Therapeutic Horizon in Multiple Myeloma: Analysis of the Emerging Landscape of Clinical Trials

Background Emerging therapies for multiple myeloma (MM) have significantly improved patient outcomes extending survival and advancing treatment toward more targeted, effective, and less toxic approaches. However, the disease remains incurable. Objectives Analyze the therapeutic landscape of MM by evaluating drug candidates in clinical trials from 2014 to 2024. Methods Data were extracted from ClinicalTrials.gov (CTG) and Cortellis Drug Discovery Intelligence (CDDI), including active and completed studies with results on newly diagnosed and refractory MM. Joinpoint regression models estimated Annual Percent Changes (APCs) and Average Annual Percent Changes (AAPC). Data were examined by clinical trial characteristics, targeting strategies, and potential impact on treatment advancements. Results A total of 1091 trials from CDDI and 1947 from CTG were screened, yielding 365 studies eligible for detailed analysis. Phase I trials were the most common ( n = 182; 49.9%), and biologic agents represented the majority of the investigational therapies ( n = 251; 68.8%). Among intervention types, cell therapies were predominant ( n = 171; 46.8%), with CAR-T products targeting BCMA ( n = 107), CD19 ( n = 16), and GPRC5D ( n = 12) emerging as the most studied in recent years. Before the joinpoint, the number of active trials grew by 1.86% per year (95% CI: 1.67–2.06), corresponding to an average increase of 11.9 studies annually. After the joinpoint, growth accelerated to 3.69% per year (95% CI: 3.19–4.19), equivalent to 26.5 additional trials each year. Across the entire study period, the weighted average annual percentage change (AAPC) was 2.63%. Conclusion The rise in MM clinical trials highlights a shift toward biologic therapies, particularly immunotherapies and gene therapies like CAR-T.