Inhibikase Therapeutics Begins Enrollment for PAH Drug Trial

Company's IKT-001 prodrug enters pivotal Phase 3 study for pulmonary arterial hypertension treatment.

Apr. 7, 2026 at 1:55pm

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A translucent, ghostly X-ray image of the internal structure of a human lung, revealing the intricate network of blood vessels and airways in a conceptual representation of pulmonary arterial hypertension.A detailed X-ray view of the human lung, highlighting the complex network of blood vessels and airways affected by pulmonary arterial hypertension.Wilmington Today

Inhibikase Therapeutics, a clinical-stage pharmaceutical company, has announced the enrollment of the first patient in its pivotal Phase 3 IMPROVE-PAH study evaluating IKT-001, a novel oral prodrug of imatinib mesylate, for the treatment of pulmonary arterial hypertension (PAH). The global IMPROVE-PAH trial is a two-part adaptive Phase 3 study designed to assess the efficacy and safety of IKT-001 in PAH patients.

Why it matters

PAH is a progressive, life-threatening disease that affects around 50,000 Americans. Current therapies have limitations, and many patients continue to experience disease progression. The IMPROVE-PAH trial represents a significant step forward, as it is designed to capture outcomes that are most important to patients and clinicians, including improvements in pulmonary vascular resistance, exercise capacity, and time to clinical worsening.

The details

The IMPROVE-PAH trial will be conducted in up to 180 sites globally and consists of two parts. Part A is a double-blind, placebo-controlled study in approximately 140 patients, with the primary endpoint of change in pulmonary vascular resistance (PVR) at Week 24. Part B will seamlessly follow Part A, adopting an identical format but with a primary endpoint of change in 6-minute walk distance (6MWD) at Week 24 in approximately 346 patients. The trial design includes a 12-week dose-titration phase to optimize the dosing of IKT-001, as well as the ability to conduct a sample size re-estimation for Part B based on the findings from Part A.

  • The first patient has been enrolled in the IMPROVE-PAH trial.
  • The IMPROVE-PAH trial is expected to be conducted over approximately 180 sites around the world.

The players

Inhibikase Therapeutics, Inc.

A clinical-stage pharmaceutical company developing therapeutics to modify the course of cardiopulmonary diseases, including pulmonary arterial hypertension (PAH).

IKT-001

Inhibikase's lead product candidate, a prodrug of imatinib mesylate designed to reduce gastrointestinal side effects commonly observed with imatinib.

Harrison Farber, M.D.

Co-director of Pulmonary Hypertension Center and Director of the Pulmonary Embolism Response Team (PERT) at Tufts Medical Center.

J. Wesley McConnell, M.D.

Director of Norton Pulmonary Specialists Pulmonary Hypertension Center in Louisville, Kentucky.

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What they’re saying

“The Phase 3 IMPRES study demonstrated that imatinib may improve key parameters associated with PAH, including exercise capacity and hemodynamics. Now, the Phase 3 IMPROVE-PAH study will help evaluate both hemodynamic and functional improvements, as well as key measures of disease progression, such as time to clinical worsening.”

— Harrison Farber, M.D., Co-director of Pulmonary Hypertension Center and Director of the Pulmonary Embolism Response Team (PERT), Tufts Medical Center

“For patients living with PAH disease progression remains a daily reality even with the therapies we have available today. The IMPROVE-PAH trial represents a meaningful step forward because the trial is designed to capture the outcomes that matter most to patients and clinicians alike. I am proud to be part of this program and optimistic about what it could mean for the future of PAH care.”

— J. Wesley McConnell, M.D., Director of Norton Pulmonary Specialists Pulmonary Hypertension Center in Louisville, Kentucky

What’s next

The IMPROVE-PAH trial is expected to be conducted in up to approximately 180 sites around the world, with Part A and Part B seamlessly transitioning to allow for uninterrupted enrollment.

The takeaway

The initiation of the IMPROVE-PAH trial represents a significant milestone in the development of IKT-001 as a potential new treatment option for patients with pulmonary arterial hypertension, a debilitating and life-threatening condition. The adaptive trial design and focus on key clinical outcomes suggest this study could provide important insights to advance PAH care.