Satellos Bioscience Reports Promising Safety, Tolerability, and Functional Benefits of SAT-3247 in Adults with Duchenne Muscular Dystrophy
Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF), a clinical-stage biotechnology company focused on developing transformative therapies for degenerative muscle diseases, has unveiled new clinical data highlighting the safety, tolerability, and initial functional impact of its investigational oral therapy, SAT-3247, in adults with Duchenne muscular dystrophy (Duchenne or DMD). The findings were presented at the 30th Annual Congress of the World Muscle Society in Vienna, Austria.
Duchenne muscular dystrophy is a rare, X-linked genetic disorder characterized by progressive muscle degeneration and weakness caused by mutations in the dystrophin gene. Current treatment options are limited and primarily aim to slow disease progression rather than restore muscle function. Satellos’ SAT-3247 represents a novel therapeutic strategy, designed to address the underlying pathophysiology of Duchenne by reactivating impaired muscle regeneration processes.
CEO Perspective and Clinical Context
Frank Gleeson, co-founder and CEO of Satellos, commented on the findings, stating, “Satellos’ new and updated clinical results from the 28-day clinical study in adults with Duchenne provide an important validation of SAT-3247’s potential to be a safe and clinically meaningful treatment. We are excited and confident in progressing into our next phase of clinical studies with the objective of demonstrating the transformative potential of SAT-3247 to the Duchenne community.”
These data provide critical insights not only into the safety profile of SAT-3247 but also into its early functional impact in adults—a population often underrepresented in Duchenne clinical trials. By focusing on adults aged 20 to 27 years, the study explores SAT-3247’s potential in patients with more advanced disease, offering valuable evidence to support broader clinical development.
Study Design and Key Findings
The data presented at the World Muscle Society Congress stem from Satellos’ Phase 1a/b trial evaluating SAT-3247 over a 28-day treatment period. The study assessed safety, tolerability, pharmacokinetics, and exploratory measures of early efficacy. Importantly, SAT-3247 demonstrated a favorable safety and tolerability profile across the study population. No drug-related adverse events of moderate severity or higher were observed, and no dose-limiting toxicities occurred.
Pharmacokinetic analyses confirmed a desirable profile, with exposure levels consistent with expected therapeutic activity. Beyond safety, the study included exploratory measures to evaluate functional outcomes, providing early signals that SAT-3247 may positively influence muscle performance.
Grip Strength Improvements Highlight Functional Potential
One of the most striking findings from the study was the impact of SAT-3247 on grip strength, a key functional measure in Duchenne muscular dystrophy. Adults treated with SAT-3247 demonstrated substantial improvements in grip strength over the 28-day study period, far exceeding expectations based on natural history data. Specifically, the mean improvement in maximum grip strength was 118.6% in the dominant hand and 97.9% in the non-dominant hand. This represents an approximate doubling of grip strength from around 2 kg to nearly 4 kg, a magnitude of improvement inconsistent with published natural history data for adults in this age range.
Further analyses revealed that these improvements correlated with higher drug concentrations on Day 15 and higher baseline creatinine levels, which serve as a surrogate for muscle mass. These correlations suggest that SAT-3247 is actively engaging its intended target pathways and driving measurable functional benefits in skeletal muscle.
Pulmonary Function Outcomes Suggest Broader Physiological Effects
In addition to gains in grip strength, participants exhibited a mean 5.8% improvement in predicted forced vital capacity (FVC), an important measure of respiratory function. This increase contrasts with the typical natural history of adults with Duchenne, in whom annual declines of approximately 5% in FVC are commonly observed. While all other exploratory measures of muscle function remained stable over the study period, the improvements in both grip strength and respiratory function provide encouraging early evidence of SAT-3247’s potential to positively influence multiple aspects of disease progression.
Mechanistic Rationale: Rebooting Impaired Muscle Regeneration
Wildon Farwell, M.D., chief medical officer of Satellos, emphasized the underlying scientific rationale for SAT-3247. “Satellos’ scientific co-founders were the first to recognize that the body’s innate process of muscle regeneration is severely impaired in Duchenne. They invented SAT-3247 with the aim of rebooting this impaired regeneration process, so that the body can once again repair and rebuild muscle,” said Dr. Farwell.
He added, “These early signs of efficacy in adults with more advanced disease are incredibly encouraging and support expanding our clinical program to the broader Duchenne community. SAT-3247 has been designed to be a convenient, oral therapy with the potential to improve outcomes regardless of an individual’s specific dystrophin mutation.”
This mechanism of action represents a significant departure from existing therapies, which often target specific genetic mutations or focus on managing symptoms. By aiming to restore the body’s natural muscle repair processes, SAT-3247 could provide a more universally applicable therapeutic approach for individuals with Duchenne.
Next Steps: Open-Label and Phase 2 Studies
Following the Phase 1b trial, the five adult participants aged 20 to 27 years are now being invited to enroll in an 11-month open-label follow-up study of SAT-3247. This extension study will also enroll additional males with DMD aged 16 to 25 years. The primary objectives of the open-label study are to evaluate long-term safety and tolerability, as well as to assess SAT-3247’s effect on fat fraction in the biceps brachii muscle—a key indicator of muscle quality and degeneration. Secondary endpoints will examine the impact on overall muscle force and functional performance.
Initial interim results from this follow-up study will be provided once participants complete their three-month visit, offering early insights into the sustainability of SAT-3247’s effects and its potential benefits over extended treatment periods.
In parallel, Satellos is advancing plans for a Phase 2 randomized, double-blind, placebo-controlled, global proof-of-concept study in ambulatory children with Duchenne muscular dystrophy. The Phase 2 study will further evaluate SAT-3247’s safety, tolerability, and functional impact, with primary endpoints focused on muscle force and secondary endpoints assessing muscle quality, regeneration, and overall function. Regulatory submissions for this study have been filed in the United States and internationally, reflecting Satellos’ commitment to rapid clinical advancement and global accessibility of SAT-3247.
Implications for the Duchenne Community
The data presented at the World Muscle Society Congress provide a critical early signal that SAT-3247 may offer meaningful clinical benefits for individuals with Duchenne muscular dystrophy. In adults with advanced disease, improvements in grip strength and respiratory function, coupled with a favorable safety profile, represent a significant achievement for a therapy in early-stage development.
By demonstrating early functional gains and the potential to enhance muscle regeneration, SAT-3247 may help fill an important unmet need in the Duchenne community. If these effects are confirmed in larger, longer-term studies, SAT-3247 could emerge as a transformative therapy capable of improving both quality of life and disease trajectory for patients across the age spectrum.
Scientific References
- Hogrel, J.-Y., et al. 2020. “Normalized grip strength is a sensitive outcome measure through all stages of Duchenne muscular dystrophy.” Journal of Neurology, 267(7):2022–2028. doi:10.1007/s00415-020-09800-9.
- McDonald, C.M., et al. 2018. “Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids.” Neuromuscular Disorders, 28(11):897–909. doi:10.1016/j.nmd.2018.07.004.
About Satellos Bioscience
Satellos Bioscience Inc. is a clinical-stage biotechnology company dedicated to developing medicines that improve the lives of patients affected by degenerative muscle diseases. Through its proprietary SAT-3247 platform, Satellos aims to restore the body’s ability to repair and rebuild muscle, addressing a critical unmet need in Duchenne muscular dystrophy and potentially other muscle-wasting disorders. The company’s mission is to provide therapies that are safe, effective, and accessible to patients worldwide.
For more information about Satellos Bioscience, upcoming clinical studies, and access to full details of the poster presentations, please visit the company’s Events and Presentations page.
