A messenger ribonucleic acid (mRNA) vaccine against cancer failed to meet a primary goal of a late-stage clinical trial, the shot’s manufacturer said on Oct. 13.
The experimental vaccine, known as BNT113, fell short of its primary endpoint of survival without progression of cancer, German-based BioNTech said in a statement.
The phase 2/phase 3 clinical trial grouped patients into two groups. One received BNT113 and pembrolizumab, a cancer immunotherapy from Merck. The other received only pembrolizumab.
Trial participants had head and neck squamous cell carcinoma that had either spread through the body or involved a tumor that could not be operated on. The type of cancer is the seventh most common cancer type across the world. They also tested positive for human papillomavirus (HPV) 16.
A main goal of the trial was to see whether patients who took the vaccine were more likely to survive without progression of their cancer. That goal was not met.
The other primary goal, overall survival, means that if patients who received the vaccine had higher survival rates, even if the freedom of progression goal was not met, the trial would be marked as positive, according to BioNTech.
The Independent Data Monitoring Committee monitoring the trial recommended advancing it to evaluate the overall survival endpoint, BioNTech said. The company is blinded to the trial data, in accordance with the rules of the trial.
Dr. Özlem Türeci, BioNTech’s co-founder and chief medical officer, said the potential of mRNA cancer immunotherapies in combination regimens lies in their ability to induce immune responses that may translate into durable clinical benefit and, ultimately, prolong survival of patients.
“While the [progression-free survival] result was not statistically significant at this first analysis, the data support continued evaluation of BNT113 plus pembrolizumab in this trial and show that it is engaging its intended therapeutic mechanism,” Türeci said in a statement.
“As immune-mediated anti-tumor effects build and deepen over time, the analysis of the second dual primary endpoint, overall survival, will be critical to fully assess the potential of this approach to deliver durable benefit for patients with HPV-driven head and neck cancer.”
BioNTech announced in the summer it was ending a trial testing a different mRNA cancer vaccine.
People in the phase 2 trial who received that shot, which targeted colorectal cancer, were more likely to die than patients who did not, the company said at the time, prompting the monitoring board for the trial to recommend halting the trial.
“While this outcome is not what we had envisioned for mRNA as a monotherapy in colorectal cancer, it provides scientific insight into the challenges of treating immunotherapy-insensitive tumor types with immune-suppressive microenvironments and will help inform the further development of investigational mRNA cancer immunotherapies,” Türeci said at the time.
Conversely, Merck and Moderna said in August that their mRNA cancer vaccine prevented skin cancer recurrence and the spread of cancer in a phase 3 clinical trial. They said that they would be filing for approval of the shot, intismeran.
The U.S. Department of Health and Human Services said recently that mRNA products, which have been associated with serious side effects such as heart inflammation, “warrant heightened scientific scrutiny,” but the department “continues to support mRNA research where the science shows promise, including for hard-to-treat cancers.”