Full Press Release Details
BioBlast Granted Patent From USPTO For
Cabaletta For The Treatment Of Spinocerebellar Ataxia (SCA)
TEL AVIV, Israel, September 14, 2015
- BioBlast Pharma Ltd. (NasdaqGM: 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 or Machado 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."
About Spinocerebellar Ataxia (SCA3, Machado Joseph disease)
SCA3, also known as Machado Joseph disease,
is the most common disease among the cerebellar ataxias, which are a group of genetic diseases that are characterized by memory
deficits, spasticity, difficulty with speech and swallowing, weakness in arms and other muscular disorders. Symptoms can begin
in early adolescence and get worse over time. Eventually SCA3 leads to paralysis, and severe cases can lead to an early death in
the fourth decade of life. SCA3 is incurable, and there is currently no approved treatment for the disease. Bioblast is currently
conducting a Phase 2 clinical study (ClinicalTrials.gov Identifier: NCT02147886) for the use of Cabaletta in SCA3.
BioBlast Pharma is a clinical-stage biotechnology
company committed to developing meaningful therapies for patients with rare and ultra-rare genetic diseases of high unmet need.
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. 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
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 anticipate that we will start pivotal Phase 3 studies subject to ongoing regulatory discussions in each of Spinocerebellar
Ataxia Type 3 and Oculopharyngeal Muscular Dystrophy in 2015, or when we discuss the potential
of our platforms to 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 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'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.
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LifeSci Advisors, LLC