AndroScience Corporation Awarded A $3.8 Million, 3-Year Milestone-Driven, Cooperative Translational Research Grant from the NIH To Develop An Oral Treatment for Spinal Bulbar Muscular Atrophy (Kennedy’s Disease)
SAN DIEGO, CA – January 25, 2011 – AndroScience Corp. (ASC), based in San Diego, California, announced receiving a $3.8 Million, 3-year milestone-driven, cooperative translational research grant from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH). Through a joint research effort with the Neurogenetics Branch of the NINDS, ASC will use the funding to pursue development of an oral drug treatment for spinal and bulbar muscular atrophy (SBMA) or Kennedy’s Disease, a rare hereditary neurodegenerative disease, which currently has no approved drug available to patients. Key pathological features of SBMA include progressive motor neuropathy and androgen insensitivity syndrome caused by a distinctive mutation within the androgen receptor (AR) gene. ASC has developed a unique platform of therapeutic small molecule drugs, which selectively and potently enhance degradation of the AR protein, termed AR degradation enhancers (ARD enhancers).
“Given encouraging pre-clinical results and the clear need for a new therapeutic option for SBMA patients, ASC is excited to continue advancing preclinical development of this promising novel drug candidate,” said ASC President Charles Shih, Ph.D. “The funding provided by the NINDS/NIH will significantly propel our efforts in validating ARD enhancers as a disease-modifying therapeutic intervention against such a rare and devastating neurodegenerative illness.”
This $3.8 Million cooperative translational research grant will leverage expertise from the NINDS and draw upon ASC’s innovative approach to targeting the mutant androgen receptor (AR). The goals of the grant will be to first validate an orally administered ARD enhancer drug is efficacious in the SBMA transgenic animal model, and further, to complete preclinical toxicology, safety pharmacology, and ADME studies necessary in supporting of an
About Spinal Bulbar Muscular Atrophy (SBMA) – Kennedy’s Disease
Spinal and bulbar muscular atrophy (SBMA or Kennedy's Disease) is a rare hereditary neurodegenerative disease that affects lower motor neurons, with progressive muscle atrophy and weakness of the bulbar, facial, and limb muscles. The disease results in progressive dysphasia, motor dysfunction, and typically affects men in the fourth or fifth decade of life. SBMA is caused by a mutation in the X-chromosome linked androgen receptor (AR) gene that results in excessive repeats of the amino acid glutamine (polyQ) within AR protein. A molecular genetic test is clinically available which has a 100% mutation detection rate to definitively diagnose SBMA. Neurotoxicity caused by these expanded polyQ AR aggregates is believed to play a pivotal role in the pathogenesis of SBMA. SBMA is considered an orphan drug indication and no approved therapy exists. Furthermore, very few drug candidates are being evaluated in clinical trials.
About AndroScience Corporation
AndroScience (ASC) is a privately held
About National Institute of Neurological Disorders and Stroke (NINDS), NIH
The Intramural Research Program of the National Institute of Neurological Disorders and Stroke (NINDS) is one of the largest neuroscience research centers in the world. Investigators in the NINDS intramural program conduct research in the basic, translational, and clinical neurosciences. Their specific interests cover a broad range of neuroscience research including molecular biophysics, synapses and circuits, neuronal development, integrative neuroscience, brain imaging and neurological disorders. Through collaboration, pre- and postdoctoral training programs, jointly sponsored seminar series and special interest groups, NINDS investigators and investigators in other intramural programs (NIMH, NEI, NIDCD and NICHD) contribute to a vital and growing neuroscience research community at the National Institutes of Health.
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