TEL AVIV, Israel, Feb. 01, 2017 (GLOBE NEWSWIRE) -- Bioblast Pharma Ltd. (NasdaqGM:ORPN), a clinical-stage, orphan disease-focused biotechnology company, announced new clinical and development updates related to its proprietary intravenous (IV) form of trehalose 90 mg/mL solution, which is being evaluated as a potential therapeutic, initially for oculopharyngeal muscular dystrophy (OPMD).

Bioblast management will provide a comprehensive overview of the Company's ongoing development work with trehalose, including OPMD activities in clinical and preclinical development, manufacturing and pre-commercial market planning. The meeting in New York City will feature a keynote presentation by Bernard Brais, MD, of McGill University and the Montreal Neurological Institute, one of the world's leading experts in the treatment of OPMD. Dr. Brais is also Co-Director of the Rare Neurological Diseases Group of the Montreal Neurological Institute, one of the leading institutions specializing in the treatment of OPMD. Since 1998, he has overseen a cohort of more than 1,400 OPMD cases.

The following five developments will be featured in the Company's presentation.
  • Patients with OPMD suffer from dysphagia (difficulty in swallowing). A post-hoc analysis of the cold water, nectar and honey-thickened drinking tests from Bioblast's Phase 2a open label OPMD trial showed a significant reduction in drinking time as compared to baseline.
Post Hoc Analysis of Trehalose in OPMD Patients
Test P Value Median Reduction In Drinking Time
Cold water <.01 40.2 %
Nectar <.01 46.5 %
Honey Thickened <.01 61.7 %

Results from these drinking tests are critical tools for assessing the progression of OPMD. This information has been incorporated into the analysis of the end points that are being developed for the Company's Phase 2b double-blind, placebo-controlled trial of trehalose in OPMD patients, scheduled to be initiated in Q2, 2017.
  • Bioblast has commissioned the undertaking of a Natural History Study (NHS) of OPMD, which is being led by Sarah Youssof, MD, MS, Assistant Professor, Department of Neurology, at the University of New Mexico Health Sciences Center and Cynthia Gagnon, PhD, Associate Professor, School of Rehabilitation, Faculty of Medicine and Health Sciences, at the University of Sherbrooke. The study has started with a retrospective review and ongoing analysis of the records of 333 OPMD patients at the Saguenay-Lac-St-Jean regional hospital, affiliated with University of Sherbrooke. The data review includes, but is not limited to: symptoms, including ptosis (eyelid drooping), dysphagia (difficulty swallowing), proximal weakness in lower limbs, fatigue, dysphonia (difficulty speaking) and pharyngeal hypersecretions; age of onset; age at diagnosis; and gene expression.

A preliminary review of this cohort of patients revealed that 96.6% of OPMD patients suffer from dysphagia. Information from the NHS will be available later this year and will be a critical part of regulatory submissions to the FDA and Health Canada.
  • A preclinical study of trehalose IV revealed that after IV administration, trehalose can be detected in the brain for up to 24 hours, suggesting that trehalose crosses the blood-brain-barrier and thus can address central nervous system effects in patients with neurodegenerative diseases. 
  • Bioblast is therefore considering an Expanded Access (Compassionate Use) program in the U.S. for trehalose in one or more diseases with cognitive as well as neuromuscular impairment, such as Amyotrophic Lateral Sclerosis (ALS) and certain Lysosomal Storage Disorders (LSDs). The Company is also investigating the use of trehalose in an undisclosed liver disease. This decision to focus on trehalose as a platform for multiple diseases is based on an analysis of published, peer-reviewed literature of the use of trehalose in preclinical studies, coupled with an assessment of recognized biomarkers for relevant diseases, plus market research evaluations of patient populations in neurodegenerative and other protein aggregation diseases. More information on this program is expected to be presented in Q2, 2017. 
  • Bioblast's investment in clinical and market development activities since the conclusion of the Phase 2a trial has led to a better understanding of the disease, appropriate clinical endpoints, and other elements of clinical design and execution. In addition, the analysis of prevalence data and patient registries suggests that the number of patients with OPMD in Canada exceeds earlier estimates, which will enable the Company to enroll patients into a well-designed double-blind, placebo-controlled Phase 2b trial more quickly than previously anticipated.

A live webcast of the event, with slides, will be available at and the slides will be archived in the Investors section of the Bioblast website at, the content of which is not incorporated by reference into this press release.

Trehalose proprietary position Bioblast has been granted two U.S. patents for parental administration of trehalose to patients with OPMD and Spinocerebellar Ataxia Type 3 (SCA3); both are expected to expire in 2033. In addition, the Company has secured Orphan Drug Designation for OPMD and SCA3 in the U.S. and in the EU.

Trehalose is a protein stabilizer that also activates autophagy and crosses the blood-brain-barrier Trehalose is a low molecular weight disaccharide (.342 kDa) that protects against pathological processes in cells. It has been shown to penetrate muscle and cross the blood brain barrier. In animal models of several diseases associated with abnormal cellular-protein aggregation, it has been shown to reduce pathological aggregation of misfolded proteins as well as to activate autophagy pathways through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for several diseases with pathologic accumulation of storage material.

About oculopharyngeal muscular dystrophy (OPMD) OPMD is an inherited myopathy characterized by dysphagia (difficulty in swallowing), eyelid drooping (ptosis) and the loss of muscle strength in multiple muscles of the limbs. Symptoms generally appear in mid-life and get worse over time. As the dysphagia becomes more severe, patients may become malnourished, may lose significant weight, and may suffer from repeated incidents of aspiration pneumonia. 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 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. Bioblast is traded on the NASDAQ under the symbol "ORPN." For more information, please visit our website:, 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, we are using forward-looking statements when we discuss future clinical studies as well as compassionate use programs, timing of milestones of such studies including timing of public announcements relating thereto, pace of enrollment of patients into clinical studies, potential new uses to the Company's drug candidate, possible revenues and profits, timing of expiry of patents and when we imply that our product candidate may successfully treat certain medical conditions. In addition, historic results of scientific research and clinical and preclinical studies do not guarantee that the conclusions of future research or studies will suggest identical or even similar conclusions or that historic results referred to in this press release would not be interpreted differently, in light of additional research and clinical and preclinical study 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. 
INVESTOR CONTACTMatthew P. DuffyManaging DirectorLifeSci Advisors LLCPhone:

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