|Post-Hoc Analysis of Trehalose in OPMD Patients|
|Test||P Value||Median Reduction In Drinking Time|
Results from a Phase 2a Trial in SCA3 patientsIn a six-month open label, Phase 2a study that also included an additional six-month follow-up period investigating trehalose in patients with SCA3, infusions of trehalose were generally safe and well tolerated. Patients remained stable over the 6-month period with no change on the Scale for Assessment and Rating of Ataxia (SARA) score - a well-accepted clinical tool for measuring the effect of this progressively debilitating disease. Trehalose's exclusivity position Bioblast has been granted three U.S. patents for parenteral administration of trehalose to patients with OPMD, SCA3 and Huntington's Disease; they 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 E.U. and has Fast Track Status in the U.S. for OPMD. Trehalose is a protein stabilizer that also activates autophagy and crosses the blood-brain barrier Trehalose is a low molecular weight disaccharide (.342 kilodaltons) that protects against pathological processes in cells. It has been shown to penetrate muscle cells 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 intracellular degradation pathways such as autophagy and lysosomal degradation. About Bioblast 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: 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. For example, we are using forward-looking statements when we discuss executing the company's business development objectives that include selecting potential development and commercial partners, future clinical studies and the timing and design thereof, and, specifically, the possibility to commence the Phase 2b clinical trial in OPMD patients discussed in this press release, if such trial is commenced at all, 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 February 24, 2017, 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 LLCMatthew@LifeSciAdvisors.comPhone: 212-915-0685JSB-Partners ContactWolfgang StoiberManaging DirectorJSB-Partnerswstoiber@jsb-partners.comPhone: 978-697-9753