Golodirsen

Six companies—Ionis Pharmaceuticals, Isarna Therapeutics, Regulus Therapeutics, Sarepta Therapeutics, Secarna Pharmaceuticals, and Wave Life Sciences—are leading in the development of antisense oligonucleotides, a targeted treatment for various diseases. Ionis, a pioneer with over 40 drugs in its pipeline, recently priced a $500 million IPO. Isarna's ISTH0036 targets TGF-β for ophthalmic conditions. Regulus focuses on microRNA targeting with its lead candidate RGLS8429 for ADPKD. Sarepta has three approved PPMO therapies for DMD and a recent $1 billion deal with Arrowhead Pharmaceuticals. Secarna's SECN-15 targets NRP1 for oncology. Wave Life Sciences' WVE-003, for Huntington's disease, recently reported positive phase 1b/2a trial results. The antisense oligonucleotide market is projected to grow to $5,519 million by 2033.

Sarepta Therapeutics has halted development of vesleteplirsen, a more potent version of its DMD therapy Exondys 51, citing risk-benefit considerations, FDA feedback, and the evolving DMD therapeutic landscape. Elevidys, Sarepta's gene therapy, is rapidly gaining ground, with $181 million in Q3 sales compared to $249 million for Exondys 51 and its counterparts.

Sarepta Therapeutics' Q3 2023 Form 10-Q highlights financial growth, key business developments, strategic initiatives, and emerging challenges. Notable financial metrics include $1,243.6 million in total revenues, $1,056.8 million in gross profit, $56.4 million in operating income, and $76.2 million in net income. Business highlights include FDA approvals for four products targeting Duchenne muscular dystrophy, new product launches, and ongoing clinical trials. Strategic initiatives focus on manufacturing expansion and capital management. Challenges include market risk, regulatory compliance, reimbursement uncertainty, and global expansion difficulties.

DelveInsight's 'Duchenne Muscular Dystrophy (DMD) Pipeline Insight, 2024' analyzes over 75 pipeline drugs from 75+ companies, focusing on recent FDA, EMA, and PMDA approvals, clinical trials, emerging therapies, and key players like Roche, Santhera, and Sarepta. The report covers various stages of development, routes of administration, and mechanisms of action, offering insights into the evolving DMD therapeutics landscape.

WaVe Life Sciences Ltd. (NASDAQ:WVE) focuses on RNA editing technology for genetic diseases, with a promising DMD program showing 53% exon skipping. The company faces competition from firms like Sarepta Therapeutics and KRRO, but recent analyst price targets suggest growing confidence. WaVe's future hinges on clinical trial success and strategic partnerships, particularly in RNA editing, which is seen as a transformative technology in genetic medicine.

EMBARK trial (SRP-9001-301) assessed delandistrogene moxeparvovec safety/efficacy in DMD patients aged 4-7. Conducted at 42 sites, it was a phase 3, two-part, multinational, randomized, double-blind, placebo-controlled trial. Patients received either delandistrogene moxeparvovec or placebo, with crossover in Part 2. Primary endpoint was change in NSAA total score from baseline to week 52. Key secondary endpoints included micro-dystrophin expression at week 12, and changes in TTR and 10MWR from baseline to week 52.