Announcement • Jul 13
CEL-SCI Corporation Launches FDA Registration-Enabling Phase 3 Confirmatory Study of Multikine for Newly Diagnosed Head and Neck Cancer
CEL-SCI Corporation had announced it is launching its global Confirmatory Registration Study of Multikine (Leukocyte Interleukin, Injection) as a neoadjuvant treatment for newly diagnosed, previously untreated, resectable, locally advanced primary head and neck cancer. The global, multi-center study will enroll patients in the United States, Europe, Asia, and South America. Orient EuroPharma, CEL-SCI's strategic partner in Taiwan, will oversee and fund patient enrollment within its territory. The study is highly optimized and designed with approximately 97% statistical power to detect a previously observed 0.34 hazard ratio based on overall survival. The Confirmatory Study will enroll approximately 212 patients presenting with newly diagnosed, previously untreated, resectable, locally advanced squamous cell carcinoma (head and neck cancer) whose tumors exhibit low or zero PD-L1 expression and who present with no clinical lymph node involvement at entry. This precise cohort represents the patient population that demonstrated the most profound clinical benefit in CEL-SCI’s completed 928-patient randomized controlled Phase 3 study. The patients in the confirmatory study will be required to possess the same disease characteristics as the patients who showed the excellent survival benefit in the completed Phase 3 study. By isolating this specific high-responder group of patients identified in the prior randomized controlled trial, the confirmatory study maintains robust statistical power while requiring a substantially smaller, more efficient patient enrollment target. Clinical Rationale & Phase 3 Data Foundation: In the target cohort of CEL-SCI's completed Phase 3 study, 114 patients who received a 3-week regimen of Multikine prior to receiving standard of care (SOC) experienced a significant reduction in the risk of death, achieving a 73% five-year overall survival rate compared to 45% for patients treated with SOC alone. Furthermore, neoadjuvant administration of Multikine induced rapid, statistically significant pre-surgical tumor responses, including complete tumor responses confirmed by pathology, within 3 weeks of Multikine treatment. In the target population, the pre-surgical objective response rate was 13% in the Multikine arm versus 0% in the control arm. Pre-surgical downstaging was observed in 35% of Multikine-treated patients compared to 13% of controls. Because these early, pre-surgical objective responses were demonstrated to be highly prognostic and appear to be predictive of long-term overall survival, CEL-SCI intends to utilize these early endpoints to support potential accelerated or conditional approval applications globally. Concurrently, overall survival follow-up will continue to be assessed in a blinded manner until the required event threshold is met. Head and neck cancer remains one of the world's most challenging oncology indications, with approximately 900,000 new diagnoses annually. Despite modern iterations of surgery and chemoradiation, long-term survival outcomes have stalled, highlighting a critical global need for non-toxic neoadjuvant immunotherapies that directly improve survival. Multikine (Leukocyte Interleukin, Injection) is a novel, patented investigational cancer immunotherapy designed to be administered immediately following diagnosis, prior to surgery, radiation, or chemotherapy. By treating patients before any other treatment, while their immune systems are still intact, Multikine aims to stimulate a robust anti-tumor immune response before the immune system is compromised by conventional treatments. Multikine has been administered to over 740 patients across its clinical development program and has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the neoadjuvant therapy of squamous cell carcinoma of the head and neck.
