Inhibikase Therapeutics Advancing IKT-001 to Global Phase 3 Study in Pulmonary Arterial Hypertension
Inhibikase Therapeutics (Nasdaq: IKT) plans to initiate a global pivotal Phase 3 study of IKT-001 in pulmonary arterial hypertension in Q1 2026. The adaptive two-part IMPROVE-PAH trial expects Part A as a double-blind, placebo-controlled study in 140 patients with primary endpoint PVR at Week 24, and Part B in 346 patients with primary endpoint 6MWD at Week 24. The design includes a 12-week dose-titration, uninterrupted enrollment between parts, and potential sample-size re-estimation; the study may run in about 180 global sites. IKT-001 is described as a prodrug of imatinib with expected improved gastrointestinal tolerability and a stated potential to accelerate an NDA timeline by ~3 years.
Inhibikase Therapeutics (Nasdaq: IKT) prevede di avviare un global pivotal Phase 3 studio di IKT-001 nell’ipertensione arteriosa polmonare nel Q1 2026. Il trial adaptativo in due parti IMPROVE-PAH prevede Part A come studio in doppio cieco controllato con placebo su 140 pazienti con endpoint primario PVR at Week 24, e Part B su 346 pazienti con endpoint primario 6MWD at Week 24. Il disegno include una dosaggio-titolazione di 12 settimane, arruolamento ininterrotto tra le parti e una possibile re-estimazione della dimensione del campione; lo studio potrebbe essere condotto in circa 180 siti globali. IKT-001 è descritto come prodrug di imatinib con una prevista maggiore tollerabilità gastrointestinale e un potenziale dichiarato di accelerare la timeline NDA di circa 3 anni.
Inhibikase Therapeutics (Nasdaq: IKT) planea iniciar un ensayo pivotal global Phase 3 de IKT-001 en la hipertensión arterial pulmonar en Q1 de 2026. El ensayo adaptativo en dos partes IMPROVE-PAH espera Part A como un estudio doble ciego y con placebo en 140 pacientes con endpoint primario PVR at Week 24, y Part B en 346 pacientes con endpoint primario 6MWD at Week 24. El diseño incluye una titulación de dosis de 12 semanas, inscripción ininterrumpida entre las partes y una posible reestimación del tamaño de la muestra; el estudio podría realizarse en aproximadamente 180 sitios globales. IKT-001 se describe como un profármico de imatinib con una tolerabilidad gastrointestinal esperada mejorada y un posible potencial para acelerar la cronología de NDA en ~3 años.
Inhibikase Therapeutics (Nasdaq: IKT)는 IKT-001에 대한 전 세계적인 결정적 Phase 3 연구를 2026년 1분기에 시작할 계획입니다. 적응형 двух 파트 IMPROVE-PAH 시험은 Part A를 140명 환자가 참여하는 위약 대조 이중 맹검 연구로, 주요 종결점은 24주 차 PVR이며, Part B는 346명의 환자에서 24주 차 6MWD를 주요 종결점으로 설정합니다. 설계에는 12주 용량 증량/감량, 파트 간 비중단적 등록, 표본 크기 재추정 가능성이 포함되며 연구는 전 세계 약 180개 사이트에서 진행될 수 있습니다. IKT-001은 이미틴ib의 프로드럭으로서 위장관 내 약물 허용성이 향상될 것으로 기대되며 NDA 일정이 약 3년 단축될 수 있다는 잠재력을 제시합니다.
Inhibikase Therapeutics (Nasdaq: IKT) prévoit de lancer une étude pivot globale Phase 3 de l’IKT-001 dans l’hypertension artérielle pulmonaire au 1er trimestre 2026. L’essai adaptatif en deux parties IMPROVE-PAH prévoit Part A comme étude en double aveugle contrôlée par placebo chez 140 patients avec l’objectif principal PVR at Week 24, et Part B chez 346 patients avec l’objectif principal 6MWD at Week 24. Le design comprend une titration de dose sur 12 semaines, un recrutement continu entre les parties et une possible ré-estimation de la taille de l’échantillon; l’étude pourrait être menée dans environ 180 sites mondiaux. LKI-001 est décrit comme un prodrogue de l’imatinib avec une tolérance gastro-intestinale attendue meilleure et un potentiel déclaré d’accélérer le calendrier NDA d’environ 3 ans.
