- Phase 1b data shows treatment with SAT-3247 was safe and well-tolerated
- Pharmacokinetic (PK) profile of SAT-3247 translated as expected to Duchenne Muscular Dystrophy (DMD) patients taking concurrent steroids
- Potential trend to improved grip strength observed, with average strength across the study participants doubling from ~2kg to ~4kg
- Study participants will have the option to enroll into an 11-month, long-term follow-up study
- Satellos plans to advance SAT-3247 into a placebo-controlled Phase 2 trial following additional data analysis and regulatory engagement
TORONTO, Canada — Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF), a biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, today announced promising Phase 1b data, in an open-label study treating five adult male DMD patients, ages 20 – 27, demonstrating early signs that SAT-3247 may have the potential to affect grip strength, which could represent a clinically meaningful measure for patients with DMD.
“We have gained valuable insights from this study of SAT-3247, and we are deeply grateful to the participants,” said Satellos Co-founder and Chief Executive Officer Frank Gleeson. “Given the short 28-day treatment window, the severity and variability of disease in this population, who have limited remaining muscle, we are encouraged by these initial data — particularly, the apparent trend of improved grip strength. Improvements in muscle strength have consistently been an early signal of a possible drug effect in our preclinical studies, where treatment with SAT-3247 led to notable increases in muscle force in both rodent and canine models of DMD. We believe the findings from this Phase 1b study support our plan to advance SAT-3247 into a placebo-controlled Phase 2 trial. We look forward to engaging with regulators and sharing more about our next steps.”
SAT-3247 is designed to address progressive muscle loss in DMD patients by aiming to restore regeneration in response to damage. The Phase 1b open-label trial of SAT-3247 was conducted in adults with DMD, with the primary endpoint being safety and tolerability. As additional endpoints, the study evaluated a number of exploratory measurements of a possible drug effect. The study focused on adults, a demographic representing individuals who have reached the later stages of the disease, where clinical management becomes increasingly complex and long-term survival is less common. All participants were maintained on their prescribed daily course of steroids during the study.
Summary of clinical results:
- SAT-3247 appeared to be safe and well tolerated in all study participants.
- The PK profile of SAT-3247 translated as expected to DMD patients on steroids, an important objective of the study.
- Grip strength was measured using the standardized MyoGrip measurement device. Across all study participants, an average doubling of strength from ~2kg to ~4kg was observed.
- Study participants appeared to remain stable in other exploratory measurement areas.
Satellos plans to begin an 11-month follow-up study, which has already received ethics committee approval in Australia. First-patient dosing is expected in Q3 2025, pending qualification of the clinical site. The study will incorporate MRI imaging to assess possible changes in muscle and will measure grip strength every three months to evaluate whether the observed improvements continue, as well as additional functional and biomarker measurements.
Satellos believes the Phase 1b findings support advancing SAT-3247 into a global, placebo-controlled Phase 2 proof-of-concept study in pediatric patients, for which global regulatory submissions are planned for the third quarter of 2025.
About Phase 1 DMD Trial with SAT-3247
The Phase 1 clinical trial of SAT-3247 was comprised of two components. In the first component, Phase 1a, 72 healthy volunteers were enrolled in a blinded, randomized, placebo-controlled, staggered, parallel design study to assess the safety and pharmacokinetic properties of SAT-3247. Participants were randomized across five SAD cohorts, four MAD cohorts, and one food effect cohort. In the second component, the Phase 1b portion of the trial, five adult volunteers with genetically confirmed DMD, all of whom were receiving their prescribed steroid treatments, were enrolled in a 28-day open-label single-dose cohort study to assess safety and pharmacokinetic properties and assess a range of exploratory measures including potential pharmacodynamic markers.
About SAT-3247
SAT-3247 is a proprietary, oral, small molecule drug being developed by Satellos as a novel treatment to regenerate skeletal muscle that is lost in Duchenne muscular dystrophy and other degenerative or injury conditions. Satellos is advancing SAT-3247 as a potential treatment for DMD, independent of dystrophin and regardless of exon mutation status.
About Satellos Bioscience Inc.
Satellos is a clinical-stage drug development company focused on restoring natural muscle repair and regeneration in degenerative muscle diseases. Through its research, Satellos has developed SAT-3247, a first-of-its-kind, orally administered small molecule drug designed to address deficits in muscle repair and regeneration. SAT-3247 targets AAK1, a key protein that Satellos has identified as capable of replacing the signal normally provided by dystrophin in muscle stem cells to effect repair and regeneration. By restoring this missing dystrophin signal in DMD, SAT-3247 enables muscle stem cells to divide properly and more efficiently, promoting natural muscle repair and regeneration. SAT-3247 is currently in clinical development as a potential disease-modifying treatment initially for DMD. Satellos also is leveraging its proprietary discovery platform MyoReGenX™ to identify additional muscle diseases or injury conditions where restoring muscle repair and regeneration may have therapeutic benefit and represent future clinical development opportunities. For more information, visit www.satellos.com.
Contacts
Investors: Liz Williams, CFO, [email protected]
Media: Emily Williams, Senior Director, Communications, [email protected]
Clinical Trial Info: [email protected]