CEL-SCI Corporation (NYSE American: CVM) has announced new breakthrough data from its Phase 3 trial of Multikine®, an innovative immunotherapy for patients with locally advanced resectable head and neck cancer. Presented at the European Society for Medical Oncology (ESMO) 2024 Congress, the data revealed that patients treated with Multikine achieved an impressive 82.6% 5-year survival rate, significantly higher than the 47.3% survival rate in patients who received standard care alone. This groundbreaking improvement highlights Multikine’s potential to transform treatment for head and neck cancers, which historically have had poor long-term survival outcomes.
The company also presented its Phase 3 data at ESMO 2022, which revealed that Neoadjuvant Multikine achieved groundbreaking tumor response and extended overall survival in locally advanced head and neck cancer patients.
On September 10, 2022, Dr. Philip Lavin, a biostatistician with 80 FDA approvals/clearances, and Dr. József Tímár, Professor and Director of the Central Pathology Laboratory at Semmelweis University presented poster presentations at the European Society for Medical Oncology (ESMO) annual Congress in Paris, France.
Summary of New Data Presented at ESMO 2024
The new findings presented at ESMO provide a more detailed analysis of the 114 patients from the completed Phase 3 study who met the selection criteria for the target population, forming the basis for the upcoming confirmatory study. The focus of this analysis was on the 79 patients considered low risk for recurrence, who were recommended to receive only radiotherapy (without chemotherapy) after surgery, as per National Comprehensive Cancer Network (NCCN) guidelines. This group was compared to the 35 high-risk patients, who were advised to receive chemotherapy in addition to radiotherapy after surgery.
The overall survival benefit for the entire target population (n=114) was both clear and statistically significant (log rank p=0.0015). However, the 79 low-risk patients showed even greater benefits from receiving Multikine prior to surgery. In this subset, the 38 patients treated with Multikine had a 5-year overall survival rate of 82.6%, compared to 47.3% for the 41 patients who received standard care. Their respective 95% confidence intervals did not overlap, underscoring the statistical strength of the results. A more recent analysis of the low-risk group (n=79) showed a hazard ratio of 0.27 (95% CI [0.12, 0.64], Wald p=0.0027), reflecting a 73% reduction in the overall risk of death.
The Burden of Head and Neck Cancer
Head and neck cancer poses a serious public health challenge. According to the American Cancer Society, approximately 66,000 new cases of head and neck cancers will be diagnosed in 2024 in the U.S., with around 14,000 deaths. These cancers, originating primarily in the oral cavity, throat, and larynx, are aggressive and their prognosis depends heavily on early detection and intervention.
Despite advancements in surgery, radiation, and chemotherapy, survival rates for locally advanced resectable head and neck cancers remain dismal. For patients receiving the standard combination of surgery and radiotherapy, the 5-year survival rate is typically below 50%. Many patients experience recurrence or relapse, underscoring the urgent need for more effective treatment options. CEL-SCI’s Multikine represents a promising new avenue for improving these outcomes.
The Promise of Immunotherapy in Cancer Treatment
In recent years, immunotherapy has become a powerful tool in oncology, offering new hope for patients with various cancers. Immunotherapies like Pembrolizumab (Keytruda) and Nivolumab (Opdivo) have already been used to treat head and neck cancers by boosting the body’s immune response to target and destroy cancer cells. These therapies have provided modest improvements in survival but often come with challenges, including high costs and variable effectiveness across patient groups.
What sets Multikine apart from other immunotherapies is its unique application as a neoadjuvant treatment, meaning it is administered before surgery to help prime the immune system. This approach aims to strengthen the body’s immune response, targeting cancer cells before they can spread further. Unlike chemotherapy, which can weaken the immune system, immunotherapies like Multikine work by activating it, potentially reducing the risk of recurrence or metastasis.
CEL-SCI’s Multikine: A Closer Look at the Phase 3 Trial Results
CEL-SCI’s Phase 3 trial, which included 923 patients, offers compelling evidence of Multikine’s efficacy. The trial focused on patients newly diagnosed with locally advanced head and neck cancer, dividing them into risk categories based on their likelihood of recurrence after surgery. The results were remarkable for patients classified as low-risk for recurrence.
