Vertex Pharmaceuticals Inc. upped its own ante with pivotal phase III data in its strong suit, cystic fibrosis (CF). Taking on Trikafta, the company’s bestseller, Vertex’s once-daily vanzacaftor/tezacaftor/deutivacaftor (vanza) combo hit all primary and key secondary endpoints in two randomized controlled studies of those ages 12 and older.
Korean bioventure Curacle Co. Ltd. reported positive top-line findings from its U.S.-based phase IIa study of CU06-1004, an oral drug for diabetic macular edema, spurring plans for a bigger phase IIb study in the second half of 2024.
Sangamo Therapeutics Inc.’s gene therapy for Fabry disease, isaralgagene civaparvovec (ST-920), is continuing to show promising efficacy and safety in the phase I/II study, Staar, but further ahead, the company needs to either attract a partner or secure financing to move to a registrational trial.
In August 2020, Johnson & Johnson paid $6.5 billion cash to acquire Momenta Pharmaceuticals Inc. That strengthened J&J’s immune-mediated disease portfolio and grew its interest in autoantibody-driven disease therapies by bringing nipocalimab into the fold. Now the investment is paying off with top-line results of phase II and III studies that hit their primary endpoints using the fully human glycosylated monoclonal antibody targeting the human neonatal Fc receptor. The studies were in treating generalized myasthenia gravis and in Sjögren’s disease.
As the company’s phase II study in idiopathic pulmonary fibrosis continues, Pliant Therapeutics Inc.’s investors looked hard at 12-week interim data from the 320-mg dose group of Integris-PSC, a multinational, randomized, double-blind, placebo-controlled phase II trial testing bexotegrast in primary sclerosing cholangitis and suspected moderate to severe liver fibrosis.
4DMT Molecular Therapeutics Inc. is looking ahead to phase III as positive data continue to roll out for gene therapy candidate 4D-150 in wet age-related macular degeneration (AMD), with the results from the phase II portion of the phase I/II Prism trial showing the single-shot treatment significantly reduced the need for chronic anti-VEGF injections. Presented over the weekend at the Angiogenesis, Exudation, and Degeneration 2024 Conference, results from the 24-week dose-expansion cohort, which comprised wet AMD patients with severe disease and high treatment burdens, showed patients receiving the high dose (3E10 vg/eye) had a 90% reduction in annualized anti-VEGF injection rates.
Biogen Inc.’s problematic trip with Aduhelm (aducanumab-avwa) for Alzheimer’s disease (AD) at last came to an end as the company, probably surprising few on Wall Street, said it’s quitting sales and development of the amyloid-beta directed antibody. The drug won accelerated approval by the U.S. FDA in July 2021. The phase III Envision study with Aduhelm will be stopped, and Cambridge, Mass.-based Biogen chalked a one-time charge of about $60 million related to close-out costs for the program in the fourth quarter of 2023. AD efforts have turned toward Leqembi (lecanemab-irmb), also an amyloid-beta directed antibody.
The topical combination therapy miconazole plus domiphen bromide (MCZ-DB) resolved signs and symptoms of acute vulvovaginal candidiasis (VVC) and eradicated the yeast Candida albicans from vaginal swabs to a greater extent than miconazole (MCZ) alone in a phase II study, according to Hyloris Pharmaceuticals SA and Purna Female Healthcare.
Despite Vertex Pharmaceuticals Inc.’s mostly positive phase III study results of a non-opioid for treating moderate to severe acute pain, the data for a key secondary endpoint show VX-548 is not as effective as Vicodin (hydrocodone, Abbvie Inc.).
In keeping with federal standards for classifying race and ethnicity data, the U.S. FDA issued a draft revision to broaden its 2016 guidance on the collection of such data in clinical trials.
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