CEL-SCI issues letter to shareholders regarding Multikine regulatory pathway

CEL-SCI released a letter to shareholders from the company’s CEO, Geert Kersten. The letter details the data reported on the efficacy of Multikine in the head and neck cancer target patient population as well as CEL-SCI’s plan to file for immediate regulatory approval. “This shareholder letter will be longer and much more detailed than prior letters. My goal is to show you the clinical data supporting Multikine’s survival benefits, explain how we identified the target population of head and neck cancer patients who should receive Multikine, and describe our efforts with the world’s leading regulatory bodies to bring Multikine to market as quickly as possible. Why are we confident that Multikine should be approved as soon as possible? First, we can clearly identify patients who should get Multikine. Second, Multikine definitely benefits patients by causing pre-surgical responses. Third, the Multikine survival benefit in the target population is outstanding. Fourth, as a statistical matter, another trial is more than 95% likely to be successful and therefore should not be necessary for approval. Fifth, there are regulatory pathways specifically designed for our situation where the target population is selected from the larger Phase 3 study population. We believe that we meet these factors with strong evidentiary support and are eager to move forward… By way of summary, Phase 3 patients in the finalized target population saw the following: risk of death cut in half at five years versus the control; 28.6% absolute 5-year overall survival benefit versus control (p=0.0015); 0.349 hazard ratio vs control (95% CIs (0.18, 0.66), Wald p=0.0012); greater than35% rate of pre-surgery tumor reductions and/or downstages (pless than0.01); and low PD-L1 tumor expression (vs high PD-L1 where Keytruda and Opdivo work best). We can identify at diagnosis the patients most likely to have pre-surgical responses to Multikine. 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. And, we have a manufacturing plant that can make an estimated $2 billion worth of Multikine annually. CEL-SCI is ready-and we believe patients and doctors are, too. A new drug for our targeted disease (previously-untreated locally advanced primary resectable squamous cell carcinoma of the head and neck) has not been approved by FDA in decades. The current standard of care provides only a 50-50 chance of living past five years. Large companies have tried and failed to improve this figure. Patients desperately need better treatments. We now have the ingredients for delivering results to investors as well. While the biotech stock market always has its ups and downs, and has been dismal lately, the clinical data will stand firm and drive the final success for CEL-SCI. We have presented our new data to regulators in Europe and the UK. Canada and the United States (FDA) have not yet seen these data, and we plan to present to them in the next quarter. CEL-SCI plans to seek approval for immediate patient access to Multikine without waiting on the results of a new trial wherever possible. There are regulatory pathways specifically designed for such approvals that CEL-SCI is pursuing worldwide. These pathways are called “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. Our situation-where we have selected a portion of the Phase 3 study for our target population-is precisely why these regulatory pathways were adopted by regulatory bodies, so that patients do not need to wait many years before gaining access to promising drugs that have already been shown to provide clinical benefit.”