Moderna/Merck’s skin cancer therapy given FDA breakthrough therapy designation

Moderna and Merck – known as MSD outside the US and Canada – have said their investigational personalised mRNA cancer vaccine, in combination with Merck’s Keytruda, has been given breakthrough therapy designation in the US as an additional treatment for high-risk melanoma patients.

The vaccine, mRNA-4157/V940, is designed to stimulate an immune response by generating specific T-cell responses based on the unique mutational signature of a patient’s tumour, while Keytruda increases the ability of the body’s immune system to help detect and fight tumour cells.

A breakthrough therapy designation is given by the US Food and Drug Administration (FDA) to accelerate the development and regulatory review of potential new medicines for serious conditions that address a significant unmet medical need.

The FDA’s decision for mRNA-4157/V940 plus Keytruda was supported by positive results from a phase 2b trial in which the combination reduced risk of recurrence or death by 44% compared with Merck’s anti-PD-1 therapy alone.

Stephen Hoge, Moderna’s president, said: “The FDA’s breakthrough designation for mRNA-4157/V940 in combination with Keytruda reflects the excitement that we have for the potential promise of individualised cancer treatments.

“mRNA-4157/V940 in combination with Keytruda provided the first demonstration of efficacy for an investigational mRNA cancer treatment in a randomised clinical trial and potentially represents a new frontier in treating melanoma and other cancers.”

The incidence of melanoma, a type of skin cancer that develops when pigment-producing cells located in the skin grow uncontrollably, has been rising steadily over the past few decades, with nearly 325,000 new cases diagnosed worldwide in 2020.

In the US, where skin cancer is one of the most common types of cancers diagnosed, it is estimated there will be nearly 100,000 new cases of melanoma and almost 8,000 deaths each year resulting from the disease.

The companies have said they will now initiate a phase 3 study of the combination therapy in adjuvant melanoma this year, as well as ‘rapidly expand’ to additional tumour types, including non-small cell lung cancer.

Dr Eric Rubin, senior vice president, global clinical development, Merck Research Laboratories, said: “We look forward to working with the FDA, in collaboration with Moderna, to conduct a rigorous and rapid clinical development programme with a focus on addressing the needs of this important patient population.”