Based on positive phase III study results, Metis Techbio is planning to file an NDA for its AI-derived orally disintegrating tablet drug candidate for pseudobulbar affect, MTS-004, in China next year.
Although Argenica Therapeutics Ltd.’s stroke drug, ARG-007, saw mixed results in top-line phase II data, new data in functional outcomes studies showed signs the drug helped patients think more clearly, regain independence, and enjoy a better quality of life after stroke.
Intellia Therapeutics Inc. followed up troubling news in May with a similar, and worse, update regarding the Magnitude and Magnitude-2 phase III trials with nexiguran ziclumeran, also known as nex-z, for patients with transthyretin amyloidosis with cardiomyopathy and polyneuropathy, respectively.
Impressive data from an interim readout of Bridgebio Pharma Inc.’s BBP-418 in limb-girdle muscular dystrophy type 2I/R9 has the company prepping to meet with the U.S. FDA to discuss plans for the upcoming NDA filing, including the possibility for seeking full approval for what could be the first therapy for the rare muscular disease.
In September 2025, BioWorld recorded 230 clinical trial updates spanning phases I through III, up sharply from 95 in August, 140 in July and just below 254 in June. Among these, 22 phase III trials reported positive outcomes, while four ended in failure and another three produced mixed results.
Tango Therapeutics Inc. plans a pivotal study next year in second-line MTAP-deleted pancreatic ductal adenocarcinoma based on positive phase I/II data with next-generation MTA-cooperative PRMT5 inhibitor vopimetostat (TNG-462) in patients with tumors of that type.
Inhibrx Biosciences Inc.’s monoclonal antibody for treating advanced or metastatic, unresectable chondrosarcoma hit its primary endpoint in a registrational phase II study, doubling the company’s stock on Oct. 24. Top-line data from the placebo-controlled study of ozekibart (INBRX-109) produced statistically significant and clinically meaningful improvement in median progression-free survival in a tough-to-treat patient population that has few remaining options.
Ventyx Biosciences Inc. rolled out positive results from the phase II study with oral, once-daily VTX-3232 in patients with obesity and cardiovascular (CV) risk factors.
Ipsen SA is expanding its cancer portfolio with the €1 billion (US$1.16 billion) acquisition of immuno-oncology specialist Imcheck SAS, and will pay €350 million up front with the balance to come in regulatory and sales-based milestones for the lead program ICT-01.
Moderna Inc. stopped its efforts to develop mRNA-1647 as a vaccine for preventing congenital cytomegalovirus infection, the most common infectious cause of birth defects in the U.S. The pivotal and placebo-controlled phase III CMVictory study of mRNA-1647 missed the primary efficacy endpoint, which was preventing the infection in seronegative females ages 16 to 40.
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