Benzinga | Dec 21, 2020 07:17AM EST

Legend Biotech Announces Initiation of Rolling Submission of Biologics License Application to U.S. FDA Seeking Approval of BCMA CAR-T Therapy Cilta-cel for the Treatment of Relapsed and/or Refractory Multiple Myeloma

Legend Biotech also achieves fifth milestone payment under its collaboration agreement with Janssen in clinical development of cilta-cel

SOMERSET, N.J.--(BUSINESS WIRE)-- 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 initiation of a rolling submission of a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for ciltacabtagene autoleucel (cilta-cel), an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell therapy, for the treatment of adults with relapsed and/or refractory multiple myeloma.

The submission is based on results from the pivotal Phase 1b/2 CARTITUDE-1 study which evaluated the efficacy and safety of cilta-cel in the treatment of patients with relapsed and/or refractory multiple myeloma.1 The latest data from the study were recently presented (Abstract #177) at the 62nd American Society of Hematology Annual Meeting.

"Initiation of the BLA submission is an important milestone in advancing this therapy for patients with multiple myeloma who are heavily pretreated and in need of treatment options," said Ying Huang, PhD, CEO and CFO of Legend Biotech. "Together with our collaborator Janssen, we look forward to working with the FDA to fulfill this unmet medical need with the goal of making this breakthrough treatment available to patients and healthcare providers in the future."

Based on this submission, Legend Biotech also announced, according to the terms and conditions of an agreement with Janssen Biotech, Inc. (Janssen), achievement of a $75M milestone payment relating to the clinical development of cilta-cel. Janssen, Legend Biotech's collaboration partner, initiated the submission of the BLA for cilta-cel. The FDA previously granted Breakthrough Therapy Designation (BTD) for cilta-cel and has agreed to a rolling review of the BLA in which completed portions of the application will be submitted and reviewed on an ongoing basis.

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], 1 proteasome inhibitor [PI] and 1 anti-CD38 antibody).1

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.1