BioBlast Pharma Ltd., a clinical-stage orphan disease-focused biotechnology company, has received a patent from the United States Patent and Trademark Office (patent no. 9,125,924) for Cabaletta (IV trehalose) entitled "Compositions and Methods for Treating Spinocerebellar Ataxia."

Cabaletta is in phase 2 clinical development for the treatment of Spinocerebellar ataxia type 3 (also known as SCA3 or Machado Joseph disease). The company anticipates that it will start pivotal phase 3 studies subject to ongoing regulatory discussions in each of Spinocerebellar ataxia type 3 and oculopharyngeal muscular dystrophy in 2015.

Colin Foster, president and CEO of BioBlast said, "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 announce patent grant for oculopharyngeal muscular dystrophy. We look forward to continuing our clinical development of Cabaletta for the benefit of patients suffering from SCA3."

SCA3 is the most common disease among the cerebellar ataxias, which are a group of genetic diseases that are characterized by memory deficits, spasticity, difficulty with speech and swallowing, weakness in arms and other muscular disorders. Symptoms can begin in early adolescence and get worse over time. Eventually SCA3 leads to paralysis, and severe cases can lead to an early death in the fourth decade of life. SCA3 is incurable, and there is currently no approved treatment for the disease. Bioblast is currently conducting a phase 2 clinical study for the use of Cabaletta in SCA3.