The most striking result was the 82.6% 5-year survival rate observed in the low-risk group treated with Multikine. By comparison, the survival rate for patients receiving standard care alone was just 47.3%. Multikine-treated patients also experienced a 73% reduction in the overall risk of death, with a hazard ratio of 0.27. In simple terms, this means that patients receiving Multikine before surgery had a significantly lower risk of death compared to those who did not receive the therapy.
Additionally, patients in the Multikine group benefited from a median overall survival advantage of 46.5 months (nearly 4 years) over the control group. This data clearly shows that Multikine has the potential to significantly extend life expectancy for head and neck cancer patients, particularly those in the low-risk category.
The Upcoming Confirmatory Registration Study
Following the success of the Phase 3 trial, CEL-SCI is preparing for an FDA-approved confirmatory Registration Study to further validate Multikine’s efficacy. This new study will focus specifically on the patient population that showed the greatest survival benefit in the previous trial—those with newly diagnosed locally advanced primary head and neck cancer, who have no lymph node involvement and low PD-L1 tumor expression.
PET scans will be a critical tool in the confirmatory study, allowing for more accurate patient selection. These scans provide detailed imaging, enabling clinicians to better identify patients who are most likely to benefit from Multikine. By refining patient selection with advanced diagnostic tools, CEL-SCI aims to push the 5-year survival rate beyond the current 82.6%.
The confirmatory study will also aim to establish a standardized protocol for selecting patients based on risk profiles. Focusing on those deemed low-risk for recurrence—who are not recommended to receive chemotherapy post-surgery—this approach could help improve survival rates while minimizing unnecessary treatments.
Expert Commentary – Oncologists Weigh In
Oncologists and researchers specializing in head and neck cancers have expressed optimism about Multikine’s potential to improve survival outcomes. Geert Kersten, CEL-SCI’s CEO, emphasized the importance of targeting the right patients for treatment, explaining that Multikine appears to offer the greatest benefit to those who are not candidates for chemotherapy. This is in line with existing knowledge that chemotherapy, while effective in some cases, can have an immunosuppressive effect, potentially reducing the efficacy of immunotherapies like Multikine.
Dr. József Tímár, a co-author of the study and Professor at Semmelweis University in Budapest, highlighted the significance of these findings at the ESMO 2024 Congress. “The additional data provides further evidence that we have identified the target population that has the greatest survival benefit from Multikine,” he stated. This insight underscores the potential of Multikine to become a first-line treatment for certain subsets of head and neck cancer patients, particularly those who are not recommended for chemotherapy.
Market and Economic Implications
Multikine’s success could have significant implications for both patients and CEL-SCI’s market position. As the global market for cancer immunotherapies continues to expand, CEL-SCI’s breakthrough therapy could make the company a major player in this sector. The global immunotherapy market, valued at $95 billion in 2023, is projected to grow to over $150 billion by 2030, driven by increased demand for effective cancer treatments.
CEL-SCI’s stock (CVM), which has already seen positive momentum following the announcement of the Phase 3 trial results, stands to benefit further as the company moves closer to Multikine FDA approval. Investors are closely monitoring the outcome of the confirmatory Registration Study, as a successful trial could lead to broad market adoption of Multikine and significantly increase the company’s valuation.
Conclusion: A New Era in Head and Neck Cancer Treatment?
CEL-SCI’s Phase 3 trial results for Multikine have set the stage for a potential paradigm shift in the treatment of locally advanced resectable head and neck cancer. With its ability to improve 5-year survival rates to 82.6%, Multikine offers a beacon of hope to patients who previously had limited treatment options. As CEL-SCI prepares for its confirmatory study and works toward FDA approval, the oncology community and patients alike are watching with anticipation. Should Multikine continue to demonstrate such profound benefits, it may soon become a cornerstone in the fight against head and neck cancer.
