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Merck, Moderna neoantigen therapy delivers landmark Phase III melanoma win

Merck, Moderna neoantigen therapy delivers landmark Phase III melanoma win

A Phase III trial of intismeran autogene, the mRNA-based individualized neoantigen therapy co-developed by Merck (NYSE: MRK) and Moderna (Nasdaq: MRNA), has met both its primary and a key secondary endpoint in patients with completely resected stage IIB-IV melanoma, the companies announced. The result marks the first Phase III success for any individualized neoantigen therapy and the first Phase III trial to demonstrate improvement over pembrolizumab monotherapy in the adjuvant melanoma setting.

The INTerpath-001 trial enrolled 1,137 patients with high-risk resected cutaneous melanoma and no prior systemic therapy, randomized 2:1 to receive intismeran in combination with Merck's Keytruda (pembrolizumab) or pembrolizumab alone for approximately one year. At a pre-specified interim analysis, the combination produced statistically significant and clinically meaningful improvements in both recurrence-free survival (RFS), the primary endpoint, and distant metastasis-free survival (DMFS), a key secondary endpoint. No specific hazard ratios or p-values were disclosed in the topline announcement; full data are expected at an upcoming international medical meeting. The safety profile was consistent with prior studies, with no new signals observed. Overall survival evaluation continues per protocol.

The result converts a durable Phase II signal into Phase III confirmation. Five-year follow-up data from the Phase IIb KEYNOTE-942 trial, presented at the 2026 ASCO Annual Meeting, showed intismeran plus pembrolizumab reduced the risk of recurrence or death by 49% (HR=0.51; 95% CI, 0.294–0.887) and the risk of distant metastasis or death by 59% (HR=0.411; 95% CI, 0.200–0.843) compared to pembrolizumab alone, with an exploratory overall survival trend also favoring the combination. INTerpath-001, with more than seven times the patient population of KEYNOTE-942, was designed to provide the registrational evidence regulators require.

Intismeran is manufactured individually for each patient: a tumor sample is sequenced to identify up to 34 patient-specific neoantigens, which are encoded in a synthetic mRNA and administered to train T cells to recognize and attack residual cancer cells bearing that mutational signature. The approach is designed to augment the checkpoint blockade provided by pembrolizumab, which blocks PD-1 to restore T-cell activity against tumors that exploit the pathway to evade immune surveillance.

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The adjuvant melanoma setting already has approved options. Pembrolizumab itself is approved for stage IIB, IIC, and III resected melanoma, and Bristol Myers Squibb's Opdivo (nivolumab) covers stages IIB through IV. Neither has been shown in a Phase III trial to improve on the other in a direct comparison, and the INTerpath-001 result is the first Phase III demonstration that a combination regimen can improve outcomes over pembrolizumab alone in this setting. Bristol Myers Squibb's nivolumab plus relatlimab combination, which adds LAG-3 blockade to PD-1 inhibition, failed to improve RFS over nivolumab alone in the adjuvant stage III/IV setting in RELATIVITY-098.

Merck and Moderna said they plan to engage with regulatory authorities on filing submissions. The INTerpath program now spans nine Phase II and Phase III trials across melanoma, non-small cell lung cancer (NSCLC), bladder cancer, and renal cell carcinoma. Two NSCLC Phase III studies — INTerpath-002 in completely resected NSCLC and INTerpath-009 in resectable NSCLC after neoadjuvant pembrolizumab plus chemotherapy — are enrolling, and a Phase III study in high-risk stage I NSCLC (INTerpath-014) recently initiated. For Moderna, which has identified intismeran as one of three commercial franchises alongside infectious disease vaccines and rare disease therapeutics, a positive regulatory submission would represent the company's first oncology product approval.


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