Satellos Announces New Data Further Demonstrating Safety, Tolerability, and Functional Impact of SAT-3247 in First-in-Human Trial of Adults with Duchenne Muscular Dystrophy

Executive Summary

• Phase 1a/b study of SAT‑3247 in five adult DMD patients showed a mean 118.6% increase in dominant‑hand grip strength and a 5.8% improvement in predicted forced vital capacity, both exceeding natural‑history expectations.
• The drug was safe and well‑tolerated with no moderate‑or‑worse adverse events or dose‑limiting toxicities observed over the 28‑day period.
• Satellos announced plans for an 11‑month open‑label follow‑up study in adults (16–25 yr) and a Phase 2 global, randomized, double‑blind, placebo‑controlled proof‑of‑concept trial in ambulatory children with DMD.

Key Details

• Study Population: 5 adult males with Duchenne muscular dystrophy, ages 20‑27 years, treated for 28 days.
• Efficacy – Grip Strength:
• Dominant hand: +118.6% mean increase (≈2 kg → ≈4 kg).
• Non‑dominant hand: +97.9% mean increase.
• Efficacy – Pulmonary Function: Predicted forced vital capacity improved by a mean of 5.8%, contrary to the typical ~5% annual decline in this age group.
• Safety: No drug‑related adverse events ≥moderate severity; no dose‑limiting toxicities reported. PK profile described as “desirable.”
• Correlative Findings: Greater grip‑strength gains correlated with higher Day 15 plasma concentrations of SAT‑3247 and higher baseline creatinine (proxy for muscle mass).
• Future Clinical Plans:
• Open‑Label Follow‑Up Study: 11‑month duration, enrolling the original five adults plus additional males aged 16‑25 years; primary endpoints – long‑term safety/tolerability and biceps brachii fat fraction; secondary endpoints – muscle force, function, and fat fraction. Interim results expected after 3‑month follow‑up visit.
• Phase 2 Proof‑of‑Concept: Global, randomized, double‑blind, placebo‑controlled trial in ambulatory children with DMD; primary endpoints – safety/tolerability and muscle force; secondary endpoints – muscle quality, function, regeneration. Regulatory filings submitted in the U.S. and internationally.
• Presentation Venue: Late‑breaking poster at the 30th Annual Congress of the World Muscle Society, Vienna, Austria.

Notable Quotes

“Satellos’ new and updated clinical results from the 28‑day clinical study in adults with Duchenne provide an important validation of SAT‑3247’s potential to be a safe and clinically meaningful treatment,” – Frank Gleeson, Co‑founder & CEO, Satellos Bioscience.

“These early signs of efficacy in adults with more advanced disease are incredibly encouraging and support expanding our clinical program to the broader Duchenne community,” – Wildon Farwell, M.D., Chief Medical Officer, Satellos Bioscience.