Bio Blast Pharma reports promising data on muscle wasting drug
Bio Blast Pharma reports promising data on muscle wasting drug
Oct 27 (Reuters) - Drug developer Bio Blast Pharma Ltd said its drug showed signs of being effective in treating patients with a rare muscle wasting disorder, according to interim results from a mid-stage study.
The company said its lead drug in development, Cabaletta, hit the main goal of proving to be safe and tolerable in the study on patient suffering from oculopharyngeal muscular dystrophy.
Bio Blast said the drug, however, also hit multiple efficacy endpoints during the trial, which was not among the main goals.
Oculopharyngeal muscular dystrophy is a rare disease where patients develop swallowing difficulties, which could lead to death in severe cases.
(Reporting by Vidya L Nathan in Bengaluru; Editing by Savio D’Souza).