Precision BioSciences has filed for an initial public offering to continue its development of gene-edited cell therapies, including a cancer treatment that is set to start tests in humans in the coming months. The company is also forging ahead in gene therapy and agricultural applications, giving it breadth that sets it apart from others in genome-editing research.
In securities documents filed late Friday, Precision Bio set a placeholder figure of $100 million for its IPO fundraising target. The Durham, NC, company has applied for a listing on the Nasdaq exchange under the stock symbol “DTIL.”
Precision Bio calls its gene-editing technology ARCUS. Like other gene-editing approaches, ARCUS uses an enzyme to make precise cuts in a cell’s genome, which then allows the DNA to be edited. ARCUS uses an enzyme called I-Crel, which is found in the genome of a type of algae, the company says in the prospectus.
CEO Matthew Kane told Xconomy in 2015 that ARCUS offers more precision and flexibility than CRISPR and TALEN, two other gene-editing technologies. Specifically, he said that by precisely hitting the intended gene, ARCUS avoids off-target effects to other genes—a concern with other gene-editing approaches. Kane said the ARCUS name was inspired by archery, and is meant to evoke the flexibility of a bow and the precision of an arrow hitting its target. Kane co-founded Precision Bio in 2006, based on technology licensed from Duke University.
Precision Bio’s most advanced therapeutic candidate, PBCAR0191, is a cell therapy developed to target the cancer protein CD19. The Precision Bio drug is a CAR-T therapy, a type of immunotherapy in which T cells are engineered to recognize cancer cells so that the immune cells can kill them.
The CAR-T treatments that the FDA has approved to date, from Novartis (NYSE: NVS) and Gilead Sciences (NASDAQ: GILD), are made from a patient’s own T cells. Precision Bio’s treatment is an allogeneic cell therapy; that is, it’s made from donor T cells. These cells are edited by ARCUS. The company says its “off-the-shelf” approach would have advantages over developing a treatment from each patient’s T cells.
“We believe our proprietary, one-step engineering process for producing allogeneic CAR-T cells at large scale in a cost-effective manner will enable us to overcome the fundamental challenges of manufacturing that have limited the CAR-T field to date,” the company says in its prospectus.
Precision Bio says in its prospectus that it plans to start a Phase 1/2a clinical trial testing PBCAR0191 in patients with acute lymphoblastic leukemia and non-Hodgkin’s lymphoma in the first half of this year. The drug, which was being developed under a partnership with a Shire subsidiary, is now partnered with French company Servier, which acquired Shire’s oncology business last year.
There are others developing allogeneic CAR-T cancer therapies. Allogene Therapeutics (NASDAQ: ALLO) is preparing to start mid-stage studies testing its treatment for acute lymphoblastic leukemia. Last fall, the South San Francisco, CA, company raised $324 million in an IPO to support its drug pipeline, which also includes treatments for non-Hodgkin’s lymphoma and multiple myeloma. Allogene is also sharing responsibility for clinical development of its lead CAR-T drug with Servier, who holds non-U.S. rights to the experimental therapy.
In addition to its allogeneic cancer cell therapies, Precision Bio is developing gene therapies. These therapies are “in vivo” treatments, in which a therapeutic gene is introduced into the body to correct genetic defects in the cells of the patients. Last September, Precision Bio announced a partnership with Gilead Sciences to develop an ARCUS-based gene therapy for chronic Hepatitis B infection. The company expects to file for the regulatory clearance to start clinical trials for that therapy next year.
Precision Bio also applies its technology to food research. The company’s agriculture division, Elo Life Systems, works with large ag companies to develop new crop varieties intended to improve plant health, nutrition, and flavor. Elo’s partners include Cargill, which is working with Elo to develop a canola oil that’s lower in saturated fatty acids.
Precision Bio spent $45.1 million on research and development in 2018, a 122 percent increase over the prior year, according to the prospectus. Last year, the company reported a net loss of $46 million on revenue of $10.8 million. The revenue came from payments made by industry partners.
Precision Bio says it will use the IPO proceeds to advance its drug pipeline and complete a manufacturing facility for its experimental therapies. That Durham site is expected to open in the second half of this year, according to the prospectus.
Since inception, Precision Bio has funded its research with approximately $300 million in investment and payments from research partners. That figure includes $136 million in venture financing, most recently a $110 million Series B investment round last June. VenBio is Precision Bio’s largest shareholder, with an 11 percent stake prior to the IPO, according to the prospectus.