CEL-SCI 2025年度报告

2025 CEL-SCICORPORATION CEL-SCI Corporation is a late clinical-stage biotechnology company dedicated to research and developmentdirected at improving thetreatment of cancer and other diseases by using the immune system,the body's naturaldefense system CEL-SCI is currently focused on the development of the following product candidates andtechnologies with emphasis on Multikine: 1Multikine, an investigational Phase II immunotherapy under development for the potential treatment ofcertain head and neck cancers; and2L.E.A.P.S. (Ligand Epitope Antigen Presentation System) technology, or LEAPS, with several productcandidates under development for the potential treatment of rheumatoid arthritis. Multikine (Leukocyte Interleukin, Injection) is the full name of this investigational therapy, which, forsimplicity, is refered to in this report as Multikine. Multikine is the trademark that CEL-SCI has registered for thisregulatory submission forapproval. Noneof CEL-SCI's product candidates havebeen approved for sale,barterorexchange by the Food and Drug Administration (FDA)or any other regulatory agency for any use to treat disease inhumans nor has the safety or efficacy of these products been established for any use. There can be no assurance thatobtainingmarketing approvalfromtheFDAin the United States andbycomparableagenciesinmostforeign countrieswillbegranted. MULTIKINE.THE PHASE II CLINICAL IRIAL RESULIS.AND PATH FORWARD Immunotherapy is a large. high growth market, Immunotherapies use the patient's own immune systemto fight disease. These"targeted therapies"are at the forefront of modern cancer research. A Bloomberg report fromJanuary2023asserted that: The global cancer immunotherapy market is expected to reach USD S196.45 billion by 2030,registering CAGR of 7.2%during theforecast period, according to a new report by Grand ViewResearch,Inc.Therisingadoptionofimmunotherapyoverothertherapyoptionsforcancerowingto its targeted action is anticipated to increase the adoption during theforecast period. Moreover,increasing regulatory approvals from authoritarian establishments for novel immunotherapy usedforoncology is alsoexpected tofurtherfuel themarketgrowth. Source:https:/www.bloomberg.com/press-releases/2023-01-18/cancer-immunotherapy-market-worth-196-45billion-byv-2030-grand-view-research-inc CEL-SCI hopes to participate in this growing market with its lead investigational therapy Multikine?(Leukocyte Interleukin, Injection). Multikine is unique among approved cancer immunotherapies because it is givenfirst, right after diagnosis, before any other treatment including surgery. Multikinehasbeen tested in approximately740 patients inPhase12and3clinical studies conducted in theU.S., Canada, Europe, Israel and Asia.In these studies,it has been administered in multiple doses by various routesand various frequencies to determine its safety and efficacy. The data from these studies allowed CEL-SCI todeterminethepatient population mostresponsive to Multikine andmost likely tobenefitfrom it.The target populationis newly diagnosed advanced primary head and neck cancer patients with no lymph node involvement (deteminedvia PET imaging), and with low PD-L1 tumor expression (determined via biopsy), two features that physiciansroutinely assess at baseline as part of standard practice. CEL-SCIcompleted abias analysis forthetargetpopulation in the PhaseII studyinpreparation forsubmission ofdata to regulatory agencies,includingtheFDA,for a confirmatoryregistration study.Thedetailed dataon parameters including patientage,sex,race,tumorlocations,and stagingdemonstratebalancebetween thetreatmentandcontrolarms.Therefore,nobiaswasfound,whichsupportsconfidenceinMultikine'sefficacyresults In thetarget patient population CEL-SCIbelieves Multikine significantlyextended life.In thePhaseII studyCEL-SCIobserveda73%survival rate with Multikine vs.only45%without Multikineat 5years aftertreatment,anda Hazard ratio of0.35 (95%CIs [0.19, 0.66]) focusing only on those patients in thetargetpopulation, which accounts for approximately 100,o00 patients worldwideregistration study of Multikine in the target population. What is Multikine and who is it for? Multikine is a biological medicinal immunotherapy comprised ofamixture ofnatural cytokines and small biological molecules.Multikine is injected around the tumor and adjacentlymph nodesfor three weeks as a first-linetreatment before the standard ofcare (SOC),which is surgeryfollowed byeither radiotherapy or chemoradiotherapy. Multikine's rationale for use is to incite a locoregional immune responseagainst the tumor before the local immune system has been compromised by the standard of care and/or diseaseprogression. The Multikine target population is not yet treated adult patients with resectable locally advanced primarysquamous cell carcinoma of the head and neck (SCCHN) in the oral cavity and who have: No lymph node involvement (via PET imaging)Low PD-L1 tumor expression (TPS≤10) (via biop