Sarepta Therapeutics

Athira Pharma's lead candidate, fosgonimeton, failed Phase II/III LIFT-AD trial for Alzheimer's, causing a 81% stock drop. Despite missing primary and secondary endpoints, Athira remains committed to fosgonimeton's development, citing potential benefits in specific subgroups. Full data will be presented at the CTAD Conference in Madrid.

Elevidys, an AAV-based gene therapy for Duchenne muscular dystrophy, received FDA approval in 2023 for ambulatory patients aged 4-5 with specific DMD gene mutations, and expanded approval in 2024 for broader patient groups. While not curative, it offers a functional gene version, but faces challenges like insurance coverage and long-term data needs. Experts highlight the importance of patient education and choice amidst expanding treatment options.

Dyne Therapeutics' DYNE-251, an investigational antisense oligonucleotide, showed significant dystrophin expression in Duchenne muscular dystrophy patients amenable to exon 51 skipping in the DELIVER trial. DYNE-251 achieved a mean absolute dystrophin expression of 3.71% of normal, surpassing the standard of care. The therapy demonstrated functional improvements and a favorable safety profile, prompting the initiation of registrational cohorts with plans for an update on the path to registration by year-end 2024.

SRP-9001 (Elevidys) received FDA approval for Duchenne muscular dystrophy (DMD) patients, including nonambulatory ones, with continued approval contingent on clinical benefit verification. CureDuchenne hosted webinars discussing this gene therapy's impact and future developments in DMD treatment.

FDA approves Elevidys, a gene therapy for Duchenne muscular dystrophy, for most patients with a specific genetic mutation. Costing $3.2M per patient, it's a one-time treatment offering hope despite risks like liver injury. It marks significant progress in treating this progressive, muscle-wasting disease.

FDA granted traditional approval for Sarepta Therapeutics’ SRP-9001 (Elevidys) for ambulatory Duchenne muscular dystrophy (DMD) patients aged 4+, expanding its use. Accelerated approval for nonambulatory patients was also announced, pending confirmatory trial results. SRP-9001, a gene therapy, showed functional improvements in trials, despite not meeting the primary endpoint in the phase 3 EMBARK study.

FDA expanded approval for Sarepta Therapeutics’ gene therapy Elevidys to DMD patients aged 4 and older, including non-ambulatory ones, based on clinical benefits despite missing primary endpoints. Sarepta’s stock rose over 32%. Pfizer’s DMD gene therapy failed in Phase III, contrasting with Sarepta’s success in secondary endpoints, highlighting the unmet need and potential market for Elevidys.

Pfizer's phase III study on fordadistrogene movaparvovec for Duchenne muscular dystrophy (DMD) failed primary and key secondary endpoints. The therapy aimed to improve motor function in boys aged 4-8. Despite safety concerns, Pfizer plans to share detailed results soon. Sarepta Therapeutics leads the DMD treatment market.

The FDA's accelerated approval of Sarepta's Elevidys, despite failing efficacy endpoints, raises concerns. Priced at $3.1M, its approval was based on micro-dystrophin levels, not proven clinical benefit, highlighting debates over drug approval standards and pricing.

Zacks.com highlights biotech stocks, focusing on Q4 earnings and regulatory updates. Moderna beat earnings estimates, reaffirmed 2024 sales guidance, and expects RSV vaccine approval. RAPT Therapeutics faced a clinical hold due to a liver failure case. Iovance surged after FDA approval for melanoma treatment. Sarepta seeks label expansion for DMD drug. Regeneron's myeloma drug gets Priority Review.