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Bioblast (ORPN) SCA3 Drug Favorable in Phase IIa Study

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Bioblast Pharma Ltd. announced positive results from a phase IIa study on its lead candidate, trehalose, for the treatment of patients with spinocerebellar ataxia type 3 (SCA3).

A look at Bioblast’s price movement during the past one year shows that the company has underperformed the Medical Drugs industry. Specifically, the stock lost 57.9%, while the industry fell 11.2%.

Coming back to the latest news, the study evaluated the safety and tolerability of two doses of trehalose IV (13.5g and 27g) on a weekly basis in SCA3 patients (n=14). The study also assessed the effect of the drug in reducing the rate of clinical decline of the disease.

Results from the study revealed that patients treated with trehalose remained stable according to a well-established clinical evaluation scale. The candidate was generally safe and well tolerated.

We note that trehalose IV (90mg/mL) is also in phase II development for the treatment of oculopharyngeal muscular dystrophy (OPMD).

Trehalose enjoys orphan drug designation both in the U.S. and the EU for the treatment of SCA3 and OPMD. The company has also received a U.S. patent for the administration of trehalose for the treatment of SCA3 and OPMD, which will expire in 2033.

Meanwhile, Bioblast is conducting analyses to determine the next orphan disease to evaluate trehalose. The company expects to reach a decision in the first half of 2017.

With no approved of treatment option for SCA3 and OPMD at present, the company has significant potential in this market. Successfully development followed by potential approval will significantly boost Bioblast.

We remind investors that a couple of days ago, Benitec Biopharma Limited’s shares received a boost after the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) granted orphan drug designation to its OPMD candidate, BB-301.

Going forward, investors are expected to remain focused on further updates pertaining to trehalose by Bioblast.

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Currently, Bioblast carries a Zacks rank #2 (Buy).

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