BioBlast Jumps On Muscular Dystrophy Drug Results

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NOTE: Dr. Haya Lorberboum-Galski, Chairperson of The Institute for Medical Research Israel-Canada (IMRIC) at The Hebrew University of Jerusalem, was a key member of the research team behind this work.

Globes header - Bioblast

The Israeli drug developer sees potential initial sales of over $1 billion for Cabaletta, which could be marketed by 2017.

BioBlast CEO Colin FosterBioBlast Pharma Ltd.(Nasdaq:ORPN) announced promising interim results on Tuesday from its Phase II study of its leading treatment, Cabaletta, with 25 patients suffering from oculopharyngeal muscular dystrophy (OPMD). Due to the success of the open label HOPEMD trial, the Israeli drug developer seeks to fast track a Phase III study in 2016.

Cabaletta showed statistically significant improvements for patients in several tests, while also hitting its marks for safety and tolerability. The study was conducted at two research centers -- Hadassah-Hebrew University Medical Center in Jerusalem and Montreal Neurological Institute at McGill University in Canada.

“The interim results for OPMD, specifically with respect to dysphagia and muscle strength and function, give us insight into the potential of Cabaletta for use in other protein aggregation-related diseases, such as spinocerebellar ataxia type 3 (SCA3 or Machado Joseph disease), another devastating hereditary disease in which we are planning a pivotal Phase 3 study in the US and EU,” said CEO Colin Foster.

There is currently no approved treatment for OPMD, a rare muscle-wasting disease characterized by swallowing difficulties. "There is a critical unmet need for patients with this rare disorder,” said Peter L. Saltonstall, the leader of the National Organization for Rare Disorders. "We are grateful to BioBlast and to the physicians and patients who have committed their time and resources to this important clinical trial."

BioBlast believes it may be able to get the treatment on the market by 2017, with initial sales potential of more than $1 billion. In pre-market trading on Nasdaq, BioBlast share price was up by over 20%.

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