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IKT-001

Phase 3

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

IKT-001 development status

Highest phase Phase 3
Registered trials 2 across 1 sponsor since Jan 2026

IKT-001 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 Trials2
Total Enrollment970

FDA Designations

ORPHAN_DRUG

IKT-001 clinical trials

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 cardiovascular condition. It is in Phase 3 clinical development and has received an orphan drug designation from the FDA. It is not yet approved for commercial use.

What does IKT-001 target?

IKT-001 targets BCR-ABL, a protein. By acting on this molecular target, the drug is being studied as a potential therapy for pulmonary arterial hypertension. It is a small molecule therapeutic candidate developed by Inhibikase Therapeutics.

Who is developing IKT-001?

IKT-001 is being developed by Inhibikase Therapeutics, Inc., which trades under the ticker IKT. The company is advancing the drug through Phase 3 clinical development for pulmonary arterial hypertension, a cardiovascular indication.

What phase is IKT-001 in?

IKT-001 is in Phase 3 clinical development for pulmonary arterial hypertension. It is an investigational therapy and has not been approved by the FDA. The program includes two active Phase 3 trials with a combined planned enrollment of 970 participants.

What clinical trials is IKT-001 in?

IKT-001 is being evaluated in two Phase 3 trials. NCT07365332 is an adaptive program in pulmonary arterial hypertension that is currently recruiting across multiple countries. NCT07632898 is an open-label study in PAH that is not yet recruiting. Both trials enroll adults aged 18 and older.

Is IKT-001 FDA approved?

IKT-001 is not FDA approved. It remains an investigational drug in Phase 3 clinical development for pulmonary arterial hypertension. The FDA has granted it an orphan drug designation, which is a status that supports development of therapies for rare diseases.