Journal article
Intratumoral vidutolimod as monotherapy or in combination with pembrolizumab in patients with programmed cell death 1 blockade-resistant melanoma: Final analysis from a phase 1b study
Cancer, Vol.131(15), e70022
08/01/2025
DOI: 10.1002/cncr.70022
PMCID: PMC12318270
PMID: 40753466
Abstract
Background
New treatment options are needed for patients with metastatic anti–programmed cell death 1 (PD-1)–resistant melanoma. The final analysis of a phase 1b study evaluating the Toll-like receptor 9 agonist vidutolimod is reported here.
Methods
This two-part, open-label, multicenter, phase 1b study in adults with metastatic/unresectable anti–PD-1–resistant melanoma evaluated the safety and clinical activity of intratumoral vidutolimod plus systemic pembrolizumab (part 1) or vidutolimod alone (part 2). Two vidutolimod formulations were evaluated with different concentrations of polysorbate (PS20-A, 0.005%–0.01% polysorbate 20; PS20-B, 0.00167% polysorbate 20). Key end points were safety and investigator-assessed objective response rate (ORR; Response Evaluation Criteria in Solid Tumors, version 1.1).
Results
A total of 159 patients were treated in part 1 (PS20-A, n = 98; PS20-B, n = 61), and 40 patients were treated in part 2. Any-grade treatment-emergent adverse events (TEAEs) occurred in 100.0% of patients. Grade ≥3 TEAEs occurred in 55.3% (part 1) and 37.5% (part 2) of patients. No treatment-related deaths occurred. Best ORR was 23.5% (95% CI, 15.5%–33.1%; complete response [CR], 7.1%) for vidutolimod PS20-A plus pembrolizumab, 11.5% (95% CI, 4.7%–22.2%; CR, 1.6%) for vidutolimod PS20-B plus pembrolizumab, and 20.0% (95% CI, 9.1%–35.6%) for vidutolimod monotherapy. Median duration of response was 25.2 months with vidutolimod PS20-A plus pembrolizumab, 11.4 months with vidutolimod PS20-B plus pembrolizumab, and 5.6 months with vidutolimod monotherapy.
Conclusions
Vidutolimod PS20-A alone or in combination with pembrolizumab had an acceptable safety profile and promising clinical activity in patients with PD-1 blockade–resistant melanoma.
Details
- Title: Subtitle
- Intratumoral vidutolimod as monotherapy or in combination with pembrolizumab in patients with programmed cell death 1 blockade-resistant melanoma: Final analysis from a phase 1b study
- Creators
- Mohammed M Milhem - University of IowaYousef Zakharia - University of IowaDiwakar Davar - University of Pittsburgh Medical CenterElizabeth I Buchbinder - Dana-Farber Cancer InstituteTheresa Medina - University of Colorado DenverAdil Daud - University of California, San FranciscoAntoni Ribas - University of California, Los AngelesBartosz Chmielowski - University of California, Los AngelesJiaxin Niu - Banner MD Anderson Cancer CenterGeoffrey T Gibney - MedStar Georgetown University HospitalKim Margolin - Saint John's Health CenterAnthony J Olszanski - Fox Chase Cancer CenterInderjit Mehmi - Angeles Clinic and Research InstituteTakami Sato - Thomas Jefferson UniversityMontaser Shaheen - The University of Texas Health Science Center at San AntonioLuping Zhao - Checkmate Pharmaceuticals, Cambridge, Massachusetts, USAHeather Kelley - Checkmate Pharmaceuticals, Cambridge, Massachusetts, USAHong Liu - Checkmate Pharmaceuticals, Cambridge, Massachusetts, USASujatha Kumar - Checkmate Pharmaceuticals, Cambridge, Massachusetts, USADmitri Bobilev - Boston FusionArthur M Krieg - Zola Therapeutics, Needham, Massachusetts, USAJames E Wooldridge - Checkmate Pharmaceuticals, Cambridge, Massachusetts, USAJohn M Kirkwood - University of Pittsburgh Medical Center
- Resource Type
- Journal article
- Publication Details
- Cancer, Vol.131(15), e70022
- DOI
- 10.1002/cncr.70022
- PMID
- 40753466
- PMCID
- PMC12318270
- NLM abbreviation
- Cancer
- ISSN
- 0008-543X
- eISSN
- 1097-0142
- Publisher
- Wiley
- Grant note
- Checkmate Pharmaceuticals
- Language
- English
- Date published
- 08/01/2025
- Academic Unit
- Hematology, Oncology, and Blood & Marrow Transplantation; Internal Medicine
- Record Identifier
- 9984937805302771
Metrics
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