(ORPN) to Present Final HOPEMD Phase 2 Clinical Study Results of Trehalose

Final Results of 24-Week Open-Label Phase 2 Trial Will be Presented by Professor Zohar Argov, MD, Senior Medical Advisor to BioBlast Pharma

NEW HAVEN, Conn., April 14, 2016  — BioBlast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan disease-focused biotechnology company, will present previously announced positive final results from its HOPEMD Phase 2 six-month open-label clinical study in patients with oculopharyngeal muscular dystrophy (OPMD) at the 2016 American Academy of Neurology (AAN) Annual Meeting, being held in Vancouver, Canada April 15-21.  OPMD is a rare progressive muscle-wasting disease characterized by swallowing difficulties (dysphagia), leading to the risk of aspiration of food into the lungs, weight loss, and generalized progressive muscle weakness.

American Academy of Neurology 2016 Annual Meeting
April 15-21 in Vancouver, Canada

Title:           Intravenous Trehalose for Treatment of Dysphagia and Muscle Function in Oculopharyngeal
Muscular Dystrophy (OPMD): Final Results of 24 Week Open-Label Phase 2 Trial
Session:     Integrated Neuroscience Session – Advances in Acquired and Genetic Muscle Diseases
Date:          Sunday, April 17, 2016
Time:          5:00pm Pacific Time
Presenter:  Prof. Zohar Argov M.D.
Senior Medical Advisor to BioBlast Pharma

Session:      S28: Experimental Therapies in Neuromuscular Diseases
Date:           Tuesday, April 19, 2016
Time:           7:15am Pacific Time
Presenter:   Prof. Zohar Argov M.D.
Senior Medical Advisor to BioBlast Pharma

Twenty-four week results from the HOPEMD Phase 2 open-label study were announced by BioBlast on March 16th, 2016. Trehalose 90mg/mL IV solution was observed to be safe and well-tolerated with no drug-related serious adverse events.  Improvements versus baseline were observed in multiple secondary efficacy endpoints related to dysphagia and muscle strength and function.

About Trehalose 90mg/mL IV Solution
Trehalose 90mg/mL IV solution is a chemical chaperone that protects against pathological processes in cells.  It has been shown to reduce pathological aggregation of proteins within cells in several diseases associated with abnormal cellular-protein aggregation as well as acting as an autophagy enhancer.  Trehalose 90mg/mL IV solution has been documented as demonstrating significant promise in preclinical animal models of OPMD and other PolyA/PolyQ diseases.

In OPMD, trehalose 90mg/mL IV solution is being developed to prevent the aggregation of the pathological protein (PABPN1) in muscle cells, the hallmark of the disease, by stabilizing the protein, reducing the formation of protein aggregations, and promoting their clearance from cells through autophagy, thus preventing muscle cell death.

About Oculopharyngeal Muscular Dystrophy (OPMD)
OPMD is an inherited myopathy characterized by dysphagia (difficulty in swallowing), eyelid drooping (ptosis), the loss of muscle strength, and weakness in multiple muscles of the body.  Symptoms generally appear in mid-life and get worse over time.  As the dysphagia becomes more severe, patients become malnourished, lose significant weight, and may suffer from repeated incidents of aspiration pneumonia.  Aspiration pneumonia and severe emaciation may result in death.  The disease is caused by a genetic mutation responsible for the creation of a mutant protein (PABPN1) with an expanded polyalanine domain that aggregates within patient muscle cells.  There is currently no approved pharmacologic treatment for OPMD.

About BioBlast Pharma Ltd.
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 portfolio of product candidates with the potential to address unmet medical needs for incurable diseases. The BioBlast platforms are based on a deep understanding of disease-causing biological processes, and potentially offer solutions for several diseases that share the same biological pathology.  BioBlast was founded in 2012 and is traded on the NASDAQ under the symbol “ORPN”. 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, potential uses of its product candidates for various indications, building a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable diseases, or that BioBlast’s 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 trial 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 29, 2016, 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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