Catabasis Pharmaceuticals Inc (NASDAQ:CATB) surged on Tuesday after the biopharma reported positive results from a mid-stage study of its Duchenne muscular dystrophy treatment.
In the trial, the company’s edasalonexent tablet helped to preserve muscle function and sustained “disease-modifying effects” in boys with DMD, while no side effects were observed.
Consistent improvements in all assessments of muscle function were observed after more than a year of treatment compared to the rate of change in the pre-specified control period.
DMD is a rare muscle-wasting disease that almost exclusively affects only young boys, for which there is currently no known cure.
Importantly, Catabasis is looking to treat all DMD sufferers with edasalonexent rather than just a subset of patients.
A pivotal phase III trial is scheduled to get underway in the first half of this year, with top-line data expected in 2020.
“We are thrilled to see this preservation of muscle function and substantial slowing of disease progression in boys following more than a year of edasalonexent treatment. This effect has the potential to be extremely impactful for boys affected by Duchenne,” said chief executive Jill Milne.
“Building on the results previously reported for edasalonexent treatment in patients up to 36 weeks, these new data at 48 and 60 weeks show that edasalonexent continued to slow progression of the disease.”
Catabasis shares soared by a quarter to US$1.57 on Tuesday.