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System for multidimensional analysis of growth and migration of neurons and neuro

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R43AI103982-01A1
Agency Tracking Number: R43AI103982
Amount: $300,000.00
Phase: Phase I
Program: SBIR
Solicitation Topic Code: NIAID
Solicitation Number: PA12-088
Timeline
Solicitation Year: 2013
Award Year: 2013
Award Start Date (Proposal Award Date): N/A
Award End Date (Contract End Date): N/A
Small Business Information
3350 Riverwood Pkwy
Atlanta, GA 30339-6451
United States
DUNS: 828325899
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 MILTON WERNER
 (678) 392-3419
 mhwerner@gmail.com
Business Contact
 MILTON WERNER
Phone: (917) 494-0831
Email: mhwerner@gmail.com
Research Institution
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Abstract

DESCRIPTION (provided by applicant): Inhibikase Therapeutics is a clinical stage, biopharmaceutical company that has developed a host- targeted mechanism of action to treat AIDS-related and drug-induced progressive multifocal leukoencephalopathy (PML). PMLis a demyelinating disease of the central nervous system and was rarely seen clinically until the era of the HIV epidemic began in the mid-1980s. During the height of the epidemic, the rate of PML occurrence rose 20-fold, with 5% of patients with a diagnosis of clinical AIDS afflicted by the disease. Indeed, the PML became the first clinical syndrome associated with the AIDS epidemic. With the introduction of immunosuppressive monoclonal antibodies (mAb), PML has become a growing concern for most mAb therapies in addition to is occurrence in patients with AIDS. PML results from pathogenic conversion of the polyomavirus JC, a virus that persistently infects adult humans. When a patient becomes immunocompromised, however, JC undergoes a genomic rearrangement,converting the virus to a pathogenic form with tropism for brain oligodendrocytes. Once infecting brain, the infection is lytic and leads to severe dementia, loss of limb function and death. Despite numerous clinical efforts, no polyoma antiviral has beenidentified, a failure that is largely due to the sparse testing landscape for drug discovery and no permissive non- human host for JC. Inhibikase Therapeutics has taken a different approach and identified host-targets that can disrupt JC reproduction inhost cells. The Company has demonstrated that host Abl-kinase inhibition can disrupt JC polyomavirus entry, using the anti-cancer agent Gleevec as a proof-of-principle drug substance to define the mechanism of action. Gleevec, however, cannot reach the effective concentration in humans to achieve this effect. It is proposed to capitalize on the outcomes of an initial in silico and SAR analysis to develop a more potent Abl-kinase inhibitor. Preliminary results identify a putative agent and the design principle that enables a successful pathway to lead identification through SAR analysis. PUBLIC HEALTH RELEVANCE PUBLIC HEALTH RELEVANCE: Inhibikase Therapeutics is a clinical stage biopharmaceutical company developing a novel strategy for treating a fatal infectious disease, progressive multifocal leukoencephalopathy, that occurs in patients treated with monoclonal antibodies for cancer and autoimmune disease. The Company's products will treat this viral infection and save up to 150,000 lives and protect more than 500,000 patients from becoming at risk for this fatal disorder.

* Information listed above is at the time of submission. *

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