BioNTech has announced positive phase 2 results for its mRNA immunotherapy candidate in a subset of advanced melanoma patients.

The EudraCT clinical study has been evaluating BNT111 in combination with Regeneron’s PD-1 checkpoint inhibitor Libtayo (cemiplimab) in patients with stage 3 or 4 melanoma whose disease had progressed following anti-PD-L1-containing treatment.

Currently among the leading causes of cancer-related deaths globally, melanoma is responsible for around 58,000 deaths annually.

The aggressive form of melanoma, anti-PD-1 refractory/relapsed unresectable stage 3 or 4 melanoma, has a particularly poor prognosis for patients.

The investigational mRNA immunotherapy is based on the company’s FixVac platform, which utilises a fixed combination of four mRNA-encoded, tumour associated antigens designed to trigger an innate and tumour-antigen-specific immune response against cancer cells that express one or more of the respective tumour antigens and is delivered as a uridine mRNA-lipoplex formulation.

The ongoing EudraCT trial showed that BNT111 in combination with Libtayo demonstrated a statistically significant overall response in patients, meeting its primary efficacy outcome measure, and was well tolerated, with the safety profile of BNT111 in combination with Libtayo being consistent with previous clinical trials assessing BNT111 in combination with anti-PD-L1-containing treatments.

Commenting on the positive results, professor Özlem Türeci, chief medical officer and co-founder, BioNTech, said: “These phase 2 results mark a significant step towards our vision of personalised cancer medicine.

“We envision mRNA as a centrepiece in future treatment paradigms for cancer, helping to address unmet medical needs, such as for patients with anti-PD-L1 refractory or resistant melanoma.”

BNT111 in combination with Libtayo previously received fast track designation by the US Food and Drug Administration in 2021 for the treatment of anti-PD-1-refractory/relapsed, unresectable stage 3 or 4 melanoma, in addition to the mRNA immunotherapy being granted orphan drug designation for the treatment of stage 2b through 4 melanoma by the US regulator.