Phase 3 trial of experimental PAH treatment enrolling participants
Study of IKT-001 now active at 79 sites globally, with more to come
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A Phase 3 clinical trial testing IKT-001, an experimental oral formulation of imatinib being developed to treat pulmonary arterial hypertension, is now enrolling participants. (Photo by iStock)
A Phase 3 clinical trial testing IKT-001, an experimental oral formulation of imatinib that Inhibikase Therapeutics is developing to treat pulmonary arterial hypertension (PAH), is now enrolling participants at dozens of sites worldwide.
According to Inhibikase, the IMPROVE-PAH trial (NCT07365332) is now active at 79 sites around the world. Additional sites are activating, with a total of 180 sites globally expected to participate in the trial. The company said it has secured regulatory clearance to launch the trial in a total of 28 countries, including the U.S., Canada, Australia, and the U.K. A list of active sites is available on the trial’s registration page.
“In recent weeks we continued to advance our Phase 3 IMPROVE-PAH study, for which we have obtained 28 country regulatory approvals in the U.S. and E.U., with 79 clinical sites activated across a range of countries. We continue to expand our clinical footprint and look forward to additional country and site activations over the coming months,” Mark Iwicki, CEO of Inhibikase, said in a company press release.
Safety update expected in first half of 2027
The IMPROVE-PAH trial is open to adults with various forms of PAH, ages 18 to 75, who meet certain clinical criteria related to body composition and blood flow. Participants can continue to take certain approved PAH therapies, though individuals taking the recently approved injection therapy Winrevair (sotatercept-csrk) are not eligible for participation.
Participants in IMPROVE-PAH will be randomly assigned to receive IKT-001 or a placebo for six months. The first part of the study will include about 140 participants and will test the therapy’s effect on pulmonary vascular resistance, an invasive assessment of how difficult it is for the heart to pump blood through the lungs. The second part of the study will enroll approximately 346 patients and will test the therapy’s effect on exercise capacity, as measured by the distance that participants are able to walk in six minutes.
Inhibikase said it expects to provide an interim safety update from the first part of the trial in the first half of 2027. To help fund the ongoing study, the company recently sold millions of its stock shares to raise $50 million.
“Supported by the $50 million proceeds from the sale of shares to RA Capital in July, Inhibikase is well-positioned to advance IKT-001 toward its potential as the first once-daily oral anti-proliferative offering significant potential benefits to the PAH patient population,” Iwicki said.
Therapy granted orphan drug status
Pulmonary hypertension refers to high pressure in the vessels that carry blood from the heart to the lungs, which strains the heart and causes symptoms like difficulty breathing. PAH is a form of the disease where the cells that line these blood vessels grow abnormally, which causes them to become narrowed.
Imatinib inhibits cell growth. It was originally developed as a cancer therapy, but in theory, its growth-blocking properties could also limit the growth of blood vessel cells that drives PAH progression. Novartis previously developed an oral version of imatinib for PAH, which improved exercise capacity in clinical testing, However, it also caused substantial safety issues, leading Novartis to discontinue development.
IKT-001 contains an inactive precursor molecule that is converted into active imatinib after it gets inside the body, which, according to Inhibikase, may help retain the therapeutic effects of the therapy with a more acceptable safety profile. Earlier this year, the U.S. Food and Drug Administration granted IKT-001 orphan drug status, which aims to incentivize the development of therapies for rare disorders.
Although the IMPROVE-PAH trial is not testing IKT-001 in people taking Winrevair, Inhibikase said it is actively running preclinical tests to explore the effects of using these two therapies in combination. The company also said it is running preclinical experiments to evaluate if IKT-001 might be a useful therapy for other types of pulmonary hypertension besides PAH. Updates on this preclinical work are expected beginning in 2027, according to Inhibikase.
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