A Phase 2 Study of EIK1001 in Combination With Pembrolizumab and Chemotherapy in Patients With Stage 4 Non-Small Cell Lung Cancer

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Overview

EIK1001 (also known as BDB001) is an intravenously administered small‑molecule co‑agonist of Toll‑like receptors 7 and 8 (TLR7/8) being developed for solid tumors, including in combinations with PD‑(L)1 inhibitors. The drug has completed Phase 1 studies as monotherapy and in combinations, and is being advanced in multiple trials, including an adaptive Phase 2/3 first‑line melanoma study in combination with pembrolizumab. The synonym relationship (EIK1001 = BDB001) is reflected in clinical‑trial listings. (ascopubs.org)

Mechanism of action

• Target: TLR7 and TLR8 co‑agonist. (ascopubs.org)
• Proposed immunologic effect: systemic activation and “reprogramming” of dendritic cells with induction of interferon‑γ and IP‑10 (CXCL10), enhancing innate and adaptive antitumor immunity. Pharmacodynamic analyses from Phase 1 showed dose‑dependent increases in IP‑10 and IFN‑γ consistent with TLR7/8 activation. (ascopubs.org)

Clinical development

Key studies (selected): - Phase 1 (NCT03486301): BDB001/EIK1001 monotherapy and in combination with pembrolizumab in advanced solid tumors; multiple interim readouts and the 2021 ASCO abstract report safety and preliminary efficacy. (cdek.pharmacy.purdue.edu) - Phase 1 (NCT04196530): BDB001/EIK1001 in combination with atezolizumab; interim results presented at SITC 2021. (ichgcp.net) - Phase 2 (NCT04819373): Monotherapy in anti‑PD‑(L)1–refractory solid tumors. (ichgcp.net) - AGADIR study (NCT03915678) Phase 2 cohort: BDB001 + atezolizumab + stereotactic body radiotherapy (SBRT) in advanced pancreatic adenocarcinoma (multi‑cohort basket). (ascopubs.org) - TeLuRide‑006 (NCT06697301): global, adaptive Phase 2/3 randomized, double‑blind study of EIK1001 + pembrolizumab vs placebo + pembrolizumab as first‑line therapy in advanced melanoma; recruiting as of August 18, 2025. (ichgcp.net)

Efficacy

• Combination with pembrolizumab (Phase 1, ASCO 2021): Among participants treated at higher dose levels (DL3–4; n=14), objective responses occurred in 4 patients (29%), with disease control in 57%; responses were seen in PD‑(L)1–refractory melanoma, hepatocellular carcinoma, cholangiocarcinoma, and platinum‑resistant ovarian carcinoma, some as early as 4 weeks. (ascopubs.org)
• Combination with atezolizumab and SBRT in pancreatic adenocarcinoma (AGADIR, ASCO 2023 abstract): disease control rate 38.0% (95% CI 18.1–61.5); the cohort met its prespecified first endpoint for DCR (additional efficacy details pending in the abstract). (ascopubs.org)

Note: Company press summaries of interim data are broadly consistent with these abstracts but should be interpreted cautiously until peer‑reviewed publications are available. (businesswire.com)

Safety

• Combination with pembrolizumab (Phase 1): No dose‑limiting toxicities; most treatment‑related adverse events (TRAEs) were grade 1–2 and transient. Common TRAEs included fever (39.1%), fatigue (39.1%), chills/rigor (34.8%), pruritus/rash (21.7%), and nausea (13.0%). Grade 3 TRAEs occurred in 13.0% (fatigue, rash, stomatitis, alkaline phosphatase elevation); no grade 4–5 TRAEs reported. (ascopubs.org)
• Pharmacokinetics/biomarkers: Linear, dose‑proportional PK with dose‑dependent cytokine induction (IP‑10, IFN‑γ) across the dose range where clinical responses were observed. (eikontx.com)

Further reading

• ASCO 2021 abstract (JCO): BDB001 + pembrolizumab Phase 1 safety/efficacy. (ascopubs.org)
• ICH GCP registry: Atezolizumab and BDB001 (EIK1001) Phase 1; name mapping. (ichgcp.net)
• ClinicalTrials.gov‑style registry: EIK1001 + pembrolizumab first‑line melanoma (TeLuRide‑006, NCT06697301). (ichgcp.net)
• ASCO 2023 abstract (JCO): AGADIR—BDB001 + atezolizumab + SBRT in pancreatic cancer. (ascopubs.org)
• AACR 2024 poster summary: PK/biomarker analysis of EIK1001 in Phase 1 studies. (eikontx.com)
• Phase 2 monotherapy study overview (anti‑PD‑(L)1‑refractory solid tumors). (ichgcp.net)

If additional peer‑reviewed manuscripts become available, these should be prioritized over press releases for future updates.

Last updated: Oct 2025

Inclusion Criteria:

1. be ≥ 18 years of age on the day of signing of informed consent.

2. confirmed Stage 4 NSCLC (squamous or non-squamous) and be considered for standard of care.

3. have confirmation that mutation-directed therapy is not indicated (documentation of absence of tumor activating mutations/fusions that are approved for first line therapy).

4. have at least 1 lesion with measurable disease at Baseline according to RECIST 1.1 as determined by the local site Investigator/radiology assessment.

5. have not received prior systemic treatment for advanced/metastatic NSCLC.

6. have an ECOG Performance Status of 0 to 1.

7. have adequate organ function.

Exclusion Criteria:

1. does not have predominantly squamous cell or non-squamous cell histology NSCLC. Mixed tumors will be categorized by the predominant cell type; if small cell elements are present, the participant is ineligible.

2. is currently participating in or has participated in a study of an investigational agent and received investigational therapy within 4 weeks or 5 half-lives (whichever is shorter) of administration of EIK1001.

3. prior to the first dose of EIK1001, has received prior systemic therapy for metastatic disease, or had major surgery (\< 3 weeks prior to the first dose).

4. has completed palliative radiotherapy within 7 days of the first dose of study drug administration.

5. has a known history of prior malignancy, except if the participant has undergone potentially curative therapy with no evidence of that disease recurrence for 5 years.

6. has an active infection requiring therapy.