Company Update (NASDAQ:ORPN): Bio Blast Pharma Ltd Augments Leadership Team and Establishes U.S. Headquarters in New Haven, Connecticut

Bio Blast Pharma Ltd (NASDAQ:ORPN), a clinical-stage biotechnology company committed to developing meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today that it has appointed Paul Firuta to the newly-created position of Chief Commercial Officer. Mr. Firuta will be a member of BioBlast’s Executive Committee and will oversee, in particular, the commercial strategy for Cabaletta (IV trehalose) – the Company’s lead product candidate set to commence Phase 3 clinical development this year in the United States and Canada.

“Paul is a seasoned biotechnology and pharmaceutical executive who brings a wealth of commercial experience to BioBlast from an impressive list of successful biopharmaceutical organizations,” said Colin Foster, President and Chief Executive Officer of BioBlast. “In particular, his significant and highly relevant commercial experience in the rare genetic disease space, overseeing commercial efforts for Cinryze® for hereditary angioedema (HAE), ideally positions him to spearhead commercial efforts for BioBlast and support our global pre-commercialization activities as we prepare to initiate two pivotal Phase 3 clinical studies for Cabaletta in oculopharyngeal muscular dystrophy (OPMD) and spinocerebellar ataxia type 3 (SCA3) this year. If results of either of these trials are positive, we would be prepared for the global launch of Cabaletta, initially in the U.S. andCanada.”

Mr. Firuta has extensive experience in biopharmaceutical commercial operations, most recently serving as President of U.S. Operations for NPS Pharmaceuticals, where he played a role in the $5.2 billion acquisition of NPS by Shire Pharmaceuticals. Prior to his role at NPS, he served as Vice President and General Manager, Americas, at ViroPharma. Prior to that, he held various senior management positions at Lev Pharmaceuticals, OraPharma and SmithKline Beecham Pharmaceuticals. Mr. Firuta has a MBA from St. Joseph’s University, and a B.Sc. from King’s College.

“It is a privilege to join BioBlast and to work closely with Colin and the executive team. With a compelling and deep pipeline targeting orphan diseases with great unmet need, and with Cabaletta specifically about to commence two pivotal Phase 3 clinical studies, there is tremendous opportunity to make a medical difference for patients and to prepare Cabaletta for the commercial market, and ultimately, to build a successful product franchise.”

Separately, the Company announced today that it has recently established a corporate office in New Haven, Connecticut, which will serve as its U.S. headquarters.

“This is a big step forward for us as we establish our corporate headquarters with a view to connecting ever more so with patients, physicians, and investors in the U.S.,” said Colin Foster. “Indeed, we anticipate that six of our nine Cabaletta clinical study centers for OPMD will be here in the U.S. and we look forward to expanding our U.S. footprint going forward.” (Original Source)

Shares of Bio Blast Pharma Ltd closed yesterday at $5.61 . ORPN has a 1-year high of $8.99 and a 1-year low of $4.50. The stock’s 50-day moving average is $5.97 and its 200-day moving average is $6.27.

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 implies an upside of 345.6% from current levels. According to, Pantginis has a total average return of -2.3%, a 38.0% success rate, and is ranked #3479 out of 3752 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.

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