CEL-SCI Corporation (“CEL-SCI” or “the Company”) is a late-stage clinical biotechnology company focused on developing immunotherapy products and technologies to treat cancer, autoimmune and infectious diseases that address significant unmet medical needs. The Company aims to develop novel therapies with the potential to activate and utilize the body’s own immune system against the disease.
The Company is focused on developing product candidates based on two innovative technologies: (1) Multikine® (Leukocyte Interleukin, Injection), a next-generation, comprehensive immunotherapy; and (2) LEAPS™ (Ligand Epitope Antigen Presentation System), an immunotherapy vaccine technology platform.
CEL-SCI’s lead product candidate, Multikine®, is an investigational immunotherapy with a completed Phase 3 study for patients with squamous cell carcinoma of the head and neck (SCCHN) (locally advanced primary head and neck cancer), for which the Company has received Orphan Drug Status from the FDA. CEL-SCI intends to demonstrate that Multikine® could become an integral first-line component of the current standard of care (SOC) regimen due to its effectiveness and safety profile. Multikine® has also been studied for the treatment of cervical dysplasia in human immunodeficiency virus (HIV) and human papillomavirus (HPV) co-infected patients.
Current drug therapies for SCCHN from Merck & Company and Bristol Myers Squibb are approved as a last resort for recurrent head and neck tumors after treatments have failed or for patients who are not candidates for surgery.
Multikine® modulates the body’s immune system through a dual mechanism of action: eliciting the direct killing of tumor cells and micrometastasis, while generating a sustainable anti-tumor response.
Multikine® is being developed as a first-line treatment for advanced primary head and neck cancer, to be given as a neoadjuvant, prior to surgery and radiation or chemoradiation. The drug has been shown to augment/stimulate the immune system before it is weakened by both the toxic therapies and the cancer itself. Multikine® could become the world’s first cancer immunotherapy drug to be administered prior to surgery.
In its Phase 2 clinical trial for Multikine®, CEL-SCI reported a 10.5% complete response rate (no clinical or pathology evidence of any remaining cancer) and a 33% improvement in overall survival. Most importantly, there was no severe adverse events associated with the use of Multikine®.
Multikine® has completed the largest and longest ever pivotal Phase 3 clinical trial (IT-MATTER) on three continents. The drug is the first of its kind with substantial survival benefit in a randomized Phase 3 trial in locally advanced primary SCCHN.
CEL-SCI published and presented data from its pivotal randomized controlled Phase 3 Multikine® head and neck cancer clinical trial at two prominent cancer conferences: 2022 ASCO Annual Meeting in Chicago, Illinois and 2022 European Society for Medical Oncology (ESMO) annual Congress in Paris, France. Additional results from the Phase 3 clinical trial of Multikine® in advanced primary head and neck cancer were posted to the U.S. government clinical trial website.
In CEL-SCI’s Phase 3 study, Multikine® produced a very significant 14.1 percentage five-year survival benefit (62.7% versus 48.6%) as well as a nearly four-year overall survival benefit in the treatment arm receiving Multikine® prior to surgery plus radiotherapy, as compared to the control group receiving only surgery plus radiotherapy.
On March 8, 2023, CEL-SCI reported new data from its Phase 3 study in a poster presentation titled “Leukocyte Interleukin Injection (LI) immunotherapy followed by radiotherapy extends overall survival (OS) in treatment naïve locally advanced primary squamous cell carcinoma of the head & neck: the IT-MATTERS Study” at the 10th European Congress on Head & Neck Oncology (ECHNO) in Lisbon, Portugal.
The Company is primarily focused on completing and submitting a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for approval of Multikine® in treating advanced primary head and neck cancer.
CEL-SCI’s second proprietary technology platform, LEAPS™, is a vaccine technology platform designed to stimulate the immune system to fight bacterial, viral, and parasitic infections more effectively, as well as autoimmune conditions, transplant rejection, and cancer. Research for the development of the LEAPS™ technology as a treatment for RA has been funded in part via two Small Business Innovation Research (SBIR) grants from the National Institutes of Health totaling $1.725 million.
The inflammatory response driving RA may be different in different individuals, with failure of some patients to respond to certain therapies reflecting these differences. The LEAPS™ platform can be designed to produce a specific natural immune response depending on the type of construct used. Thus, once the nature of the inflammatory response has been identified, the patient can be treated with the appropriate LEAPS™ vaccine.
The Company operates a dedicated state-of-the art manufacturing facility with over 73,000 square feet of manufacturing and research and development (R&D) space, noting that the production capacity has been doubled to meet anticipated market demand for Multikine® once it receives regulatory approval. The Phase 3 trial was supplied from this facility, reducing regulatory risks at time of approval.
CEL-SCI’s manufacturing facility is being validated following the completion of its commercial scale build out during the first quarter of 2022. The construction was designed to ensure the facility will be compliant with both FDA GMP and European regulations.
CEL-SCI has added highly experienced individuals to its team, including scientists, biostatisticians, regulatory counsel who worked at the FDA, an oncologist who worked at the FDA as a clinical reviewer, and more than half a dozen independent oncologists to its Scientific Advisory Board, including key opinion leaders from major U.S. medical centers. Biographies of these key individuals are provided on pages 8-11.
As of December 31, 2022, CEL-SCI’s cash position was $18 million.