Legend Biotech Announces Submission of European Marketing Authorisation Application for BCMA CAR-T Therapy Ciltacabtagene Autoleucel (cilta-cel) for the Treatment of Relapsed and/or Refractory Multiple Myeloma

Rhea-AI Summary

Legend Biotech Corporation (NASDAQ: LEGN) has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency for the approval of ciltacabtagene autoleucel (cilta-cel) as a treatment for relapsed and/or refractory multiple myeloma. This investigational CAR-T therapy is supported by positive results from the CARTITUDE-1 study, presented at the ASH 2020 Annual Meeting. The EMA granted accelerated assessment for the MAA, highlighting its public health significance. A Biologics License Application is also under review by the FDA.

Positive

• Successful submission of MAA to EMA for cilta-cel.
• Positive efficacy and safety results from CARTITUDE-1 study.
• EMA granted accelerated assessment, indicating major public health interest.
• Ongoing FDA review for Biologics License Application.

Negative

• None.

Legend Biotech Corporation (NASDAQ: LEGN) (“Legend Biotech”), a global clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications, announced today the submission of a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of ciltacabtagene autoleucel (cilta-cel) for the treatment of patients with relapsed and/or refractory multiple myeloma.

Cilta-cel is an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor T cell (CAR-T) therapy being investigated as a treatment for multiple myeloma. The MAA is based on positive results from a Phase 1b/2 CARTITUDE-1 study, which were presented at the American Society of Hematology (ASH) 2020 Annual Meeting.¹ The submission was filed to the EMA by Janssen-Cilag International N.V., an affiliate of Janssen Biotech, Inc., Legend’s collaboration partner for cilta-cel.

“Today’s submission is a testimony to the promising results we have seen from the CARTITUDE-1 study showing the efficacy and safety of cilta-cel for treating patients with multiple myeloma who are heavily pretreated and in need of treatment options,” said Ying Huang, PhD, CEO and CFO of Legend Biotech. “We are proud of our collaboration with Janssen and look forward to bringing this personalized treatment to patients in the EU following the accelerated assessment.”

The EMA’s Committee for Medicinal Products for Human Use (CHMP) granted accelerated assessment for this MAA. An accelerated assessment is granted when the CHMP determines that a medicinal product is of major public health interest and therapeutic innovation and can significantly reduce the review timelines to evaluate an MAA.² Cilta-cel previously received a PRIority MEdicines (PRIME) designation from the EMA. A Biologics License Application (BLA) seeking approval of cilta-cel based on the CARTITUDE-1 study is currently under review with the United States Food and Drug Administration.

About CARTITUDE-1

CARTITUDE-1 (NCT03548207) is an ongoing Phase 1b/2, open-label, multicenter study evaluating the safety and efficacy of cilta-cel in adults with relapsed and/or refractory multiple myeloma, 99 percent of whom were refractory to the last line of treatment; 88 percent of whom were triple-class refractory (to at least 1 immunomodulatory drug [IMiD], proteasome inhibitor [PI] and 1 anti-CD38 antibody).³ The primary objective of the Phase 1b portion of the study was to characterize the safety and confirm the dose of cilta-cel, informed by the first-in-human study with LCAR-B38M CAR-T cells (LEGEND-2). The Phase 2 portion further evaluated the efficacy of cilta-cel with overall response rate as the primary endpoint.

About Ciltacabtagene autoleucel (cilta-cel)

Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy, formerly identified as JNJ-4528 outside of China and LCAR-B38M CAR-T cells in China, that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed and/or refractory multiple myeloma and in earlier lines of treatment. Cilta-cel is a differentiated CAR-T therapy with two BCMA-targeting single domain antibodies. In December 2017, Legend Biotech, Inc. entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. to develop and commer

FAQ

What is the significance of Legend Biotech's MAA submission for LEGN?

The MAA submission for cilta-cel represents a significant step in seeking regulatory approval in the EU, potentially expanding market access for LEGN.

What is cilta-cel and how does it work?

Cilta-cel is a CAR-T therapy targeting BCMA, designed for treating relapsed and/or refractory multiple myeloma.

What were the results of the CARTITUDE-1 study?

The CARTITUDE-1 study demonstrated positive efficacy and safety results for cilta-cel in heavily pretreated multiple myeloma patients.

What does the accelerated assessment by the EMA mean for LEGN?

The accelerated assessment indicates that cilta-cel is of major public health interest, potentially speeding up the approval process.

Is there any review from the FDA for LEGN?

Yes, a Biologics License Application for cilta-cel is currently under review by the FDA.