BioBlast Pharma announced promising results from a Phase 2 clinical study of its lead experimental drug to treat patients with rare muscle wasting disorder.
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.
BioBlast said the drug improved patients' muscle function while also reducing difficulty in swallowing.
Oculopharyngeal muscular dystrophy is a rare disease where patients develop swallowing difficulties, which could lead to death in severe cases.
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.