Bioblast Pharma

Hope should not be rare

IV trehalose

Trehalose 90 mg/mL IV solution

Lead drug candidate in Phase 2 clinical development for people living with OPMD or SCA3

Trehalose 90 mg/mL IV solution, a first-in-class and first therapeutic to potentially treat a number of devastating PolyA/PolyQ diseases, is a chemical chaperone that protects against pathological processes in cells.

Widely known to act as a protein stabilizer and autophagy enhancer, trehalose 90 mg/mL IV solution 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 90 mg/mL IV solution has been documented as demonstrating significant promise in preclinical animal models of OPMD; spinocerebellar ataxia, type 3 (SCA3); and other PolyA/PolyQ diseases.

Trehalose 90 mg/mL IV solution has received orphan drug designation for OPMD and SCA3 in the US and EU and has been designated Fast Track for OPMD in the US.

Learn about the OPMD clinical trial
Learn about the SCA3 clinical trial