Satellos Advances Duchenne Pill Trial in Children; Idorsia Raises $81 Million
Satellos Bioscience has submitted an Investigational New Drug (IND) application to the U.S. FDA and parallel submissions in the UK, Europe, Serbia, and Australia to launch a Phase 2 clinical trial of SAT-3247 in ambulatory children with Duchenne muscular dystrophy (DMD)1216.
SAT-3247 is an oral small molecule designed to restore impaired muscle regeneration in DMD; the Phase 2 trial will evaluate safety, pharmacokinetics, dose, muscle biopsies, and functional outcomes over three months in children who are able to walk91116.
The trial will be randomized and placebo-controlled, with a nine-month open-label extension also planned; initial patient enrollment is expected by the end of 2025, pending regulatory approvals1216.
These submissions follow promising safety and tolerability data from the Phase 1 trial of SAT-3247 in adults and healthy volunteers, including functional improvements exceeding the disease’s natural history517.
SAT-3247 has received Rare Pediatric Disease and Orphan Drug designations from the FDA, recognizing its novel therapeutic approach in DMD414.
Satellos has raised additional funds recently through warrant exercises, supporting ongoing clinical development1216.
Idorsia, another biotech company, has successfully raised $81 million (no further details were provided in the search results, and this news is distinct from Satellos).
Sources:
4. https://www.clinicaltrialsarena.com/news/satellos-commence-phase-i-trial-dmd/
5. https://www.biospace.com/press-releases/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
9. https://musculardystrophynews.com/news/satellos-asks-fda-clear-phase-2-trial-dmd-treatment/
11. https://www.contemporarypediatrics.com/view/satellos-submits-sat-3247-ind-to-fda-for-phase-2-study-in-children-with-duchenne-muscular-dystrophy
12. https://www.morningstar.com/news/business-wire/20250922219476/satellos-announces-ind-submission-to-the-us-fda-and-global-regulatory-filings-to-advance-a-phase-2-clinical-trial-of-sat-3247-in-children-with-duchenne-muscular-dystrophy
14. https://synapse.patsnap.com/article/satellos-gets-rare-pediatric-disease-designation-from-fda-for-sat-3247-to-treat-duchenne-muscular-dystrophy
16. https://investingnews.com/satellos-announces-ind-submission-to-the-u-s-fda-and-global-regulatory-filings-to-advance-a-phase-2-clinical-trial-of-sat-3247-in-children-with-duchenne-muscular-dystrophy/
17. https://www.parentprojectmd.org/satellos-announces-positive-functional-data-from-phase-1b-open-label-trial-of-sat-3247-in-adults-with-duchenne/