Precigen Announces First Patient Dosed in Phase 1/1b Study of PRGN-3006 UltraCAR-T™ in Patients with Relapsed or Refractory Acute Myeloid Leukemia (AML) or Higher Risk Myelodysplastic Syndrome (MDS)

GERMANTOWN, Md., June 26, 2019 /PRNewswire/ -- Precigen, Inc., a wholly-owned subsidiary of Intrexon Corporation (NASDAQ: XON) and a biopharmaceutical company specializing in the development of innovative gene and cellular therapies to improve the lives of patients, today 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) (clinical trial identifier: NCT03927261).

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."

About Acute Myeloid Leukemia (AML)
AML is a cancer that starts in the bone marrow, but most often moves into the blood¹. Though considered rare, AML is among the most common types of leukemia in adults². In 2019, it is estimated that 21,450 new cases of AML will be diagnosed in the US². AML is uncommon before the age of 45, and the average age of diagnosis is about 68². 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 65³. 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 age³.

About Myelodysplastic Syndromes (MDS)
MDS are cancerous conditions of the bone marrow⁴ generally found in adults in their 70s⁵. Incidence in the US is not known for sure, but estimates range from 10,000 each year and higher⁵. Sometimes referred to as pre-leukemia, about 1 in 3 MDS patients progress to AML⁴. 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)⁶. Using the IPSS-R⁶, 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 group⁷.

Precigen : Advancing Medicine with Precision^TM
Precigen is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cellular therapies using precision technology to target the most urgent and intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases. Precigen also follows the science opportunistically in pursuit of promising programs in emerging therapeutics. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. Precigen was founded as a wholly-owned subsidiary of Intrexon Corporation (NASDAQ: XON) and leverages a diverse portfolio of technology platforms to advance human health. For more information about Precigen, visit www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

Precigen's UltraCAR-T^TM Therapeutic Platform
Precigen's UltraCAR-T platform has the potential to disrupt the CAR-T treatment landscape by increasing patient access through shortening manufacturing time, decreasing manufacturing-related costs, and improving outcomes using advanced approaches for precise tumor targeting and control of the immune system. The platform brings several key advancements: 1) Non-viral gene transfer using multigenic vectors for expression of multiple effector genes leads to better precision and control of tumor targeting and eliminates the need for virus; 2) Sustained persistence and desired phenotype of infused UltraCAR-T helps address T-cell exhaustion, a common issue with current CAR-T therapies; 3) T-cell control by incorporation of kill switch technology to potentially improve the safety profile; and 4) Rapid manufacturing of UltraCAR-T cells using our proprietary non-viral gene transfer process, which eliminates the need for ex vivo propagation, thus dramatically reducing wait times for patients from weeks to fewer than two days.

Safe Harbor Statement
Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to plans, objectives and expectations for the development of our business, including the timing and progress of preclinical and clinical trials and discovery programs. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.

¹ American Cancer Society. What is Acute Myeloid Leukemia (AML)? (Accessed June 2019)
² American Cancer Society. Key Statistics for Acute Myeloid Leukemia (AML) (Accessed June 2019)
³ Thein, M., et al., Outcome of older patients with acute myeloid leukemia: an analysis of SEER data over 3 decades. Cancer, 2013. 119(15): p.2720-7.
⁴ American Cancer Society. What are Myelodysplastic Syndromes? (Accessed June 2019)
⁵ American Cancer Society. Key Statistics for Myelodysplastic Syndromes (Accessed June 2019)
⁶ American Cancer Society. Myelodysplastic Syndrome Prognostic Scores (Accessed June 2019)
⁷ American Cancer Society. Survival Statistics for Myelodysplastic Syndromes (Accessed June 2019)

Precigen Media Contact:
Donelle M. Gregory
[email protected]

SOURCE Precigen, Inc.

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