AndroScience Awarded NIH Grant to Develop Oral Treatment for Kennedy's Disease
AndroScience Corp. recently 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 preclinical 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, PhD. “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. 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
AndroScience is a privately held San Diego, CA-based pharmaceutical company applying expertise in natural product chemistry to develop proprietary small molecule drug compounds targeting disease-dependent intracellular signaling events related to the Androgen Receptor and selective Signal Transducer and Activation of Transcription (STAT) pathways. The company is focused on therapeutic indications of significant medical and commercial importance, including androgen-dependent diseases in both topical and systemic treatment settings and novel approaches to the treatment of cancer. Active ARD enhancer therapeutic programs include topical indications: acne (in Phase IIb clinical trail), alopecia (male pattern baldness), and wound healing; and systemic indications: SBMA, prostate cancer, HBV-induced HCC, and bladder cancer. Currently, the newly discovered STAT inhibitor compounds demonstrate potent anti-tumor properties across a wide range of solid and hematological tumors. ASC is actively seeking licensing partners for multiple products at various stages of program development with both domestic and international-based pharmaceutical companies.
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