Israeli biotech company BioBlast Pharma Ltd. has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application and is now authorized to advance development of lead product Cabaletta. The chemical chaperone is expected to become a therapeutic option for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD).
With the clearance from the FDA, BioBlast will now initiate its phase 2 /3 clinical trial as part of the multi center Phase 2 /3 HOPEMD study in collaboration with a center located in California. The HOPEMD study is planned to include patients in Canada, which are still being enrolled, as well in Israel, whose enrollment is already completed. The company will initiate patient enrollment in the United States by the second quarter of the year.
“The FDA’s clearance to conduct a Phase 2/3 clinical trial for OPMD is a critical step in advancing our development plan,” said the president and CEO of BioBlast Pharma, Colin Foster, in a press release. “OPMD is a severe and debilitating disease for which there is currently no effective treatment options. We believe the Cabaletta platform can be an effective treatment for this devastating disease that would bring hope to the patients and their families.”
The company had already been grated Orphan Drug Designation by the FDA to Cabaletta for addressing OPMD. In addition, the United States Patent and Trademark Office has granted BioBlast an allowance regarding Cabaletta.
Previous studies already demonstrated the drug’s effectiveness in patients who suffer from numerous diseases with abnormal cellular-protein aggregation or autophagy enhancers. BioBlast recently presented preliminary from HOPEMD Phase 2/3 study, demonstrating the therapy’s capacity to reduce pathological aggregation of proteins within cells, as well as significant efficacy in preclinical animal models of Occulopharyngeal Muscular Dystrophy and other PolyA/PolyQ diseases.
BioBlast aims to reduce the burden of patients who suffer from OPMD, which is an inherited and genetic disease that causes difficulties in swallowing, as well as loss of muscle strength. When swallowing difficulty, called dysphagia, is severe it can cause other problems such as malnutrition, weight loss, dehydration, and repeated aspiration pneumonia. Dehydration and aspiration pneumonia are often the cause of death.