Inhibikase Therapeutics (Nasdaq: IKT) plantiert, im 1. Quartal 2026 eine globale pivotal Phase-3-Studie von IKT-001 zur pulmonalen arteriellen Hypertonie zu starten. Die adaptive Zwei-Teil-IMPORVE-PAH-Studie erwartet, dass Teil A eine doppelblinde, placebokontrollierte Studie mit 140 Patienten hat und der primäre Endpunkt PVR at Week 24 ist, und Teil B bei 346 Patienten mit dem primären Endpunkt 6MWD at Week 24. Das Design umfasst eine 12-Wochen-Dosis-Titration, eine ununterbrochene Rekrutierung zwischen den Teilen und eine potenzielle Stichprobengrößen-Neinschätzung; die Studie könnte weltweit in rund 180 Standorten durchgeführt werden. IKT-001 wird als Prodrug von Imatinib beschrieben, mit voraussichtlich verbesserter gastrointestinale Verträglichkeit und dem angegebenen Potenzial, den NDA-Zeitplan um ca. 3 Jahre zu beschleunigen.
Inhibikase Therapeutics (Nasdaq: IKT) تخطط لبدء دراسة عالمية محورية من المرحلة Phase 3 لـ IKT-001 في ارتفاع ضغط الدم الرئوي الشرياني في الربع الأول 2026. الاختبار التكيفي ذو جزأين IMPROVE-PAH يتوقع Part A كدراسة مزدوجة التعمية خاضعة للدواء الوهمي لـ 140 مريضًا مع نقطة النهاية الأساسية PVR at Week 24، و Part B في 346 مريضًا مع نقطة النهاية الأساسية 6MWD at Week 24. التصميم يشمل معدلة جرعة لمدة 12 أسبوعًا، وتسجيل مستمر بين الجزأين، وإعادة تقدير محتملة لحجم العينة؛ قد تبلغ الدراسة حوالي 180 موقعًا عالميًا. يوصف IKT-001 بأنهدواء خام من إماتينيب مع تحمل معوي مع تحسين متوقع ووجود إمكانية لتسريع جدول NDA بنحو 3 سنوات.
- Phase 3 targeted to start in Q1 2026
- Adaptive two-part design with sample-size re-estimation
- Part A: 140 patients; primary endpoint PVR at Week 24
- Part B: 346 patients; primary endpoint 6MWD at Week 24
- Planned conduct in ~180 global sites
- Includes 12-week dose-titration to reach higher tolerable doses
- Prior imatinib IMPRES study had high discontinuations impacting results
- IKT-001 remains investigational; efficacy and tolerability unproven in Phase 3
Insights
Advancing IKT-001 directly into a global adaptive Phase 3 shortens the regulatory path and signals meaningful clinical development progress.
The company plans to start the two‑part adaptive Phase 3 study IMPROVE-PAH in
Dependencies and risks remain clear and contained within the disclosed facts: the plan relies on successful execution of the adaptive design, enrollment across up to ~180 global sites, and the assumption that IKT-001 will lower discontinuations versus prior imatinib studies. Watch for initiation milestones in
Phase 3 Study initiating in First Quarter of 2026
Single Pivotal Study Accelerates Potential FDA Approval
Timeline by Approximately 3 Years
BOSTON and ATLANTA, Nov. 20, 2025 (GLOBE NEWSWIRE) -- Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (“Inhibikase” or “Company”), a clinical-stage pharmaceutical company developing therapeutics to modify the course of cardiopulmonary diseases namely, Pulmonary Arterial Hypertension (“PAH”), today announced that it expects to advance IKT-001 to a global pivotal Phase 3 clinical study in PAH. The Phase 3 study, named IMPROVE-PAH (IKT-001 for Measuring Pulmonary Vascular Resistance and Outcome Variables in a Phase 3 Evaluation of PAH), is expected to be initiated in the first quarter of 2026.
IKT-001 is an investigational novel pro-drug of imatinib mesylate (“imatinib”). Imatinib is an anti-proliferative tyrosine kinase inhibitor, TKI, with potential best-in-class improvements in pulmonary vascular resistance (“PVR”) and 6-minute walk distance (“6MWD”) of 45 meters(1) based on Phase 3 IMPRES and Phase 2 studies(2). Despite the IMPRES Phase 3 study of imatinib demonstrating improved exercise capacity and hemodynamics in patients with advanced PAH, high discontinuations impacted the results. IKT-001 is a prodrug of imatinib which is engineered to realize the potential of imatinib in PAH and lower discontinuations in the forthcoming Phase 3 study.
The Company previously planned to initiate a Phase 2b study in 150 subjects in PAH prior to advancing to a pivotal Phase 3 study. However, the Company submitted a Type C Meeting request to the U.S. Food & Drug Administration (“FDA”) to, among other things, obtain feedback on an immediate transition to a pivotal Phase 3 study design.
