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Satellos Bioscience Inc. Reports Six-Month Interim Trailhead Data for Sat-3247 in Adults with Duchenne Muscular Dystrophy
Satellos Bioscience Inc. announced six-month interim data from TRAILHEAD evaluating its investigational drug candidate SAT-3247 in adults living with Duchenne muscular dystrophy. The results, in four adults aged 21-28 who had previously completed the Phase 1a/b CL-101 study, showed reduced muscle fat fraction, increased total effort, stable strength, lower CK, and a safety and tolerability profile consistent with previously reported data. All four participants showed a decline in fat fraction as measured by MRI, with a mean improvement of 3.7% from 49.7% at TRAILHEAD baseline to 46.0% at month 6, suggesting improved muscle composition. All four participants showed an increase in TE99C, a measure of maximum effort in upper limbs, with a mean improvement of approximately 34% from a baseline of 16.1 joules/kg to 21.6 joules/kg at TRAILHEAD month 6, suggesting enhanced upper limb activity. Near doubling of handgrip strength measured during the 28-day CL-101 study was maintained through month 6 of TRAILHEAD. Mean creatine kinase (CK), a biomarker of muscle damage, declined 38% from baseline through month 6 of TRAILHEAD. SAT-3247 was well tolerated; the safety profile was consistent with previously reported data, with no serious treatment emergent adverse events (TEAEs), no TEAEs leading to withdrawal or discontinuation, and 100% compliance over an average of 186 days of drug exposure. All four participants showed a decline in muscle fat fraction as measured by MRI, with a mean improvement of 3.7% from 49.7% at TRAILHEAD baseline to 46.0% at month 6, suggesting improved muscle composition. All four participants showed an increase in TE99C, a measure of maximum effort in the upper limbs assessed using SYSNAV Syde®, a wearable device widely used in clinical trials to track patient movement in real-world settings, with a mean improvement of approximately 34% from a CL-101 baseline of 16.1 joules/kg to 21.6 joules/kg at TRAILHEAD month 6, suggesting enhanced upper limb activity. Across all measures of upper extremity strength (muscle force), including handgrip and handheld dynamometry of the elbow and shoulder, participants demonstrated stability through the end of six months of treatment; for clarity, the near-doubling of handgrip strength reported during the 28-day CL-101 study was maintained through to the end of month 6 of the TRAILHEAD follow-up period. Mean CK declined 38% from CL-101 baseline (2130 u/l) through month 6 in TRAILHEAD (1315 u/l). Mean PUL2.0 (Performance of the Upper Limb 2.0) increased by one point in two participants and remained stable in two participants from TRAILHEAD baseline through month 6. In the natural history of DMD, upper limb function is generally expected to decline over time. Mean PedsQL-MFS scores, a patient reported quality-of-life measure designed to quantify fatigue and adapted for adults, increased 6.94 points from CL-101 baseline (71.53 pts.) to month 6 in TRAILHEAD (78.47 pts.). The TRAILHEAD study is expected to enroll up to 30 male participants in Australia and the United States aged =16 years with a definitive diagnosis of DMD and a confirmed mutation in the DMD gene. Participants will receive SAT-3247 at a dose of 60 mg administered orally using a 5-days-on/2-days-off (weekday) dosing regimen for up to 12 months. The primary objectives include evaluation of long-term safety and tolerability of SAT-3247, as well as changes in fat fraction of the biceps brachii muscle measured by magnetic resonance imaging (MRI) following 12 months of treatment. Key secondary and exploratory objectives include quantitative assessments of muscle composition via MRI, muscle force by dynamometry, physical activity and function via limb sensors and PUL 2.0, pulmonary function, participant and caregiver quality of life, health economic impact, and proteomic biomarkers. BASECAMP is a global, Phase 2, randomized, double-blind, placebo-controlled, proof-of-concept clinical study evaluating SAT-3247 in ambulatory boys with DMD aged 7 to 9 years. The study includes an initial 12-week randomized, double-blind, placebo-controlled treatment period, followed by a 36-week active-treatment period in which all participants receive SAT-3247. The primary objectives are to evaluate the safety and tolerability of SAT-3247, along with its effects on muscle function using standardized dynamometry-based assessments of muscle force. Secondary and exploratory endpoints include assessments of muscle quality, functional outcomes, and biomarkers of muscle regeneration, including Satellos' proprietary Regenerative Index. BASECAMP is being conducted across multiple clinical sites in North America, Europe, the United Kingdom, and other international regions, and is designed to provide proof-of-concept data supporting the potential of SAT-3247 as a potentially disease-modifying therapy for Duchenne muscular dystrophy. SAT-3247 is an investigational, proprietary, oral, small molecule drug candidate being developed by Satellos as a novel approach to regenerating skeletal muscle lost in DMD and other degenerative muscle diseases or injury conditions. Satellos is advancing SAT-3247 as a potential treatment for DMD that is independent of dystrophin regardless of exon mutation status, with ongoing Phase 2 clinical studies, including TRAILHEAD, an open-label study in adult participants, and BASECAMP, a global, randomized, placebo-controlled study in pediatric participants. SAT-3247 targets AAK1, a protein that is a key regulator of the body’s natural muscle repair and regeneration biology, which Satellos discovered is disrupted in DMD and other degenerative conditions. By inhibiting AAK1, SAT-3247 is designed to re-establish a critical biochemical signal needed to guide this process, in a dystrophin-independent manner.