Cel-Sci withdraws 2016 study protocol amendment
- Cel-Sci has withdrawn its trial amendment, submitted to the Food and Drug Administration in 2016, according to a shareholder letter released by the company yesterday. The company will continue to follow the 928 patients enrolled so far in the Phase 3 trial of its lead immunotherapeutic, Multikine, until the endpoint of 298 deaths between the two groups in the study. The future of the study had been under some doubt since the FDA put a partial clinical hold in place in September 2016.
- Cel-Sci is in arbitration against the clinical research organization that originally ran the Phase 3 trial, seeking at least $50 million damages over alleged breach of contract and fraud. The trial began in mid-2016 and Cel-Sci expects it to conclude in second quarter 2017.
- Cel-Sci's shares closed almost 6% higher on the news, but the share price is down almost 83% from the initial announcement of the partial clinical hold, and over 99% down from its opening value in December 1983. The company's current market cap is $20.32 million.
Without providing further details, Cel-Sci said: "In light of new information we have recently decided to withdraw the study protocol amendment for additional patients that we had submitted to the FDA last summer. It is now possible that we may not need to add more patients to the study or that only a smaller number of patients need to be added to the study to complete it in a reasonable period of time. Should additional patients be needed, we will submit a future study amendment to the FDA to seek their clearance to proceed."
Cel-Sci's Multikine is a cancer immunotherapy that triggers both the active and passive immune system, and is designed to be given before adjuvant radiotherapy or chemoradiotherapy. As the company's only clinical stage drug, it has been a long story so far, with clinical trials beginning in 2003, and it looks like it's only going to run longer.
In 2016, Cel-Sci hit its 880-patient recruitment target in head and neck cancer, but spotted a lower than expected death rate, meaning that it would be more difficult to see the primary endpoint of an increase of 10% in overall survival within a realistic timescale. So, Cel-Sci asked the Food and Drug Administration if it could increase the study size, and recruitment continued, rising to 928.
However, in September 2016, within two months of submitting the protocol amendment, the Food and Drug Administration hit the pause button for the Multikine Phase 3 clinical trial, putting it on partial clinical hold. Cel-Sci had to stop recruitment but was allowed to continue treatment and follow-up for patients already recruited.
In February 2017, Cel-Sci had a Type A meeting with the FDA, and has responded to the two action items to address the partial clinical hold: updating the investigator's brochure and compliance procedures and providing a list of major protocol deviations that could affect the study results, as well as a plan to identify such deviations across all the enrolled patients.
"We have been in a continuing dialogue with [the FDA] to address their questions and to supply them with supplemental information," said Cel-Sci in a statement. "We are working extremely hard to bring our Phase 3 trial to a successful conclusion, to have the FDA's partial clinical hold lifted, and to win the arbitration case against the former CRO that originally ran our Phase 3 trial."
Multikine has also been assessed in Phase 1 in cervical dysplasia in women co-infected with HIV and HPV, and in peri-anal warts in co-infected men and women.
- Cel-Sci Shareholder letter
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