Satellos Bioscience Doses First Adult Patient in LT-001, a Long-Term Follow-Up Study of SAT-3247 in Duchenne Muscular Dystroph
Toronto, Ontario — [Insert Date], 2025 — Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a clinical-stage biotechnology company pioneering regenerative therapies for degenerative muscle diseases, today announced that the first adult patient has been dosed in its open-label, long-term follow-up study (LT-001) of SAT-3247 in males with Duchenne muscular dystrophy (Duchenne or DMD).
This milestone marks an important next step in the company’s mission to advance innovative treatments that address the root causes of muscle degeneration in Duchenne, a devastating genetic disease characterized by progressive loss of muscle strength and function.
Advancing a Regenerative Approach to Duchenne
SAT-3247 is a first-in-class, oral small molecule therapeutic candidate designed to stimulate the body’s innate muscle repair and regeneration mechanisms. Unlike existing therapies that primarily aim to slow muscle damage or compensate for dystrophin deficiency, SAT-3247 targets a newly identified biological pathway discovered by Satellos scientists that regulates asymmetric muscle stem cell division. By restoring balance to this regenerative process, SAT-3247 aims to promote the creation of new muscle fibers and repair tissue lost to the disease — offering the potential for meaningful, long-term improvement in muscle structure and function.
Preclinical studies have demonstrated SAT-3247’s ability to restore regenerative muscle repair in animal models of Duchenne. Early clinical data from the company’s Phase 1b study further supported its safety, tolerability, and potential efficacy, setting the stage for this longer-term evaluation.
About the LT-001 Study
The LT-001 study is an open-label, long-term follow-up extension enrolling adult males with Duchenne muscular dystrophy who previously completed Part D of Satellos’ 28-day Phase 1b clinical trial. Participants in LT-001 will receive ongoing treatment with SAT-3247 and undergo comprehensive assessments every three months. These evaluations will focus on long-term safety, changes in muscle composition via magnetic resonance imaging (MRI), and various functional outcomes that measure physical performance and quality of life.
The study’s design reflects Satellos’ commitment to patient-centered clinical development, emphasizing continued access to investigational therapy for participants who completed prior trials while enabling a deeper understanding of SAT-3247’s sustained effects over time.
To broaden the study’s impact and data scope, Satellos plans to expand the LT-001 protocol to include up to ten new participants in Australia. The company also intends to initiate trial sites in the United States, both expansions subject to regulatory review and clinical site approvals.
A Milestone in the Journey Toward Long-Term Treatment
“Dosing the first patient in LT-001 represents a significant milestone for Satellos and for the Duchenne community,” said Frank Gleeson, Co-founder and Chief Executive Officer of Satellos Bioscience. “Our vision has always been to develop a therapy that not only halts the progression of Duchenne but actually restores muscle health and function. This study allows us to explore the durability and depth of SAT-3247’s effects, with the goal of understanding how it might bring lasting benefit to those living with this devastating disease.”
Gleeson added that the transition from short-term to long-term evaluation underscores the Company’s confidence in SAT-3247’s therapeutic potential:
If the improvements we observed in the Phase 1b adult cohort are strengthened and sustained through long-term follow-up, we will be one step closer to demonstrating that muscle regeneration — not just protection — is possible in Duchenne. That could mark a paradigm shift in how this disease is treated, offering hope to patients and families who have long awaited new options.
Clinical Collaboration and Investigator Insights
The LT-001 study is being conducted at St Vincent’s Hospital in Melbourne, Australia, under the direction of Dr. Gayatri Jain, a clinical neurologist and neurophysiologist with extensive experience in neuromuscular disorders and clinical research.
Dr. Jain expressed optimism about the start of the trial and its implications for the Duchenne community:
Seeing the first patient enrolled and dosed in LT-001 is a hopeful and inspiring moment. Duchenne muscular dystrophy remains one of the most challenging genetic diseases to manage, and new therapeutic strategies are urgently needed. We look forward to studying the long-term safety of SAT-3247 and exploring how it may sustain or enhance functional improvements while positively affecting muscle composition over time. The data we gather could provide valuable insights into the drug’s ability to improve patients’ daily lives.
The Broader Vision for SAT-3247 and Muscle Regeneration
Duchenne muscular dystrophy affects approximately one in every 3,500 to 5,000 male births worldwide, leading to progressive muscle weakness, loss of ambulation, and life-threatening complications. Current treatment options primarily focus on managing symptoms or slowing disease progression. Despite decades of research, there remains a critical unmet need for therapies that can restore or replace muscle tissue lost to the disease.
Satellos’ regenerative medicine platform seeks to address that need by targeting the biological mechanism of muscle stem cell polarity and regeneration. By activating this process pharmacologically with SAT-3247, the Company aims to restore continuous muscle repair — even in the absence of dystrophin — thereby complementing or enhancing the benefits of other therapeutic approaches, such as gene or exon-skipping therapies.
With the LT-001 study now underway, Satellos is positioned to gather pivotal data on how SAT-3247 performs over extended treatment durations and across multiple patient cohorts. This research will inform the design of future clinical trials intended to evaluate efficacy in broader Duchenne populations, including younger individuals and those at earlier stages of disease progression.
About Satellos Bioscience Inc.
Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) is a clinical-stage biotechnology company focused on developing life-improving medicines that restore or enhance muscle regeneration in degenerative muscle diseases. Guided by proprietary discoveries in muscle stem cell biology, Satellos is advancing its lead candidate, SAT-3247, for the treatment of Duchenne muscular dystrophy and exploring additional applications across muscle-wasting conditions.
The company’s mission is to unlock the body’s natural capacity for repair by targeting novel biological pathways that govern muscle regeneration. Satellos is headquartered in Toronto, Canada, and operates globally through research and clinical collaborations.
