Satellos’ small-molecule therapy for Duchenne muscular dystrophy (DMD) has shown signals of improving grip strength in adult patients in a Phase I trial.
The Phase Ib portion of the open-label study (NCT06565208) is investigating SAT-3247 in five males aged between 20 and 27 years. This followed a Phase Ia portion of the study in healthy volunteers.
In the Phase Ib portion, an average doubling of grip strength, from 2kg to 4kg, was observed in all patients, as measured by the MyoGrip measurement device, after 28 days. As well as this, the pharmacokinetic PK profile was translated as expected to DMD patients on steroids.
SAT-3247 appeared to be safe and well tolerated in all patients, with the males appearing to remain stable in other exploratory measurement areas.
Due to this data, Satellos plans to begin an 11-month follow-up study for the DMD therapy. This trial has already received ethics committee approval in Australia, with first-patient dosing expected in Q3 2025.
This study will incorporate MRI imaging to assess possible changes in muscle and measure grip strength every three months to evaluate whether the improvements continue, as well as additional functional and biomarker measurements.
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By GlobalDataSatellos is also planning a global, placebo-controlled Phase II proof-of-concept study in paediatric patients, for which global regulatory submissions are planned for Q3 2025.
Satellos CEO Frank Gleeson said: “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 this initial data – particularly, the apparent trend of improved grip strength.”
In March 2025, Satellos announced data from a Phase Ia study of the DMD therapy, which found it to be safe and well tolerated across all healthy volunteer cohorts, as well as displaying PK data consistent with preclinical studies.
SAT-3247 is a small molecule that acts by inhibiting adaptor-associated kinase 1 (AAK1) to modulate muscle stem cell polarity, leading to the repair and regeneration of functional muscle tissue.
Several gene therapies approved for DMD
There are some approved gene therapies for DMD, including Roche and Sarepta’s Elevidys (delandistrogene moxeparvovec-rokl), which gained accelerated approval by the US Food and Drug Administration (FDA) in June 2023. Earlier this month, Sarepta announced data from a cohort of children aged between two and four years.
Sarepta has faced struggles with its therapy, including a recent clinical hold on the therapy after a teenage patient died after the therapy was administered. The hold has since been lifted.
As well as this, Pfizer has pulled its DMD gene therapy fordadistrogene movaparvovec after a Phase III failure and a Phase II patient fatality.
There are also ongoing trials of other DMD therapies, including Capricor’s deramiocel, Regenxbio’s RGX-202 and Avidity Bioscience’s del-zota.
Research by GlobalData estimates that across the seven major markets (7MM: US, France, Germany, Italy, Spain, the UK, and Japan), the DMD treatment market is expected to rise from sales of $2.3bn in 2023 to $5.2bn by 2033. This is mostly driven by Elevidys and Santhera Pharmaceuticals’ Agamree (vamorolone).
