Precision BioSciences Announces Dosing of First Patient in Phase 1/2a Clinical Trial 

Precision BioSciences, Inc. recently announced that the first patient has been dosed in a Phase 1/2a clinical trial of PBCAR269A, its third allogeneic chimeric antigen receptor (CAR) T cell therapy candidate. Wholly owned by Precision, PBCAR269A targets the B-cell maturation antigen (BCMA) and is being evaluated for the treatment of relapsed/refractory multiple myeloma.

“PBCAR269A is our third off-the-shelf CAR T candidate to advance into the clinic; the second within the last 2 months. Despite the uncertain impact of COVID-19 on patients and the healthcare community at large, we maintained our focus and dedication that have enabled continued execution during the pandemic,” said Matt Kane, CEO and Co-founder of Precision Biosciences. “Notably, this will be our first study for which all clinical trial material will be produced at our in-house manufacturing facility.”

“There remains significant unmet need for a broadly available and well-tolerated treatment for patients with relapsed or refractory Multiple Myeloma,” said Chris Heery, MD, Chief Medical Officer of Precision BioSciences. “We are committed to improving the access of CAR T therapies for more patients. We appreciate the commitment of our clinical sites to start enrollment ahead of schedule, even during these difficult times, and the willingness of patients to take part in this trial.”

In preclinical disease models, PBCAR269A demonstrated potent in vivo clearance of BCMA+ tumor cells and overall tumor volume reduction, with no evidence of graft-versus-host disease (GVHD). Clinical trial material for this study is generated at the Company’s in-house Manufacturing Center for Advanced Therapeutics (MCAT) in Durham, NC. PBCAR269A has received Orphan Drug Designation from the FDA for the treatment of multiple myeloma.

PBCAR269A is being evaluated in a Phase 1/2a multicenter, nonrandomized, open-label, parallel assignment, single-dose, dose-escalation, and dose-expansion study to evaluate the safety and clinical activity of PBCAR269A in adults with relapsed/refractory multiple myeloma. The starting dose of PBCAR269A will be 6 x 10⁵ CAR T cells/kg body weight. Subsequent cohorts will be treated with escalating doses to a maximum dose of 6 x 10⁶ CAR T cells/kg body weight. The trial will be conducted at multiple US sites. For more information, visit www.clinicaltrials.gov, study identifier number NCT04171843.

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.

Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS genome editing platform. ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the company’s pipeline consists of multiple “off-the-shelf” CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates to cure genetic and infectious diseases where no adequate treatments exist. Elo Life Systems is a wholly-owned subsidiary of Precision BioSciences also using ARCUS to benefit human health and wellness with novel food products that enhance the nutrition and diversity of global food supply. For more information, visit www.precisionbiosciences.com.