Precigen Announces First Patient Dosed in PRGN-3006 UltraCAR-T™ Study

Precigen announced that the first patient has been dosed with PRGN-3006, a first-in-class investigational therapy using Precigen's non-viral UltraCAR-T™ therapeutic platform. PRGN-3006 UltraCAR-T is an autologous chimeric antigen receptor T (CAR-T) cell therapy under investigation for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) or higher risk myelodysplastic syndrome (MDS).

PRGN-3006 utilizes Precigen's transformative UltraCAR-T therapeutic platform, which eliminates ex vivo expansion and reduces manufacturing time to fewer than two days following non-viral gene transfer at the cancer center. PRGN-3006 UltraCAR-T is a multigenic CAR-T cell treatment utilizing Precigen's advanced non-viral gene delivery system to co-express a chimeric antigen receptor, membrane-bound interleukin‐15 (mbIL15), and a kill switch for better precision and control in targeting relapsed or refractory AML and higher risk MDS. The study is conducted in collaboration with the Moffitt Cancer Center.

"The first patient dosed using Precigen's UltraCAR-T therapeutic platform is an important milestone for our company," said Helen Sabzevari, PhD, President of Precigen. "Timing is critical for this patient population and the ability to manufacture PRGN-3006 UltraCAR-T overnight without ex vivo expansion accelerates timing to provide treatment to patients."

"AML is an aggressive disease with very poor prognosis," said James J. Mulé, PhD, Associate Center Director and Michael McGillicuddy, Endowed Chair in Melanoma Research/Treatment at the Moffitt Cancer Center. "The first patient dosed with investigational PRGN-3006 UltraCAR-T represents a significant development for this challenging patient population with high unmet medical need."

The PRGN-3006 UltraCAR-T clinical study is a single center, nonrandomized, investigator‐initiated Phase 1/1b safety and tolerability study. The safety and tolerability of PRGN‐3006 UltraCAR-T will be assessed following intravenous administration of escalating doses in patients with relapsed or refractory AML or higher risk MDS.

"AML and MDS patients have few treatment options, and time is critical when selecting the best treatment path," said David A. Sallman, MD, lead investigator for the PRGN-3006 study at the Moffitt Cancer Center. "We are hopeful that this study will be the beginning of the development of a therapeutic that may result in a critically needed new safe and efficacious treatment option that allows for rapid treatment."

AML is a cancer that starts in the bone marrow, but most often moves into the blood. Though considered rare, AML is among the most common types of leukemia in adults. In 2019, it is estimated that 21,450 new cases of AML will be diagnosed in the US. AML is uncommon before the age of 45, and the average age of diagnosis is about 68. The prognosis for patients with AML is poor with an average 5‐year survival rate of approximately 25 percent overall and less than a 5 percent 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.

MDS are cancerous conditions of the bone marrow generally found in adults in their 70s. Incidence in the US is not known for sure, but estimates range from 10,000 each year and higher. Sometimes referred to as pre-leukemia, about 1 in 3 MDS patients progress to AML. As diseases of the bone marrow, MDS outlook is not based on the size of a tumor or tumor metastasis, and other factors are used to predict outlook and inform the treatment plan. Some factors have been combined to develop scoring systems, such as the Revised International Prognostic Scoring System (IPSS-R). Using the IPSS-R, median survival for MDS patients can be estimated to vary from less than one year for the "very high" IPSS-R risk group to more than eight years for the "very low" IPSS-R group.

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