The long-term use of corticosteroids for treating Duchenne muscular dystrophy (DMD) is tied to several undesired effects, compromising the patient’s quality of life; hence, the use of nonsteroidal drugs is highly desirable in the treatment of DMD. Metriopharm AG is investigating the nonsteroidal drug MP-1032 for DMD and recently presented data from studies in a model of DMD.
Sarepta Therapeutics Inc.’s next-generation peptide-conjugated PMO therapy, SRP-5051 (vesleteplirsen), looks set to stake its claim in the Duchenne muscular dystrophy (DMD) space, as phase II data unveiled dystrophin expression that outperforms first-generation exon-skipping drug Exondys 51 (eteplirsen). The question is what that space might look like in the wake of a U.S. FDA decision whether to expand labeling and convert to full approval Sarepta’s DMD gene therapy, Elevidys (delandistrogene moxeparvovec).
After almost 10 years on the market, the EMA is to withdraw its conditional approval of the Duchenne muscular dystrophy treatment Translarna (ataluren), after concluding there is still not enough evidence of its effectiveness.
Edgewise Therapeutics Inc. priced an underwritten offering of 21.8 million shares at $11 each as it looks for about $240 million in gross proceeds to develop its Duchenne and Becker muscular dystrophies treatment. The offering propelled the company’s stock (NASDAQ:EWTX) Jan. 19 to close 34.5% higher at $13.04 each, their highest valuation in the past 12 months.
Solid Biosciences Inc.’s SGT-003 has been granted orphan drug designation by the FDA. The company’s next-generation Duchenne muscular dystrophy gene therapy candidate was also granted fast track designation last month.
“This is a tough business. It’s never a straight line from start to success.” Those words, from Exelixis Inc. CEO Michael Morrissey, during a presentation at the J.P. Morgan Healthcare Conference (JPM), could easily sum up any aspect of the biopharma industry. But with more biopharma firms than ever having reached commercial status, along with the introduction of new therapeutic modalities into the health care market, many are finding the toughest part comes after regulatory approval, whether it’s navigating a competitive landscape, getting payers and physicians on board, or satisfying regulators’ stringent postmarketing requirements. As industry players and observers head home after a busy week in San Francisco, BioWorld offers a brief glimpse at a few firms taking on those post-approval challenges in 2024.
Factor Bioscience Inc. has been awarded a grant by the U.S. Department of Defense to develop next-generation cell therapy candidates using Factor's patented mRNA, cell-reprogramming, and gene-editing technologies.
With positive initial phase I/II data in hand from two trials, Dyne Therapeutics Inc. plans to report more findings and start enrolling registrational cohorts in both studies by the end of this year for DYNE-101 in myotonic dystrophy type 1 (DM1) and a study called Deliver with DYNE-251 in Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
