BioBlast receives patent for Cabaletta in SCA treatment

BioBlast Pharma Ltd. on Monday announced it has been granted a U.S. patent for Cabaletta (IV trehalose) titled "Compositions and Methods for Treating Spinocerebellar Ataxia (SCA)."

"We are pleased to have been granted this patent for SCA which adds to our portfolio of intellectual property and the protections we have for Cabaletta, including the previously announced patent grant for Oculopharyngeal Muscular Dystrophy,” BioBlast CEO and President Colin Foster said.

Cabaletta is in Phase 2 clinical development for the treatment of SCA3, also known as Machado Joseph disease. SCA3 is the most common disease among the cerebellar ataxias, a group of genetic diseases characterized by memory deficits, spasticity, difficulty with speech and swallowing, weakness in arms and other muscular disorders.

BioBlast expects to start pivotal Phase 3 studies this year, subject to ongoing regulatory discussions in each of Spinocerebellar Ataxia Type 3 and Oculopharyngeal Muscular Dystrophy.

“We look forward to continuing our clinical development of Cabaletta for the benefit of patients suffering from SCA3," Foster said.

BioBlast Pharma is a clinical-stage biotechnology company committed to developing therapies for patients with rare and ultra-rare genetic diseases of high unmet need.