Table of Contents Hide
Biotech company CEL-SCI announced today the long awaited results of their Phase 3 clinical trial for their immunotherapy product Multikine. The company believes that boosting the patient’s immune system while still intact prior to surgery, radiotherapy or chemotherapy can provide the greatest impact on overall survival and the latest trial works to enforce those beliefs.
Cel-Sci Multikine Study Overview
In the recent Phase 3 study, the largest ever for head & neck cancer patients, Cel-Sci treated newly diagnosed cancer patients with advanced primary squamous cell carcinoma of the head and neck with their investigational product Multikine prior to any surgical procedures, radiotherapy or chemotherapy. Surgery, followed by radiotherapy and chemotherapy is the current standard of care for these cancer patients.
Cel-Sci’s approach to cancer is unique and has been met with skepticism because to date few companies have set out to change the standard of care for cancer treatment. Most other cancer immunotherapies are only administered after conventional therapies, not prior. Cel-Sci believes that the immune system wears down during the current SOC which is why initial treatment is crucial.
Multikine (Leukocyte Interleukin, Injection) previously received Orphan Drug designation from the FDA for neoadjuvant therapy in patients with squamous cell carcinoma (cancer) of the head and neck.
The Phase 3 study began in Spring 2011 and saw a full enrollment of 928 patients by September 2016. This worldwide study encompassed 78 total medical sites spanning three continents of the globe. The ITT population was a total of 923 patients, 5 randomized patients were never treated. The primary focus comparisons of the study were the Multikine treatment regimen plus SOC versus SOC alone. To prove an overall survival benefit, the study required CEL-SCI to wait until at least 298 (deaths) events had occurred among the two main comparator groups. This study milestone occurred in late April 2020 and database lock occurred in December 2020.
Cel-Sci Multikine Phase 3 Study Results
On June 28, 2021 Cel-Sci announced the results of a near 10 year pivotal Phase 3 study for the immunotherapy Multikine leukocyte interleukin injection in the treatment of advanced primary squamous cell carcinoma of the head and neck.
Cel-Sci’s data release stated that for those patients where there was intent to treat the advanced primary squamous cell carcinoma of the head and neck, the study showed a statistically significant overall survival benefit of 14.1% with overall survival of 62.7% at five years for the group of patients receiving the Multikine treatment regimen followed by surgery and radiotherapy therapy, but not chemotherapy, as part of their standard of care treatment. The OS benefit increased over time, Cel-Sci said.
For patients treated with the Multikine regimen prior to surgery and radiotherapy, the results are a potential breakthrough and a near 5% increase is a celebrated win for any current patients, families or survivors who understand the battles of cancer.
Patients treated with the Multikine treatment regimen plus SOC vs. SOC alone had an overall survival benefit of 14.1% at five years which exceeded the pre-defined 10% overall survival benefit set out for the study population as a whole. Patients that received chemotherapy did not exhibit the same survival advantage as those with surgery and radiotherapy alone.
Multikine FDA Approval
There were no safety issues found for Multikine in the treated population during or as a result of administration. There were also no late effects of the overall treated population. Furthermore, with the demonstrated success in the separate group that did not receive chemotherapy and that analysis, CEL-SCI plans to seek U.S. Food and Drug Administration (FDA) approval for Multikine immunotherapy based on the demonstrated significant OS benefit and favorable safety profile in the unmet medical need head and neck cancer patients who received Multikine, surgery and radiotherapy as part of their SOC.
Cel-Sci believes this analysis and the positive results will meet regulatory requirements for FDA submission based on the protocol and Statistical Analysis Plan, which were prospectively concluded before database lock and unblinding.. The company also believes that these results mark the first-ever success of a neoadjuvant cancer immunotherapy in advanced primary head and neck cancer.
It should be noted that Multikine was tested in a trial focusing on cancers of the head and neck, but the immunotherapy has a tremendous range of potential for many types of cancer treatment in the future.