FDA Approves CEL-SCI's Multikine Study for Head & Neck Cancer

CEL-SCI Corporation has announced a significant milestone following a meeting with the U.S. Food and Drug Administration (FDA) regarding its investigational cancer immunotherapy, Multikine (Leukocyte Interleukin, Injection). The FDA has given CEL-SCI the green light to proceed with a confirmatory registration study, aimed at patients newly diagnosed with advanced primary head and neck cancer, who have no lymph node involvement and exhibit low PD-L1 tumor expression.

The decision follows the analysis of CEL-SCI’s completed Phase 3 trial involving 928 patients, which demonstrated strong safety and efficacy data. In this trial, patients in the target population who received Multikine treatment before surgery showed a 73% five-year survival rate, compared to a 45% rate in those who did not receive Multikine. This significant difference has paved the way for the smaller, 212-patient confirmatory study that will focus specifically on the subgroup that benefited the most from Multikine.

CEL-SCI's CEO, Geert Kersten, emphasized the importance of this milestone, stating that the confirmatory study needs to enroll only 212 patients to verify the positive results previously achieved. He expressed eagerness to commence the study promptly.

The upcoming confirmatory study will be designed as a randomized controlled trial with two arms: one group will receive Multikine in addition to the standard of care, while the other group will receive only the standard of care. This design is intended to further validate the survival benefits observed in the Phase 3 study.

Traditionally, patient selection for different treatments in newly diagnosed head and neck cancer occurs post-surgery. Given that Multikine must be administered pre-surgery, CEL-SCI faced a unique challenge. By examining Multikine's biological mechanism, supported by Phase 3 data, the company developed criteria to identify patients who would benefit most from Multikine before surgery. The FDA has accepted these selection criteria and the study design, allowing CEL-SCI to proceed with patient enrollment.

The FDA's stringent requirements for newly diagnosed cancer patients, who are considered more delicate compared to terminal patients, highlight the robustness of CEL-SCI’s data. A regulator noted the higher standard needed for such studies, given that these patients are not all expected to die.

Statisticians and physicians advising CEL-SCI believe the confirmatory study has a high likelihood of success, given the significant survival benefit already demonstrated in the Phase 3 trial. The smaller size of the confirmatory study, focusing on the most responsive subgroup, should enable quicker and more conclusive results.

Approval of Multikine as a pre-surgical treatment could potentially integrate it into the standard of care for the identified patient population. Furthermore, the FDA acknowledged during the meeting the significant unmet need for improved therapies in this target group, an important consideration favoring Multikine’s approval.

CEL-SCI’s confidence in its de-risked value proposition for investors stems from the extensive data showing Multikine’s tumor response and long-term survival benefits in the target population. The smaller confirmatory study aims to reinforce these positive results, offering a unique investment opportunity in a Phase 3 oncology company with compelling efficacy and safety data.

CEL-SCI Corporation operates from Vienna, Virginia, and near Baltimore, Maryland. The company’s approach of boosting patients’ immune systems while still intact aims to enhance survival rates. Multikine is designed to help the immune system target tumors more effectively before conventional therapies, such as surgery, radiation, or chemoradiation, are applied. In the Phase 3 study, Multikine was administered first to newly diagnosed patients with locally advanced primary squamous cell carcinoma of the head and neck, leading to a significant survival advantage.

Multikine has received Orphan Drug designation from the FDA for neoadjuvant therapy in head and neck cancer, underscoring its potential significance in cancer treatment.