Satellos’ ‘intriguing’ Duchenne data raise hopes ahead of key readout

Satellos Bioscience has shared evidence that its Duchenne muscular dystrophy (DMD) drug candidate is active in adults, leading analysts to predict the molecule “could re-imagine the ... therapeutic landscape” if the biotech replicates the data in children. The adult data come from four people aged 21 to 28 years who completed a phase 1a/b study of SAT-3247. The orally administered small molecule targets AAK1 to address problems with stem cell division caused by dystrophin deficiency, promoting muscle repair and regeneration. Satellos previously shared (PDF) early evidence of improvements in the grip strength and lung capacity of adult patients taking SAT-3247. Toronto-based Satellos used the International Congress on Neuromuscular Diseases to share (PDF) six-month data on the adults. All four patients showed improvements across multiple measures of DMD, including decreased fat fraction (FF) in the elbow flexor. The DMD data impressed Guggenheim Securities analysts, who called the results intriguing and consistent. “In our view, the consistency of effect across this diverse set of endpoints is suggestive of a true drug effect—an impressive feat given the advanced disease state of these patients,” the analysts said in a note to investors. “To the best of our knowledge, other DMD therapies have only been able to slow the rate of fat deposition but did not result in a reduction in FF.” Satellos is most encouraged by the change in muscle FF as measured by MRI scan, Wildon Farwell, M.D., the biotech’s chief medical officer, said on a call with investors to discuss the data. The FF measure is the primary endpoint of the Trailhead phase 2 trial that Satellos is running to assess SAT-3247 in adults. DMD patients are more likely to lose function when FF increases, Farwell said. The adult data are “a good harbinger for the forthcoming Basecamp study” in boys aged 7 to 10 years, Guggenheim analysts said. Satellos expects to complete enrollment in Basecamp in the third quarter and post initial topline data from the study in the fourth quarter. One-year data on the four adult patients are also due in the fourth quarter.