Full Press Release Details
BioBlast Pharma Announces Positive in
vivo Preclinical Proof-of-Concept Results of its Cabaletta Platform in Spinocerebellar Ataxia Type 3 (SCA3)
TEL AVIV, Israel, March 30, 2015 - BioBlast Pharma
Ltd. (NasdaqGM: ORPN), a clinical-stage, orphan disease-focused biotechnology company announced today positive in vivo proof-of-concept
results for Cabaletta for Spinocerebellar Ataxia Type 3 (also known as SCA3 or Machado Joseph disease). Results were presented
at the International Ataxia Research Conference held in Windsor UK, March 25-28, 2015.
Cabaletta was tested in two different animal models of the disease:
Colin Foster, BioBlast's President and CEO commented:
"Our in vivo proof-of-concept studies demonstrate Cabaletta's efficacy in reducing Ataxin3 aggregates, reducing
neural markers for neural damage and preventing typical phenotypic deterioration in animal models. As a whole, this is powerful
support for our ongoing SCA3 Phase 2 clinical trial program underway in Israel. We anticipate expanding the number of SCA3 clinical
study centers later this year.
"There are approximately two dozen diseases that Cabaletta
may address, given a common disease-causing mechanism related to intracellular and intranuclear protein aggregation. With Cabaletta,
we have previously reported on our ongoing oculopharyngeal muscular dystrophy (OPMD) Phase 2/3 multicenter clinical trial and our
preclinical work in spino bulbar muscular atrophy. Today, given the SCA3 proof-of-concept results, we share the fervent hopes of
the many SCA3 patients waiting anxiously for a treatment for this devastating disease."
Cabaletta is BioBlast's proprietary intravenous (IV) solution
of trehalose, a disaccharide, known for its capabilities in stabilizing intracellular and intranuclear proteins and enhancing autophagy.
About Spinocerebellar Ataxia Type 3 (SCA3 or Machado Joseph
SCA3, also known as Machado Joseph disease, is the most common
among the cerebellar ataxias, which are a group of genetic diseases that are characterized by gait and movement disorders memory
deficits, spasticity, difficulty with speech and swallowing, weakness in arms and other muscular anomalies. Symptoms can begin
in early adolescence and get worse over time. Eventually SCA3 leads to paralysis and severe disability. The disease typically appears
in the third or fourth decades of life and is associated with early death, on average within 15-20 years of diagnosis. There is
currently no approved treatment for the disease.
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 has a diverse
portfolio of product candidates with the potential to address unmet medical needs for incurable genetic orphan diseases.
The BioBlast platforms are based on deep understanding of the
disease-causing biological processes, and potentially offer solutions for several diseases that share the same biological pathology.
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, we are using forward looking statements when we discuss making further progress toward providing
a therapy to help SCA3 patients, that our platforms potentially offer solutions for several diseases that share the same biological
pathology, including approximately two dozen diseases in the case of our Cabaletta product candidate, or when we discuss expanding
the number of SCA3 clinical studies and timing of such expansion. 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 that historic results 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 Bio Blast 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 prospectus dated July 30, 2014 filed with the Securities and Exchange Commission ("SEC")
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