Precigen CEO Envisions a Transformative 2021 with Precision Medicine Therapy

Precigen Chief Executive Officer Helen Sabzevari pictured above. 

The year 2020 and the COVID-19 pandemic created a number of challenges for individuals and companies. Despite these hurdles, Maryland-based Precigen still managed to achieve its clinical milestones in oncology.

The company is advancing its UltraCAR-T cell therapy approach to treating cancer that Chief Executive Officer Helen Sabzevari believes will be transformative to the personalized cell therapy landscape for cancer patients.

“This is what precision medicine in the twenty-first century should look like,” Sabzevari told BioSpace in an interview.

Sabzevari joined Precigen in 2017 after undergoing a rebranding effort that included a name change from Intrexon Corporation, as well as the divestiture of non-healthcare assets, such as AgBiotech and Intrexon Produce Holdings. The company now focuses on the development of next-generation gene and cell therapies that have potential to change the treatment paradigm in immuno-oncology, autoimmune disorders and infectious diseases.

While there are multiple companies pursuing programs of these types, Sabzevari said Precigen can differentiate itself through a unique precision medicine approach that could simultaneously impact different types of genetic expressions on cancer cells, both hematological and solid tumors. Additionally, Precigen believes its UltraCAR-T immunotherapies can be developed with fewer toxicity issues and manufactured at a lower cost than currently available CAR-T treatments.

She added the company has the most differentiated platform in cell and gene therapy compared to other companies in the space.

The company is developing two UltraCAR-T platforms. The first, PRGN-3005 UltraCAR-T, is a first-in-class investigational therapy currently being assessed in a Phase I/Ib clinical study for the treatment of advanced, recurrent platinum resistant ovarian, fallopian tube or primary peritoneal cancer.

In December, Precigen reported preliminary data from the study that showed a favorable safety profile with no dose-limiting toxicities (DLTs), neurotoxicity or cytokine release syndromes. What may be most important of all, Sabzevari said the study showed the UltraCAR-T treatment generated expansion and persistence after low dose IP infusion without lymphodepletion.

Precigen intends to begin a dose expansion of this study in the second half of the year. For the first time, Sabzevari said, they were able to show clinical efficacy in a solid tumor. Approximately 50% of patients showed a decrease in tumor lesions.

Precigen is also developing PRGN-3006 UltraCAR-T, a first-in-class investigational therapy currently under clinical evaluation in a Phase I/Ib study for the treatment of patients with relapsed or refractory (r/r) acute myeloid leukemia (AML) or higher-risk myelodysplastic syndromes (MDS).

Preliminary Phase I data reported in December showed a favorable safety profile with no DLTs or neurotoxicity. Encouraging expansion and persistence of PRGN-3006 UltraCAR-T was observed in both lymphodepletion and non-lymphodepletion cohorts and across all dose levels. PRGN-3006 treatment indicated clinical activity as evidenced by reduction in AML tumor blast levels, the company said. Data from the PRGN-3006 study also revealed that UltraCAR-T cells persisted for more than seven months after a very low dose of 24 million total UltraCAR-T cells in a patient. Sabzevari said this highlights the difference between Precigen’s UltraCAR-Ts and other CAR-T programs, which introduce hundreds of millions of CARs in to patients during an infusion. Those CARs also have a shorter life than the Precigen assets, which means they can have a sustainable impact on cancer.

 Following the revelation of the preliminary data, the U.S. Food and Drug Administration awarded PRGN-3006 with Orphan Drug Designation for AML.

While CAR-T therapies are not yet considered front-line defenses in cancer, Sabzevari predicted that cell and gene therapies will eventually move to first-line options.

“Checkpoint inhibitors have become frontline therapies and that’s the vision for CAR-T and cell therapies,” she said.

Sabzevari likened the possibilities of Precigen’s UltraCAR-T therapies to antibiotics. If a doctor prescribes one and it does not produce the desired therapeutic effect, there will be another type available from the company’s library that could treat the cancer.

”If one target, for whatever reason isn’t sufficient, you can switch overnight to change it with another. In our vision, a patient can come in and an oncologist could identify the indication of the cancer through screening, order a treatment from our UltraCar-T library and get it done, infuse the patient,” Sabzevari said.

Because of the potential Precigen’s UltraCAR-T program has in oncology, Sabzevari believes the company’s stock is significantly undervalued. In January, Precigen raised about $130 million. She believes the stock could go much higher and noted that Precigen’s market cap has tripled in the past year.

“2021 promises to be another transformative year for our company with important data readouts and trial initiations anticipated for our key programs. We have a very good stretch of growth in front of us,” Sabzevari said.