- 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|
- 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 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.
INVESTOR CONTACTMatthew P. DuffyManaging DirectorLifeSci Advisors LLCPhone: 212-915-0685Matthew@lifesciadvisors.com