BioBlast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage orphan disease-focused biotechnology company whose mission is to transform excellent science into safe and effective therapies for rare genetic diseases, has recently presented pre-clinical proofs of concept for two of its therapeutic platforms at two important scientific meetings that focus on rare genetic diseases.
The meetings included:
The 19th International SMA Researcher Meeting, June 18-20, 2015 Westin Crown Center, Kansas City, Missouri, where BioBlast presented its proof of concept for BBrm02 (intrathecally-administered azithromycin) for Spinal Muscular Atrophy, its Read-through Platform lead drug candidate.
The United Mitochondrial Disease Foundation’s Mitochondrial Medicine 2015 Meeting, June 17-20, 2015, Hyatt Dulles, Herndon, Virginia, where BioBlast presented its proof of concept for BB-FA (frataxin protein replacement) for Friedreich’s Ataxia, its lead drug candidate in its Mitochondrial Protein Replacement Platform (mPRT).
In a company press release on the BBrm02 presentation, Colin Foster, BioBlast’s President and CEO stated “We are excited to share this comprehensive proof-of-concept as it establishes the efficacy of our lead drug candidate in our Read-through Platform for Spinal Muscular Atrophy. Importantly, given that BBrm02 is a repurposed drug (azithromycin), we believe there is a reasonable possibility for a shorter developmental process. We plan to start a Phase 2a study with BBrm02 at year-end, 2015.”
In a comment concerning the BB-FA proof of concept, Mr. Foster, added, “We are excited by this breakthrough proof-of-concept of our mitochondrial protein replacement platform. This is a strong evidence of the immense potential of our platform, which aims to address the needs of thousands of patients suffering from these debilitating and fatal diseases. We are eager to move BB-FA forward into clinical testing following our anticipated completion of the preclinical program in late 2016.”
About Friedreich’s Ataxia
FA is a rare inherited disorder that is caused by defects in an important gene that encodes for the protein, frataxin. Frataxin is a protein that regulates the amount of iron in the mitochondria, and the defects caused by FA lead to iron overload and irreversible damage to the mitochondria’s DNA. Patients diagnosed with FA have a progressive degeneration of the central and peripheral nervous systems, impaired coordination and gait, and fatigue from energy deprivation and muscle loss. According to the National Institutes of Health (NIH), 20,000 people in the US and Europe have FA. There are currently no FDA approved therapies for FA.