Precision BioSciences Announces FDA Acceptance of IND for PBCAR269A, a BCMA Targeted Genome Edited Allogeneic CAR T Therapy Candidate for Multiple Myeloma

-Phase 1 clinical trial of off-the-shelf (allogeneic) anti-BCMA CAR T therapy candidate for patients with relapsed/refractory multiple myeloma expected to begin dosing patients in 2020-

-PBCAR269A has received Orphan Drug Designation from the FDA for the treatment of multiple myeloma-

-First program for which clinical trial material will be generated fully in-house at Precision’s Manufacturing Center for Advanced Therapeutics (MCAT) in Durham, N.C.-

DURHAM, NC, USA I January 13, 2020 I Precision BioSciences, Inc. (Nasdaq: DTIL), a genome editing company dedicated to improving life through the application of its pioneering, proprietary ARCUS® platform, today announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for PBCAR269A, the Company’s third allogeneic chimeric antigen receptor (CAR) T cell therapy candidate. The FDA has also granted Orphan Drug Designation to PBCAR269A for the treatment of multiple myeloma. Wholly-owned by Precision, PBCAR269A is an allogeneic CAR T therapy candidate which targets the B-cell maturation antigen (BCMA) and is in development for the treatment of relapsed/refractory multiple myeloma. Precision plans to initiate dosing in this Phase 1 clinical trial in 2020.

“FDA acceptance of the IND for PBCAR269A further underscores the ongoing progress in our allogeneic CAR T pipeline,” commented Matt Kane, Chief Executive Officer of Precision BioSciences. “We have now moved three CAR T programs from preclinical to clinical stage development since April 2019, and we look forward to continuing to advance our allogeneic CAR T portfolio to bring these novel therapeutic candidates to patients. The IND for PBCAR269A builds on the initial clinical data we presented in late 2019 for our lead program, PBCAR0191, and the FDA’s acceptance of the IND for our second program, PBCAR20A. It’s a testament to the hard work and expertise of the Precision team that we will be able to generate the clinical trial material for the PBCAR269A trial in-house at our MCAT manufacturing facility.”

“In preclinical disease models, PBCAR269A has demonstrated no evidence of graft-versus-host disease at doses that resulted in potent anti-tumor activity,” commented Chris Heery, Chief Medical Officer of Precision BioSciences. “There remains significant unmet need in the treatment of relapsed/refractory multiple myeloma, and we are excited to begin clinical trials with an off-the-shelf CAR T therapy candidate in this setting.”

About the PBCAR269A Clinical Trial
PBCAR269A will be evaluated in a Phase 1 multicenter, open-label dose-escalation and dose-expansion clinical trial in adult relapsed/refractory multiple myeloma patients. The trial will be conducted at multiple U.S. sites. For more information, visit www.clinicaltrials.gov, study identifier number NCT04171843.

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 by improving access to care through a well-tolerated lymphodepletion regimen, high quality cell products derived from carefully selected healthy donors, and a consistent final cell product with attributes in line with those previously observed to result in optimal safety and activity profiles. 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 in a single step, 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.

SOURCE: Precision BioSciences

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