Only two days after Bridgebio Pharma Inc. impressed investors with data from BBP-418 in limb-girdle muscular dystrophy type 2I/R9, the company was back at it again, this time reporting positive top-line results from its global phase III study of encaleret in autosomal dominant hypocalcemia type 1, a genetic form of hypoparathyroidism.
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.
Hanmi Pharmaceutical Co. Ltd. announced Oct. 27 that its glucagon-like peptide-1 receptor agonist, efpeglenatide (HM-11260C), met the co-primary endpoints in a phase III study of obese adults without diabetes.
An examination of X-ray and MRI scans of 518 patients before and after treatment with Levicept Ltd.’s Levi-04 has shown that, in addition to providing significant analgesia, the selective neurotrophin-3 inhibitor may have a disease-modifying effect in osteoarthritis of the knee.
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.
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