Bio Blast Pharma reports promising data on muscle wasting drug

Reuters

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.

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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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http://www.reuters.com/article/2015/10/27/us-bio-blast-pharma-study-idUSKCN0SL14O20151027#MrVYQdKUvWOoXyXL.99

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