Biotech CEL-SCI gets FDA go-ahead to conduct investigational cancer immunotherapy study

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CEL-SCI Corporation, the Vienna, Virginia-based biotechnology company with operations in Baltimore, Wednesday received approval from the U.S. Food and Drug Administration to move forward on an investigational cancer immunotherapy study for its drug Multikine.

The project will study the drug’s effectiveness in newly diagnosed advanced primary head and neck cancer patients with no lymph node involvement, determined via PET scan, and with low PD-L1 tumor expression, determined via biopsy.

The FDA agreed to a 212-person confirmatory Registration Study based on the strength of the safety and survival benefit data in the selected target population from the prior 928-person Phase 3 study. The confirmatory study will be a randomized controlled trial with two arms: Multikine treatment plus standard of care versus standard of care alone. As presented at the ESMO cancer conference in October 2023, Multikine-treated patients in the selected group had a 73% 5-year survival vs a 45% 5-year survival in the control group who did not receive Multikine.

If approved as a pre-surgical treatment, Multikine should be added to the standard of care for the target population. The FDA also acknowledged in the meeting that there is a great unmet need in the target population for improved therapies. This is an important factor that weighs in favor of approval for Multikine.

CEL-SCI officials believe that its de-risked value proposition for investors presents a unique opportunity to invest in a Phase 3 oncology company with a large body of data demonstrating not only tumor responses, but also long-term survival, in the target patient population. The goal of our smaller confirmatory study is to confirm these positive results in a prospectively defined target population.

Multikine received Orphan Drug designation from the FDA for neoadjuvant therapy in patients with squamous cell carcinoma of the head and neck.