CEL-SCI Launches Confirmatory Study for Multikine in Head and Neck Cancer

CEL-SCI Corporation has announced the initiation of an FDA registration-enabling confirmatory study for leukocyte interleukin injection (Multikine) as a neoadjuvant treatment for newly diagnosed, previously untreated, resectable, locally advanced primary head and neck cancer. The study aims to validate findings from a previously completed Phase 3 trial and serves as a definitive step toward establishing the immunotherapy as a new standard of care.

Targeted Patient Population and Study Design

The confirmatory trial is designed to enroll approximately 212 patients across clinical centers in the United States, Europe, and Asia, with expansion into South America planned. Within Taiwan, enrollment will be overseen and funded by Orient EuroPharma. The study focuses on a specific patient subset identified as having the most profound clinical benefit in the earlier 928-patient IT-MATTERS Phase 3 trial. Eligible participants must have newly diagnosed, previously untreated, resectable, locally advanced squamous cell carcinoma of the head and neck. Crucially, the trial is limited to patients whose tumors exhibit low or zero PD-L1 expression and who present with no clinical lymph node involvement at the time of entry. In May 2024, the FDA accepted these selection criteria for the trial’s target population. The study is powered at approximately 97% to confirm a previously observed hazard ratio of 0.34 for overall survival (OS).

Clinical Rationale and Phase 3 Data

The current study is rooted in data from the IT-MATTERS trial, where a target subgroup of 114 patients received a 3-week course of leukocyte interleukin injection prior to standard of care (SOC). This group achieved a 73% 5-year OS rate, compared with 45% for those who received SOC alone. Furthermore, the treatment demonstrated early biological activity. In that same subgroup, the pre-surgical objective response rate was 13% for the leukocyte interleukin arm, compared with 0% for the control arm. Pre-surgical downstaging occurred in 35% of the treated patients versus 13% of the control group. Because these early responses correlate with long-term survival, the developers plan to use them as early endpoints to support potential accelerated or conditional approval applications while OS follow-up continues in a blinded manner. “Our confirmatory registration study marks a definitive milestone toward establishing [leukocyte interleukin] as the new immunotherapy standard of care for newly diagnosed head and neck cancer with low or zero levels of PD-L1,” said Geert Kersten, CEO of CEL-SCI.

Mechanism and Context of Treatment

The broader IT-MATTERS trial, which involved 923 evaluable patients, did not meet statistical significance for its primary endpoint of OS across the full intent-to-treat population. However, investigators noted that in the high-risk subgroup, SOC alone outperformed the leukocyte interleukin combination. Researchers attributed this to the 3-week treatment delay required for dosing, which disadvantaged patients with faster-growing disease. Conversely, the treatment showed an OS advantage in a broader “low-risk” subset of the original trial, further informing the design of the current, more precise confirmatory study.

CEL-SCI Advances Multikine Toward FDA Approval with Confirmatory Phase 3 Head & Neck Cancer Trial

Strategic Expansion and Market Initiatives

Beyond the U.S. regulatory pathway, CEL-SCI is pursuing international commercialization. The company has entered into a strategic partnership with Saudi Amarox to facilitate regulatory approval, distribution, and commercialization of Multikine in Saudi Arabia. This agreement includes a 50/50 net revenue share. As the company proceeds with these clinical and commercial efforts, it continues to manage its financial position. Despite these initiatives, the company faces ongoing financial challenges, including a current ratio of 0.6 and negative levered free cash flow of $16.7 million over the last twelve months.

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