Mesoblast and Satellos advance Duchenne muscular dystrophy treatment trials
Mesoblast received FDA clearance for a registrational DMD trial of remestemcel-L-rknd, while Satellos began dosing boys in the Phase 2 BASECAMP study of SAT-3247. The studies are enrolling children ages 5-9 and 7-9, respectively.
Mesoblast has received U.S. Food and Drug Administration clearance to launch a registrational clinical trial of remestemcel-L-rknd in children with Duchenne muscular dystrophy, and dosing has begun in a Phase 2 trial of SAT-3247 in boys with the disease. The Mesoblast study aims to enroll 76 children ages 5-9, while the BASECAMP study of SAT-3247 aims to enroll 51 boys aged 7-9 who can walk.
Mesoblast said participants in its study will be randomly assigned to receive infusions of the experimental cell therapy or a placebo seven times over the course of nine months. The study’s main goal is to determine whether cell therapy improves performance on the time-to-stand test, a standard measure of motor function. The study will be designed as registrational, meaning that if results are positive, Mesoblast hopes to use the findings as a basis for applying for FDA approval of the therapy.
The FDA’s authorization of the DMD trial was based in part on safety data showing that the therapy was generally well tolerated in children with graft-versus-host disease. The FDA’s decision also took into account preclinical data indicating efficacy in models of DMD. Remestemcel-L-rknd contains mesenchymal stromal cells derived from the bone marrow of healthy donors and is approved by the FDA under the name Ryoncil for children ages 2 months and older with hard-to-treat acute graft-versus-host disease. Mesoblast said it will collaborate with advocacy group Parent Project Muscular Dystrophy to help identify trial participants.
In the separate BASECAMP trial, participants will be randomly assigned to take SAT-3247 at one of two doses, 60 or 120 mg, or a placebo, daily for about three months. The study’s main goals are to evaluate the safety and tolerability of SAT-3247 and to assess the therapy’s effect on muscle strength, as measured by dynamometry. The company said the trial is designed to be pivotal, meaning that positive results could support regulatory approval applications for SAT-3247.
Patients who have received treatments such as exon skippers, corticosteroids, Duvyzat and Elevidys may be eligible for BASECAMP. The study will include sites across several countries, and some sites in the U.S. have begun recruitment. SAT-3247 aims to restore muscle stem cells’ ability to produce new healthy muscle tissue.
Satellos previously conducted a Phase 1 trial that tested SAT-3247 in five adults with DMD aged 20 or older, all of whom had relatively advanced disease at the trial’s start. Results indicated the therapy led to significant improvements in muscle function, most notably a roughly doubling in patients’ grip strength. A long-term study is now underway to collect additional data on the therapy’s use in these adults.