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 the U.S. Food and Drug Admin­istra­tion (FDA) has granted Fast Track Desig­na­tion to PBCAR269A for the treat­ment of re­lapsed / re­frac­tory mul­ti­ple myeloma. This is the com­pany’s sec­ond allo­geneic chi­meric an­ti­gen re­cep­tor (CAR T) cell ther­apy to re­ceive Fast Tack Desig­na­tion for which the FDA pre­vi­ously granted Orphan Drug Desig­na­tion.

“Fast Track Desig­na­tion will help us expedite our allo­geneic CAR T cell ther­apy pro­gram aimed to address the unmet med­i­cal need among patients with re­lapsed / re­frac­tory mul­ti­ple myeloma,” said Chris Heery, M.D., Chief Medical Of­fi­cer of Pre­ci­sion BioSciences. “We look for­ward to work­ing more closely with the FDA as we con­tinue to in­ves­ti­gate PBCAR269A as a po­ten­tial new treat­ment op­tion that can be more broadly avail­able to patients who other­wise may be in­eli­gible or unable to access existing au­tol­o­gous CAR T ther­a­pies.”

About Fast Track Desig­na­tion

Fast Track Desig­na­tion facilitates the expedited de­vel­op­ment and re­view of a new drug, whether alone or in com­bi­na­tion with another drug that may address unmet med­i­cal needs and treats a serious or life-threatening dis­ease. Benefits of this desig­na­tion in­clude more fre­quent engagements with the FDA to discuss the drug’s clin­i­cal de­vel­op­ment plan, eligibility for Accelerated Approval and Priority Review, and a Rolling Review in which Pre­ci­sion can submit com­pleted sections of its New Drug Appli­ca­tion (NDA) for re­view by the FDA rather than waiting to com­plete the NDA be­fore it can be sub­mitted for re­view.

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 is 6 x 105 CAR T cells/kg body weight with sub­se­quent cohorts treated with es­ca­lat­ing doses to a max­i­mum dose of 6 x 106 CAR T cells/kg body weight. The trial is being con­ducted at mul­ti­ple U.S. sites with clin­i­cal trial ma­teri­al gen­er­ated in-house at the Manu­fac­tur­ing Center for Advanced Thera­peutics (MCAT) in Durham, North Carolina. 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 that 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. This brief video illustrates the process.

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 known adequate treat­ments exist. 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, re­­sults from and in­for­ma­tion involving the Com­pany’s clin­i­cal trials involving PBCAR269A, and details of our manu­fac­tur­ing process 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,” “de­signed to”, “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 and effects of restrictions thereunder; risks asso­ci­ated with raising addi­tional capital; 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 lim­ited ability or in­abil­ity to assess the safety and ef­fi­cacy of our prod­uct can­di­dates; 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 ob­tain an adequate supply of T cells from qualified donors; our ability to achieve our antic­i­pated op­er­at­ing efficiencies at our manu­fac­tur­ing facility; delays or dif­fi­culties in our and our col­lab­o­rators’ ability to en­roll patients; changes in interim “top-line” and initial data that we an­nounce or pub­lish; if our prod­uct can­di­dates do not work as in­tended or cause un­de­sir­able side effects; risks asso­ci­ated with appli­cable health­care, data pro­tec­tion, 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 and reliance on 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 exec­u­tives and per­son­nel; mar­ket and eco­nom­ic con­di­tions; effects of sys­tem failures and se­cu­ri­ty breaches; 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; effects of tax rules; risks re­lated to ownership of our common stock 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, in­clud­ing, but not lim­ited to, our Quar­ter­ly Report on Form 10-Q for the quar­ter­ly period ended June 30, 2020, 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.