• PBCAR269A Targets BCMA for the Treatment of Re­lapsed / Re­frac­tory Multiple Myeloma and is the Com­pany’s Third Inves­ti­ga­tional Allogeneic CAR T Candidate Advanced to the Clinic
• PBCAR269A is the First Off-the-Shelf Candidate Produced at In-House Manufacturing Center

Durham, NC (Press Release) – Pre­ci­sion BioSciences, Inc. (Nasdaq: DTIL), a clin­i­cal stage bio­technol­ogy com­pany ded­i­cated to im­prov­ing life with its novel and pro­pri­e­tary ARCUS® genome edit­ing plat­form, to­day 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 two 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,” commented 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, North Carolina. PBCAR269A has re­ceived Orphan Drug Desig­na­tion from the FDA for the treat­ment of mul­ti­ple myeloma.

About the PBCAR269A Clinical Trial

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 U.S. sites. For more in­for­ma­tion, visit www.clinicaltrials.gov, study identifier num­ber NCT04171843.

About Pre­ci­sion’s Allogeneic 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, 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 about Pre­ci­sion BioSciences please visit www.precisionbiosciences.com.

Forward-Looking State­ments

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, without lim­i­ta­tion, state­ments re­gard­ing the Com­pany’s timing of clin­i­cal trials and re­­sults there­from involving PBCAR269A and the ex­pected ben­e­fits of producing clin­i­cal trial ma­teri­al at the Com­pany’s in-house manu­fac­tur­ing facility. 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.

Forward-looking state­ments are based on man­age­ment’s cur­rent ex­pec­ta­tions, 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 im­por­tant factors, in­clud­ing, but not lim­ited to: our ability to be­come profitable; our ability to procure suf­fi­cient funding and re­quire­ments under our cur­rent debt in­stru­ments; our op­er­at­ing ex­penses and our ability to predict what those ex­penses will be; our lim­ited op­er­at­ing history; the success of our pro­grams and prod­uct can­di­dates in which we expend our resources; our de­pendence on our ARCUS tech­nology; the initiation, cost, timing, progress, achieve­ment of mile­stones and re­­sults of re­search and de­vel­op­ment ac­­tiv­i­ties, pre­clin­i­cal or greenhouse stud­ies and clin­i­cal or field trials; pub­lic perception about genome edit­ing tech­nology and its appli­ca­tions; com­pe­ti­tion in the genome edit­ing, bio­pharma­ceu­tical, bio­technol­ogy and agricultural bio­technol­ogy fields; our or our col­lab­o­rators’ ability to identify, de­vel­op and com­mer­cialize prod­uct can­di­dates; pend­ing and poten­tial liability lawsuits and penalties against us or our col­lab­o­rators re­lated to our tech­nology and our prod­uct can­di­dates; the U.S. and foreign regu­la­tory landscape appli­cable to our and our col­lab­o­rators’ de­vel­op­ment of prod­uct can­di­dates; our or our col­lab­o­rators’ ability to obtain and main­tain regu­la­tory ap­prov­al of our prod­uct can­di­dates, and any re­lated restrictions, lim­i­ta­tions and/or warnings in the label of an approved prod­uct can­di­date; our or our col­lab­o­rators’ ability to ad­vance prod­uct can­di­dates into, and suc­cess­fully design, implement and com­plete, clin­i­cal or field trials; poten­tial manu­fac­tur­ing prob­lems asso­ci­ated with the de­vel­op­ment or com­mer­cial­iza­tion of any of our prod­uct can­di­dates; our ability to achieve our antic­i­pated op­er­at­ing efficiencies at our manu­fac­tur­ing facility; delays or dif­fi­culties in our and our col­lab­o­rators’ ability to en­roll patients; if our prod­uct can­di­dates do not work as in­tended or cause undesirable side effects; risks asso­ci­ated with appli­cable health­care, data privacy and se­cu­ri­ty reg­u­la­tions and our compliance there­with; the rate and degree of mar­ket ac­ceptance of any of our prod­uct can­di­dates; the success of our existing col­lab­o­ration agree­ments, and our ability to enter into new col­lab­o­ration arrangements; our cur­rent and future rela­tion­ships with third parties in­clud­ing suppliers and manu­fac­turers; our ability to obtain and main­tain in­tel­lec­tual property pro­tec­tion for our tech­nology and any of our prod­uct can­di­dates; poten­tial lit­i­ga­tion relating to in­fringe­ment or misappropriation of in­tel­lec­tual property rights; our ability to ef­fec­tively man­age the growth of our op­er­a­tions; our ability to attract, retain, and motivate key scientific and man­age­ment per­son­nel; mar­ket and economic con­di­tions; effects of natural and man­made disasters, pub­lic health emergencies and other natural catastrophic events effects of the out­break of COVID-19, or any pan­dem­ic, epi­demic or out­break of an infectious dis­ease; insurance ex­penses and exposure to uninsured liabilities; and other im­por­tant factors discussed under the cap­tion “Risk Factors” in our Quar­ter­ly Report on Form 10-Q for the quar­ter­ly period ended March 31, 2020, as any 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 and the Investors & Media page of our website at in­vestor.precisionbiosciences.com.

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.