BioNTech said that its BNT111 candidate in combination with Regeneron’s Libtayo (cemiplimab) has met the primary endpoint of overall response rate (ORR) in a Phase 2 clinical trial of patients with unresectable stage 3 or 4 melanoma.
BNT111 is an investigational mRNA cancer immunotherapy developed using BioNTech’s proprietary FixVac platform.
In 2021, BNT111, in combination with cemiplimab, received fast track designation from the US Food and Drug Administration (FDA) for treating anti-PD-1-refractory or relapsed, unresectable stage 3 or 4 melanoma.
Additionally, the FDA granted orphan drug designation to BNT111 for the treatment of Stage 2B through 4 melanoma.
The randomised Phase 2 trial assessed the BNT111 investigational combination in advanced melanoma patients whose disease had progressed following anti-PD-(L)1-containing treatment.
It assessed the clinical activity and safety of the combination, as well as the effects of each of the two single agents individually.
According to the results, the study achieved showed a statistically significant improvement in the ORR for patients treated with BNT111 in combination with cemiplimab compared to historical controls in this context.
Both monotherapy arms demonstrated clinical activity, with the ORR in the cemiplimab monotherapy arm aligning with historical data for anti-PD-(L)1 or anti-CTLA-4 treatments in this patient group.
The treatment was well tolerated, and the safety profile of BNT111 combined with cemiplimab was consistent with earlier trials of BNT111 with anti-PD-(L)1 treatments.
BioNTech will continue the Phase 2 trial to further evaluate the secondary endpoints, which were not yet mature at the time of the primary analysis.
BioNTech chief medical officer and co-founder Özlem Türeci said: “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-(L)1 refractory or resistant melanoma.
“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.”
In 2022, BioNTech expanded its partnership with Regeneron to advance the development of FixVac candidate BNT116, in combination with Libtayo to treat advanced non-small cell lung cancer (NSCLC).
