Inhibikase Therapeutics Receives FDA Clearance for Phase II Trial of IkT-001 to treat JC virus infection, the cause of Progressive Multifocal Leukoencephalopathy (PML) in Multiple Sclerosis Patients

August 16, 2011

Host-Targeted Antiviral Blocks JC Virus Entry

Inhibikase Therapeutics, Inc., an emerging leader in infectious disease, announced today that it has received FDA clearance to commence a Phase II proof-of-concept trial for its lead compound IkT-001. IkT-001, a host-directed kinase inhibitor, is intended to clear JC polyomavirus (JCV) infection, the causative agent of Progressive Multifocal Leukoencephalopathy (PML). This proof-of-concept trial will be conducted in multiple sclerosis (MS) patients on natalizumab (TYSABRI®), a
patient group that is at significant risk for developing the potentially fatal PML disease. JCV progresses to PML only in patients with chronic or drug-induced immune suppression, to include patients diagnosed with clinical AIDS or who are receiving monoclonal antibody treatment for MS, lupus, rheumatoid arthritis, leukemia or lymphoma.

The Phase II trial will be a four-week, open-label, sequential-dose-ranging study of the compound’s safety and its antiviral effects against JCV in 48 patients with a relapsing form of MS who receive  TYSABRI® infusion therapy. In addition to closely monitoring the safety and pharmacokinetic profile of IkT-001, the study also will determine the potential antiviral effect of IkT-001 against JCV as measured by the suppression of urinary excretion of JCV. Led by Jeffrey English, MD, of the Multiple Sclerosis Center of Atlanta, the study will be performed at three to five U.S. treatment centers for MS. All participating centers must be registered with the TOUCH Program for the prescription and infusion of TYSABRI® to patients with MS.

“The launch of this study is a pivotal event for Inhibikase Therapeutics, just 23 months into operations, as we advance a new approach to treating bacterial and viral infectious diseases into the clinic,” said Milton H. Werner Ph.D., Chief Executive Officer and President of Inhibikase   Therapeutics. “If IkT-001 proves to be efficacious at suppressing JCV in MS patients on TYSABRI®, then a much broader population of patients at potential risk of contracting PML could benefit from this approach to preempting the virus from reaching the brain and causing the disease. The  common mechanism of action utilized by IkT-001 enables treatment for bacterial and viral infectious disease, thus an efficacious outcome for this Phase II study could clear a path for multiple  indications using the same pharmaceutical approach.”

About JC Virus and Progressive Multifocal Leukoencephalopathy (PML)
The John Cunningham virus (JCV) is a type of human polyomavirus. It was isolated in 1971 from a patient with progressive multifocal leukoencephalopathy (PML). Human polyomaviruses, like JC and BK, cause PML and other diseases only in the context of immunodeficiency, as in AIDS, or  drug-related immune suppression, either for treatment of autoimmune disease, cancer or for solid organ transplant maintenance. JCV infection is very common in the general population, with roughly 60 percent of adult humans likely to have been exposed to the virus; most people acquire JCV in childhood or adolescence.(1) When immunodeficiency or immunosuppression allows JCV to
‘reactivate’, the virus preferentially migrates to the brain, destroying oligodendrocytes and astrocytes. This lytic infection leads to cognitive and motor dysfunction (the syndrome PML) and is fatal in 50 percent or more of patients. The best approach to treating the disease is to attack the virus in its quiescent state before it enters the brain. IkT-001 blocks viral entry into host cells, and therefore could suppress an active JCV infection in at risk patients prior to virus migration into the brain. There is no currently approved treatment for PML.

About Inhibikase Therapeutics
Founded in 2008, Inhibikase Therapeutics is a private biopharmaceutical company developing novel, small-molecule compounds to treat bacterial and viral infectious diseases through a know-risk development strategy, one that advances a compound against a clinically validated target to proof-of-concept stage on an accelerated timeline. Its pipeline of validated targets utilizes known chemical entities, as well as novel chemical entities, to treat diseases therapeutically and prophylactically. By inhibiting human pathways used by multiple bacteria and viruses for reproduction in the patient, the Company’s compounds block viral and bacterial reproduction with a lower likelihood of stimulating resistance. Inhibikase Therapeutics is also developing a broad-spectrum antiviral specific for enveloped viruses, opening new avenues for vaccine development
and biodefense. For more information, please visit the Company’s website at www.inhibikase.com.

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