MAINZ – BioNTech SE (Nasdaq: BNTX, “BioNTech” or “the Company”) today announced positive topline data from the ongoing Phase 2 clinical trial (EudraCT No.: 2020-002195-12; NCT04526899) in patients with unresectable stage III or IV melanoma whose disease had progressed following anti-PD-(L)1-containing treatment. The randomized trial evaluates the clinical activity and safety of the investigational mRNA cancer immunotherapy BNT111 in combination with Libtayo® (cemiplimab), an anti-PD-1 monoclonal antibody being developed by Regeneron, and assesses the two single agents alone.
The trial met its primary efficacy outcome measure, demonstrating a statistically significant improvement in ORR in patients treated with BNT111 in combination with cemiplimab as compared to historical control in this indication and treatment setting. Both randomized monotherapy arms showed clinical activity. The ORR in the cemiplimab monotherapy arm was in line with the historical control of anti-PD-(L)1 or anti-CTLA-4 treatments in this patient group. The treatment was well tolerated and the safety profile of BNT111 in combination with cemiplimab in this trial was consistent with previous clinical trials assessing BNT111 in combination with anti-PD-(L)1-containing treatments. The Phase 2 trial will continue as planned to further assess the secondary endpoints, which were not mature at the time of the primary analysis.
“These Phase 2 results mark a significant step towards our vision of personalized cancer medicine. We envision mRNA as a centerpiece in future treatment paradigms for cancer, helping to address unmet medical needs, such as for patients with anti-PD-(L)1 refractory or resistant melanoma,” said Prof. Özlem Türeci, MD, Chief Medical Officer and Co-Founder at BioNTech. “These data are a proof of concept for us in three dimensions: First, for our decade-long improved mRNA cancer vaccine technology that uses uridine mRNA chemistry, a non-coding backbone that is engineered for optimal translational performance and our proprietary lipoplex formulation for delivery. Second, for our computational approaches for selecting suitable tumor antigens for our cancer indication-specific FixVac platform candidates. Third, for our strategy to combine synergistic modalities, in this case BNT111 with an established immune checkpoint treatment.”
BioNTech and Regeneron plan to present data from this trial at a forthcoming medical conference. Further, the companies also intend to submit these data for publication in a peer reviewed scientific journal.
BNT111 is based on BioNTech’s fully owned FixVac platform that utilizes a fixed combination of four mRNA-encoded, tumor-associated antigens designed to trigger an innate and tumor-antigen-specific immune response against cancer cells expressing one or more of the respective tumor antigens. In 2021, BNT111 in combination with cemiplimab received Fast Track designation by the FDA for the treatment of anti-PD-1-refractory/relapsed, unresectable Stage III or IV melanoma. In the same year, the FDA granted Orphan Drug designation for BNT111 the treatment of stage IIB through IV melanoma.