Full Press Release Details
Pharma Augments Leadership Team and Establishes
in New Haven, Connecticut
Former NPS and ViroPharma Executive, Paul Firuta as Chief Commercial Officer
New Haven, CT - September 10,
2015 - BioBlast Pharma Ltd., (NasdaqGM: 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. and Canada."
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
"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
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."
BioBlast Pharma is a clinical-stage biotechnology
company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases. The company
is rapidly building a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable diseases.
The BioBlast platforms are based on deep understandings of the disease-causing biological processes, and potentially offer solutions
for several diseases that share the same biological pathology. For more information please visit the Company's website, www.bioblast-pharma.com,
the content of which is not incorporated herein by reference.
Forward-Looking Statements
This press release contains forward-looking
statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995
and other Federal securities laws. For example, the Company is using forward looking statements when it discusses making a medical
difference for patients, preparing Cabaletta for the commercial market, building a successful product franchise, initiating two
pivotal Phase 3 clinical studies for Cabaletta in OPMD and SCA3 and timing of such initiation, the global launch of Cabaletta,
the locations of certain clinical studies, building a diverse portfolio of products candidates with the potential to address unmet
medical needs for incurable diseases, or that our platforms potentially offer solutions for several diseases that share the same
biological pathology. In addition, historic results of scientific research and clinical and preclinical trials do not guarantee
that the conclusions of future research or trials would not suggest different conclusions or those historic results referred to
in this press release would not be interpreted differently in light of additional research and clinical and preclinical trials
results. Because such statements deal with future events and are based on BioBlast Pharma Ltd.'s current expectations, they are
subject to various risks and uncertainties and actual results, performance or achievements of BioBlast Pharma could differ materially
from those described in or implied by the statements in this press release, including those discussed under the heading "Risk
Factors" in BioBlast Pharma's Annual Report on Form 20-F filed with the Securities and Exchange Commission ("SEC")
on March 31, 2015, and in any subsequent filings with the SEC. Except as otherwise required by law, BioBlast Pharma disclaims any
intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as
a result of new information, future events or circumstances or otherwise.
U.S. Investor Contact
LifeSci Advisors, LLC