Inhibikase Therapeutics Receives FDA Orphan Drug Designation For IKT-001 In Pulmonary Arterial Hypertension

Inhibikase Therapeutics, Inc., a clinical-stage pharmaceutical company developing therapies for pulmonary arterial hypertension (PAH), has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead product candidate, IKT-001, for the treatment of pulmonary arterial hypertension. IKT-001 is a prodrug of imatinib mesylate and is being developed as a potential once-daily oral therapy for patients living with this rare and progressive disease.

The FDA's decision represents an essential regulatory milestone for the Company as it advances the clinical development of IKT-001. Orphan Drug Designation is intended to encourage the development of treatments for rare diseases that affect fewer than 200,000 people in the United States. Pulmonary arterial hypertension is a serious, life-threatening condition characterized by increased blood pressure in the arteries of the lungs, leading to reduced quality of life and, if left untreated, progressive heart and lung complications.

Mark Iwicki, Chief Executive Officer of Inhibikase Therapeutics, said the designation highlights the significant unmet medical need among the approximately 50,000 people in the United States living with pulmonary arterial hypertension. He explained that the disease is driven by the abnormal growth of vascular cells within the lungs and remains associated with poor patient outcomes despite currently available therapies. Iwicki also pointed to recently presented preclinical data for IKT-001 at the American Thoracic Society International Conference, which demonstrated improvements in pulmonary vascular and hemodynamic markers associated with PAH. In addition, the data suggested a lower potential for gastrointestinal toxicity compared with imatinib mesylate, supporting the Company's belief that IKT-001 could offer meaningful clinical benefits as a once-daily oral treatment for patients with PAH.

According to the FDA, the Orphan Drug Designation applies to the active ingredient of IKT-001, imatinib, rather than to a specific formulation of the drug. The designation provides several potential development incentives designed to support therapies targeting rare diseases. These include eligibility for tax credits related to qualified clinical trial expenses, exemption from certain FDA application fees, and the possibility of receiving seven years of market exclusivity in the United States if the therapy ultimately receives regulatory approval.

The FDA's Office of Orphan Products Development grants Orphan Drug Designation to investigational therapies intended to treat rare medical conditions affecting fewer than 200,000 patients in the United States. By receiving this designation, Inhibikase Therapeutics gains additional regulatory support as it continues the development of IKT-001 with the goal of providing a new treatment option for patients with pulmonary arterial hypertension.