The FDA has granted accelerated designation to PRGN-3006 for the treatment of adults with relapsed or refractory acute myeloid leukemia. PRGN-3006 (Precigen Inc.) is a CAR T cell therapy that targets the CD33 protein on the surface of cancer cells. This investigational therapy, designed to reduce manufacturing time to 1 day, is based on Precigen’s UltraCAR-T therapeutic platform. The therapy uses the Sleeping Beauty non-viral gene editing system to simultaneously express CD33-targeted CAR, membrane-bound interleukin-15 and a therapeutic “kill switch” that conditionally eliminates CAR T cells for increased safety. PRGN-3006 is being evaluated in a single-center Phase 1/phase 1B dose escalation/extension study to determine safety and recommended Phase 2 dose for adults with relapsed or refractory AML, high-risk myelodysplastic syndrome or chronic myelomonocytic leukemia. Study participants will receive a single IV dose of the treatment with or without prior lymphodepletion. Interim results from the study, presented last year at the ASH Annual Meeting, showed an overall response rate of 50% in the first six patients in the lymphodepletion cohort. Safety results showed no dose-limiting toxicities. One of the 15 patients developed a grade 3 cytokine release syndrome.
