BioBlast Pharma (NASDAQ:ORPN) has submitted its Phase II interim data for Cabaletta (intravenous trehalose) in opculopharyngeal muscular dystrophy (OPMD) to the American Academy of Neurology (AAN) annual meeting and the 5th International Congress of Myology for presentation, said CEO Colin Foster. The AAN conference takes place 1521 April 2016 in Vancouver, Canada whilst Myology 2016 takes place 1418 March 2016 in Lyon, France.
The company announced positive interim results for Cabaletta on 27 October. Ten out of 12 patients (83.3%) were observed to stabilize or improve on an efficacy endpoint, the Penetration Aspiration Score, related to dysphagia (difficulty in swallowing), as measured by Video Fluoroscopy (VFSPAS). There was a statistically significant improvement in the timed drinking test, a swallowing qualityoflife symptom score, as measured by the SWALQOL questionnaire. Patients also showed a statistically significant improvement in their lower extremities’ muscle strength versus baseline, and demonstrated numerical improvement in other muscle strength and function tests.
The company is now in the process of planning a Phase III trial for early 2016 contingent on FDA discussions , said Foster. There will be three sites in the Canada as well as six in the US enrolling around 80 patients in total, he noted. The primary endpoint will be Penetration Aspiration Score as measured by VFSPAS, he added.
At the end of 2Q15, BioBlast had USD 26m in cash which should see the company through the Phase III trial, said Foster. In the future the company may look to business development partnerships or a capital raise in order to develop its other drug candidates, he said, declining to comment further on these points. BioBlast has recently hired a chief corporate development officer to assist the company with M&A and business development, Foster added.
OPMD is an inherited myopathy characterized by dysphagia and the loss of muscle strength, and weakness in multiple muscles of the body. Symptoms generally appear in midlife and get worse over time. 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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Cabaletta is a chemical chaperone that protects against pathological processes in cells. It has been shown to reduce pathological aggregation of proteins within cells in several diseases associated with abnormal cellularprotein aggregation as well as acting as an autophagy enhancer, according to the company website. BioBlast is also investigating Cabaletta in spinocerebellar ataxia type 3, said Foster. A Phase II trial is underway in Israel and is fully enrolled with an expected December completion date, he added.
The company is opportunistic regarding inlicensing additional orphan disease drug candidates that fit strategically with the company’s current platforms, said Foster. A candidate with clinical proof of concept would be preferred, however, if the data is good enough, preclinical candidates may be considered, he added.
BioBlast’s market cap is USD 70.44m.
by Hamish McDougall in London