Bio Blast Pharma Ltd (NASDAQ:ORPN), a clinical-stage biotechnology company developing meaningful therapies for patients with rare and ultra-rare genetic diseases, today provided an update on its research and development programs.

Recently, the Company announced positive, open-label, interim HOPEMD Phase 2 clinical data for Cabaletta in oculopharyngeal muscular dystrophy (OPMD), in which proof-of-concept efficacy signals were observed in patients early in treatment. Based on the positive data from the HOPEMD study, the Company intends to conduct a double-blind, placebo-controlled, Phase 2b study to augment the existing data package for Cabaletta and to further guide the design of a future pivotal Phase 3 study. Therefore, at this time, and in advance of discussions with regulatory authorities, the Company does not anticipate commencing a Phase 3 Cabaletta clinical study in 2016 in either OPMD or spinocerebellar ataxia type 3 (SCA3) patients.

“Our primary objective is to develop a robust clinical program and regulatory dossier for Cabaletta,” said Colin Foster, President and Chief Executive Officer of the Company. “With the accumulating knowledge from our open-label, interim HOPEMD Phase 2 clinical study, we believe that a double-blind, placebo-controlled Phase 2b clinical study will enhance our clinical development program for Cabaletta.”

Following the conclusion of an ongoing assessment, the Company anticipates providing updated guidance on its overall R&D strategy in early 2016. (Original Source)

Shares of Bio Blast Pharma closed yesterday at $4.48, down $0.11 or -2.50%. ORPN has a 1-year high of $8.85 and a 1-year low of $3.92. The stock’s 50-day moving average is $4.78 and its 200-day moving average is $5.72.

On the ratings front, Bio Blast Pharma has been the subject of a number of recent research reports. In a report issued on November 19, Oppenheimer analyst Ling Wang reiterated a Buy rating on ORPN, with a price target of $32, which implies an upside of 614.3% from current levels. Separately, on November 17, Roth Capital’s Joseph Pantginis reiterated a Buy rating on the stock and has a price target of $26.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Ling Wang and Joseph Pantginis have a total average return of 2.0% and -2.8% respectively. Wang has a success rate of 43.7% and is ranked #1419 out of 3640 analysts, while Pantginis has a success rate of 39.0% and is ranked #3408.

Bio Blast Pharma Ltd is a development-stage biopharmaceutical company. It is engaged in the identification, licensing, acquisition, development and commercialization of drugs for rare and ultra-rare genetic and metabolic diseases.