PML antiviral for AIDS

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DESCRIPTION (provided by applicant): Inhibikase Therapeutics is an early stage biopharmaceutical company developing a novel strategy for treating infectious disease that occurs in AIDS patients. The Company seeks to create an anti-infective product for treatment of JC viral infection, which leads to progressive multifocal leukoencephalopathy (PML), an AIDS- defining disease. PML results from JC virus reactivation in the aforementioned patients, inducing an inflammatory disease in the central nervous system that has proven to be >70% fatal. Because the disorder occurs in a wide variety of patients, has forced such benchmark drugs as Tysabri (for MS) and Raptiva (for psoriasis) off the market due to the sporadic occurrence of the disease, Inhibikase has the opportunity to help a very large patient population now at risk for PML. While the proposed product to be developed could help people outside of the AIDS patient population, the occurrence of PML in AIDS patients represents the best patient population for which to develop a product to treat PML. AIDS patients are activist and they are largely treated in a uniform fashion, therefore, the response rate in these patients is likely to be less affected by variations in health, in immunity status, etc. The causative agent for PML, the polyomavirus JC, is one of a small family of human pathogens that can lead to debilitating or fatal diseases of the genitourinary tract and the central nervous system. The total patient population affected by polyomaviral diseases is well in excess of 150,000 patients annually, thus, the Company's development of an anti-JC antiviral could have the added benefit of providing a new therapeutic paradigm for a number of fatal diseases. The Company has identified a family of small molecules, several which could be re-purposed and have been shown to be safe-for-human use, that are capable of interfering with mouse polyoma virus infection in cell culture and in the mouse. These small molecules represent a dramatic departure from current treatment paradigms for infectious disease, because these molecules target host pathways upon which bacterial and viral pathogens depend, the Company's treatment strategy has the potential to be applied to multiple infectious diseases simultaneously in a single patient. In the present case, the Company has screened its compound portfolio against TB, flu (H5N1), pox, mouse polyoma and herpes viruses and seen in vitro antiviral activity. Mouse polyoma is of the same family of polyomaviruses that JC belongs to, suggesting that the Company's lead compounds could be active against JC virus. The Company proposes to assess the antiviral efficacy of its compound portfolio against JC virus in culture and to establish the utility of its compound portfolio as drugs that could enter the clinic. No animal models exist for the evaluation of anti-JC compound efficacy in vivo. Thus, compounds with anti-JC efficacy in culture will be further evaluated for safety and drugability prior to filing Initial New Drug applications to trial the effective compounds against PML in the clinical setting. PUBLIC HEALTH RELEVANCE: Inhibikase Therapeutics is an early stage biopharmaceutical company developing a novel strategy for treating a fatal infectious disease, progressive multifocal leukoencephalopathy, that occurs in AIDS patients. 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.