CEL-SCI Receives FDA Go-Ahead for Its Confirmatory Study of Multikine in the Treatment of Head & Neck Cancer

Rhea-AI Summary

CEL-SCI has received FDA approval for a confirmatory study of its cancer immunotherapy Multikine for head and neck cancer patients, based on strong Phase 3 data showing a 73% survival rate with Multikine. The study will enroll 212 patients and aims to confirm the results of the Phase 3 trial, paving the way for potential approval. The FDA acknowledged the unmet need for improved therapies in this patient population, supporting approval for Multikine.

Positive

• FDA approved CEL-SCI's confirmatory study for Multikine based on strong Phase 3 data showing a 73% survival rate with Multikine.
• The confirmatory study will enroll 212 patients, demonstrating a clear path forward for potential approval.
• CEL-SCI's de-risked value proposition for investors presents a unique opportunity in the oncology sector with a large body of data showing positive results in the target patient population.

Negative

• None.

Oncology Doctor

The recent FDA approval for CEL-SCI to proceed with its confirmatory study of Multikine is a significant development in the oncology field, particularly for the treatment of head and neck cancer. The observed survival rates in the Phase 3 trial indicate a strong potential for efficacy. In oncology, survival rates provide substantial evidence of treatment effectiveness. The selected patient profile, restricted to those with no lymph node involvement and low PD-L1 tumor expression, is characteristic of a precision medicine approach, aiming to maximize therapeutic impact while minimizing unnecessary exposure. From an oncological standpoint, this study stands to confirm a targeted intervention strategy, which could translate into a new standard of care if results are replicated and the treatment is approved. The emphasis on pre-surgery administration of Multikine could also pioneer a shift in treatment paradigms.

Medical Research Analyst

The design and scale of the confirmatory Registration Study are noteworthy. In clinical research, the reduction from a 928-patient to a 212-patient study without compromising statistical power is indicative of the robustness of the prior results. A smaller cohort means that CEL-SCI is confident in Multikine's efficacy and the ability to demonstrate it in a confirmatory study. This approach is cost-effective and time-efficient, which could accelerate the path to market upon successful results. Additionally, the strict selection criteria that align with the FDA's stringent standards for newly diagnosed patients reflect a rigorous scientific and regulatory strategy that aims to ensure patient safety and a clear demonstration of benefit.

Financial Analyst

The unmet need acknowledged by the FDA suggests that if Multikine's confirmatory trials are successful and result in approval, CEL-SCI could capture significant market share in the head and neck cancer treatment space. This could potentially lead to a substantial increase in the company's valuation. However, investors must consider the inherent risks of investing in biotechnology firms, as the path from trial to commercial success is fraught with regulatory hurdles and market adoption challenges. Furthermore, the 'de-risked' narrative should be weighed against the biotech sector's unpredictability. In the short term, positive interim data from the confirmatory study could act as value inflection points for the stock, while long-term value is contingent upon regulatory approval and successful commercialization.

• Critical milestone achieved—the FDA accepted CEL-SCI’s selection criteria defining the Multikine target population before surgery
• Selected patient population had 73% survival with Multikine vs. 45% without Multikine based on analysis of completed 928-patient Phase 3 randomized controlled trial
• 212-patient confirmatory study is de-risked, backed by robust efficacy and safety data from completed Phase 3 trial, the largest in advanced primary head and neck cancer
• View CEL-SCI’s comprehensive report regarding the FDA’s go-ahead and the Company’s value proposition for investors here: https://cel-sci.com/wp-content/uploads/2024/05/CEL_SCI-Report-on-FDA-Path-Forward-May-2024-FINAL.pdf

VIENNA, Va.--(BUSINESS WIRE)-- CEL-SCI Corporation (NYSE American: CVM) today announced a significantly positive outcome from its recent meeting with the U.S. Food and Drug Administration (FDA) regarding the path to approval for its first-line investigational cancer immunotherapy Multikine* (Leukocyte Interleukin, Injection). Based on strong safety and efficacy data from CEL-SCI’s completed Phase 3 head and neck cancer study, the FDA indicated CEL-SCI may move forward with a confirmatory Registration Study of Multikine in newly diagnosed advanced primary head and neck cancer patients with no lymph node involvement (determined via PET scan) and with low PD-L1 tumor expression (determined via biopsy).

“Through this discussion and agreement with the FDA, we have achieved a tremendous milestone for people who are newly diagnosed with head and neck cancer. The survival benefit was so strong and clear in the target patient population that our confirmatory study needs to enroll only 212 people to confirm what was already achieved in the Phase 3 study. This gives us a clear path forward,” stated CEL-SCI CEO, Geert Kersten. “We are eager to begin the study as soon as possible.”

