After a long regulatory road that included a complete response letter in May, Stealth Biotherapeutics Inc. finally got its Barth syndrome drug across the finish line, with the U.S. FDA granting accelerated approval to Forzinity (elamipretide HCl) to improve muscle strength in those with the ultra-rare pediatric mitochondrial cardioskeletal disease.
“People have some inability to focus on [Regeneron Pharmaceuticals Inc.’s] pipeline,” which stands as “the most prolific in the industry, I would dare to say,” CEO Leonard Schleifer remarked during the Morgan Stanley health care conference Sept. 8. Most recently, Regeneron bragged on two prospects. The ultra-rare disease fibrodysplasia ossificans progressiva (FOP) took center stage Sept. 17 with news that the phase III Optima trial testing fully human monoclonal antibody garetosmab met its primary endpoint. Separately, Regeneron provided updated analyses of the phase II Courage trial that tested new pairings of GLP-1 receptor agonist semaglutide plus the anti-GDF8/anti-myostatin compound trevogrumab, with or without garetosmab, in obesity.
As Avidity Biosciences Inc. brought the second-largest follow-on offering of the year to the market, the company also released positive early and midstage stage results of del-zota, an antibody-oligonucleotide conjugate, in treating Duchenne muscular dystrophy. Phase I/II results showed a reversal of disease progression in patients who have been continuously treated for a year, plus improvements in several functional measures.
At the American Society for Bone and Mineral Research (ASBMR) in Seattle, researchers from Ashibio Inc. reported preclinical efficacy data on vantictumab, a human monoclonal IgG2 lambda antibody that binds to multiple frizzled (FZD) receptors.
Heme oxygenase 1 (HO-1) is a stress-inducible enzyme that plays a critical role in the pathogenesis of rheumatoid arthritis, a systemic autoimmune disease characterized by chronic synovial inflammation, immune cell infiltration and progressive joint destruction.
Onco3r Therapeutics BV has obtained clinical trial application (CTA) approval by the Belgian regulatory authorities for its SIK3 inhibitor O3R-5671. A first-in-human trial will be conducted in Belgium and is expected to open enrollment in the coming weeks. Final data are expected in the first half of next year and will inform subsequent patient trials across a range of autoimmune diseases, which are planned to commence next year.
Researchers have identified a potential therapeutic target for muscular dystrophies, a group of genetic disorders characterized by progressive muscle weakness and degeneration. The study reveals that inhibiting the microRNA (miRNA) known as miRNA-33 can significantly improve muscle regeneration and ameliorate the dystrophic phenotype in animal models.
Ribomic’s umedaptanib pegol (RBM-007) looks to have some advantages compared to competitors in the achondroplasia space, and the company plans to progress the oligonucleotide-based aptamer that targets anti fibroblast growth factor 2 to phase III trials, Ribomic’s business development head, Kihei Yamashita, told BioWorld.
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.
Ribomic’s umedaptanib pegol (RBM-007) looks to have some advantages compared to competitors in the achondroplasia space, and the company plans to progress the oligonucleotide-based aptamer that targets anti fibroblast growth factor 2 to phase III trials, Ribomic’s business development head, Kihei Yamashita, told BioWorld.
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