FDA gives OK, Cel-Sci clinical hold finally lifted
- Cel-Sci Corp. received long-awaited good news — a letter from the Food and Drug Administration that lifts the almost year-long hold on the Phase 3 Multikine study in first-line treatment of head and neck cancer.
- The study had enrolled 928 patients and all those who had completed treatment continue to be followed. The study's primary endpoint is a 10% increase in overall survival for patients treated with Multikine with standard of care (SOC), compared with SOC only, and will be met when there have been a total of 298 deaths in the two groups. Cel-Sci doesn't plan to enroll any further patients.
- Cel-Sci hasn't confirmed what the next step for the study will be, but has stated the FDA has agreed that "all clinical trial activities under this Investigational New Drug application (IND) may resume." The market shares the company's relief, with the share price opening over 40% higher.
Editor's Note: This article was updated after publication with comments from the CEO.
It's been a long trek for Cel-Sci, with a drug that was in clinical trials back in 2003. To cut a long story not particularly short, the FDA put the Multikine head and neck cancer Phase 3 on a partial clinical hold, stopping further enrollment. The letter cited unreasonable and significant risk of illness, the absence of prompt reports to the Independent Data Monitoring Committee (IDMC), errors in the investigator brochure and deficiencies in the protocol. In November 2016, Cel-Sci responded, and in December 2016, the FDA stated that the response was incomplete.
In January 2017, the discussions continued; Cel-Sci submitted data, records and information, and following "an open and frank discussion of the clinical hold issues raised by the FDA" with the regulatory authority, Cel-Sci then submitted an updated investigators brochure and a list of major protocol deviations that could affect the study results in April 2017. A further response in June 2017 took it a step closer to what the FDA was looking for and resulted in an uptick in the share price.
"We were able to show the FDA that the clinical hold was not justified," said Geert R. Kersten, director and CEO, in a call with BioPharma Dive this morning.
Because of the low death rate in both arms of the study, which has been ongoing for around seven years, Cel-Sci submitted an amendment in 2016 to expand the study. The amendment was withdrawn in 2017, because, as Kersten explained, changes in the death rates in the last year has meant that there was no longer a need to recruit more patients.
"We can't predict a date for when the study will complete, as it is based on the death rate, and we need to carry out more analysis of the data so far," said Kersten.
"Treating patients with an immunotherapy while they still have a healthy immune system, rather than waiting until they are very sick, makes logical sense. Cancer immunotherapy is unlikely to work at a late stage when patients have a heavy tumor burden, and so we see this as an option before surgery and radiotherapy. The key to treating cancer is likely to be a combination of approaches," added Kersten
Multikine is Cel-Sci's only clinical stage product, and it is also in development for the treatment of perianal warts in people coinfected with HIV and HPV, in collaboration with the University of California San Francisco.
"The study is ongoing but enrollment has been very slow, as people do not necessarily see anal warts as something that requires urgent treatment. Head and neck cancer, which is a horrible disease, remains our main driver," said Kersten.
CEL-1000, which uses Cel-Sci's LEAPS technology, is in preclinical development as a vaccine adjuvant and to prevent against infection from unknown pathogens.
- Cel-Sci Statement
Follow Suzanne Elvidge on Twitter