Focused on providing solutions for patients with significant unmet medical needs.

Our lead investigational product, tilsotolimod, demonstrated activity in numerous pre-clinical models and is in clinical development across several oncology indications. These indications include anti-PD-1 refractory advanced melanoma, microsatellite-stable colorectal cancer (MSS-CRC; phase 2), and recurrent/metastatic head & neck squamous cell carcinoma (HNSCC, sponsored by AbbVie; phase 1/2).

Tilsotolimod received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for the treatment of anti-PD-1 refractory melanoma, in combination with ipilimumab, as well as Orphan Drug Designation for the treatment of stage IIb-IV melanoma.1

Tilsotolimod also has intellectual property protection under a composition of matter patent, which provides exclusivity until 2030 (est.), and under method-of-use patents covering certain melanoma, CRC, & HNSCC treatments in combination with therapies targeting CTLA-4, PD-1, or PD-L1, which provide exclusivity through September 2037 (est.).

Tilsotolimod (IMO-2125) Program

Tumor Type
Trial Regimen Collaborator Phase 1 Phase 2 Pivotal Approved
Anti-PD-1 Refractory Advanced Melanoma
ILLUMINATE-301 tilsotolimod +
ipilimumab		 Phase 2
Microsatellite-Stable Colorectal Cancer
ILLUMINATE-206 tilsotolimod +
ipilimumab/ nivolumab		 Phase 1
Resistant Solid Tumors
ILLUMINATE-101 tilsotolimod
monotherapy		 Discovery
Recurrent/Metastatic Head & Neck Squamous Cell Carcinoma
AbbVie-Sponsored Trial tilsotolimod +
ABBV-368 (OX-40)		 Discovery
Anti-PD-1 Refractory Advanced Melanoma
ILLUMINATE-301
tilsotolimod +
ipilimumab
Pivotal
Microsatellite-Stable Colorectal Cancer
ILLUMINATE-206
tilsotolimod +
ipilimumab/ nivolumab
Phase 2
Resistant Solid Tumors
ILLUMINATE-101
tilsotolimod
monotherapy
Phase 1
Recurrent/Metastatic Head & Neck Squamous Cell Carcinoma
AbbVie-Sponsored Trial
tilsotolimod +
ABBV-368 (OX-40)
Phase 1
1 Tilsotolimod is an investigational agent which has not been approved by any regulatory authority

Clinical Trials

A randomized, multicenter phase 3 trial to compare the efficacy of tilsotolimod in combination with ipilimumab versus ipilimumab alone in patients with anti-PD-1 refractory advanced melanoma.

This study enrolled approximately 480 patients, with key topline data reported in March 2021. For more information on ILLUMINATE-301, please refer to ClinicalTrials.gov.

A phase 1/2 trial that tested the safety and efficacy of tilsotolimod in combination with ipilimumab or pembrolizumab in patients with anti-PD-1 refractory metastatic melanoma.

Tilsotolimod in combination with ipilimumab has shown promising efficacy, including complete responses, and has been generally well tolerated. Topline data from the study were reported in 2020, with final data to be published in 2021. For more information on ILLUMINATE-204, please refer to ClinicalTrials.gov.

A phase 2, open-label, multi-cohort, multicenter study to test the safety and efficacy of tilsotolimod in combination with ipilimumab and nivolumab for the treatment of solid tumors.

Though checkpoint inhibitors are approved for use in microsatellite instability-high colorectal cancer (CRC), patients with microsatellite-stable (MSS) CRC do not appear likely to respond to these regimens. We’re examining the efficacy of the triple combination of tilsotolimod, ipilimumab, and nivolumab in MSS-CRC with the goal of extending the option of immunotherapy to these patients and the physicians treating them. For more information on ILLUMINATE-206, please refer to ClinicalTrials.gov.

A phase 1b study of the safety, efficacy, and immune effects of intratumoral tilsotolimod in patients with refractory solid tumors.

Tilsotolimod was generally well tolerated and, in alignment with pre-clinical findings, induced alterations in the tumor microenvironment, including immune checkpoint upregulation, activation of dendritic cells, and induction of Type-I IFN signaling. Final data from the study were reported in April 2020. For more information on ILLUMINATE-101, please refer to ClinicalTrials.gov.

AbbVie collaboration

A study to determine the safety, tolerability, pharmacokinetics, and preliminary efficacy of ABBV-368 plus tilsotolimod and other therapy combinations in patients with recurrent/metastatic head and neck squamous cell carcinoma. AbbVie is solely responsible for the conduct of the study. For more information on this study, please refer to ClinicalTrials.gov.


For information on clinical trials with tilsotolimod, please visit ClinicalTrials.gov or contact us at clinicaltrials@iderapharma.com.