Cilta-cel Approval Requested in Japan for Heavily-treated Myeloma

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by Patricia Inacio PhD |

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Janssen has submitted an application to regulators in Japan seeking the approval of ciltacabtagene autoleucel, an investigational CAR T-cell therapy also known as cilta-cel, to treat relapsed or refractory multiple myeloma patients.

The submission to the Japanese Ministry of Health, Labour and Welfare (MHLW) specifically asks that cilta-cel be approval for patients who received at least three prior lines of therapies, including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD), and a CD38 inhibitor.

“Today’s submission is an encouraging step in our mission to provide a potentially transformative cell therapy option to patients with multiple myeloma,” Ying Huang, PhD, CEO and chief financial officer of Legend Biotech, said in a press release. Legend entered into an exclusive worldwide license and collaboration agreement with Janssen to develop and commercialize cilta-cel in 2017.

Cilta-cel is a form of immunotherapy in that involves collecting a patient’s own T-cells — immune cells with anti-cancer activity — and genetically modifying them to boost their ability to fight cancer.

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Specifically, the T-cells in cilta-cel are engineered to eliminate cells containing the B-cell maturation antigen (BCMA) — a protein found at high levels on the surface of myeloma cells — while leaving healthy cells unharmed. The modified cells are expanded in the lab before being infused into the patient.

A Phase 1b/2 clinical trials, called CARTITUDE-1 (NCT03548207), is testing cilta-cel in people with relapsed or refractory multiple myeloma. Participants had received three or more prior lines of therapy, including a PI, an IMiD, and a CD38 inhibitor, or had failed to respond to a PI or IMiD. It is due to conclude in August.

Among the 97 patients in the study, the median number of prior therapies was six, and most had failed to respond to the three classes of treatment.

Interim data, collected after a median follow-up of 12.4 months, showed that 97% of patients experienced a reduction in cancer burden, and 67% had a stringent complete response, or no detectable cancer after a single infusion. Over half of patients (72%) at this time were still responding to the therapy.

More recent findings, with a median follow-up of 22 months, were shared in an oral presentation at the American Society of Hematology’s 2021 Annual Meeting.

Results showed that responses tended to deepen over time, with the amount of patients showing a stringent complete response raising from 67% at 12.4 months to 80% after 18 months, and to 83% at the time of the latest analysis.

Among the 61 patients examined for minimal residual disease (MRD) — the small number of cancer cells that remain in circulation after treatment and can cause a relapse — 92% were MRD-negative.

At the time of the analysis, most patients were still alive and progression-free. Data also demonstrated a safety profile consistent with prior studies of cilta-cel, and no signs of new safety concerns.

“These data add to the growing body of evidence supporting the potential clinical benefit of cilta-cel in the treatment of patients with relapsed and/or refractory multiple myeloma, a population in need of new options,” Sen Zhuang, MD, PhD, a vice president of clinical research and development at Janssen, said in another press release.

“We look forward to closely collaborating with our partner Janssen and the MHLW in order to make cilta-cel available to patients living with relapsed or refractory multiple myeloma, who have exhausted several standard-of-care treatments and are facing poor prognoses,” Huang said.

A similar application has been submitted to the U.S. Food and Drug Administration (FDA) and to the European Medicines Agency. An FDA decision is expected by the close of February.