DEFA14A: CEL-SCI Identifies Target Patient Population for Multikine, Advances Regulatory Filings

CEL-SCI has identified a target patient population for its Multikine cancer therapy and is advancing regulatory filings after overcoming manufacturing challenges.

Summary

• CEL-SCI has identified a target patient population for Multikine, its investigational cancer therapy, based on Phase 3 trial results showing significant survival benefits in head and neck cancer patients treated before surgery with Multikine followed by surgery and radiotherapy, but not chemotherapy.
• The target population, identified by no lymph node involvement and low PD-L1 tumor expression, saw a 5-year risk of death cut in half, with a 73% survival rate compared to 45% in the control group, representing a 28.6% absolute 5-year overall survival benefit (p = 0.0015, hazard ratio = 0.35).
• The company has completed commissioning its cGMP manufacturing facility and is pursuing regulatory approvals from the FDA, EMA, MHRA, and Health Canada.
• CEL-SCI has submitted target population data and a proposed confirmatory study protocol to the FDA in Q1 2024.
• Health Canada has advised CEL-SCI to request advance consideration for approval under a Notice of Compliance with Conditions (NOCC) policy.
• The UK's NICE has selected Multikine for evaluation as a potential new standard of care for SCCHN.
• The EMA's Paediatric Committee granted CEL-SCI a product-specific waiver, removing a hurdle for commercialization in Europe.
• Meetings with UK regulators and the EMA are expected in H1 2024.
• CEL-SCI has filed a patent for the use of Multikine in tumors expressing low levels of PD-L1.
• CEL-SCI plans to seek approval for immediate patient access to Multikine without waiting on the results of a new trial wherever possible.

Sentiment

Score: 8

Explanation: The document presents a positive outlook due to the identification of a target patient population, promising clinical data, progress in manufacturing, and advancements in regulatory filings. The tone is optimistic, emphasizing the potential of Multikine to improve patient outcomes and deliver results to investors.

Positives

• Multikine demonstrated a significant survival benefit in a specific head and neck cancer patient population.
• The target patient population can be identified using routine tests.
• CEL-SCI has completed commissioning its cGMP manufacturing facility.
• The company is pursuing regulatory approvals from multiple agencies (FDA, EMA, MHRA, Health Canada).
• The EMA granted a product-specific waiver, easing the path to commercialization in Europe.
• Multikine shows promise in patients with low PD-L1 expression, a group where checkpoint inhibitors are less effective.
• CEL-SCI plans to seek approval for immediate patient access to Multikine without waiting on the results of a new trial wherever possible.

Negatives

• The initial Phase 3 trial results presented a conundrum regarding which patients would benefit from Multikine.
• Delays were experienced in commissioning the manufacturing facility due to prioritization of COVID-related medical supplies.

Risks

• The company faces the risk of not duplicating the clinical results demonstrated in clinical studies.
• There is a risk of not receiving necessary regulatory approvals.
• Difficulties in manufacturing any of the Company's potential products could arise.
• The company may face an inability to raise the necessary capital.

Future Outlook

CEL-SCI plans to seek approval for immediate patient access to Multikine without waiting on the results of a new trial wherever possible. The company is pursuing conditional approvals (or, in the U.S., accelerated approval) which means you can be approved first while a confirmatory study is ongoing and before that study is completed.

Management Comments

• The survival statistics in this target population are so good that it is hard to imagine how they could be challenged or ignored.
• Multikine's safety profile is very favorable compared to other oncology agents.
• CEL-SCI is ready and we believe patients and doctors are, too.
• While the biotech stock market always has its ups and downs, the clinical data will stand firm and drive the final success for CEL-SCI.

Industry Context

The document highlights the need for new treatments for previously untreated locally advanced primary resectable squamous cell carcinoma of the head and neck, as the current standard of care provides only a 50-50 chance of living past five years. Multikine's potential to improve survival rates in a specific patient population positions it as a possible competitor to existing treatments like Keytruda and Opdivo, especially in patients with low PD-L1 expression.

Comparison to Industry Standards

• The document mentions Keytruda and Opdivo as approved checkpoint inhibitors that typically show longer survival in patients with higher levels of tumor cell PD-L1 expression.
• Multikine's efficacy in patients with low PD-L1 expression differentiates it from these checkpoint inhibitors, suggesting a potential advantage in this specific patient population.
• The 5-year survival rate of 73% with Multikine in the target population is significantly higher than the current standard of care, which provides only a 50-50 chance of survival.

Stakeholder Impact

• Shareholders: Positive impact due to potential for increased value if Multikine receives regulatory approval and achieves commercial success.
• Patients: Potential for improved treatment outcomes and increased survival rates for head and neck cancer patients.
• Physicians: Potential for a new treatment option for a specific patient population with limited alternatives.
• Employees: Positive impact due to potential for company growth and job security.

Next Steps

• Pursue regulatory approvals from the FDA, EMA, MHRA, and Health Canada.
• Conduct a confirmatory study of 212 patients.
• Seek approval for immediate patient access to Multikine without waiting on the results of a new trial wherever possible.
• Meetings with UK regulators and the EMA are expected H1 2024.

Key Dates

Keywords

Multikine, CEL-SCI, Head and Neck Cancer, SCCHN, Immunotherapy, PD-L1, Clinical Trial, Regulatory Approval, FDA, EMA, Manufacturing, Oncology