Bio Blast Pharma Ltd (NASDAQ:ORPN), a clinical-stage orphan disease-focused biotechnology company, announced today that it was granted a patent from the United States Patent and Trademark Office (patent no. 9,125,924) for Cabaletta (IV trehalose) entitled “Compositions and Methods for Treating Spinocerebellar Ataxia.”

Cabaletta is in Phase 2 clinical development for the treatment of Spinocerebellar Ataxia Type 3 (also known as SCA3 orMachado Joseph disease). The Company anticipates that it will start pivotal Phase 3 studies subject to ongoing regulatory discussions in each of Spinocerebellar Ataxia Type 3 and Oculopharyngeal Muscular Dystrophy in 2015.

Colin Foster, President and CEO of BioBlast said, “We are pleased to have been granted this patent for SCA which adds to our portfolio of intellectual property and the protections we have for Cabaletta, including the previously announce patent grant for Oculopharyngeal Muscular Dystrophy. We look forward to continuing our clinical development of Cabaletta for the benefit of patients suffering from SCA3.” (Original Source)

Shares of Bio Blast Pharma closed last Friday at $5.47. ORPN has a 1-year high of $8.85 and a 1-year low of $4.50. The stock’s 50-day moving average is $5.89 and its 200-day moving average is $6.25.

On the ratings front, Roth Capital analyst Joseph Pantginis maintained a Buy rating on ORPN, with a price target of $25, in a report issued on August 21. The current price target represents a potential upside of 357.0% from where the stock is currently trading. According to, Pantginis has a total average return of -1.2%, a 40.1% success rate, and is ranked #3206 out of 3755 analysts.

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.