Satellos Bioscience announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA), along with parallel regulatory filings in the United Kingdom, Europe, Serbia and Australia, to initiate a Phase 2 clinical trial of SAT-3247 in ambulatory children with Duchenne muscular dystrophy (Duchenne or DMD).
The planned Phase 2 trial will enroll children with Duchenne in the U.S., following FDA approval, and globally, following country health authority approvals. The three-month randomized, placebo-controlled study will assess safety and tolerability as well as key measures of strength, function, biomarkers, and muscle health. A nine-month open-label extension for this study is also being planned.
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- Regulatory submissions filed in the U.S., UK, Europe, Serbia and Australia
- Three-month randomized placebo-controlled study to assess safety, pharmacokinetics, dose, muscle biopsies, and measures of function in ambulatory children
- First patient expected to be enrolled into the study by the end of 2025
- Long-term extension study approved in Australia with adult patients from the Phase 1b trial; additional adult patients also planned
“Filing our Phase 2 clinical trial submissions in the US and globally marks a major milestone for Satellos in advancing SAT-3247’s potential to treat Duchenne in a novel way,” said Satellos Co-Founder and CEO Frank Gleeson. “Current therapies do not address the fundamental challenge in Duchenne, which we have identified — the body’s impaired muscle-repair process. With SAT-3247, our goal is to re-boot that regenerative cycle with the potential to restore muscle, improve functional outcomes and truly change lives.”
Clinical Trials of SAT-3247
SAT-3247 is an oral small-molecule therapy designed to restore the body’s ability to regenerate muscle, a process that is impaired in Duchenne. In a recently completed Phase 1b study in five adults with Duchenne, SAT-3247 was safe and well tolerated and the pharmacokinetics of SAT-3247 in adults with Duchenne mirrored that of healthy volunteers. Importantly, efficacy was explored, and an approximate doubling of grip strength was observed over 28 days along with a 5% improvement in force vital capacity (lung function). Satellos has initiated an 11-month extension study in Australia to evaluate long-term safety and efficacy among individuals who participated in the Phase 1b trial. Further expansions of this trial are planned. Read More: Satellos SAT-3247 Approach
Please do this quickly.
Verujem u uspeh leka. Nadam se šansi za izlečenje.
Where in Europe is the second phase? Is there one in Austria? How can a child with Duchenne be admitted to the second phase of the Satellos program ?