Positive Topline Phase 2 Results Announced for mRNA Immunotherapy Candidate in Patients With Advanced Melanoma

BioNTech announced positive topline data from the ongoing phase 2 clinical trial evaluating the clinical activity and safety of the investigational mRNA cancer immunotherapy BNT111 in combination with cemiplimab (Libtayo; Regeneron), according to a news release.¹

Image credit: David A Litman | stock.adobe.com

The trial is assessing the drugs in patients with unresectable stage 3 or 4 melanoma whose disease had progressed following anti-PD-(L)1-containing treatment. Cemiplimab is an anti-PD-1 monoclonal antibody.¹

A statistically significant improvement in overall response rate (ORR) in patients treated with BNT111 in combination with cemiplimab, as compared to historical control in this indication and treatment setting, was achieved. Importantly, the ORR in the cemiplimab monotherapy arm was in line with the historical control of treatments in this patient group.¹

BNT111 in combination with cemiplimab was well-tolerated, and had a consistent safety profile with previous clinical trials that assessed BNT111 in combination with anti-PD-(L)1-containing treatments.¹

In the news release, the company said that the phase 2 trial will continue to further assess secondary end points, which were not mature at the time of the completion of the primary analysis.¹

“These phase 2 results mark a significant step towards our vision of personalized cancer medicine,” Özlem Türeci, MD, chief medical officer at BioNTech, said in the news release. “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.”¹

Türeci said that the data are proof of concept for their decade-long work on an improved mRNA cancer vaccine that uses uridine mRNA chemistry, which is a non-coding backbone engineered for optimal translational performance and the proprietary lipoplex formulation for delivery.¹

Additionally, Türeci lauded the computational approaches that selected suitable tumor antigens for their cancer indication-specific FixVac platform candidates. Lastly, he praised BioNTech’s strategy of combining synergistic modalities—in this study, they combined BT111 with an established immune checkpoint treatment.¹

BioNTech and Regeneron plan to present the data from this trial at a future medical conference and aim to submit these data for publication in a peer-reviewed scientific journal.¹

BNT111 in combination with cemiplimab was granted fast Ttack designation by the FDA for the treatment of anti-PD-1-refractory/relapsed, unresectable stage 3 or stage 4 melanoma in 2021. That same year, the FDA granted orphan drug designation to BNT11 for the treatment of stage 2B through 4 melanoma.²

BNT111 uses a fixed combination of 4 mRNA-encoded, tumor-associated antigens that are designed to trigger an innate and tumor-antigen specific immune response against cancer cells that express 1 or more of the respective tumor antigens.¹

The trial evaluating BNT111 is being conducted across approximately 60 sites in 7 different countries. Additional end points of the study include duration of response, disease control rate, overall survival, safety, and tolerability.¹

References

1. BioNTech. BioNTech announces positive topline phase 2 results for mRNA immunotherapy candidate BNT111 in patients with advanced melanoma. News release. Released July 30, 2024. Accessed August 1, 2024. https://investors.biontech.de/news-releases/news-release-details/biontech-announces-positive-topline-phase-2-results-mrna

2. BioNTech. BioNTech receives FDA fast track designation for its FixVac candidate BNT111 in advanced melanoma. News release. Released November 19, 2021. Accessed August 1, 2024. https://investors.biontech.de/news-releases/news-release-details/biontech-receives-fda-fast-track-designation-its-fixvac