Inhibikase Therapeutics Receives FDA Orphan Drug Designation for Risvodetinib for the Treatment of Multiple System Atrophy

BOSTON and ATLANTA, Oct. 04, 2023 (GLOBE NEWSWIRE) -- Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (Inhibikase or Company), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Parkinson's disease ("PD"), Parkinson's-related disorders and other diseases of the Abelson Tyrosine Kinases, today announced that risvodetinib (IkT-148009) has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of Multiple System Atrophy (MSA).

“We are pleased to receive orphan drug designation for risvodetinib in MSA underscoring the high unmet medical need in this patient population. There are currently no approved symptomatic or disease-modifying therapies for MSA on the market. MSA is an aggressive form of Parkinsonism, leading to death four times faster than a diagnosis of Parkinson’s disease and we believe that risvodetinib could potentially slow or halt the progression of disease. As we look ahead, we are advancing our ongoing animal model studies of risvodetinib to determine its therapeutic potential to block progression and correct functional loss in MSA. These studies will form the basis of our planned Phase 2 clinical study and we look forward to providing further updates on the potential timing of the Phase 2 trial in the coming quarters. We believe proof that risvodetinib is clinically beneficial in MSA will form a basis for potential success in other forms of Parkinsonism,” noted Dr. Milton Werner, Chief Executive of Inhibikase Therapeutics.

In 2022, Inhibikase published data demonstrating that c-Abl is activated in the brains of patients diagnosed with MSA and that c-Abl modifies alpha-synuclein aggregates as part of the disease process. In March 2023, the Company received IND clearance for its planned Phase 2 clinical studies in MSA and presented preclinical data in August 2023, showing that risvodetinib could be therapeutically active in models of disease, at the Movement Disorder Society Congress in Copenhagen, Denmark.

The FDA’s Orphan Drug Designation program provides orphan status to drugs or biologics intended for the prevention, diagnosis, or treatment of diseases that affect fewer than 200,000 people in the United States. Sponsors of medicines that are granted Orphan Drug Designation are entitled to certain incentives and regulatory assistance, including tax credits for qualified clinical trials, prescription drug user-fee exemptions, and potential seven-year marketing exclusivity upon FDA approval.