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EIK1001

Phase 2

Advanced Melanoma | Small molecule | Oncology |Eikon Therapeutics, Inc.|Last Updated: Sep 2, 2026

Target and mechanism

Molecular targetTLR7/8
Target classToll-like Receptor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindACTIVE_CONTROLLEDDMC
Total Trials1
Total Enrollment740

FDA Designations

No designations recorded

Clinical trial landscape

EIK1001 · 3 trials · 4 indications

Phase 2 3
NCT07365319A Safety and Efficacy Study of EIK1001 in Combination With Pembrolizumab and Chemotherapy in Participants With Stage 4 Non-Small Cell Lung Cancer.Non Small Cell Lung Cancer (Squamous or Non Squamous)
RECRUITING750 Analytics
NCT06697301Safety and Efficacy of EIK1001 in Combo With Pembro Versus Placebo and Pembro as First-Line Therapy in Patients With Advanced Melanoma.Advanced Melanoma
RECRUITING740 Analytics
NCT06246110A Phase 2 Study of EIK1001 in Combo With Pembrolizumab and Chemotherapy in Patients With Stage 4 NSCLCNSCLC
ACTIVE NOT_RECRUITING70 Analytics
PHASE2RECRUITING
A Safety and Efficacy Study of EIK1001 in Combination With Pembrolizumab and Chemotherapy in Participants With Stage 4 Non-Small Cell Lung Cancer.
Non Small Cell Lung Cancer (Squamous or Non Squamous)Unlock trial analytics
PHASE2RECRUITING
Safety and Efficacy of EIK1001 in Combo With Pembro Versus Placebo and Pembro as First-Line Therapy in Patients With Advanced Melanoma.
Advanced MelanomaUnlock trial analytics
PHASE2ACTIVE NOT_RECRUITING
A Phase 2 Study of EIK1001 in Combo With Pembrolizumab and Chemotherapy in Patients With Stage 4 NSCLC
NSCLCUnlock trial analytics

Study Endpoints

Primary Endpoints

Progression-free survival (PFS)
Through study completion, up to 6 years

Progression-free survival (PFS) is defined as the time from the first dose of the study medication to the first documented disease progression according to RECIST 1.1 by BICR, or death due to any cause, whichever occurs first

Overall survival (OS)
Through study completion, up to 10 years

OS defined as the time from the first dose of study medication to death due to any cause

Objective Response (OR)
Through study completion, up to 6 years

Objective response (OR) is defined as participants who demonstrate complete response (CR) or partial response (PR) by RECIST 1.1 as assessed by the Investigator, adverse events (AEs), and discontinuation of study intervention due to an AE (Dose Optimization Only).

Progression Free Survival (PFS)
up to 5 years

Progression-free survival (PFS) is defined as the time from the date of randomization to documented progressive disease per RECIST 1.1 by BICR or death due to any cause, whichever occurs first.

Objective Response (OR) (Dose Optimization Only)
up to 5 years

Objective Response (OR; defined as participants who demonstrate confirmed complete response \[CR\] or partial response \[PR\] by Response Evaluation Criteria in Solid Tumors \[RECIST\] version 1.1 as assessed by the Investigator) (Dose Optimization Only).

Adverse Events (AEs) (Dose Optimization Only)
up to 2.5 years

Adverse events (AEs), and discontinuation of study treatment due to an AE (Dose Optimization Only). An AE is defined as any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment.

Percentage of participants with safety event during treatment
Up to 2 years

Defined AEs that occur during treatment and are deemed to be related EIK1001 or combination with SOC or due to disease progression or toxicity.

Secondary Endpoints

Objective response (OR)
Up to 6 years
Duration of response (DOR)
Up to 6 years
Progression-free survival (PFS) by Investigator
Up to 6 years
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Arm 1 (Placebo in Combination with SOC)PLACEBO_COMPARATORParticipants in this arm will receive EIK1001 Placebo + Standard of Care (SOC).
Arm 2 (EIK1001 in Combination with SOC)EXPERIMENTALParticipants in this arm will receive EIK1001 (Selected Dose 1) + Standard of Care (SOC).
Arm 3 (EIK1001 in Combination with SOC)EXPERIMENTALParticipants in this arm will receive EIK1001 (Selected Dose 2) + Standard of Care (SOC).
Arm 1ACTIVE_COMPARATORParticipants in this arm will receive Placebo and Standard of Care (Pembrolizumab).
Arm 2EXPERIMENTALParticipants in this arm will receive EIK1001 (selected dose 1) + Standard of Care (Pembrolizumab).
Arm 3EXPERIMENTALParticipants in this arm will receive EIK1001 (selected dose 2) + Standard of Care (Pembrolizumab).
Cohort A - Participants with non-squamous NSCLCEXPERIMENTALParticipants in this arm will receive EIK1001 + Standard of Care (SOC).
Cohort B - Participants with squamous NSCLCEXPERIMENTALParticipants in this arm will receive EIK1001 + Standard of Care (SOC).

