Inhibikase seeks FDA feedback about proposed trial for IkT-001Pro

Inhibikase meeting with FDA to discuss its imatinib formulation for PAH

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Inhibikase Therapeutics is meeting with the U.S. Food and Drug Administration (FDA) April 5 to discuss the potential of its experimental formulation of imatinib, dubbed IkT-001Pro, for pulmonary arterial hypertension (PAH).

“Following our … discussion with the FDA related to the path to approval for IkT-001Pro in up to 11 blood and stomach cancers in January, we requested an additional FDA meeting with the Division of Cardiology and Nephrology to discuss Pro as a treatment for Pulmonary Arterial Hypertension,” Milton Werner, PhD, president and CEO of Inhibikase, said in a company press release.

As part of the meeting, Inhibikase will be asking the FDA for feedback on a proposed clinical trial to test the efficacy and safety of IkT-001Pro in PAH. Based on what the agency says, the company will decide whether or not to pursue such a trial.

“We look forward to the FDA’s input on a proposed late-stage trial design and to the FDA’s viewpoint on if Pro could be treated as a branded product for this indication,” Werner said.

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Imatinib originally developed to treat cancer

Originally created as a cancer therapy, imatinib is a drug that works to stop cell growth. In PAH, it’s thought the therapy may limit the abnormal growth of blood vessel cells that drives disease progression.

An oral formulation of the drug called imatinib mesylate, developed by Novartis, was previously tested in PAH trials. Findings indicated the therapy had good efficacy for improving patients’ exercise ability. In fact, the therapeutic effect of imatinib was on par with results seen in trials of the recently approved PAH treatment Winrevair (sotatercept).

However, even though efficacy data were promising, oral imatinib mesylate also caused substantial side effects, leading many patients to discontinue treatment. Ultimately, the therapy was deemed too toxic for use in clinical practice, and Novartis decided to stop developing it for PAH.

According to Inhibikase, the novel formulation of imatinib in IkT-001Pro may be able to deliver similar efficacy with less toxicity, though the company notes this idea has yet to be proven in trials.

“Previous clinical research with imatinib, the active ingredient in Pro, was shown to be potentially disease-modifying for PAH, however, the adverse event profile of imatinib mesylate in this patient population could not support approval by the FDA,” Werner said. “We believe that Pro may be a be a safer and better tolerated therapeutic option for imatinib treatment in PAH.”

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