Inhibikase Therapeutics Reveals Pre-IND Meeting Results for IkT-001Pro in Pulmonary Arterial Hypertension

Inhibikase Therapeutics, Inc., a clinical-stage pharmaceutical company focused on developing treatments for Parkinson's disease and related disorders, has announced the receipt of final meeting minutes from a pre-IND meeting with the U.S. Food and Drug Administration (FDA). This meeting was pivotal in discussing IkT-001Pro (Pro) as a potential treatment for Pulmonary Arterial Hypertension (PAH). The FDA confirmed that IkT-001Pro would be considered a New Molecular Entity (NME) in the treatment of PAH, which paves the way for exclusivity designations and patent opportunities.

Dr. Milton Werner, President and CEO of Inhibikase, commented on the significance of this designation, emphasizing that it could solidify IkT-001Pro as a branded product with substantial value in addressing a high unmet medical need. Despite the FDA’s agreement that the 505(b)(2) approval pathway is suitable, the NME status affords additional market protection. PAH is a severe, progressive disease that primarily affects women aged 30 to 60 and has a high mortality rate. Although there are existing treatments for PAH symptoms, only one has so far been recognized as disease-modifying. Imatinib, the active component in IkT-001Pro, has previously demonstrated potential as a disease-modifying agent in PAH.

Inhibikase is optimistic that IkT-001Pro could provide a safer and more tolerable therapeutic option compared to existing imatinib treatments. The company is actively seeking partnerships to advance IkT-001Pro into clinical development.

The FDA's final minutes from the pre-IND meeting also supported the company's bridging strategy of using imatinib for blood and gastrointestinal cancers to its application in PAH. The FDA endorsed Inhibikase’s proposed Phase 2/3 clinical trial design but requested a pre-clinical study on the hERG ion channel to compare IkT-001Pro to imatinib due to previously noted cardiovascular risks associated with imatinib. Inhibikase plans to complete this study before submitting the IND application and is also in discussions with potential strategic partners to further the development of IkT-001Pro for PAH.

Pulmonary Arterial Hypertension is a rare disease affecting the pulmonary microvasculature. It can arise spontaneously or due to genetic mutations, drug exposure, environmental toxins, connective tissue diseases, congenital heart disease, or HIV infection. Current treatments mainly address the symptoms of PAH, but the FDA's recent approval of Winrevair® shows that disease-modifying treatments are possible. There are around 30,000 cases of PAH in the U.S., and the global market for PAH was valued at $7.66 billion in 2023, with a projected annual growth rate of 5.4% from 2024 to 2030.

Inhibikase Therapeutics, headquartered in Atlanta, Georgia, with offices in Lexington, Massachusetts, continues to focus on developing therapeutics aimed at Parkinson's disease and related disorders. Their lead program, risvodetinib, targets the treatment of Parkinson's disease both inside and outside the brain, as well as other diseases linked to Abelson Tyrosine Kinases. The company’s pipeline includes a range of therapeutics for Parkinson’s-related disorders, orphan indications like Multiple System Atrophy, and drug delivery technologies for kinase inhibitors, such as IkT-001Pro. Inhibikase's RAMP™ medicinal chemistry program has identified several follow-on compounds that could potentially address other cognitive and motor function diseases of the brain.