CEL-SCI published a report on the FDA’s agreement and Multikine’s path forward. Please click on this link https://cel-sci.com/wp-content/uploads/2024/05/CEL_SCI-Report-on-FDA-Path-Forward-May-2024-FINAL.pdf to read the full report. Highlights include:

• The FDA agreed to a 212-person confirmatory Registration Study based on the strength of the safety and survival benefit data in the selected target population from the prior 928-person Phase 3 study. The confirmatory study will be a randomized controlled trial with two arms: Multikine treatment plus standard of care versus standard of care alone. As presented at the ESMO cancer conference in October 2023, Multikine-treated patients in the selected group had a 73% 5-year survival vs a 45% 5-year survival in the control group who did not receive Multikine.
• Generally, patient selection for different treatments in newly diagnosed head and neck cancer is done only after surgery. That presented CEL-SCI with a challenge, because Multikine has to be given before surgery. By analyzing Multikine's biological mechanism of action, as supported by the completed Phase 3 study, CEL-SCI developed criteria for selecting, before surgery, those patients who would have the best survival from Multikine. The FDA accepted the selection criteria and the proposed study design, which now permits CEL-SCI to enroll patients in the confirmatory study.
• CEL-SCI met a very high bar set by the FDA, which requires more stringent analysis for newly-diagnosed patients than for terminal cancer patients. One regulator called these newly-diagnosed cancer patients “much more delicate” and explained that the standard for permitting a new study with these patients has to be more stringent, since they are not all expected to die.
• CEL-SCI has been advised by statisticians and physicians that the confirmatory study has a high likelihood of success because a large survival benefit has already been demonstrated in the target population in the completed Phase 3 study. The much smaller confirmatory study—less than a quarter the size of the prior study—will focus on the patients who saw the greatest survival benefit when treated with Multikine.
• If approved as a pre-surgical treatment, Multikine should be added to the standard of care for the target population.
• The FDA also acknowledged in the meeting that there is a great unmet need in the target population for improved therapies. This is an important factor that weighs in favor of approval for Multikine.
• CEL-SCI believes that its de-risked value proposition for investors presents a unique opportunity to invest in a Phase 3 oncology company with a large body of data demonstrating not only tumor responses, but also long-term survival, in the target patient population. The goal of our smaller confirmatory study is to confirm these positive results in a prospectively defined target population.

About CEL-SCI Corporation

CEL-SCI believes that boosting a patient’s immune system while it is still intact should provide the greatest possible impact on survival. Multikine is designed to help the immune system "target" the tumor at a time when the immune system is still relatively intact and thereby thought to be better able to mount an attack on the tumor. In the completed Phase 3 study, CEL-SCI studied patients who were newly diagnosed with locally advanced primary squamous cell carcinoma of the head and neck with the investigational product Multikine administered first, before they received the standard of care, which involved surgery followed by either radiation or chemoradiation. The Phase 3 study enrolled 928 patients. Our approach is unique because most other cancer immunotherapies are administered only after conventional therapies have been tried and/or failed.

After analyzing data from the Phase 3 study, we have better defined the target population for Multikine, which is locally advanced primary head and neck cancer patients with no lymph node involvement and with low PD-L1 tumor expression. In the Phase 3 study, we observed statistically significant survival in this target population, showing that Multikine cut the risk of death in half at five years vs control.

Multikine (Leukocyte Interleukin, Injection) received Orphan Drug designation from the FDA for neoadjuvant therapy in patients with squamous cell carcinoma (cancer) of the head and neck.

The Company has operations in Vienna, Virginia, and near/in Baltimore, Maryland.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. When used in this press release, the words "intends," "believes," "anticipated," "plans" and "expects," and similar expressions, are intended to identify forward-looking statements. Such statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Such statements include, but are not limited to, statements about the terms, expected proceeds, use of proceeds and closing of the offering. Factors that could cause or contribute to such differences include an inability to duplicate the clinical results demonstrated in clinical studies, timely development of any potential products that can be shown to be safe and effective, receiving necessary regulatory approvals, difficulties in manufacturing any of the Company's potential products, inability to raise the necessary capital and the risk factors set forth from time to time in CEL-SCI's filings with the Securities and Exchange Commission, including but not limited to its report on Form 10-K for the year ended September 30, 2023. The Company undertakes no obligation to publicly release the result of any revision to these forward-looking statements which may be made to reflect the events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.

* Multikine (Leukocyte Interleukin, Injection) is the trademark that CEL-SCI has registered for this investigational therapy. This proprietary name is subject to FDA review in connection with the Company's future anticipated regulatory submission for approval. Multikine has not been licensed or approved for sale, barter or exchange by the FDA or any other regulatory agency. Similarly, its safety or efficacy has not been established for any use.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240508140076/en/

Gavin de Windt

CEL-SCI Corporation

(703) 506-9460

Source: CEL-SCI Corporation