Bioblast Pharma Ltd., a clinical-stage, orphan disease-focused biotechnology company, has engaged JSB-Partners, a global life sciences advisor, to assist the company in executing its business development objectives that include selecting potential development and commercial partners for its investigational proprietary intravenous (IV) form of trehalose 90 mg/mL solution (trehalose), which has been studied in humans with Ocular Pharyngeal Muscular Dystrophy (OPMD) and Spinocerebellar Ataxia Type 3 (SCA3).
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 Health Canada that enables the commencement of a Phase 2b double-blind, placebo-controlled clinical trial of trehalose in patients with OPMD. Such a trial is designed to enroll up to 48 patients at three sites in Canada. The study protocol provides for patients to be randomized in a 1:1 ratio to receive either trehalose or placebo for a period of 24 weeks, following a 4-week screening period and then for all patients to continue to receive trehalose for an additional 24-week period in an open label extension period.
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.