Interventions

NameTypeDescription
EIK1001DRUGEIK1001 is a Toll like receptor 7/8 (TLR 7/8) dual agonist
Pembrolizumab (KEYTRUDA®)DRUGPD-1 inhibitor
PlaceboDRUGPlacebo control
Paclitaxel + CarboplatinDRUGSOC Chemotherapy for squamous NSCLC
Nab-paclitaxel + CarboplatinDRUGSOC Chemotherapy for squamous NSCLC
Pemetrexed + Cisplatin /CarboplatinDRUGSOC Chemotherapy for non-squamous NSCLC
Pembrolizumab (KEYTRUDA® )DRUGPembrolizumab is a PD-1 inhibitor.
PembrolizumabDRUGPD-1 inhibitor
PaclitaxelDRUGChemotherapy
PemetrexedDRUGChemotherapy
CarboplatinDRUGChemotherapy
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites23

Key Inclusion Criteria: 1. Participant must be ≥ 18 years old at the time of signing the informed consent. 2. Participant has a life expectancy of at least 3 months. 3. Participant has histologically or cytologically confirmed Stage 4 NSCLC predominately squamous or non-squamous) and is considered ...

Countries:United StatesAustraliaSpainAustriaBelgiumCanadaCzechiaDenmarkFinlandFranceGermanyHungaryIsraelItalyNew ZealandNorwayPolandPortugalSerbiaSouth AfricaSouth KoreaSwedenSwitzerlandUnited Kingdom
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Recent Changes (Last 90 Days)

LOWSep 2, 2026NCT07365319lastUpdatePostDate: changed
LOWSep 2, 2026NCT07365319lastUpdatePostDate: changed
LOWSep 2, 2026NCT07365319lastUpdatePostDate: changed
LOWAug 20, 2026NCT07365319lastUpdatePostDate: changed
LOWAug 20, 2026NCT07365319lastUpdatePostDate: changed
LOWAug 20, 2026NCT07365319lastUpdatePostDate: changed
LOWAug 20, 2026NCT07365319lastUpdatePostDate: changed
LOWAug 6, 2026NCT07365319lastUpdatePostDate: changed
LOWAug 6, 2026NCT07365319lastUpdatePostDate: changed
LOWJul 15, 2026NCT07365319startDate: changed
LOWJul 15, 2026NCT07365319startDate: changed

Frequently asked questions about EIK1001

What is EIK1001 used for?

EIK1001 is an investigational small molecule being studied for the treatment of advanced melanoma and non-small cell lung cancer (NSCLC), including squamous and non-squamous subtypes. It is currently in Phase 2 clinical development for these oncology indications.

What does EIK1001 target?

EIK1001 targets TLR7/8, which are toll-like receptors involved in immune activation. By targeting these receptors, the drug is designed to modulate the immune system's response against cancer cells.

Who makes EIK1001?

EIK1001 is being developed by Eikon Therapeutics, Inc., a biopharmaceutical company. The company's stock is traded under the ticker symbol EIKN.

What phase is EIK1001 in?

EIK1001 is currently in Phase 2 clinical trials. It is an investigational drug and has not yet been approved by regulatory authorities. The ongoing trials are evaluating its safety and efficacy in patients with advanced melanoma and NSCLC.

What clinical trials is EIK1001 in?

EIK1001 is being studied in three Phase 2 trials: NCT06246110 combines EIK1001 with pembrolizumab and chemotherapy in stage 4 NSCLC; NCT06697301 compares EIK1001 plus pembrolizumab versus placebo plus pembrolizumab in advanced melanoma; and NCT07365319 evaluates EIK1001 with pembrolizumab and chemotherapy in stage 4 NSCLC.

Is EIK1001 the same as other drugs?

EIK1001 is a distinct investigational drug developed by Eikon Therapeutics. It is not known to be the same as any other approved or investigational medication, and it is being studied as a novel TLR7/8 agonist for cancer treatment.