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KEYNOTE-942: a personalised mRNA cancer vaccine plus pembrolizumab after melanoma surgery

A vaccine custom-made from each patient's own tumour mutations, given with pembrolizumab after surgery for high-risk melanoma, reduced recurrence by about 44% compared with pembrolizumab alone in a mid-sized randomised trial, the first sign that personalised cancer vaccines can work.

Open-label randomised phase 2b trial of 157 patients with completely resected stage IIIB-IV melanoma randomised 2:1 to mRNA-4157 (V940, intismeran autogene, encoding up to 34 patient-specific neoantigens) plus pembrolizumab or pembrolizumab alone for about a year. Primary endpoint was recurrence-free survival.

Recurrence-free survival HR was 0.56 (18-month RFS 78.6% vs 62.2%) and distant metastasis-free survival HR 0.35, with benefit regardless of tumour mutational burden or PD-L1. Toxicity was mainly injection-site reactions and flu-like symptoms. It triggered the phase 3 INTerpath-001 trial and a wave of investment in individualised neoantigen vaccines.

Randomised controlled trialHas not changed practice yet157 participants
Authors
Weber JS, Carlino MS, Khattak A, et al.
Published
What it found
  • Recurrence-free survival HR 0.561 (95% CI 0.309-1.017); 18-month RFS 78.6% vs 62.2%.
  • Distant metastasis-free survival HR 0.347 (95% CI 0.145-0.828).
  • Benefit was seen in both high and low tumour mutational burden and in PD-L1-negative tumours.
  • Three-year update: RFS HR 0.51, with the curves continuing to separate.
  • Grade 3 or higher treatment-related adverse events 25% vs 18%; vaccine-related events were mostly grade 1-2 fatigue, injection-site pain and chills.
  • Manufacturing took about six to eight weeks per patient from biopsy sequencing to first dose.
What it means

For the first time a randomised trial suggests that a vaccine tailored to an individual's tumour can reduce relapse when combined with immunotherapy, which is a proof of concept for a field that had failed for decades. Nothing changes for patients yet: the trial was small, the confidence interval crossed one, and the phase 3 trial in melanoma (and parallel trials in lung and other cancers) must confirm it. If it does, personalised mRNA vaccines could become a routine adjunct to checkpoint inhibitors after surgery.

Be careful
  • Phase 2b with 157 patients and an upper confidence limit above 1.0; the prespecified one-sided p-value was met but the result needs confirmation.
  • Open-label; recurrence assessments were investigator-based.
  • Individualised manufacturing is slow and expensive and has never been scaled to thousands of patients.
  • No biomarker predicts who benefits, and the mechanism (neoantigen-specific T-cell expansion) has been shown in only a subset.

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