivate’s Drugs to Watch 2024 list, is expected to become a best-in-class trophoblast cell surface antigen-2 (TROP2)-directed cancer drug, after Gilead Sciences Inc.’s Trodelvy, for both non-small-cell lung cancer and hormone receptor-positive/HER2-negative and triple-negative breast cancer. While high TROP2 expression is associated with increased tumor progression and poor survival, no TROP2-directed ADC is yet approved for lung cancer.

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Anagenex, Antibe, Apollomics, Aruna, Bayer, Biomunex, Biovie, Bridgebio, C4, Coherus, Elevation Oncology, Eyepoint, Genesis, Heidelberg, Merck, Nektar, Nocion, Nuance, Oragenics, Regeneron, Revance, Sandoz, Shattuck Labs, Sitryx, Skye, Tome, Verona, Xortx, Y-Mabs