CEL-SCI provides rationale for use of Multikine in head, neck cancer study

CEL-SCI (CVM) highlighted biological rationale for the use of Multikine in the confirmatory registration head and neck cancer study. This study of 212 newly diagnosed locally advanced, resectable head and neck cancer patients was given the go-ahead as a confirmatory registration study by FDA and will focus on those patients who showed a 73% survival with Multikine vs. a 45% for the control patients not treated with Multikine in the prior Phase 3 study. The Phase 3 study missed the primary endpoint of 10% improvement in overall survival in the ITT population but showed a 46.5-month OS benefit vs control in the patients who received Multikine followed by surgery and radiotherapy. In the other group of about 50% of patients who had chemotherapy added to radiotherapy after surgery, there was no survival benefit. Because the decision to administer chemotherapy is made after surgery, CEL-SCI had to develop selection criteria that would identify at screening those patients who would be most likely to benefit from Multikine neoadjuvant treatment. After this analysis was done and the evidence collected, CEL-SCI presented these selection criteria to FDA. The agency accepted these selection criteria and gave CEL-SCI the go-ahead to conduct a 212-patient confirmatory registration study in the patient population defined by the selection criteria. The study will include patients with newly diagnosed locally advanced primary head and neck cancer presenting with: No lymph node involvement and having low PD-L1 tumor expression. Multikine was shown to be highly active in the full Phase 3 study population, leading to significant rates of tumor regression, including 5 complete regressions, before surgery, following just 3 weeks of Multikine treatment. In this advanced disease stage, tumors do not shrink spontaneously, and the control group in the study had no reported pre-surgical tumor regressions. Therefore, the regressions had to be due to Multikine. Pre-surgical tumor regressions were confirmed by pathology at surgery, where biological evidence of Multikine’s activity, inducing cellular infiltration of anti-tumor immune cells, could be seen at the tumor microenvironment. Pre-surgical tumor regressions were also seen in prior published Phase 2 studies, further supporting the validity of the Phase 3 results showing Multikine’s anti-cancer activity. The pre-surgical tumor regressions forecasted increased long-term survival benefit in responders. That is, subjects who had tumor regressions lived longer than those who did not. In the target population selected for the confirmatory study the rate of pre-surgical tumor regressions was substantially higher than what was seen in the full Phase 3 population. This shows that the selected population is highly likely to show a significant survival prolongation in the confirmatory registration study.