Satellos Bioscience announces data on tolerability, initial efficacy of SAT-3247

Satellos Bioscience (MSCLF) announced new data further demonstrating tolerability and initial efficacy of SAT-3247 in adults with Duchenne muscular dystrophy at the 30th Annual Congress of the World Muscle Society in Vienna, Austria. Data presented at the meeting demonstrate that SAT-3247 was safe and well-tolerated across the Phase 1a/b study with a desirable pharmacokinetic profile. The presentation also included new analyses of exploratory measures of early drug effect. Individuals treated with SAT-3247 over a 28-day period demonstrated an increase in grip strength far greater than seen in the Duchenne natural history in this age group. Specifically, a 118.6% mean improvement in maximum grip strength was observed in the dominant hand and 97.9% mean improvement in the non-dominant hand, representing an approximate doubling of grip strength from ~2 kg to ~4 kg. These improvements are inconsistent with published natural history1 and were correlated with higher drug concentrations on Day 15 and higher baseline creatinine, which we believe indicates our drug is having the desired impact on muscle. Furthermore, participants exhibited a 5.8% mean improvement of predicted forced vital capacity; such an increase is also inconsistent with natural history with declines about 5% annually among adults with Duchenne. All other measures remained stable over the study period. No drug-related adverse events of moderate severity or higher were observed in either study, and no dose-limiting toxicities occurred. The five adult patients, aged 20-27 years, who participated in the Phase 1b trial are now invited to enroll in an 11-month open-label, follow-up study of SAT-3247, which will also enroll additional males with DMD, aged 16-25 years. The primary endpoints of this study are to evaluate long-term safety and tolerability, as well as the effect of SAT-3247 on fat fraction in biceps brachii muscle. Secondary endpoints include the effect of SAT-3247 on fat fraction, and impact on muscle force and function. Once participants have completed their 3-month follow-up visit, the company will provide initial interim results. Based on the initial safety and efficacy data from the Phase 1a/b trial, Satellos is also planning a Phase 2 randomized, double-blind, placebo-controlled, global, proof-of-concept study of SAT-3247 in ambulatory children with DMD. Primary endpoints will evaluate safety and tolerability of SAT-3247 and effect on muscle force. Secondary endpoints will evaluate SAT-3247’s impact on muscle quality, function, and regeneration. The company has recently submitted regulatory filings in the U.S. and globally to advance this study.