CEL-SCI Corporation is advancing its investigational cancer therapy, Multikine (Leukocyte Interleukin, Injection), into a confirmatory Phase 3 registration study following promising results in pre-surgical tumor regression and alignment with FDA on patient selection criteria. The study aims to solidify Multikine's potential as a novel treatment for head and neck cancer, particularly in patients with low PD-L1 expression.
The FDA has concurred with CEL-SCI's strategy to utilize PD-L1 expression as a biomarker to identify patients most likely to benefit from Multikine therapy. This decision is based on data from a previous Phase 3 trial, which demonstrated a 73% 5-year survival rate in patients with low PD-L1 expression treated with Multikine, compared to 45% in the control group (p-value of 0.0015).
Confirmatory Registration Study
CEL-SCI is initiating a 212-patient confirmatory registration study to further evaluate Multikine in newly diagnosed, locally advanced head and neck cancer patients. The study will focus on patients with no lymph node involvement (N0) and low PD-L1 tumor expression, a population that represents approximately 100,000 patients annually. Full enrollment is anticipated by Q2 2026, with potential for early approval based on pre-surgical response rates.
The design of the Registration Study is based on the prior Phase 3 study results, where Multikine demonstrated significant rates of tumor regression prior to surgery, a finding not observed in the control group. These pre-surgical tumor regressions, confirmed at surgery, were indicative of a survival benefit. CEL-SCI believes the study has a high chance of success, with potential for accelerated or conditional approval for Multikine in the coming year, should the pre-surgical tumor responses mirror those seen in the Phase 3 data.
Mechanism of Action and Clinical Rationale
Multikine is designed to boost the patient's immune system to target the tumor before surgery, radiation, and chemotherapy compromise immune function. The therapy has shown particular promise in patients with low PD-L1 expression, a population that typically does not respond well to checkpoint inhibitors. CEL-SCI's approach offers a potential new treatment avenue for this subset of head and neck cancer patients.
Financial Overview
CEL-SCI reported its fiscal Q1 2025 financial results, with research and development expenses at $4.4 million, consistent with the previous year. The net loss for the quarter was $7.1 million, narrowing to $0.11 per common share, a 21% improvement compared to the prior year. For fiscal year 2024, CEL-SCI reported a net loss of $26.9 million, with R&D expenses of $18.2 million.
Collaboration and Manufacturing
CEL-SCI has partnered with Ergomed, a clinical research organization (CRO), for the confirmatory registration study. Ergomed previously collaborated with CEL-SCI on the Phase 3 study. CEL-SCI has also completed the commissioning of its cGMP state-of-the-art dedicated manufacturing facility, a key step toward regulatory approval of Multikine.
Study Design
The confirmatory study is titled:
"A Phase III, Open Label, Randomized, Controlled, Multi-Center Study of the Effects of Neoadjuvant Leukocyte Interleukin, Injection (LI) Plus Standard of Care Versus Standard of Care Only in Treatment Naïve Adults With Resectable Locally Advanced Primary Squamous Cell Carcinoma of the Head and Neck (Oral Cavity) Who Present With No Nodal Involvement and Low Tumor Programmed Death Ligand 1 Expression (Defined as Tumor Proportion Score <10)"
The trial will be conducted across multiple centers in various countries, with the first clinical site expected to open in the United States.
