Satellos Bioscience, announced that the first patient has been dosed in the open-label, long-term follow-up study (LT-001) of SAT-3247 in adult males with Duchenne muscular dystrophy (Duchenne, or DMD). SAT-3247 is a novel oral small molecule therapeutic candidate designed to repair and regenerate muscle that is lost in people living with Duchenne.
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The study will evaluate longer-term safety, changes in muscle composition by MRI, and functional outcomes in participants with Duchenne who previously completed Part D of the Phase 1b 28-day study. The design allows patient assessments and reporting every three months during treatment. The company is working to expand the protocol to include up to 10 new participants in Australia and plans to open the study in the U.S. — both subject to regulatory and clinical site approvals.
“Dosing the first patient in LT-001 marks an important milestone as we work to unlock the long-term potential of SAT-3247 for people living with Duchenne,” said Satellos Co-founder and CEO Frank Gleeson. “If we find the benefits observed in the initial Phase 1b adult study are strengthened and maintained in our long-term follow up, we will be another step closer to the possibility of a therapy that can reverse the effects of Duchenne — thereby making a real and lasting difference for patients and families living with this devastating disease.”
The trial is underway at St Vincent’s Hospital in Melbourne, Australia, in collaboration with clinical neurologist and neurophysiologist Gayatri Jain, MD. “Seeing the first patient enrolled in LT-001 is a hopeful moment for the Duchenne community,” said Dr. Jain. “We look forward to evaluating long-term safety of SAT-3247, as well as how SAT-3247 may sustain functional improvements and positively affect muscle composition over time, providing valuable insights into its potential to improve patients’ daily lives.”
Read More: Clinical Trials for Duchenne (List of All Researches)
