On May 8, 2024 CEL-SCI Corporation (NYSE American: CVM) reported a significantly positive outcome from its recent meeting with the U.S. Food and Drug Administration (FDA) regarding the path to approval for its first-line investigational cancer immunotherapy Multikine* (Leukocyte Interleukin, Injection) (Press release, Cel-Sci, MAY 8, 2024, View Source [SID1234642912]). Based on strong safety and efficacy data from CEL-SCI’s completed Phase 3 head and neck cancer study, the FDA indicated CEL-SCI may move forward with a confirmatory Registration Study of Multikine 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).

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"Through this discussion and agreement with the FDA, we have achieved a tremendous milestone for people who are newly diagnosed with head and neck cancer. The survival benefit was so strong and clear in the target patient population that our confirmatory study needs to enroll only 212 people to confirm what was already achieved in the Phase 3 study. This gives us a clear path forward," stated CEL-SCI CEO, Geert Kersten. "We are eager to begin the study as soon as possible."

CEL-SCI published a report on the FDA’s agreement and Multikine’s path forward. Please click on this link View Source to read the full report. Highlights include:

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 (Free ESMO Whitepaper) 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.
Generally, patient selection for different treatments in newly diagnosed head and neck cancer is done only after surgery. That presented CEL-SCI with a challenge, because Multikine has to be given before surgery. By analyzing Multikine’s biological mechanism of action, as supported by the completed Phase 3 study, CEL-SCI developed criteria for selecting, before surgery, those patients who would have the best survival from Multikine. The FDA accepted the selection criteria and the proposed study design, which now permits CEL-SCI to enroll patients in the confirmatory study.

CEL-SCI met a very high bar set by the FDA, which requires more stringent analysis for newly-diagnosed patients than for terminal cancer patients. One regulator called these newly-diagnosed cancer patients "much more delicate" and explained that the standard for permitting a new study with these patients has to be more stringent, since they are not all expected to die.

CEL-SCI has been advised by statisticians and physicians that the confirmatory study has a high likelihood of success because a large survival benefit has already been demonstrated in the target population in the completed Phase 3 study. The much smaller confirmatory study—less than a quarter the size of the prior study—will focus on the patients who saw the greatest survival benefit when treated with 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 believes 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.