FDA and CEL-SCI Approve PD-L1 Biomarker for Head and Neck Cancer Study Starting Q1 2025

CEL-SCI Corporation, a biotechnology company listed on the NYSE American under the ticker CVM, has received confirmation from the U.S. Food and Drug Administration (FDA) on their proposed approach for selecting patients for a major upcoming clinical study. This study, which will begin in the first quarter of 2025, focuses on the use of CEL-SCI’s Multikine® (Leukocyte Interleukin, Injection) for treating patients with newly diagnosed locally advanced primary head and neck cancer. Selection will be based on patients exhibiting low PD-L1 tumor expression, specifically those with a Tumor Proportion Score (TPS) of less than 10.

The Registration Study will involve approximately 212 patients and aims to verify the favorable safety profile and effectiveness of Multikine observed in previous trials. The prior Phase 3 study, which included 928 participants, suggested that Multikine could particularly benefit patients with low PD-L1 expression, a characteristic present in about 70% of head and neck cancer cases.

PD-L1 is a significant biomarker in cancer treatment, particularly with checkpoint inhibitors like pembrolizumab (Keytruda) and nivolumab (Opdivo), which are more effective in patients with high PD-L1 expression. The global market for checkpoint inhibitors was around $48 billion in 2023, with Keytruda leading sales projections for 2024 at $27 billion. However, Multikine has shown potential advantages for patients with lower PD-L1 expression, positioning it as a promising alternative for a substantial subset of head and neck cancer patients.

Geert Kersten, the CEO of CEL-SCI, emphasized the uniqueness of Multikine as a neoadjuvant immunotherapy that has demonstrated a survival benefit for head and neck cancer patients with low and negative PD-L1 expression. He highlighted the importance of the FDA’s agreement on the patient selection criteria, which will enhance the upcoming confirmatory Registration Study's success. This agreement underscores CEL-SCI’s collaborative efforts with the FDA to address the unmet medical needs of cancer patients with low PD-L1 expression.

CEL-SCI Corporation focuses on enhancing the immune system to provide the most significant possible survival benefit when it is still relatively intact. Multikine aims to stimulate the immune system to target tumors at an early stage, improving the body's ability to combat cancer. To date, Multikine has been administered to over 740 patients and has received Orphan Drug designation from the FDA for neoadjuvant therapy in head and neck cancer patients with squamous cell carcinoma.

The confirmatory Registration Study will include patients with newly diagnosed, locally advanced, and treatment-naive resectable head and neck cancer, who have no lymph node involvement and low PD-L1 tumor expression. This group represents about 100,000 patients annually. The study’s objective is to substantiate the positive safety and efficacy results previously observed in CEL-SCI’s Phase 3 trial.

CEL-SCI operates from Vienna, Virginia, with additional facilities near Baltimore, Maryland.