FDA has authorized Precision to begin dosing with PBCAR20A at Dose Level 2
DURHAM, N.C., April 07, 2020 (GLOBE NEWSWIRE) -- Precision BioSciences, Inc. (Nasdaq: DTIL), a life sciences company dedicated to improving life through the application of its pioneering, proprietary ARCUS® genome editing platform, today announced the initiation of patient dosing in a Phase 1/2a clinical trial of its second off-the-shelf (allogeneic) chimeric antigen receptor (CAR) T cell therapy candidate, PBCAR20A.
Wholly owned by Precision, PBCAR20A is an investigational allogeneic anti-CD20 CAR T therapy in development for the treatment of two separate cohorts of patients. The first cohort will enroll patients with relapsed/refractory (R/R) non-Hodgkin lymphoma (NHL), and the second will include patients with R/R chronic lymphocytic leukemia (CLL) or R/R small lymphocytic lymphoma (SLL). The NHL cohort will include patients with mantle cell lymphoma (MCL), an aggressive subtype of NHL, for which Precision BioSciences has received orphan drug designation from the United States Food and Drug Administration (FDA).
"As we navigate the unprecedented COVID-19 pandemic, we are incredibly grateful to patients and their families, investigators and their clinical study sites, and our employees and partners for advancing a study that has the potential to treat very challenging and life threatening cancers," commented Matt Kane, CEO and co-founder of Precision BioSciences. "It is very encouraging that, based on the safety profile we have so far observed with our first CAR T candidate, PBCAR0191, the FDA authorized us to begin dosing PBCAR20A at what was expected to be our second dose level. It is a testament to the progress we have made over the past year that Precision now has two CAR T programs in clinical trials."
"We believe that PBCAR20A provides the opportunity to apply our ARCUS technology and cell engineering platform to target CD20, a validated antigen in NHL and CLL/SLL, which often remains expressed in patients previously treated with CD19-targeted therapies. This approach may allow us to address some patients who failed CD19 CAR T treatment and/or consider earlier line treatment to achieve meaningful clinical benefit in patients suffering with these diseases," noted Chris Heery, MD, Chief Medical Officer of Precision BioSciences. "Additionally, receiving orphan drug designation from the FDA for MCL provides a special opportunity to serve a group of patients who have a poor prognosis with currently available treatments."
In preclinical disease models, PBCAR20A demonstrated potent in vivo clearance of CD20+ tumor cells and overall tumor volume reduction, with no evidence of graft-versus-host disease. Based on the safety profile observed from early dose levels in the Company's ongoing Phase 1/2a study of PBCAR0191, the FDA authorized dosing in this Phase 1/2a study of PBCAR20A to begin at what was originally designed to be Dose Level 2 (1x106 cells/kg), with the subsequent dose level expected to be 3x106 cells/kg.
About the PBCAR20A Clinical Trial
PBCAR20A is being evaluated in a Phase 1/2a multicenter, nonrandomized, open-label, dose-escalation and dose-expansion clinical trial in adult NHL and CLL/SLL patients. The trial will be conducted at multiple U.S. sites. For more information visit www.clinicaltrials.gov, study identifier number NCT04030195.
Precision's Off-The-Shelf CAR T Platform
Precision is advancing a pipeline of cell-phenotype optimized allogeneic CAR T therapies, leveraging fully scaled, proprietary manufacturing processes. The platform is designed to maximize the number of patients who can potentially benefit from CAR T therapy. Precision carefully selects high-quality T cells derived from healthy donors as starting material, then utilizes its unique ARCUS genome editing technology to modify the cells via a single-step engineering process. By inserting the CAR gene at the T cell receptor (TCR) locus, this process knocks in the CAR while knocking out the TCR, creating a consistent product that can be reliably and rapidly manufactured and is designed to prevent graft-versus-host disease. Precision optimizes its CAR T therapy candidates for immune cell expansion in the body by maintaining a high proportion of naïve and central memory CAR T cells throughout the manufacturing process and in the final product.
About Precision BioSciences, Inc.
Precision BioSciences is dedicated to improving life (DTIL) through its proprietary genome editing platform, "ARCUS." Precision leverages ARCUS in the development of its product candidates, which are designed to treat human diseases and create healthy and sustainable food and agriculture solutions. Precision is actively developing product candidates in three innovative areas: allogeneic CAR T immunotherapy, in vivo gene correction, and food. For more information regarding Precision, please visit www.precisionbiosciences.com.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding the Company's plans for its Phase 1/2a clinical trial for PBCAR20A, the potential results with respect to PBCAR20A and the expectation for additional CAR T candidates to enter clinical trials. In some cases, you can identify forward-looking statements by terms such as "anticipate," "believe," "could," "expect," "should," "plan," "intend," "estimate," "target," "mission," "may," "will," "would," "should," "could," "target," "project," "predict," "contemplate," "potential," or the negative thereof and similar words and expressions.
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