Cilta-cel ( Ciltacabtagene autoleucel ) is an investigational BCMA-directed CAR-T therapy in development for the treatment of adults with relapsed and/or refractory multiple myeloma.
CAR‑T therapy is a highly personalised technology where a patient’s own T-cells are re-programmed to target and eradicate cancer.
The Cilta-cel is supported by the positive results from CARTITUDE-1 study. The latest results from the CARTITUDE-1 study were presented at the American Society of Hematology ( ASH ) 2020 Annual Meeting, and have continued to demonstrate a very high overall response rate of 97%, which deepened over time with 67% of patients achieving a stringent complete response.
With a median follow-up of 12.4 months, median duration of response and progression-free survival ( PFS ) were not reached.
CARTITUDE-1 is an ongoing phase 1b/2, open-label, multicentre study evaluating the safety and efficacy of Cilta-cel in adults with relapsed and/or refractory multiple myeloma, 99% of whom were refractory to the last line of treatment; 88% of whom were triple-class refractory, meaning their cancer did not, or no longer responds to an immunomodulatory agent ( IMiD ), a proteasome inhibitor ( PI ) and an anti-CD38 antibody.
The primary objective of the CARTITUDE-1 study is to characterise the safety and confirm the dose of Cilta-cel, informed by the first-in-human study with LCAR-B38M CAR-T cells ( LEGEND-2 ) and to evaluate the efficacy of Cilta-cel with overall response as the primary endpoint.
Cilta-cel is an investigational chimeric antigen receptor T cell ( CAR-T ) therapy for the treatment of patients with multiple myeloma.
The design comprises a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies.
CAR-T cells are an innovative approach to eradicating cancer cells by harnessing the power of a patient’s own immune system.
BCMA is a protein that is highly expressed on myeloma cells.
Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterised by an excessive proliferation of plasma cells.
In Europe, more than 48,200 people were diagnosed with multiple myeloma in 2018, and more than 30,800 patients died.
Around 50% of newly diagnosed patients do not reach 5-year survival, and almost 29% of patients with multiple myeloma will die within one year of diagnosis.
Although treatment may result in remission, patients will most likely relapse as there is currently no cure.
Refractory multiple myeloma is when a patient’s disease progresses within 60 days of their last therapy.
Relapsed cancer is when the disease has returned after a period of initial, partial or complete remission.
While some patients with multiple myeloma have no symptoms at all, others are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.
Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and require new therapies for continued disease control. ( Xagena )
Source: Janssen Pharmaceutical, 2021