• 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 (Press Release) – 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.

Forward-Looking State­ments

Information con­tained in this press release con­tains for­ward-looking state­ments. All state­ments other than state­ments of present and historical facts con­tained in this press release, in­clud­ing without lim­i­ta­tion, state­ments re­gard­ing the poten­tial for the suc­cess­ful devel­op­ment of PBCAR269A for patients living with mul­ti­ple myeloma and the timing of trials for this ther­apy can­di­date and the pre­lim­i­nary data there­from, may be for­ward looking state­ments. Without limiting the foregoing, the words “anticipate,” “believe,” “could,” “expect,” “should,” “plan,” “intend,” “estimate,” “target,” “may,” “will,” “would,” “potential,” the neg­a­tive thereof and similar words and ex­pres­sions are in­tended to identify for­ward-looking state­ments. These for­ward-looking state­ments reflect var­i­ous assump­tions of Precision’s man­agement that may or may not prove to be correct. No for­ward-looking state­ment is a guar­an­tee of future results, per­for­mance, or achieve­ments, and one should avoid placing undue reliance on such state­ments.

Forward-looking state­ments are based on our man­agement’s beliefs and assump­tions and on in­for­ma­tion cur­rently avail­able to us. Such state­ments are subject to a number of known and unknown risks, un­cer­tainties and assump­tions, and actual results may differ ma­teri­ally from those ex­pressed or im­plied in the for­ward-looking state­ments due to var­i­ous factors, in­clud­ing, but not limited to, our ability to be­come profitable; our ability to procure suf­fi­cient funding; our limited op­er­at­ing history; our ability to identify, de­vel­op and com­mer­cial­ize our prod­uct can­di­dates; our dependence on our ARCUS tech­nology; the initiation, cost, timing, progress and results of re­search and devel­op­ment ac­­tiv­i­ties, pre­clin­i­cal or greenhouse studies and clin­i­cal or field trials; our or our col­lab­o­rators’ ability to identify, de­vel­op and com­mer­cial­ize prod­uct can­di­dates; our or our col­lab­o­rators’ ability to ad­vance prod­uct can­di­dates into, and suc­cess­fully com­plete, clin­i­cal or field trials; our or our col­lab­o­rators’ ability to obtain and main­tain regu­la­tory ap­prov­al of future prod­uct can­di­dates, and any related restrictions, lim­i­ta­tions and/or warnings in the label of an approved prod­uct can­di­date; the regu­la­tory landscape that will apply to our and our col­lab­o­rators’ devel­op­ment of prod­uct can­di­dates; our ability to achieve our antic­i­pated op­er­at­ing efficiencies as we com­mence manu­fac­tur­ing op­er­a­tions at our new facility; our ability to obtain and main­tain in­tel­lec­tual property pro­tec­tion for our tech­nology and any of our prod­uct can­di­dates; the poten­tial for off-target edit­ing or other adverse events, undesirable side effects or unexpected char­ac­ter­istics asso­ci­ated with any of our prod­uct can­di­dates; the success of our existing col­lab­o­ration agree­ments; our ability to enter into new col­lab­o­ration arrangements; pub­lic perception about genome edit­ing tech­nology and its appli­ca­tions; com­pe­ti­tion in the genome edit­ing, bio­pharma­ceu­tical, bio­technology and agricultural bio­technology fields; poten­tial manu­fac­tur­ing problems asso­ci­ated with any of our prod­uct can­di­dates; poten­tial liability lawsuits and penalties related to our tech­nology, our prod­uct can­di­dates and our current and future rela­tion­ships with third parties; and other im­por­tant factors discussed under the caption “Risk Factors” in our quar­ter­ly report on Form 10-Q filed for the quar­ter­ly period ended Sep­tem­ber 30, 2019, as such factors may be up­dated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov.

All for­ward-looking state­ments speak only as of the date of this press release, and except as re­quired by appli­cable law, we do not plan to pub­licly up­date or revise any for­ward-looking state­ments con­tained herein, whether as a result of any new in­for­ma­tion, future events, changed cir­cum­stances or other­wise.

Source: Precision Biosciences.