FDA grants regenerative medicine advanced therapy designation to ALLO-715 for RRMM

On April 21, 2021, it was announced that the U.S. Food and Drug Administration (FDA) granted regenerative medicine advanced therapy (RMAT) designation to ALLO-715 for the treatment of relapsed/refractory multiple myeloma (RRMM).¹ Designation was based on promising data from the ongoing phase I UNIVERSAL trial (NCT04093596), which has been summarized on the Multiple Myeloma Hub. Initial results demonstrated deep clinical responses and a promising safety profile when using a combination of ALLO-715 with the CD52-targeting monoclonal antibody, ALLO-647, for heavily pretreated patients with RRMM.¹

The advantages of the RMAT designation include all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with the FDA, which may be used to discuss potential surrogate or intermediate endpoints to support accelerated approval. You can find the requirements for RMAT designation here.²

ALLO-715 is an allogeneic chimeric antigen receptor (CAR) T-cell product that targets the B-cell maturation antigen (BCMA). Importantly, these cells lack CD52, allowing for lymphodepletion with ALLO-647 with no effect on the CAR-T product. Additionally, they overcome some of the manufacturing challenges of using autologous CAR T cells, reducing the timelines significantly and eliminating the need for bridging therapy.¹

The UNIVERSAL trial continues to enroll patients to optimize the dosage for ALLO-715 and ALLO-647 and explore the combination of ALLO-715 with the investigational gamma secretase inhibitor, nirogacestat, which increases BCMA cell-surface levels.¹

An updated announcement on August 12, 2021, declared that the product has been granted orphan drug designation by the FDA for the treatment of patients with RRMM.