- OPMD: with emphasis on its pathogenesis involving accumulation of an abnormal PABN1 protein. The main features of the disease will be presented, including the progressive swallowing difficulties (dysphagia) and pattern of muscle weakness.
- Trehalose: its newly understood mechanism of action that includes activation (enhancing) of autophagy and lysosomal activity. The basic common mechanism is presumed to be through cellular 'glucose starvation' which leads to the activation of these cellular pathways.
- Results of the phase 2 trial of trehalose in OPMD including an extension study: The main results include improvement in swallowing functions as measured by drinking times of fluids as well as by swallowing quality of life measurements. Trends of positive response to trehalose in various muscle functions will be presented as well.
- Pharmacokinetics: trehalose in normal animals and humans as well as in OPMD patients. Bioblast's preclinical and clinical work emphasizes that the mode of administration of trehalose is critical to achieving therapeutic serum concentrations. Due to the active metabolism in the gut, trehalose must be administered intravenously (IV). In rats, IV administration after a single dose results in high plasma levels of trehalose for 8 hours, while muscle tissue levels remain high for more than 48 hours. In normal human subjects, trehalose has linear kinetics with exposure increasing proportionally with increasing dose. In OPMD patients in a Phase 2 trial, trehalose levels after a single 27 gram IV dose reach equivalent plasma levels to those in the animal model, with a half-life of 1.8 hours.
- Safety: in an OPMD phase 2 clinical trial in which patients received once weekly IV dosing of 27 grams for up to 52 weeks, trehalose was generally safe and well tolerated. There were no drug-related serious adverse events and no changes in safety labs including ECG. Trehalose is a disaccharide that is metabolized to glucose; however, there was no clinically meaningful change in plasma glucose levels during or following the infusion of trehalose.
- Preclinical: the animal basis for the action of trehalose in a mouse model of OPMD.
Professor Argov, a board-certified neurologist, earned his medical degree from Hebrew University Hadassah Medical School in Jerusalem, Israel.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 limbs. 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 the Muscle Study Group Society The Muscle Study Group Society (MSGS), is a consortium of scientific investigators from academic and research centers who are committed to the cooperative planning, implementation, analysis and reporting of controlled clinical trials and of other research for muscle and other neuromuscular diseases. The MSGS aims to advance knowledge about the cause(s), pathogenesis, epidemiology, and clinical manifestations of muscle disease and related neuromuscular disorders and to develop and implement strategies to examine promising therapeutic interventions.
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, with a lead drug candidate -- trehalose 90mg/mL solution -- in Phase 2 development. Bioblast was founded in 2012 and is traded on the NASDAQ under the symbol "ORPN". For more information, please visit our website, www.bioblastpharma.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. 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.
INVESTOR CONTACT: Matthew P. DuffyManaging DirectorLifeSci Advisors, LLCTelephone: 212-915-0685