VIENNA, Va. - CEL-SCI Corporation (NYSE American: CVM) has announced the FDA's concurrence with its approach to patient selection for a confirmatory Registration Study of Multikine, focusing on newly diagnosed head and neck cancer patients with low PD-L1 tumor expression. The study, set to begin in the first quarter of 2025, aims to confirm the safety and efficacy results seen in a previous Phase 3 study, where patients with low PD-L1 expression showed a significant survival benefit when treated with Multikine.
The Phase 3 study had 928 participants, with results indicating a 5-year survival rate of 73% for patients with low PD-L1 expression who received Multikine, compared to 45% in the control group. The upcoming study will enroll approximately 212 patients and seeks to prospectively confirm these outcomes.
PD-L1, a biomarker used in cancer treatment, is typically associated with patient selection for checkpoint inhibitors, a class of drugs in a $48 billion market. While drugs like pembrolizumab (Keytruda) and nivolumab (Opdivo) are generally more effective for patients with high PD-L1 expression, Multikine has shown promise for patients with low PD-L1 expression, which constitutes about 70% of head and neck cancer cases.
CEL-SCI's CEO Geert Kersten highlighted the importance of the FDA's agreement on patient selection methodology, emphasizing Multikine's unique position as the only neoadjuvant immunotherapy to demonstrate an overall survival benefit in this patient population. The company aims to address an unmet need for cancer patients with low PD-L1 expression.
Multikine is designed to activate the immune system against cancer early on when it is still intact. It has been dosed in over 740 patients and received Orphan Drug designation from the FDA for its use in treating squamous cell carcinoma of the head and neck.
The information in this article is based on a press release statement from CEL-SCI Corporation. The company, headquartered in Vienna, Virginia, with operations near Baltimore, Maryland, is preparing for a focused confirmatory study following the strong data from its completed randomized controlled Phase 3 study. Multikine has not yet been approved for sale by the FDA or any regulatory agency.
In other recent news, CEL-SCI Corporation's immunotherapy, Multikine, has been making significant strides in the treatment of head and neck cancer. Notably, the FDA has approved the company's patient selection criteria for an upcoming confirmatory Registration Study. The study, targeting patients with low PD-L1 tumor expression and no lymph node involvement, is anticipated to enroll 212 patients.
Additionally, the company has received a waiver from the UK's Healthcare Products Regulatory Agency, eliminating the need for pediatric trials of Multikine in the UK marketing approval process. Furthermore, CEL-SCI Corporation has announced a public offering of 10,845,000 shares, priced at $1.00 each, aiming to raise gross proceeds of $10.8 million. The funds are projected to be used for the further development of Multikine and general corporate needs.
In recent developments, the U.S. FDA's Oncologic Drugs Advisory Committee (ODAC) expressed concerns over the use of certain immune checkpoint inhibitors in patients with low PD-L1 expression. The ODAC's analysis may influence the future use of these treatments, potentially paving the way for alternative treatments like Multikine.
Finally, the company reported positive outcomes from a comprehensive bias analysis for its Phase 3 study of Multikine, supporting its clinical effect in extending patient survival. It is important to note that Multikine is still under investigation, and its safety and efficacy have not yet been established for any use.
InvestingPro Insights
CEL-SCI Corporation's ambitious plans for Multikine come at a challenging time for the company's financial performance. According to InvestingPro data, CEL-SCI has faced significant headwinds in the market, with its stock price declining 54.5% over the past six months and 72.06% year-to-date as of the latest available data.
The company's financial metrics reveal some concerns. CEL-SCI reported a negative gross profit of $18.95 million and an operating loss of $27.7 million over the last twelve months. These figures align with an InvestingPro Tip indicating that the company suffers from weak gross profit margins and is not profitable over the last twelve months.
Despite these challenges, CEL-SCI's focus on addressing an unmet need in head and neck cancer treatment for patients with low PD-L1 expression could potentially turn the tide. The upcoming Registration Study, set to begin in Q1 2025, may be a critical inflection point for the company's future prospects.
Investors should note that analysts do not anticipate the company will be profitable this year, as highlighted by another InvestingPro Tip. However, with the next earnings date scheduled for December 18, 2024, investors will have an opportunity to reassess the company's progress towards its clinical and financial goals.
For those interested in a deeper dive into CEL-SCI's financial health and market performance, InvestingPro offers 11 additional tips, providing a more comprehensive analysis of the company's position and potential.
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