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 is also testing the drug as a treatment for
spinocerebellar ataxia type 3, another genetic condition that
affects the nerves in the brain.
Up to Monday's close of $5.04, the Israeli company's U.S.-listed
shares have lost a little over half their value since they went
public in July last year.
(Reporting by Vidya L Nathan in Bengaluru; Editing by Savio D'Souza)
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