The prospect of a gene therapy for Alzheimer’s disease has kept Wall Street steadily interested in Lexeo Therapeutics Inc., and another increment of intrigue was added when the New York-based firm offered positive interim results from the 52-week, 15-subject phase I/II study of LX-1001 for the treatment of the condition when APOE4-associated.
Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York. The agency is trying to set up cell and gene companies for success and that’s a very different agency than what it was years ago, said Paul Bresge, CEO of Ray Therapeutics Inc.
While phase II results of Coya Therapeutics Inc.’s low-dose IL-2 drug, COYA-301, showed promise in Alzheimer’s disease patients when dosed every four weeks, it was the more frequent dosing of every two weeks that led to exhausted regulatory T cells and no benefits, driving down the company’s stock by nearly 28%.
Semaglutide, the glucagon-like peptide 1 receptor agonist from Novo Nordisk A/S, which has seemingly improved every disease it’s been tested on, was a focus at Kidney Week 2024, where researchers presented data from multiple clinical studies in patients with kidney diseases.
In September 2024, BioWorld recorded 252 clinical trial updates, up from 92 in August, due in part to news from the European Society for Cardiology Congress, the IASCLC World Conference on Lung Cancer and the European Association for the Study of Diabetes, among others. The month included 35 successful phase III trial updates and four failures.
Despite an outcome that TD Cowen analyst Tyler Van Buren called ”fantastic,” shares of Tyra Biosciences Inc. (NASDAQ:TYRA) closed Oct. 25 at $21.93, down $6.68, or 23%, as Wall Street digested new phase I/II data with FGFR3 inhibitor TYRA-300 in metastatic urothelial cancer from the Surf301 phase I/II study in progress.
Visterra Inc., a subsidiary of Otsuka Pharmaceutical Co. Ltd., reported positive top-line data from the ongoing Visionary phase III study of sibeprenlimab, an anti-APRIL monoclonal antibody for immunoglobulin A nephropathy (IgAN).
For a company that was running out of money, a missed phase III endpoint for its only development product knelled a death blow for Marinus Pharmaceuticals Inc., tanking its stock by 82%. The Radnor, Pa.-based company will no longer develop oral ganaxolone for seizures associated with tuberous sclerosis complex, or for any other indication, as it reduces its workforce and explores strategic alternatives.
Hope Medicine Inc. reported positive interim results for monoclonal antibody HMI-115 in a phase II endometriosis trial that saw the mean non-menstrual pelvic pain score reduced by 50%. “HMI-115 is a prolactin receptor blocker, and we're using it to treat endometriosis and some other diseases. It is a first-in-class new mechanism to treat endometriosis,” Hope Medicine CEO Nathan Chen told BioWorld.
Despite positive findings from an earlier trial, Alto Neuroscience Inc.’s BDNF-targeting candidate, ALTO-100, failed to best placebo in a phase IIb study in major depressive disorder, sending shares of the company to their lowest price since going public in a February 2024 IPO, as investors worried about readthrough to Alto’s biomarker-based approach for treating psychiatric disorders.
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