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BioBlast Pharma reports Phase 3 pivotal study of Cabaletta in Oculopharyngeal Muscular Dystrophy patients in the US and Canada.

M2 EQUITYBITES-June 16, 2015-BioBlast Pharma reports Phase 3 pivotal study of Cabaletta in Oculopharyngeal Muscular Dystrophy patients in the US and Canada

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Biotechnology company BioBlast Pharma (NasdaqGM:ORPN) reported on Monday the launch of a Phase 3 pivotal study of Cabaletta (trehalose) in Oculopharyngeal Muscular Dystrophy (OPMD) in the US and Canada.

According to Colin Foster, president and CEO, the company see positive signals in a consequential number of the 25 treated patients currently enrolled in its open label HOPEMD study, with respect to various muscle and other performance tests.

Based upon various in vivo and cellular models of disease, the company has had initially expected to slow the deterioration of the disease over a period of 18 months in the HOPEMD study. Instead, it has seen evidence of improvement in a number of the functional measures within approximately the first six months, said Foster.

The company has decided to initiate a 12 month, placebo-controlled, multi-centre Phase 3 study in the next quarter and will begin enrolling 60 OPMD patients in the USA and Canada into this new trial. It has stopped enrollment in the HOPEMD trial at 25 patients from 70 patients, stated Foster.

New OPMD patients will now be enrolled in the Phase 3 pivotal study. This Phase 3 study will be completed by the end of 2016 and because of its shorter duration will provide results at the same time as the HOPEMD study. This new Phase 3 study will expedite its OPMD programme towards a regulatory filing, according to Foster.

OPMD is a rare inherited myopathy characterized by dysphagia (difficulty in swallowing), the loss of muscle strength and weakness in multiple parts of the body. As the dysphagia becomes more severe, patients become malnourished, lose significant weight, become dehydrated and suffer from repeated incidents of aspiration pneumonia.

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Date:Jun 16, 2015
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