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IKT-001

Phase 3

Pulmonary Arterial Hypertension | Small molecule | Cardiovascular |Inhibikase Therapeutics, Inc.|Last Updated: Sep 2, 2026

Target and mechanism

Molecular targetBCR-ABL
Target classProtein
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment486

FDA Designations

ORPHAN_DRUG

Clinical trial landscape

IKT-001 · 2 trials · 2 indications

Phase 3 2
NCT07632898Open-Label Study of IKT-001 in Pulmonary Arterial Hypertension (PAH)Pulmonary Arterial Hypertension (PAH)
NOT YET_RECRUITING484 Analytics
NCT07365332An Adaptive Program of IKT-001 in Pulmonary Arterial Hypertension (PAH)Pulmonary Arterial Hypertension
RECRUITING486 Analytics
PHASE3NOT YET_RECRUITING
Open-Label Study of IKT-001 in Pulmonary Arterial Hypertension (PAH)
Pulmonary Arterial Hypertension (PAH)Unlock trial analytics
PHASE3RECRUITING
An Adaptive Program of IKT-001 in Pulmonary Arterial Hypertension (PAH)
Pulmonary Arterial HypertensionUnlock trial analytics

Study Endpoints

Primary Endpoints

Incidence of Treatment-emergent adverse events (TEAEs), serious TEAEs, and TEAEs of special interest
From baseline to end of study (up to 2 years or availability of commercial product)

The number of participants who experience a TEAE, serious TEAE or TEAE of special interest will be reported.

[Part A] To evaluate the effect on Pulmonary Vascular Resistance (PVR) in participants with WHO Group 1 PAH treated with IKT-001 compared to placebo
Baseline to Week 24

Change in Pulmonary Vascular Resistance (PVR)

[Part B] To characterize the effects of IKT-001 on symptoms and characteristics of Pulmonary Arterial Hypertension compared to placebo
Baseline to Week 24

Change in 6-minute walk distance (6MWD)

Secondary Endpoints

Change from baseline in 6-minute walk distance (6MWD)
From baseline to end of study (up to 2 years or availability of commercial product)
Change from baseline in NT-proBNP
From baseline to end of study (up to 2 years or availability of commercial product)
To characterize the effects of IKT-001 on WHO Functional Class
Baseline up to Week 48
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
IKT-001EXPERIMENTALIKT-001 tablets for PO administration
PlaceboPLACEBO_COMPARATORMatching placebo to IKT-001 tablets for PO administration

Interventions

NameTypeDescription
IKT-001DRUGIKT-001 tablets for PO administration
PlaceboDRUGPlacebo to IKT-001 tablets for PO administration
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Eligibility Criteria

Age Range18 Years to 75 Years
SexALL
Healthy VolunteersNo

Inclusion Criteria: * Capable of giving signed informed consent. * Participants who have completed Study IKT-001-201 or any other Phase 2 or 3 controlled clinical study with IKT-001. Exclusion Criteria: * Any of the following clinical laboratory values at screening: * ALT or AST levels \>3× th...

Countries:United StatesArgentinaAustraliaBelgiumCanadaFranceGermanyItalyNetherlandsNew ZealandPortugalSouth KoreaSpainUnited Kingdom
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Recent Changes (Last 90 Days)

LOWSep 2, 2026NCT07365332lastUpdatePostDate: changed
LOWSep 2, 2026NCT07365332lastUpdatePostDate: changed
LOWSep 2, 2026NCT07365332lastUpdatePostDate: changed

Frequently asked questions about IKT-001

What is IKT-001 used for?

IKT-001 is an investigational small molecule being developed for the treatment of Pulmonary Arterial Hypertension (PAH), a form of high blood pressure in the arteries of the lungs. It is currently in Phase 3 clinical development and has not yet been approved by regulatory authorities.

What does IKT-001 target?

IKT-001 targets BCR-ABL, a fusion protein kinase. By inhibiting this target, the drug is being studied for its potential effects in Pulmonary Arterial Hypertension (PAH). The drug is currently in Phase 3 clinical trials to evaluate its safety and efficacy in patients with this condition.

Who makes IKT-001?

IKT-001 is being developed by Inhibikase Therapeutics, Inc., a biopharmaceutical company listed on the stock exchange under the ticker symbol IKT. The company is conducting Phase 3 clinical trials to evaluate the drug for the treatment of Pulmonary Arterial Hypertension (PAH).

What phase is IKT-001 in?

IKT-001 is currently in Phase 3 clinical development for Pulmonary Arterial Hypertension (PAH). It is an investigational drug and has not been approved by the FDA. The drug has received Orphan Drug designation, which is granted to therapies intended for rare diseases.

What clinical trials is IKT-001 in?

IKT-001 is being studied in two Phase 3 clinical trials. The first trial, NCT07365332, is an adaptive study in Pulmonary Arterial Hypertension (PAH) that is currently recruiting participants across multiple countries. The second trial, NCT07632898, is an open-label study in PAH that has not yet started recruiting.

Is IKT-001 the same as imatinib?

IKT-001 is a distinct investigational drug developed by Inhibikase Therapeutics. While it targets BCR-ABL, a kinase also targeted by imatinib, IKT-001 is being studied specifically for Pulmonary Arterial Hypertension (PAH) and is not the same compound as imatinib. It is currently in Phase 3 clinical trials.