Satellos Bioscience announced new data further demonstrating tolerability and initial efficacy of SAT-3247 in adults (aged 20-27 years) with Duchenne muscular dystrophy (Duchenne or DMD) at the 30th Annual Congress of the World Muscle Society in Vienna, Austria.
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Key Findings
- Adults with Duchenne muscular dystrophy demonstrated functional improvements exceeding natural history, following 28-day treatment with SAT-3247
- Grip strength increased 118.6% and predicted forced vital capacity increased 5.8%; whereas, both decline in natural history
- SAT-3247 demonstrated safety, tolerability, and expected pharmacokinetics (PK) across all parts of Phase 1 a/b study
- New data presented in late-breaking poster session at the 30th Annual Congress of the World Muscle Society
“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,” said Frank Gleeson, co-founder and CEO of Satellos. “We are excited and confident in progressing into our next phase of clinical studies with the objective of demonstrating the transformative potential of SAT-3247 to the Duchenne community.”
Data presented at the meeting demonstrate that SAT-3247 was safe and well-tolerated across the Phase 1a/b study with a desirable pharmacokinetic (PK) profile. The presentation also included new analyses of exploratory measures of early drug effect.
Increase in Grip Strength
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 (a surrogate for increased muscle mass), 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.
Independent of Specific Exon Mutation
“Satellos scientific co-founders were the first to recognize that the body’s innate process of muscle regeneration is severely impaired in Duchenne. They invented SAT-3247 with the aim of rebooting this impaired regeneration process, so that the body can once again repair and rebuild muscle,” said Wildon Farwell, M.D., Satellos chief medical officer. “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. SAT-3247 has been designed to be a convenient, oral therapy with the potential to improve outcomes, regardless of an individual’s specific dystrophin mutation.”
Future Clinical Trial Plans
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.
