Sarepta Therapeutics

Regenxbio has initiated the pivotal phase of its clinical trial for RGX-202 gene therapy in boys with Duchenne muscular dystrophy (DMD), aiming for accelerated FDA approval. New data from the Phase 1/2 study shows RGX-202 led to physical function gains, with microdystrophin expression levels at 10% or more of normal dystrophin in all participants. The pivotal study will enroll 30 DMD patients, including those as young as 1 year old, and is actively recruiting participants in multiple U.S. states.

REGENXBIO has initiated a pivotal Phase I/II/III trial for its gene therapy RGX-202 for Duchenne muscular dystrophy (DMD), following positive Phase I/II results. RGX-202, using a novel microdystrophin construct, demonstrated functional improvements and a favorable safety profile. The therapy is expected to file a Biologics License Application (BLA) in 2026 under the accelerated approval pathway, positioning REGENXBIO as a significant competitor to Sarepta Therapeutics.

Regenxbio aligns with FDA for an abbreviated approval path for its Duchenne muscular dystrophy gene therapy, expanding a Phase 1/2 study into a pivotal trial. The therapy aims to produce at least 10% normal levels of microdystrophin, with early data showing higher microdystrophin levels than Elevidys. The trial will not include an active placebo, focusing on microdystrophin production as the main goal, unlike previous studies by Pfizer and Sarepta.

Delandistrogene moxeparvovec (Elevidys) failed to meet primary endpoint in phase 3 EMBARK trial for Duchenne muscular dystrophy, showing no significant functional improvement after 52 weeks. Despite this, the gene therapy gained full FDA approval in 2023 for patients aged 4 and older, supported by secondary endpoint improvements and a manageable safety profile.

DelveInsight's 'Multiple Sclerosis Pipeline Insight 2024' report highlights 75+ companies developing 80+ therapies, including promising candidates like GA Depot, Remibrutinib, and IMU 838. Key events include Immunic's positive Phase 3 ENSURE trial interim analysis, Roche's FDA approval for Ocrevus injectable, and Sanofi's mixed results for tolebrutinib in Phase III trials. The report covers global pipeline stages, product types, molecule types, mechanisms of action, and routes of administration.

Life sciences and biopharma hedge funds saw gains in October, led by Casdin Capital's 16% surge. RTW Investments rose 13%, Madrigal Pharmaceuticals surged 22%, and Janus Henderson Biotech Innovation Fund climbed 2%. RA Capital Management gained 2%, while Avoro Capital Advisors and Soleus Capital added 1.6% and 2.1% respectively. Averill Partners lost 2.8%, and EcoR1 Capital dropped 3.4%, primarily due to Apellis Pharmaceuticals.

Acadia Pharmaceuticals sold its rare pediatric disease priority review voucher (PRV) for $150 million, with Neuren Pharmaceuticals set to receive $50 million. PRVs, awarded by the FDA for developing drugs for rare pediatric diseases, can be sold to accelerate drug reviews, highlighting their value in licensing deals.

Sarep­ta Ther­a­peu­tics is stop­ping de­vel­op­ment of vesleteplirsen, an ex­per­i­men­tal ex­on 51-skip­ping ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy pa­tients with cer­tain mu­ta­tions.

Sarepta Therapeutics discontinues SRP-5051 for Duchenne muscular dystrophy due to safety concerns and FDA feedback, as Elevidys gene therapy sales surge to $181 million in Q3. Elevidys revenue expected to exceed $2 billion next year, overshadowing the end of SRP-5051 development.