Improved Activity and Durability New Generation of Ready-to-Use CAR-T Therapy Receives FDA Orphan Drug Designation

Allogene Therapeutics is a clinical-stage biotechnology company developing allogeneic chimeric antigen receptor T-cell (AlloCAR T) products targeting cancer. Recently, the company announced that the U.S. FDA has granted orphan drug designation (ODD) for the investigational CAR-T therapy ALLO-605 for the treatment of multiple myeloma. ALLO-605, currently in Phase 1 clinical development, is part of Allogene's multi-pronged strategy to target B cell maturation antigen (BCMA). BCMA is a ubiquitously expressed antigen on multiple myeloma cells and a proven anticancer target. It is worth mentioning that ALLO-605 is the first investigational therapy developed by the company's TurboCAR technology platform, which is expected to improve the function of T cells.

Allogene's TurboCAR platform specifically activates signaling of specific cytokines in CAR-T cells. A variety of cytokines play an important role in stimulating T cell activity and maintaining T cell persistence, but systemic use of cytokines often brings toxic side effects. By expressing recombinant proteins on the surface of CAR-T cells, the TurboCAR platform activates the cytokine signaling pathway only in CAR-T cells, thereby improving the function of CAR-T therapy without activating host immune cells and possibly avoiding systemic toxicity.

Preclinical results from ALLO-605, presented at the American Society of Hematology (ASH) annual meeting in December 2020, show that TurboCAR technology may improve cell therapy colonization and persistence and delay T cell exhaustion. Additionally, the U.S. FDA granted Fast Track designation to ALLO-605 in the second quarter of 2021 based on the product candidate's potential to address an unmet need in patients who have failed other standard multiple myeloma treatments.

Rafael Amado, MD, executive vice president of research and development and chief medical officer at Allogene, said: "Orphan drug designation marks an important step toward developing an anti-BCMA portfolio for patients with multiple myeloma and providing patients with an allogeneic CAR-T product. step."