Precigen Announces Clearance of IND to Initiate Phase 1 Study for First-in-Class PRGN-3005 UltraCAR-T™ to Treat Advanced Stage Platinum Resistant Ovarian Cancer Patients

- First UltraCAR-T targeting solid tumors to enter the clinic

- First-in-class therapy using autologous CAR-T cells targeting advanced ovarian cancer

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Feb 11, 2019, 07:30 ET

GERMANTOWN, Md., Feb. 11, 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 US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for Precigen's PRGN-3005, a first-in-class investigational therapy using autologous chimeric antigen receptor T (CAR-T) cells to treat advanced-stage platinum-resistant ovarian cancer patients.

PRGN-3005 is the second candidate to clear IND utilizing Precigen's transformative UltraCAR‑T™ platform that reduces manufacturing time to less than two days following non-viral gene transfer. PRGN-3005 UltraCAR-T is a multigenic CAR-T cell treatment utilizing Precigen's clinically-validated Sleeping Beauty system to co-express a chimeric antigen receptor, membrane-bound interleukin‐15 (mbIL15), and a kill switch for better precision and control in targeting advanced ovarian cancer. This is an open-label, first-in-human Phase 1 dose escalation study to evaluate the safety and maximal tolerated dose of PRGN‐3005 UltraCAR-T.

"This patient population has limited treatment options and ovarian cancer remains one of the most challenging with minimal advances in long-term survival for patients with advanced disease," said Mary L. (Nora) Disis, MD, professor of medicine at the University of Washington and lead investigator for the PRGN-3005 study. "Clinical advancements and new research, including the PRGN-3005 UltraCAR-T study, are needed to make progress in fighting this intractable disease."

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:

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;
Sustained persistence and desired phenotype of infused UltraCAR-T helps address T-cell exhaustion, a common issue with current CAR-T therapies;
T-cell control by incorporation of kill switch technology to potentially improve the safety profile; and
Rapid manufacturing of UltraCAR-T cells using our proprietary non-viral gene transfer process, eliminates the need for ex vivo propagation, thus dramatically reducing wait times for patients from weeks to less than two days.

"We are pleased our PRGN-3005 UltraCAR-T investigational therapy received clearance from the FDA to advance into the clinic. This is an exciting milestone as it represents the first UltraCAR-T to enter the clinic targeting solid tumors," said Helen Sabzevari, PhD, President of Precigen. "This is an important step for patients and our dedicated employees who continue to work with urgency to execute our important mission of creating innovative medicines with meaningful benefit for patients."

About Ovarian Cancer
Worldwide, nearly 300,000 women are diagnosed with ovarian cancer every year¹ and around 22,000 are diagnosed in the US². Five-year survival rates depend on stage and type of ovarian cancer; however, stage IV survival can drop to as low as 20 percent.^3.

Precigen : Advancing Medicine with Precision^™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 Intrexon's proprietary technology platforms to advance human health. For more information about Precigen, visit www.precigen.com.

¹World Health Organization, International Agency for Research on Cancer, Global Cancer Observatory. Cancer Today, Estimated number of new cases in 2018, worldwide, both sexes, all ages. Accessed December 2018 via WHO IARC GCO website.
²American Cancer Society Ovarian Cancer Special Section. Access December 2018 via ACS website.
³American Cancer Society. Survival Rates for Ovarian Cancer, by Stage. Accessed December 2018 via ACS website.

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.

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

SOURCE Precigen, Inc.