AndroScience has been awarded a $3.8 million, milestone-driven cooperative translational research grant from the NINDS and NIH. The funds will support a collaborative project by AndroScience and researchers at the Neurogenetics Branch of the NINDS that aims to develop an oral therapy for spinal and bulbar muscular atrophy (SBMA), a rare hereditary neurodegenerative disorder also known as Kennedy disease.
SBMA is caused by a specific mutation within the X chromosome-linked androgen receptor (AR) gene, and leads to progressive motor neuropathy and androgen insensitivity syndrome. There is currently no approved treatment for the condition, although a molecular genetic test is clinically available, AndroScience points out.
The firm has developed a platform of therapeutic small molecules that selectively enhance degradation of the androgen receptor protein. The collaboration with NINDS will exploit AndroScience’s technology to target the mutant AR that causes SBMA. The overall aim of the grant-funded work will be to validate the activity of an orally administered AR degradation enhancer (ARD enhancer) in an SBMA transgenic animal model, and to complete preclinical studies needed to support an IND filing. AndroScience says it has already generated proof-of-concept data demonstrating that its ARD enhancer compound positively impacts the main features of SBMA neuromuscular pathology, restores functional activity, and improves survival in an SBMA transgenic mouse model.
“Given encouraging preclinical results and the clear need for a new therapeutic option for SBMA patients, AndroScience is excited to continue advancing preclinical development of this promising novel drug candidate,” comments the firm’s 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.”
AndroScience is focused on developing small molecule therapeutics targeting disease-dependent intracellular signaling events critical to the AR and signal transducer and activation of transcription (STAT) pathways.
The compound already in preclinical development for the potential treatment of SBMA is ASC-J9®, which is separately undergoing Phase II clinical development in a topical formulation for the treatment of acne, and Phase I trials for the treatment of alopecia. Topical ASC-J9 is, in addition, in preclinical development for potential wound-healing applications.
AndroScience says ARD enhancer compounds may also have utility in the treatment of other systemic indications such as prostate cancer, benign prostatic hyperplasia, and AR-related solid tumors including hepatocellular carcinoma and bladder cancer.
The firm’s separate STAT inhibitor pipeline is undergoing final lead identification, and could have applications against a wide range of aggressive solid and hematologic cancers, it claims. The ASC-JP compounds are being designed as synthetic small molecule inhibitors of STAT that exhibit potent activity against STAT 3 and 5.
In May 2010 AndroScience inked strategic partnership with Taiwanese firm Orient Europharma (OEP), centered on the clinical development and commercialization of ASC-J9 as a topical anti-acne product. Under terms of the deal, OEP secured commercialization rights to the ASC-J9 drug for the acne indication in Taiwan, Hong Kong, S. Korea, New Zealand, Australia, and other select markets served by OEP throughout Asia Pacific.