CEL-SCI's multidecade Multikine journey extends with FDA request for new trial

CEL-SCI will need to conduct another clinical trial for its Multikine head and neck cancer drug—which the company has long trumpeted as a promising treatment, despite data that say otherwise.

After Multikine failed to improve on overall survival achieved by standard of care in the overall population of head and neck cancer patients during a phase 3 trial back in 2021, CEL-SCI began looking at a subgroup population. The company pointed to data showing that a group of patients who hadn’t yet received chemotherapy had overall survival at five years that was 14 percentage points higher than the control group. And so, CEL-SCI’s strategy shifted to this group.

With that focus in mind, CEL-SCI has been meeting with regulators around the world to try and shore up an approval. The company provided an update on the U.S. effort today, saying that the FDA has agreed on the patient selection process, but would like a new confirmatory clinical trial to prove efficacy in newly diagnosed locally advanced squamous cell carcinoma of the head and neck.

That means CEL-SCI’s many years-quest to get an FDA nod will take a few years longer. The company was formed around Multikine in 1983. 

CEO Geert Kersten pledged to work with the FDA to design a study protocol “that will allow us to generate, as expeditiously as possible, the confirmatory data they require for approval of Multikine.”

“This includes confirming the pre-surgical tumor responses that arise within just weeks of Multikine treatment in the agreed upon population,” Kersten said in a statement.

CEL-SCI is now prepping more information about its development plan for the next FDA meet-up.

Meanwhile, CEL-SCI is trying to convince regulators elsewhere that Multikine is ready to be approved as is. These efforts are furthest along in Canada, where the company was told an advanced consideration of approval may be possible. If Health Canada were to grant the early nod, Multikine could arrive in the country as early as 2024, CEL-SCI said.

CEL-SCI is hoping for the same advanced nods in both Europe and Britain, with a promise to either conduct a confirmatory trial after approval or enroll a small number of patients to show the work is underway.

It’s been a long journey for Multikine. Keersten has long said that the therapy will lead to a “promising future” for the company, but the data has yet to prove that. CEL-SCI’s shares have plummeted from $24 apiece in July 2021 prior to the phase 3 data drop to $2.26 as of Friday morning.