Satellos releases new Duchenne trial data

Mr. Frank Gleeson reports 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 Satellos Bioscience Inc. has released new data further demonstrating tolerability and initial efficacy of SAT-3247 in adults (aged 20 to 27 years) with Duchenne muscular dystrophy (Duchenne or DMD) at the 30th Annual Congress of the World Muscle Society in Vienna, Austria. "Satellos's 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 chief executive officer 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. 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-per-cent mean improvement in maximum grip strength was observed in the dominant hand and 97.9-per-cent mean improvement in the non-dominant hand, representing an approximate doubling of grip strength from approximately two kilograms to approximately four kg. These improvements are inconsistent with published natural history and were correlated with higher drug concentrations on day 15 and higher baseline creatine (a surrogate for increased muscle mass), which the company believes indicates its drug is having the desired impact on muscle. Furthermore, participants exhibited a 5.8-per-cent mean improvement of predicted forced vital capacity; such an increase is also inconsistent with natural history with declines about 5 per cent 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. "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, MD, 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." The five adult patients, aged 20 to 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 to 25 years. The primary end points 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 end points include the effect of SAT-3247 on fat fraction, and impact on muscle force and function. Once participants have completed their three-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 end points will evaluate safety and tolerability of SAT-3247 and effect on muscle force. Secondary end points 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. Full details from the poster presentations will be available on the events and presentations page of the Satellos website. About SAT-3247 SAT-3247 is a proprietary, oral, small molecule drug being developed by Satellos as a novel treatment to regenerate skeletal muscle that is lost in Duchenne muscular dystrophy and other degenerative or injury conditions. Satellos is advancing SAT-3247 as a potential treatment for DMD, independent of dystrophin and regardless of exon mutation status. About Satellos Bioscience Inc. Satellos is a clinical-stage drug development company focused on restoring natural muscle repair and regeneration in degenerative muscle diseases. Through its research, Satellos has developed SAT-3247, a first-of-its-kind, orally administered small molecule drug designed to address deficits in muscle repair and regeneration. SAT-3247 targets AAK1, a key protein that Satellos has identified as capable of replacing the signal normally provided by dystrophin in muscle stem cells to effect repair and regeneration. By restoring this missing dystrophin signal in DMD, SAT-3247 enables muscle stem cells to divide properly and more efficiently, promoting natural muscle repair and regeneration. SAT-3247 is currently in clinical development as a potential disease-modifying treatment initially for DMD. Satellos also is leveraging its proprietary discovery platform MyoReGenX to identify additional muscle diseases or injury conditions where restoring muscle repair and regeneration may have therapeutic benefit and represent future clinical development opportunities. We seek Safe Harbor.