Satellos Bioscience has dosed the first subject with Duchenne muscular dystrophy (DMD) in a Phase Ib trial of its oral small molecule drug candidate, SAT-3247.
The study aims to evaluate the pharmacokinetics (PK) of the therapy in up to ten adult subjects with genetically confirmed DMD over 28 days.
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The Phase I clinical trial of SAT-3247 is structured in two components. Initially, 72 healthy adult volunteers participated in a blinded, randomised, placebo-controlled study.
They were divided into various cohorts to determine the safety and PK profile of the drug. The trial has now progressed to the Phase Ib component, focusing on DMD patients.
The clinical data reported by the company has so far revealed that healthy volunteers who received the therapy across single-ascending dose (SAD) cohorts and the first cohort of the multiple-ascending dose (MAD) have reported no adverse side effects.
The safety findings are notable for the absence of drug-related adverse events, abnormal clinical labs, vitals, ECG, and physical exam results.
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By GlobalDataFurthermore, the PK profile of the therapy observed in healthy volunteers has effectively translated from pre-clinical models to human subjects.
Satellos Bioscience co-founder and CEO Frank Gleeson said: “The dosing of our first DMD participant is a major milestone in evaluating SAT-3247’s potential benefit for DMD patients.
“We are very pleased to have safely completed the full enrolment and dosing of all five cohorts in the SAD study and the first two MAD cohorts.
“This reinforces our view that SAT-3247 has the potential to be a safe and tolerable, once-daily, oral treatment option for DMD patients. We look forward to continuing to advance SAT-3247 as a potential disease-modifying therapy for degenerative muscle conditions.”
SAT-3247 aims to reinstate the regeneration of skeletal muscle in DMD.
The company is leveraging its discovery platform, MyoReGenX, to detect other degenerative muscle diseases that may benefit from therapeutic intervention and expand future clinical development.
