Satellos’ proprietary MyoReGenX™ platform identified a protein kinase (codenamed “K9”) as a potential drug target to modulate polarity in muscle stem cells. Using a known inhibitor of this kinase target as a reference compound for generating Proof of Concept to guide future drug development efforts with its own compounds, Satellos treated Mdx mice, a gold-standard experimental model for studying Duchenne muscular dystrophy, for a period of two weeks.
Drug-treated muscles were larger in size than untreated control muscles by an average of 40% and displayed about a 25% increase in ability to generate force, approaching levels seen in normal mice.
“To the best of our knowledge, these pre-clinical results are unprecedented for a small molecule therapeutic approach to treat Duchenne as they represent the first time a small molecule has induced these levels of muscle growth that translate to clear functional benefit – and so rapidly. We are so energized by seeing such an effect on regeneration because of the potential it represents for a future treatment to regenerate functional muscle in patients.”Frank Gleeson, President and CEO of Satellos
Defeat Duchenne Canada funded researcher and Satellos’ co-founder and Chief Scientific Officer, Dr. Michael Rudnicki, added:
“We believe these results continue to solidify our mechanistic approach of targeting the restoration of muscle regeneration through modulating muscle stem cell polarity.”
Satellos believes that a dysfunction in the normal process of stem cell polarity in response to muscle damage represents a previously unrecognized root cause of Duchenne. The goal of correcting polarity in Duchenne is to restore the body’s innate ability to regenerate muscle in response to the ongoing damage experienced by people living with Duchenne. SAT-3153 has been designated by Satellos as its lead drug candidate, and the Company is pursuing pre-IND development activities.