Bio Blast Pharma Ltd said its lead experimental drug showed signs of being effective in treating patients with a rare muscle wasting disorder, besides also hitting the main goals of safety and tolerability.
The company said the experimental drug, Cabaletta, hit multiple efficacy endpoints during a mid-stage trial on patients suffering from oculopharyngeal muscular dystrophy, according to interim results from study.
Oculopharyngeal muscular dystrophy is a rare disease where patients develop swallowing difficulties, which could lead to death in severe cases.
Bio Blast said the drug improved patients’ muscle function while also reducing difficulty in swallowing.
The company said it would discuss results from this study and a second mid-stage study with the U.S. Food and Drug Administration before it started a late-stage study.
Bio Blast pharma is also testing the drug as a treatment for spinocerebellar ataxia type 3, another genetic condition that affects the nerves in the brain.
(Reporting by Vidya L Nathan in Bengaluru; Editing by Savio D’Souza)