Bioblast Pharma to Seek Strategic Alternatives Including Partnership and Merger Opportunities
Company Receives Regulatory Clearance to Initiate a Phase 2b trial of Trehalose in OPMD patients
At the present time, Bioblast is not encumbered by any license, partnership or royalty arrangements with third parties for development or commercialization of trehalose.
Phase 2b trial in OPMD patients is ready to initiate
In addition, Bioblast has received confirmation from
OPMD is a rare, debilitating, life-threatening disorder. 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
Results from these drinking tests were critical tools for assessing the progression of OPMD. This information has been incorporated into the analysis of the end points that were developed for the Company’s Phase 2b double-blind, placebo-controlled trial of trehalose in OPMD patients. In both the six-month treatment period as well as in the extension phase of up to an additional 12 months, infusions of trehalose were generally safe and well tolerated.
Results from a Phase 2a Trial in SCA3 patients
In 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.
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
Matthew P. DuffyManaging Director LifeSci Advisors LLCMatthew@LifeSciAdvisors.com Phone: 212-915-0685 JSB-Partners Contact Wolfgang StoiberManaging Director JSB-Partnerswstoiber@jsb-partners.com Phone: 978-697-9753