According to the announcement made by Satellos Bioscience Inc., in an experiment conducted on dogs with DMD, it was seen that dystrophin was produced at near normal levels in the subjects with the therapy called SAT-3247. This development gave hope to families going through stressful days with Duchenne disease.
- Analysis showed improvement in muscle morphology and increased levels of regeneration in muscle groups, including the diaphragm, quadriceps, and calf
- No adverse events and no significant changes in hematology or clinical chemistry were observed after four months of daily, oral treatment with SAT-3247, Satellos’ proprietary small molecule
- Data to be presented at the 2024 World Muscle Society Annual Congress
Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL, OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, today announced a presentation of data at the 29th Annual Congress of the World Muscle Society taking place October 8-12, 2024, in Prague. The presentation will provide an overview of key data collected during the open-label pilot study of SAT-3247 in a canine model of Duchenne muscular dystrophy (“Duchenne” or “DMD”).
An initial summary of the data is presented below. After four months of treatment with SAT-3247:
- Treated animals showed a return to muscle function near healthy, non-diseased, age-matched animal levels when evaluated against historical comparator data.
- The animals showed increases in Regenerative Index (RI) in diaphragm, gastrocnemius medialis (calf), and vastus lateralis (quadriceps). This is in addition to previously reported improvements in RI demonstrated in the bicep femoris.
- There were no adverse events and no significant changes in hematology or clinical chemistry observed.
- Trends to lower creatine kinase levels were noted, a finding that could be consistent with a Duchenne disease-modifying treatment.
“We believe the improvements observed in regeneration and muscle force to close to healthy levels within four months of treatment, when compared to published natural history data and other therapeutic interventions in the canine model of DMD, are groundbreaking,” said Frank Gleeson, Co-founder and CEO of Satellos Bioscience. “The canine model is considered more severe than mouse models, and potentially represents a greater test of a treatment’s utility to translate to humans. We are ecstatic that treatment with our oral small molecule, SAT-3247, showed such dramatic improvements. These highly promising results reinforce our belief in the potential of SAT-3247 to offer a meaningful disease-modifying medicine to help patients living with Duchenne.”
The canine model of muscular dystrophy represents a more severe clinical phenotype and reflects the disease progression observed in people with DMD. In this pilot study, each animal (n=2) was treated for four months with a daily oral dose of SAT-3247. This multiparameter pilot study measured clinical chemistry, hematology, muscle function, and included a large-scale muscle histology workup. From the muscle histology, a calculation was made of the Regenerative Index (RI), a measure of the number of newly regenerated muscle fibers over the number of damaged and dying muscle fibers.
The data from this study will be presented in a poster entitled, “SAT-3247: An Oral Small Molecule Inhibitor Targeting AAK1, a Critical Effector of Skeletal Muscle Regeneration.” The poster is available on the Events & Presentations page of the Satellos website located at ir.satellos.com .
