The U.S. Food and Drug Administration (FDA) has concurred with CEL-SCI Corporation's approach to patient selection using low PD-L1 tumor expression for its confirmatory Registration Study of Multikine® (Leukocyte Interleukin, Injection). This study, focusing on newly diagnosed locally advanced primary head and neck cancer patients with no lymph node involvement and low PD-L1 tumor expression, is slated to commence in the first quarter of 2025. It aims to enroll approximately 212 patients to confirm the safety profile and efficacy results demonstrated in a prior Phase 3 randomized study of 928 patients.
Patients with low PD-L1 expression treated with Multikine showed a 5-year survival rate of 73%, compared to 45% in the control group. PD-L1, a biomarker used for selecting cancer patients for checkpoint inhibitors, is significant as Multikine has been shown to be more effective in patients with low PD-L1 expression, positioning it as a potentially more effective drug for head and neck cancer patients, about 70% of whom have low PD-L1 expression.
CEL-SCI CEO Geert Kersten highlighted the importance of the agreement with the FDA on patient selection criteria, emphasizing that Multikine is the only neoadjuvant immunotherapy showing overall survival benefit in the low and negative PD-L1 head and neck cancer population. The company believes that boosting a patient’s immune system while it is still intact should provide the greatest possible impact on survival, with Multikine designed to help the immune system target the tumor at a time when it is still relatively intact.
Multikine has been dosed in over 740 patients and received Orphan Drug designation from the FDA for neoadjuvant therapy in patients with squamous cell carcinoma of the head and neck. The upcoming confirmatory Registration Study will focus on newly diagnosed locally advanced primary treatment naïve resectable head and neck cancer patients with no lymph node involvement and low PD-L1 tumor expression, representing about 100,000 patients annually.
