Precigen’s AML Candidate Gets Fast Track Designation from FDA

Precigen’s AML Candidate Gets Fast Track Designation from FDA
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Gene and cell therapies developer Precigen Inc., announced today that its drug candidate for the treatment of acute myeloid leukemia (AML), PRGN-3006 UltraCAR-T, has received Fast Track Designation by the U.S. Food and Drug Administration (FDA), for patients with patients with relapsed or refractory AML.

PRGN-3006 UltraCAR-T is a multigenic autologous chimeric antigen receptor(CAR)-T cell treatment utilizing Precigen’s non-viral system that simultaneously expresses a CAR targets the CD33, which is over expressed on AML blasts and is present in 85% to 95% of all AML cases; membrane bound IL-15 for enhanced in vivo expansion and persistence; and employs a “kill switch” that can conditionally eliminate CAR-T cells to potentially curb the known adverse effects of CAR-T cell therapy for some patients.

Precigen’s UltraCAR-T platform is designed to overcome limitations of currently available CAR-T therapies by using an advanced overnight non-viral gene delivery manufacturing process conducted on premises at a medical center’s cGMP facility without the need for ex vivo expansion. The overnight production of the CAR-T therapy is accomplished using Precigen’s proprietary UltraPorator system, which employs a high-throughput, semi-closed electroporation technique. It is designed to provide a decentralized CAR-T manufacturing platform for the rapid manufacture of CAR-T therapies—a process that is typically lengthy due to the need for ex vivo expansion at a centralized facility.

“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, Ph.D., president and CEO of Precigen in a press release. “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.”

AML is a cancer that starts in the bone marrow, but most often moves into the blood. Though considered rare—PRGN-3006 UltraCAR-T had previously received FDA Orphan Drug designation in January 2020—AML is among the most common types of leukemia in adults. It is uncommon before the age of 45 and the average age at diagnosis is around 62 years. The prognosis for patients with AML is poor with an average 5‐year survival rate of approximately 25% overall, with a less than a 5% 5‐year survival rate for patients older than 65.  Amongst elderly AML patients (≥ 65 years of age), median survival is short, ranging from 3.5 months for patients 65 to 74 years of age to 1.4 months for patients ≥ 85 years of age.