What is Multikine®


Multikine, CEL-SCI's lead investigational immunotherapy, is an immunotherapeutic agent that is being developed as a potential first-line neoadjuvant therapy in patients with squamous cell carcinoma of the head and neck, and potentially, as a treatment for cervical dysplasia or peri-anal warts in HIV/HPV co-infected patients. If it were to be approved for use following completion of CEL-SCI's clinical development program, Multikine would be a different kind of therapy in the fight against cancer; one that appears to have the potential to work with the body's natural immune system in the fight against tumors. Multikine may also have the potential to be a first in a new class of cancer immunotherapy drugs. It is the first investigational combination immunotherapy thought to have both passive and active immune properties and has displayed the following characteristics in studies to date:

  • Potential first-line treatment: Multikine is administered prior to any other therapy because CEL-SCI believes that this is the period when there is a greater potential likelihood of activating an anti-tumor immune response. Once the patient has had surgery or has received radiation and/or chemotherapy, the immune system may become weakened and may be less able to mount an anti-tumor immune response.
  • Potential to work with the body: Multikine investigational immunotherapy is directed at activating and possibly boosting the body's own anti-tumor immune response to potentially help the body in addressing the tumor.
  • A mixture of different immune molecules, potentially simulating the natural immune system: Multikine is composed of a defined mixture of naturally occurring molecules which in early-phase studies have been reported to be biologically/immunologically active.
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Multikine

Multikine (Leukocyte Interleukin, Injection) is the full name of this investigational therapy, which, for simplicity, is referred to in the remainder of this page as Multikine. Multikine has not been licensed or approved by the FDA or by any other regulatory agency. Similarly, its safety or efficacy has not been established for any use. Moreover, no definitive conclusions can be drawn from the early-phase, clinical-trials data summarized on this page or elsewhere on this website involving this investigational therapy. Further research is required, and early-phase clinical trial results must be confirmed in the well-controlled, Phase III clinical trial of this investigational therapy that is currently in progress.

In the Phase III clinical trial, Multikine is administered to cancer patients prior to their receiving any treatment for cancer, including surgery, radiation and/or chemotherapy

Clinical Trial Status


The first indication we are pursuing for our Multikine investigational product candidate is an indication for the neoadjuvant therapy in patients with squamous cell carcinoma of the head and neck, or SCCHN. The Phase III Clinical trial of the Multikine investigational neoadjuvant therapy in SCCHN. SCCHN is a type of head and neck cancer, and CEL-SCI believes that, in the aggregate, there is a large, unmet medical need among head and neck cancer patients. To the best of our knowledge, the last FDA approval of a therapy indicated for the treatment of advanced primary head and neck cancer was over 50 years ago. In the aggregate, head and neck cancer represents about 6% of the world's cancer cases, with approximately 650,000 patients diagnosed worldwide each year, and nearly 60,000 patients diagnosed annually in the United States. Multikine investigational immunotherapy was granted Orphan Drug designation for neoadjuvant therapy in patients with SCCHN by the FDA in the United States.

  • Reported average of 50% reduction in tumor cells in Phase II trials (based on 19 patients evaluable by pathology, having OSCC)
  • Reported 10.5% complete response in the Phase II trial (based on 19 patients evaluable by pathology, having OSCC)

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Phase III Trial Design


The Phase III Clinical Trial: Multikine has been cleared for a global Phase III trial in advanced primary head and neck cancer. It has received a go-ahead from 24 Regulatory Agencies worldwide including leading regulators in the U.S., Canada, France and the United Kingdom. The Phase III accrued its first patient in early 2011 and complete enrollment in September 2016.

  • Number of Subjects Enrolled: 928
  • Clinical Centers: approximately 100 globally distributed; North America, Europe, Asia

For more information about the Phase III Study please see www.clinicaltrials.gov (NCT number: NCT01265849)

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Study Summary: A Pivotal Phase III, Open-label, Randomized, Controlled Multi-center Global Study of the Effects of Multikine Plus Standard of Care (Surgery + Radiotherapy or Surgery + Concurrent Chemoradiotherapy) in Subjects with Advanced Primary Squamous Cell Carcinoma of the Oral Cavity and Soft Palate Versus Standard of Care Only.

Current Understanding of the Potential Mechanism of Action of the Investigational Therapy Multikine (Leukocyte Interleukin, Injection)

Mechanism of Action


Clinical and pathology data from Phase I and Phase II clinical trials with the Multikine investigational therapy published in peer reviewed scientific journals (Feinmesser et al Arch Otolaryngol. Surg. 2003; Timar et al 2003 and 2005) suggest that Multikine has the potential to produce an anti-tumor response, as the reported data from these early-stage studies suggests:

  • Multikine potentially may recognize and/or bind to multiple (different) antigens (or receptors) on the cancer cells.
  • Multikine potentially may directly affect/kill cancer cells: The various cytokines present in the Multikine investigational therapy, such as TNF, IL-2, IFN, along with other cytokines, could be responsible for this potential activity.
  • Multikine may signal the immune system to produce an anti-tumor immune response: Clinical data (reported from Phase II studies) also suggest that Multikine could augment the type of cells that infiltrate and attack the tumor from CD-8 cells to predominantly CD-4 cells. These CD-4 cells have the potential to bring about an anti-tumor immune response (Timar et al 2005).

Multikine may have the potential to render the remaining cancer cells susceptible to radiation and chemotherapy treatment (Timar et al 2003).

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Potential indications/
relevance to the medical community


The first indication we are pursuing for our Multikine product candidate is for the neoadjuvant therapy used as first-line treatment in patients newly diagnosed with squamous cell carcinoma of the head and neck, or SCCHN. SCCHN is a type of head and neck cancer. In Phase I and Phase II clinical trials and in the ongoing Phase III clinical trial, Multikine was being administered prior to any other cancer therapy because CEL-SCI believes that this is the period when there is a greater potential likelihood of activating an anti-tumor immune response.

  • Safety reports from Phase I and Phase II clinical trials (as reported by the clinical investigators)
  • During the early investigational phase, in Phase I and Phase II clinical trials in over 220 subjects who received the investigational therapy Multikine in (daily) doses of 200 to 3200 IU as IL-2, no serious adverse events were reported as being expressly due to administration of this investigational therapy.
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Potential Indications of our Investigational Therapy Multikine to the Medical Community