Legend Biotech Reports Positive Overall Survival Results of Phase 3 CARTITUDE-4 Trial in Multiple Myeloma

Legend Biotech Corporation, a renowned entity in the field of cell therapy, has announced encouraging overall survival findings from their CARTITUDE-4 study. This ongoing, global, randomized, open-label Phase 3 trial is assessing the efficacy and safety of CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) compared to two other treatment regimens—pomalidomide, bortezomib and dexamethasone (PVd) and daratumumab, pomalidomide, and dexamethasone (DPd)—in adult patients with relapsed and lenalidomide-refractory multiple myeloma who have received one to three prior lines of therapy, including a proteasome inhibitor and an immunomodulatory agent.

At the pre-specified second interim analysis, CARVYKTI® showed a statistically significant and clinically meaningful enhancement in overall survival. The safety results were consistent with the known safety profile of CARVYKTI®, with no new safety signals emerging from the data.

Ying Huang, Ph.D., CEO of Legend Biotech, expressed satisfaction with the observed survival benefit from a single infusion of CARVYKTI® in the CARTITUDE-4 study. He noted that the data further supports CARVYKTI® as a promising treatment option for multiple myeloma patients.

These findings will be showcased in an upcoming medical meeting and shared with regulatory bodies worldwide to potentially update the drug's labeling.

On April 5, 2024, the U.S. Food and Drug Administration (FDA) approved CARVYKTI® for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide. CARVYKTI® is currently the only BCMA-targeted CAR-T cell therapy approved by the FDA for this patient population.

CARVYKTI® is indicated for the treatment of adult patients with relapsed or refractory multiple myeloma with at least one prior therapy line, including a proteasome inhibitor and an immunomodulatory agent, who are refractory to lenalidomide. However, it comes with several important safety warnings.

CARVYKTI® treatment can lead to Cytokine Release Syndrome (CRS), which may include fatal or life-threatening reactions. Patients should not receive the therapy if they have active infections or inflammatory disorders. Severe neurologic toxicities, including Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS), have also occurred. Monitoring and supportive care are recommended for severe cases.

Among other potential side effects are Hemophagocytic Lymphohistiocytosis/Macrophage Activation Syndrome (HLH/MAS), prolonged and recurrent cytopenias, serious infections, hypogammaglobulinemia, and hypersensitivity reactions. There is also a risk of secondary malignancies and a need for life-long monitoring for such conditions.

CARVYKTI® is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) due to the risks of CRS and neurologic toxicities. Patients receiving CARVYKTI® should refrain from driving and operating heavy machinery for at least eight weeks post-infusion due to potential neurologic events.

Legend Biotech, headquartered in Somerset, New Jersey, is dedicated to developing advanced cell therapies to treat and potentially cure life-threatening diseases. They are leveraging a variety of technology platforms, including autologous and allogeneic chimeric antigen receptor T-cell, gamma-delta T cell, and natural killer cell-based immunotherapy, to discover cutting-edge therapeutics for patients worldwide.

The CARTITUDE-4 study is a significant part of Legend Biotech's research efforts, comparing the efficacy of cilta-cel against other established therapies in patients with relapsed and refractory multiple myeloma. This study is conducted on a global scale, involving multiple research sites.

Multiple myeloma is an incurable blood cancer originating in the bone marrow and characterized by the proliferation of plasma cells. It is estimated that in 2024, over 35,000 people in the U.S. will be diagnosed with multiple myeloma, with more than 12,000 fatalities expected from the disease. Symptoms often include bone issues, low blood counts, elevated calcium levels, kidney problems, or infections.

In conclusion, Legend Biotech's CARTITUDE-4 study offers promising results for CARVYKTI®, potentially providing a significant therapeutic advantage for patients battling multiple myeloma. The continued development and regulatory discussions around CARVYKTI® may lead to further advancements in the treatment landscape for this challenging disease.