Press Release (ePRNews.com) - CHARLOTTESVILLE, Va. - Jan 12, 2017 - Biovista Inc. today announced that two drugs targeting Niemann-Pick Disease Type A (NPA), have shown strong positive results in the Acid Sphingomyelinase ASM knock-out (ASMKO) mouse model. Both repositioning candidates (BVA-901A and BVA-901B) tested by the laboratory of Dr. M.D. Ledesma at the Madrid Center for Molecular Biology “Severo Ochoa” (CBMSO) promoted significant sphingomyelin hydrolysis in vitro in hippocampal ASMKO mouse neurons. The drug candidates were identified using Biovista’s Project Prodigy big data inference engine in drug repositioning mode.
“We are pleased with this latest positive outcome from Project Prodigy and these initial results that confirm the predicted efficacy of these two candidates, in particular because we are seeing significant hydrolysis of sphingomyelin. We will continue working with both candidates to further confirm the purported mechanism of action and quantify expected efficacy before we proceed with our IND application” said Andreas Persidis, Ph.D., CEO of Biovista. “We are also happy that our collaboration with Wylder Nation Foundation (WNF) that started with the 2015 BeHeard science challenge hosted by the Rare Genomics Institute has yielded these early success results” added Dr. Persidis.
The results were obtained using the ASMKO model of NPA. “We are excited by these results obtained using an animal model that faithfully reproduces the human disease. We believe they open new perspectives for finding a cure for NPA patients” said Dr Ledesma. “We remain very optimistic about the potential that one of these drug candidates will significantly improve the most severe neurological manifestations of Niemann-Pick Type A, and we are currently in the process of planning in vivo studies to further validate these initial in vitro results,” said Steven Laffoon, Co-Founder and President of Wylder Nation Foundation.
About Biovista’s trial in the ASMKO model of NPA
Niemann-Pick Disease Type A (NPA), is the early-onset, fatal form of a rare progressive genetic disorder known as Acid Sphingomyelinase Deficiency (ASMD), characterized by the deficiency of acid sphingomyelinase, a key enzyme in the sphingolipid metabolic pathway. This results in harmful sphingomyelin accumulation in various tissues of the body. Symptoms of NPA include failure to thrive, hepatosplenomegaly, interstitial lung disease and progressive neurodegeneration that leads to death by age 2-3 years. In the in vitro trial, BVA-901A and BVA-901B were found to induce a statistically significant increase in sphingomyelin hydrolysis in hippocampal ASMKO mouse neurons without inducing neuronal toxicity.
Biovista develops advanced technologies that use big data and next generation analytics to address major needs in biomedical research, development, and clinical practice. Project Prodigy is a new category of inference generation and validation system. It operates by generating extremely large numbers of new potential clinical outcomes and ranking their real-world validity. Project Prodigy is different to what are called “cognitive systems” that work as search engines on known datasets and require significant training, since Project Prodigy’s recommendations are not already known. Biovista is using Project Prodigy to advance its own drug repositioning programs in rare diseases, as well as in programs with biopharmaceutical companies, regulators, and patient advocacy groups. To date, Biovista has delivered previously unanticipated and now validated drug positioning options for leading pharmaceutical and biotechnology companies and patient advocacy groups.
About Wylder Nation Foundation:
Wylder Nation Foundation is a non-profit organization focused on improving the lives of children diagnosed with Lysosomal Storage Disorders by accelerating the discovery and development of treatment options. Their current work revolves around the most severe form of Acid Sphingomyelinase Deficiency also known as Niemann-Pick Type A (NPA), and focuses on the translation of basic discoveries into potential treatment options.
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Dr. Andreas Persidis, CEO Biovista Inc.