Inhibikase Therapeutics, Inc. (Nasdaq: IKT) has reached a significant milestone in its clinical development program, announcing the completion of enrollment in its Phase 2 '201' trial evaluating risvodetinib for untreated Parkinson's disease. The company expects to report trial results in the fourth quarter of 2024 and plans to discuss Phase 3 protocols with the FDA by year-end.
The 12-week, randomized, double-blind, multi-center, placebo-controlled clinical trial has enrolled all 120 participants across 32 U.S. sites, with plans to randomize a total of 126 patients to accommodate already screened participants. As of June 17, 2024, 69 participants have completed the 12-week dosing period, with only 32 mild and 5 moderate adverse events potentially related to risvodetinib treatment reported. Four participants withdrew before completing the full treatment course.
"The completion of enrollment for the 201 Trial in untreated Parkinson's disease represents a major milestone for Inhibikase," said Dr. Milton H. Werner, President and Chief Executive Officer of Inhibikase. "This achievement reflects the hard work of our team, the success of our proprietary and innovative patient identification and referral service and the commitment of our 32 U.S. study sites to evaluating potential disease-modifying solutions for patients with this debilitating disease."
Pipeline Expansion and Strategic Updates
In parallel with its Parkinson's disease program, Inhibikase is expanding its therapeutic pipeline across multiple areas. Following a pre-IND meeting with the FDA in April 2024, the company has redirected its efforts with IkT-001Pro toward cardiopulmonary disease, specifically Pulmonary Arterial Hypertension (PAH).
The company plans to submit an IND application to the FDA for IkT-001Pro as a PAH treatment early in the third quarter of 2024. The active ingredient in IkT-001Pro, imatinib, has previously demonstrated disease-modifying potential for PAH, and Inhibikase believes its formulation could offer improved safety and tolerability compared to standard imatinib.
"At Inhibikase we have let fundamental scientific discoveries drive identification of new product candidates that could transform the lives of patients," commented Dr. Werner. "In 2025, we anticipate having multiple late-stage ready assets across our therapeutic pipeline."
Multiple System Atrophy Program
Inhibikase is also pursuing development in Multiple System Atrophy (MSA), a Parkinson's-related disorder. The company is seeking grant funding for its '202 Trial' in MSA through The National Institute of Neurological Diseases and Stroke (NINDS) under a new funding mechanism called the Other Transaction Authority (OTA), which begins in June 2024.
Manufacturing Scale-Up
Following a pre-NDA meeting with the FDA in January 2024, Inhibikase is scaling its process development efforts for IkT-001Pro to support late-stage clinical development and NDA batch requirements. These activities include developing new dosage forms to differentiate 001Pro tablets from generic imatinib mesylate, in alignment with FDA feedback.
Strategic Focus on Core Programs
As part of its strategy to focus on late-stage clinical assets in neurodegeneration, cancer, and cardiopulmonary disease, Inhibikase will discontinue development of treatments for Progressive Multifocal Leukoencephalopathy (PML).
About Risvodetinib and Inhibikase's Approach
Risvodetinib is a potent, selective c-Abl inhibitor designed to target the treatment of Parkinson's disease both inside and outside the brain. The company's approach focuses on protein kinase inhibitor therapeutics to potentially modify the course of Parkinson's disease and related disorders.
Inhibikase's multi-therapeutic pipeline includes programs for Parkinson's-related disorders of the brain and GI tract, orphan indications such as Multiple System Atrophy, and drug delivery technologies for kinase inhibitors. The company's RAMP™ medicinal chemistry program has also identified several follow-on compounds to risvodetinib that could potentially address other cognitive and motor function diseases of the brain.
With the 201 Trial now fully enrolled, Inhibikase is positioned to advance its lead program toward late-stage development, potentially offering a new therapeutic option for patients with Parkinson's disease who currently lack disease-modifying treatments.
