Stealth BioTherapeutics Reaches 50% Enrollment Milestone in Phase 3 Trial for Novel Dry AMD Treatment
• Stealth BioTherapeutics has reached 50% enrollment in its Phase 3 ReNEW study evaluating elamipretide, a first-in-class mitochondria-targeted therapy for dry age-related macular degeneration.
• The trial will assess elamipretide's ability to slow photoreceptor loss through daily self-administered subcutaneous injections, with data expected in 2026.
• Dry AMD affects approximately 19.8 million Americans aged 40 and older, with current treatment options limited for this leading cause of irreversible blindness.
FDA Extends Review of Elamipretide for Barth Syndrome Treatment
• The FDA has extended the PDUFA action date for elamipretide to April 29, 2025, allowing more time to review supplemental data for Barth syndrome treatment.
• The extension follows a positive FDA advisory committee meeting that supported elamipretide's effectiveness for treating Barth syndrome.
• The FDA has not raised any safety concerns or requested new pre-marketing studies, reconfirming post-marketing commitments for elamipretide.
• If approved, elamipretide would be the first FDA-approved therapy for Barth syndrome, addressing a significant unmet medical need.
Ashvattha Therapeutics' Migaldendranib Shows Promise in Reducing Treatment Burden for Wet AMD and DME
• Ashvattha Therapeutics announced positive Phase 2 data for Migaldendranib (MGB), a subcutaneous nanomedicine, in treating wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME).
• The study showed a significant reduction in the need for anti-VEGF intravitreal injections in patients treated with MGB, with a 69% and 76.5% reduction observed in wet AMD and DME, respectively.
• MGB demonstrated maintenance of visual acuity and reduction of central subfield thickness, suggesting potential for convenient at-home treatment and reduced clinic visits for patients.
• Ashvattha Therapeutics also secured $50 million in funding to advance the Phase 2 ophthalmology trial and Phase 1/2 neuroinflammation trial.
FDA Accepts Abeona Therapeutics' BLA Resubmission for Prademagene Zamikeracel in RDEB Treatment
• The FDA has accepted Abeona Therapeutics' resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB).
• Pz-cel, an autologous cell-based gene therapy, aims to address the unmet needs of RDEB patients by providing collagen VII expression at wound sites.
• The BLA is supported by data from the Phase 3 VIITAL study and a Phase 1/2a study with up to 8 years of follow-up, showcasing clinical efficacy and safety.
• The FDA has set a PDUFA target action date of April 29, 2025, with potential for Abeona to receive a Priority Review Voucher upon approval.
FDA Advisory Committee Backs Elamipretide for Barth Syndrome Despite Data Concerns
• The FDA's Cardiovascular and Renal Drugs Advisory Committee voted 10-6 in favor of elamipretide for treating Barth syndrome, a rare genetic disorder.
• The decision was made despite concerns about the strength and design of the clinical trial data presented by Stealth BioTherapeutics.
• If approved, elamipretide would be the first FDA-approved therapy for Barth syndrome, addressing a significant unmet medical need.
• The FDA's final decision on elamipretide is expected by January 29, 2025, and will consider the advisory committee's recommendation.
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