Following receipt from the FDA of the Written Response from the Type C interaction, the Company now plans to initiate a two-part adaptive Phase 3 study. We expect Part A of IMPROVE-PAH will be a double blind, placebo-controlled study in 140 patients with a primary endpoint of PVR at Week 24. We expect Part B will adopt an identical format to Part A except the primary endpoint will be 6MWD at Week 24 in 346 patients. We believe this adaptive Phase 3 study design has important advantages including; (1) permitting a 12-week dose-titration phase designed to get patients to the highest tolerable dose of IKT-001; (2) uninterrupted enrollment between Part A and Part B; and (3) the ability to, if necessary, undertake a sample size re-estimation for Part B based on Part A findings. Given the Company was well-advanced in initiating the previous Phase 2b study design, the Company expects to initiate IMPROVE-PAH in the first quarter of 2026, with this study expected to be conducted in up to approximately 180 sites around the world.
“Our revised study plan for IKT-001 in PAH permits a single pivotal study format and thereby has the potential to advance our timeline to Phase 3 topline data readout and a potential NDA filing by approximately three years. We are excited about the recent progress we have made,” said Mark Iwicki, Chief Executive Officer of Inhibikase. “Importantly, the written responses from FDA will allow the Company to adopt a 12-week dose-titration phase which we believe will potentially allow patients to get to the higher tolerable doses where imatinib has previously demonstrated the greatest benefit in exercise capacity and hemodynamics. With over two decades of imatinib clinical experience, together with IKT-001’s expected improved gastrointestinal tolerability profile and adaptive study design for IMPROVE-PAH, we believe this supports a potential higher probability of success and we are looking forward to study initiation next quarter.”
About Inhibikase (www.inhibikase.com)
Inhibikase Therapeutics, Inc. (Nasdaq: IKT) is a clinical-stage pharmaceutical company developing therapeutics to modify the course of cardiopulmonary diseases namely, PAH, that arise from aberrant signaling through the Abelson Tyrosine Kinase, and type III receptor tyrosine kinases including platelet derived growth factor receptors and c-KIT. Our lead product candidate is IKT-001, a prodrug of imatinib mesylate, for PAH which is an orphan indication. PAH is a progressive, life-threatening disease characterized by pulmonary vascular remodeling and elevated pulmonary vascular resistance that affects approximately 50,000 Americans.
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Investors and others should note that the Company announces material financial information to investors using its investor relations website, press releases, SEC filings and public conference calls and webcasts. The Company intends to also use LinkedIn as a means of disclosing information about the Company, its services and other matters and for complying with its disclosure obligations under Regulation FD.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking terminology such as “believes,” “expects,” “may,” “will,” “should,” “anticipates,” “plans,” or similar expressions or the negative of these terms and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements that express the Company’s intentions, beliefs, expectations, strategies, predictions or any other statements related to the potential effects of IKT-001; expectations regarding the Company’s Phase 3 trial of IKT-001 in PAH, including design, timing of initiation and its impact on the Company’s timeline; and the Company’s future activities, or future events or conditions. These forward-looking statements are based on Inhibikase’s current expectations and assumptions. Such statements are subject to certain risks and uncertainties, which could cause Inhibikase’s actual results to differ materially from those anticipated by the forward-looking statements. Important factors that could cause actual results to differ materially from those in the forward-looking statements include our ability to initiate and execute a global Phase 3 trial to evaluate IKT-001 as a treatment for PAH, as well as such other factors that are included in our periodic reports on Form 10-K and Form 10-Q that we file with the U.S. Securities and Exchange Commission. Any forward-looking statement in this release speaks only as of the date of this release. Inhibikase undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws.
Contacts:
Investor Relations:
Michael Moyer
LifeSci Advisors
mmoyer@lifesciadvisors.com
(1) Placebo-adjusted 6MWD improvement of 45 meters at week 24 in patients at 400 mg for >
FAQ
When will Inhibikase (IKT) start the Phase 3 IMPROVE-PAH study?
What is the design of IKT-001's IMPROVE-PAH Phase 3 trial (IKT)?
How could the IMPROVE-PAH design affect Inhibikase's (IKT) regulatory timeline?
What is IKT-001 and how does it differ from imatinib (IKT)?
What key operational elements are included in IMPROVE-PAH (IKT)?
How many sites will participate in the IMPROVE-PAH Phase 3 study for IKT?
