Satellos Announces First Adult Patient Dosed in LT-001, an Open-Label, Long-Term Follow-Up Study of SAT-3247 in Duchenne Muscular Dystrophy

Executive Summary

• First adult patient dosed in the open‑label, long‑term follow‑up study (LT‑001) of SAT‑3247 for Duchenne muscular dystrophy.
• LT‑001 will assess safety, muscle composition by MRI, functional outcomes and serum biomarkers every three months, with results expected starting early 2026 and then quarterly.
• Company plans to expand the protocol to enroll up to 10 additional adult participants in Australia and to open the study in the United States pending regulatory and site approvals.

Key Details

• Study Milestone: First patient dosed in LT‑001, marking the transition from Phase 1b (28‑day) to long‑term follow‑up.
• Therapeutic Candidate: SAT‑3247 – oral small‑molecule designed to repair and regenerate skeletal muscle independent of dystrophin status.
• Target Population: Adult males with Duchenne muscular dystrophy who completed Part D of the Phase 1b study.
• Study Design: Open‑label, 11‑month duration; assessments (safety, MRI muscle composition, functional outcomes, serum biomarkers) every three months.
• Data Release Schedule: Initial 3‑month results anticipated early 2026; subsequent updates on a quarterly basis thereafter.
• Geographic Expansion: Protocol being broadened to enroll up to 10 new adult participants in Australia and to launch the study in the United States, subject to regulatory and clinical site approvals.
• Site & Collaboration: Conducted at St Vincent’s Hospital, Melbourne, Australia, in collaboration with Dr. Gayatri Jain (clinical neurologist/neurophysiologist).
• Management Quote: CEO Frank Gleeson highlighted the dosing as an “important milestone” toward unlocking long‑term potential of SAT‑3247 for Duchenne patients.

Notable Quotes

“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,” – Frank Gleeson, Co‑founder & CEO

“Seeing the first patient enrolled in LT-001 is a hopeful moment for the Duchenne community,” – Dr. Gayatri Jain, Clinical Neurologist/Neurophysiologist

Materiality Assessment: Material – Positive** (initiation of a pivotal long‑term clinical study for a novel therapy targeting an unmet need).