FDA Fast Tracks PRGN-3006 For R/R Acute Myeloid Leukemia Treatment

The FDA has granted fast track designation to PRGN-3006 UltraCAR-T®, for the treatment of patients with relapsed or refractory AML (R/R AML), announced Precigen, Inc, in a press release.¹

The multigenic autologous chimeric antigen receptor (CAR)-T cell agent was previously granted orphan drug designation by the FDA for the treatment of R/R AML. PRGN-3006 is currently being evaluated in a first-in-human, single-center, nonrandomized, investigator-initiated phase 1/1b study (NCT03927261). The study includes both patients with R/R AML and those with high-risk myelodysplastic syndrome (MDS).^1,2

"We are very pleased to receive the FDA's fast track designation, which facilitates development and expedites the review process of drugs that address serious conditions and high unmet medical needs," said Helen Sabzevari, PhD, president, and chief executive officer, Precigen, in a press release.¹

The study of PRGN-3006 is actively recruiting patients to receive PRGN-3006 in a dose-escalation phase to determine the maximum-tolerated dose (MTD) of CAR T cells. The coprimary end points of the study are the number of patients who experience dose-limiting toxicities (DLTs) and the number of patients who experience treatment-related adverse events. As secondary end points, the study is investigating disease progression in patients with AML, disease response in patients with MDS, absolute lymphocyte count, and the number of PRGN-3006 T cells present in patients treated with the agent.²

To be eligible for inclusion in the study, patients must be diagnosed with R/R AML or high-risk MDS, have an absolute lymphocytes count ≥ 0.2 k/μL, a Karnofsky performance score ≥ 60%, and a life expectancy of at least 12 weeks. Patients must also meet the required levels for serum bilirubin alanine and aspartate aminotransferase, and ejection fraction. Those who have undergone allogeneic stem cell transplant must be at least 3 months post-transplant before enrolling in the study.

The study excluded patients with a diagnosis of acute promyelocytic leukemia, as well as those with known central nervous system involvement, Human immunodeficiency virus seropositivity, active hepatitis B or C infection, history of allergic reaction to a compound similar to the cetuximab (Erbitux), active autoimmune disease, or uncontrolled serious infection or comorbidity that may interfere with study treatment. No patient can be previously treated with CAR T-cell therapy for any disease,

"AML is a rapidly progressing disease with a very poor prognosis. The fast track designation will help facilitate the timely development of this program and we look forward to working more closely with the FDA to potentially bring this new and highly differentiated overnight UltraCAR-T therapy to patients,” Sabzevari stated.

_REFERENCES:

_{1. Precigen receives fast track designation for PRGN-3006 UltraCAR-T® in patients with relapsed or refractory acute myeloid leukemia. News release. April 4, 2022. Accessed April 4, 2022. https://bit.ly/3NN4KWJ}

_{2. PRGN-3006 adoptive cellular therapy for relapsed or refractory AML or high risk MDS. Clinicaltrials.gov. Updated October 19, 2021. Accessed April 4, 2022. https://bit.ly/3u5d6kW}