The winding regulatory road for the BLA to Capricor Therapeutics Inc.’s cell therapy for Duchenne muscular dystrophy has more clarity. Out of a recent type A meeting between Capricor and the U.S. FDA, prompted by a complete response letter in July regarding lead asset CAP-1002 (deramiocel), the two are in agreement about a path to potential approval.
Amyotrophic lateral sclerosis (ALS), formerly known as Lou Gehrig’s disease, is a progressive and fatal neurodegenerative disorder with no known cure. While three therapies have gained U.S. FDA approvals to date, including Rilutek (riluzole), Radicava/Radicava ORS (edaravone) and tofersen (BIIB-067, the lack of a disease-modifying drug has spurred the continual search for novel therapies.
A failed July inspection of manufacturer Catalent Indiana LLC has delayed another U.S. FDA approval, the latest being that of Scholar Rock Inc.’s selective anti-latent myostatin antibody, apitegromab, which was expected to become the first therapy to enhance skeletal muscle in patients with spinal muscular atrophy.
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.
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.
Alterity Therapeutics Ltd. helped develop a new neuroimaging biomarker called the multiple system atrophy index (MSA-AI), which looks to be a more reliable biomarker for tracking disease progression of MSA. Developed using deep learning methods, the MSA-AI offers a superior, objective and quantifiable measure of brain atrophy in MSA patients.
Sarepta Therapeutics Inc.’s adventure with the Duchenne muscular dystrophy (DMD) AAV-based gene therapy Elevidys (delandistrogene moxeparvovec) continued as the firm said it would restart shipments of the compound for ambulatory patients “imminently,” with the U.S. FDA’s blessing.
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