Satellos has announced promising results from their Phase 1b study of SAT-3247 in treating five adult males with Duchenne muscular dystrophy (DMD). People with Duchenne are lacking in dystrophin, which provides the structural support to muscle fibers, and also helps tell muscle stem cells to make new or repair existing damaged muscle. SAT-3247 is an oral drug designed to target the root cause of muscle loss in degenerative diseases, initially Duchenne, by reactivating the disrupted muscle regeneration process. In studies of animals with Duchenne, SAT-3247 resulted in increases in muscle cells and strength. In the Phase 1 portion of this Phase 1/1b study, SAT-3247 was found to be safe in people who did not have Duchenne. Now, it has been found to be safe and well tolerated in people with Duchenne and resulted in doubled grip strength, which could be an important measure during future studies, including the anticipated global Phase 2 study which will be active in Canada.
“… We are encouraged by these initial data — particularly, the apparent trend of improved grip strength. Improvements in muscle strength have consistently been an early signal of a possible drug effect in our preclinical studies, where treatment with SAT-3247 led to notable increases in muscle force in both rodent and canine models of DMD. We believe the findings from this Phase 1b study support our plan to advance SAT-3247 into a placebo-controlled Phase 2 trial.”
Frank Gleeson, Satellos Co-founder and Chief Executive Officer
SAT-3247 presents a new treatment to address muscle loss in people with DMD, to support new and repair damaged muscle caused by the absence of functional dystrophin. Satellos believes the Phase 1b findings support advancing SAT-3247 into a global Phase 2 study in pediatric patients, for which global regulatory submissions are planned for the third quarter of 2025.
What is SAT-3247?
SAT-3247 is an oral, small molecule drug candidate designed to target the root cause of muscle loss in degenerative diseases, initially in Duchenne. SAT-3247 presents a novel mechanism of action to restore impaired muscle regeneration caused by the absence of functional dystrophin.
Defeat Duchenne Canada is pleased to be currently funding the work of Dr. Mike Rudnicki, who is the Director of the Regenerative Medicine Program at the Ottawa Research Institute and is also the Chief Scientific Officer of Satellos Bioscience Inc.
For more information, please read the full press release below.
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: