BioBlast Pharma, a clinical-stage orphan disease-focused biotechnology company, announced positive preclinical in vitro and in vivo proof-of-concept study results for its Read-through drug candidate (BBrm02) for Spinal Muscular Atrophy (SMA). BBrm02 is a proprietary, intrathecal formulation of azithromycin (19th International SMA Researcher Meeting, Kansas City, MO).
In a series of cell and mouse studies BBrm02 was effective in creating high levels of full length SMN2 protein (Survival Motor Neuron 2) in the central nervous system resulting in a high degree of cellular and disease phenotypic rescues. Significant levels of full length SMN2 protein were detected following treatment with BBrm02 in several different patient cell lines representing different stages of disease severity. This SMN2 protein was tested for biological activity and was found to be highly functional.
In two different mouse models of Spinal Muscular Atrophy, BBrm02 was effective in preventing weight loss, increasing animal survival, and in normalizing a range of neurological functions, including, among others, the ability of the mouse to return to four feet from a supine position (“time-to-right” test) and to walk on a narrow tube (“tube-test”), both of which measure equilibrium and muscle strength.
Colin Foster, BioBlast’s President and CEO commented: “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.”
Mr. Foster also noted that there are no drugs approved today for SMA, which is an orphan disease with approximately 30,000 patients in the U.S. and an equal number in Europe.
In a series of cell and mouse studies BBrm02 was effective in creating high levels of full length SMN2 protein (Survival Motor Neuron 2) in the central nervous system resulting in a high degree of cellular and disease phenotypic rescues. Significant levels of full length SMN2 protein were detected following treatment with BBrm02 in several different patient cell lines representing different stages of disease severity. This SMN2 protein was tested for biological activity and was found to be highly functional.
In two different mouse models of Spinal Muscular Atrophy, BBrm02 was effective in preventing weight loss, increasing animal survival, and in normalizing a range of neurological functions, including, among others, the ability of the mouse to return to four feet from a supine position (“time-to-right” test) and to walk on a narrow tube (“tube-test”), both of which measure equilibrium and muscle strength.
Colin Foster, BioBlast’s President and CEO commented: “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.”
Mr. Foster also noted that there are no drugs approved today for SMA, which is an orphan disease with approximately 30,000 patients in the U.S. and an equal number in Europe.
Comments
Post a Comment