FDA OK’s Phase 2/3 trial design for experimental imatinib IkT-001Pro
FDA agreed Inhibikase connected imatinib’s use in cancers, PAH
The U.S. Food and Drug Administration (FDA) has supported Inhibikase Therapeutics’ Phase 2/3 trial of its experimental formulation of imatinib, called IkT-001Pro, for pulmonary arterial hypertension (PAH), the company reports.
In a meeting, the FDA asked the company to complete a preclinical study in cells to compare IkT-001Pro to imatinib. Cardiovascular abnormalities have been reported with imatinib, a molecule that’s been explored for PAH. The FDA agreed Inhibikase had shown a connection between imatinib’s use in blood and gastrointestinal cancers and PAH, according to the company.
Inhibikase intends to complete the experiments before it formally seeks permission to start a clinical trial.
“Following our … discussion with the FDA related to the chemical entity status and path to approval for Pro [IkT-001Pro] as a treatment for PAH, the FDA confirmed in final meeting minutes that Pro would be considered a New Molecular Entity (NME) in PAH,” Milton Werner, PhD, Inhibikase’s president and CEO, said in a company press release. “If approved, IkT-001Pro could be a branded product with all the value drivers of a novel treatment for an indication of high unmet medical need.”
PAH is caused by the narrowing of pulmonary arteries, the blood vessels that transport blood through the lungs, leading to high blood pressure and causing the heart to work harder to pump blood.
“PAH is a rapidly progressing, often fatal disease affecting primarily women between the ages of 30 and 60. There are several products on the market that address symptoms of the disease, but only one disease-modifying therapy has been approved to date,” Werner said.
Imatinib and IkT-001Pro
Imatinib is designed to stop cell growth and was originally developed as a cancer therapy. In PAH, it’s thought to limit the abnormal growth of blood vessel cells, which contributes to the disease’s progression.
An oral formulation of the therapy, called imatinib mesylate, developed by Novartis, improved PAH patients’ exercise ability in clinical trials. Its therapeutic effects were equivalent to the recently approved disease-modifying PAH treatment Winrevair (sotatercept). Significant side effects led Novartis to halt imatinib’s development for PAH, however.
IkT-001Pro is an imatinib precursor, that is, a molecule with little or no pharmacological activity that’s converted into imatinib in the body. It was generated using the company’s Re-engineering Approach with Metabolism Preserved (RAMP) technology and leverages the clinical experience gained from approved or clinically evaluated small molecule therapies, Inhikibase said.
The use of RAMP has the potential to enhance drug absorption and delivery, therefore enabling dose reduction, while improving the therapy’s safety and reducing dose-related side effects, according to the company.
“In previous clinical trial work, imatinib, the active ingredient in Pro, demonstrated that it could be disease-modifying for PAH. We believe that Pro has the potential to be a safer and better tolerated therapeutic option for imatinib treatment and are actively pursuing partnership discussions to advance Pro into clinical development,” Werner said.
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