FDA Approves Investigative New Drug Application for AUTO1 CAR-T Therapy for ALL

The FDA has accepted the Investigational New Drug (IND) application of CD19-targeting chimeric antigen receptor (CAR) T-cell therapy AUTO1 for the treatment of adults with acute lymphoblastic leukemia (ALL).

Approval of the IND application allows for the initiation of the AUTO-AL1 study, which will enroll 100 patients with relapsed or refractory ALL from across the U.S. and Europe, with overall response rate as its primary endpoint. Secondary endpoints include duration of response and measurable residual disease−negative complete response rate. The study will have a short phase Ib component before proceeding to a single arm phase II study. Enrollment in the second phase is scheduled to begin later this year to minimize impact from the COVID-19 pandemic.

AUTO1 is a “fast-off” CAR-T cell therapy designed to minimize excessive activation and associated cytokine release, which may reduce toxicity. It also claims to reduce T cell exhaustion and enhance engraftment. The cell product is also being evaluated in a phase I study as a treatment for pediatric ALL.

Source: Autolus Therapeutics press release, April 16, 2020.