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Allan Ryan

Investigational melanoma vaccine receives breakthrough designation from U.S. FDA

Data from a Phase 2b study indicates that an investigational personalized mRNA cancer vaccine known as mRNA-4157/V940, in combination with pembrolizumab, may potentially provide an additive benefit in melanoma and enhance T cell-mediated destruction of tumour cells. The combination is being studied for the adjuvant treatment of patients with high-risk melanoma following complete resection. Based on these findings, the U.S. FDA has granted a Breakthrough Therapy designation to the combination.


A Breakthrough Therapy designation is intended to expedite the development and review of therapies for serious conditions when preliminary clinical evidence indicates the product may demonstrate substantial improvement over available therapies on at least one clinically significant endpoint.


According to a press release, this study is the first randomized trial to show a personalized mRNA cancer vaccine improved clinical outcomes for patients versus an active comparator.


The KEYNOTE-942 study is an ongoing randomized, open-label Phase 2b trial involving 157 patients with stage III/IV melanoma. Following complete surgical resection, patients were randomized to receive Moderna’s mRNA-4157/V940 (nine total doses of mRNA-4157) and pembrolizumab (200 mg every three weeks up to 18 cycles [for approximately one year]) versus pembrolizumab alone for approximately one year until disease recurrence or unacceptable toxicity. The release notes the primary study endpoint is recurrence-free survival (RFS); secondary endpoints include distant metastasis-free survival and safety.


In a previous announcement, investigators reported that patients who received the mRNA vaccine in addition to adjuvant pembrolizumab had a 44% reduction in risk of recurrence or death (HR, 0.56; 95% CI, 0.31-1.08; one-sided p=0.0266) versus pembrolizumab alone following resection of high-risk melanoma.


“The results of this randomized phase 2b trial are exciting for the field,” Jeffrey S. Weber, MD, PhD, principal investigator of the study and deputy director of the Perlmutter Cancer Center at NYU Langone Health, said in the December statement. “These data provide the first evidence that we can improve on the rates of RFS achieved by PD-1 blockade in resected high-risk melanoma.”


Key eligibility criteria for the ongoing trial includes: patients with resectable cutaneous melanoma at high risk of recurrence, patients with complete resection within 13 weeks prior to the first dose of pembrolizumab, patients were disease free at study entry (after surgery) with no loco-regional relapse or distant metastasis and no clinical evidence of brain metastases.


A Phase 3 study of this study in melanoma will be launched in 2023, and investigations may expand to include other cancer types, including non-small cell lung cancer.

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