With new and positive phase III data in treating agitation in patients – at home instead of in a clinic – with bipolar disorders or schizophrenia, Bioxcel Therapeutics Inc. is preparing to submit an sNDA in the first quarter of 2026 to expand the label of the company’s Igalmi (BXCL-501). These data also bring the company closer to having the first such treatment that is approved by the U.S. FDA for home use. The unmet need for patients is huge, Vimal Mehta, the CEO of Bioxcel, told the BioWorld Insider podcast. Watch the interview, below.
Akeso Pharmaceuticals Inc.’s gumokimab (AK-111) and manfidokimab met all primary efficacy endpoints in its phase III trials in active ankylosing spondylitis and in moderate to severe atopic dermatitis, respectively.
French vaccines specialist Osivax SAS has been awarded $19.5 million by the U.S. Biomedical Advanced Research and Development Authority (BARDA) to fund work on its universal influenza A vaccine.
Eli Lilly and Co.’s regulatory ducks are lined up nicely with the latest positive top-line results from the phase III Attain-2 trial testing orforglipron, an oral glucagon-like peptide 1 (GLP-1) receptor agonist, in adults with obesity or overweight and type 2 diabetes.
Amid the increasingly competitive myasthenia gravis drug development space, siRNA candidate cemdisiran met phase III endpoints, with the monotherapy showing numerically higher results than a combination product. Regeneron Pharmaceuticals Inc., which has a worldwide license to cemdisiran from Alnylam Pharmaceuticals Inc., plans to file for U.S. approval in generalized myasthenia gravis, a rare and chronic autoimmune disease leading to life-threatening muscle weakness, in the first quarter of 2026.
The frontiers of xenotransplantation have been further extended with a pig-to-human lung transplant, the first time an organ that is directly exposed to the external environment – with the associated risk of respiratory pathogens – has been transplanted. The genetically modified pig lung remained viable and functional for nine days, after it was transplanted into a 39-year-old man who was declared brain dead following a hemorrhagic stroke.
Showing similar results as the first phase III trial completed in 2022, Nicox SA’s nitric oxide-donating bimatoprost eye drop, NCX-470, met the primary endpoint in the phase III Denali trial for open-angle glaucoma or ocular hypertension, clearing the way for regulatory filings in both the U.S. and China.
With a few tweaks to the protocol, Rocket Pharmaceuticals Inc.’s phase II trial testing RP-A501 in Danon disease is expected to resume following the lifting of the clinical hold, issued by the U.S. FDA in May in response to the death of a patient in the pivotal gene therapy study.
Celldex Therapeutics Inc. will not advance barzolvolimab (CDX-0159, barzo) in eosinophilic esophagitis (EoE) after taking a look at top-line data from the ongoing phase II effort. The study met its primary endpoint, proving barzo’s efficacy in depleting mast cells in the gastrointestinal (GI) tract, but this didn’t improve symptoms of EoE – a chronic inflammatory disease of the esophagus – which means mast cells are not a primary driver. Hampton, N.J.-based Celldex noted that barzo did turn up a favorable safety profile.
Rates of adverse events (AEs) gave pause to some on Wall Street, and shares of Viking Therapeutics Inc. (NASDAQ:VKTX) fell 42%, or $17.73, to close Aug. 19 at $24.36 after the firm reported 13-week data from the phase II trial with the oral tablet form of VK-2735, the company's dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors.
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