CAR-T BCMA in De­vel­op­ment for Patients with Multiple Myeloma

Horsham, PA (Press Release) – Janssen Bio­tech, Inc. (“Janssen”), a Janssen Pharma­ceu­tical Com­pany of John­son & John­son, an­nounced to­day that it has entered into a world­wide col­lab­o­ration and license agree­ment with Legend Bio­tech USA Inc. and Legend Bio­tech Ireland Limited (“Legend”), sub­sid­i­aries of Genscript Bio­tech Corpo­ra­tion, to de­vel­op, manu­fac­ture and com­mer­cial­ize a chi­meric an­ti­gen re­cep­tor (CAR) T-cell drug can­di­date, LCAR-B38M, which spe­cif­i­cally targets the B-cell maturation an­ti­gen (BCMA). LCAR-B38M is cur­rently ac­cepted for re­view by the China Food and Drug Admin­istra­tion (CFDA) and in the planning phase of clin­i­cal stud­ies in the United States for mul­ti­ple myeloma.

“Despite sig­nif­i­cant ad­vances, mul­ti­ple myeloma re­mains an incurable dis­ease for most patients, creating the need for addi­tional, highly active op­tions. LCAR-B38M provides an inno­va­tive ap­proach with the poten­tial to trans­form the treat­ment of myeloma,” said Peter F. Lebowitz, M.D., Ph.D., Global Thera­peutic Area Head, Oncology, Janssen Re­search & De­vel­op­ment, LLC. “We look for­ward to col­lab­o­rating with the pio­neer­ing scientific team at Legend and applying our ex­per­tise to the devel­op­ment of this cell ther­apy, with the goal of build­ing regi­mens aiming for a cure.”

LCAR-B38M is the first CAR-T ther­apy ac­cepted for re­view by the CFDA. Under terms of the agree­ment, Legend will grant Janssen a world­wide license to jointly de­vel­op and com­mer­cial­ize LCAR-B38M in mul­ti­ple myeloma with the Legend team of experts. Janssen will record world­wide net trade sales, except for sales made in Greater China. The com­pa­nies have entered into a 50/50 per­cent cost-sharing / profit-split arrange­ment, except in Greater China, where Janssen and Legend have a 30/70 per­cent cost-sharing / profit-split arrange­ment. Janssen will make an up­front pay­ment of $350 mil­lion that will be recorded in the fourth quarter and addi­tional pay­ments based upon the achieve­ment of cer­tain devel­op­ment, regu­la­tory and sales mile­stones.

Johnson & John­son reaffirms its pre­vi­ously an­nounced adjusted earn­ings guidance for full-year 2017 of $7.25-$7.30 per share.

“We are pleased to enter into a part­ner­ship with Legend to gain access to their CAR-T plat­form, an im­por­tant future thera­peutic modality for Janssen,” says Mathai Mammen, M.D., Ph.D., Global Head, Science & De­vel­op­ment, Janssen Re­search & De­vel­op­ment, LLC. “Legend is an inno­va­tive bio­tech com­pany that has devel­oped a dif­fer­en­ti­ated CAR-T ther­apy, which has shown promising re­­sults in early-stage mul­ti­ple myeloma trials con­ducted in China. We are ex­cited to bring Janssen’s global ex­per­tise in drug devel­op­ment to ad­vance this inno­va­tion into poten­tial new treat­ment op­tions for patients around the world.”

About CAR-T and BCMA

CAR T-cells are an inno­va­tive ap­proach to eradicate can­cer cells by har­nessing the power of a patient’s own im­mune sys­tem. BCMA is a pro­tein that is highly ex­pressed on myeloma cells. By targeting BCMA via a CAR-T ap­proach, CAR-T ther­a­pies may have the poten­tial to redefine the treat­ment par­a­digm for mul­ti­ple myeloma and poten­tially ad­vance to­wards cures for patients with the dis­ease.

About Multiple Myeloma

Multiple myeloma is an incurable blood can­cer that oc­curs when malignant plasma cells grow un­con­trol­lably in the bone mar­row.^1,2 While some patients with myeloma have no symp­toms at all, most patients are diag­nosed due to symp­toms, which can in­clude bone fracture or pain, low red blood counts, fatigue, cal­cium elevation, kidney prob­lems or in­fec­tions.³ Despite sig­nif­i­cant treat­ment ad­vances in the past 10 years, many myeloma patients relapse after initial or sec­ond­ary treat­ment and/or be­come re­sis­tant to ther­a­pies. There is a sig­nif­i­cant unmet med­i­cal need for more efficacious treat­ments that can over­come re­sis­tance asso­ci­ated with stan­dard of care based on an immuno­modu­la­tory agent and/or a pro­te­a­some in­hib­i­tor to induce deeper and durable re­sponses.^1,4 Globally, it is esti­mated that 124,225 people were diag­nosed and 87,084 died from mul­ti­ple myeloma in 2015.^5,6

Janssen in Oncology

In on­col­ogy, our goal is to fundamentally alter the way can­cer is under­stood, diag­nosed and man­aged, rein­forcing our com­mitment to the patients who in­spire us. In looking to find inno­va­tive ways to address the can­cer chal­lenge, our pri­mary efforts focus on sev­er­al treat­ment and prevention solu­tions. These in­clude a focus on hema­to­logic malig­nan­cies, prostate can­cer and lung can­cer; can­cer interception with the goal of devel­op­ing prod­ucts that in­ter­rupt the carcinogenic process; bio­­markers that may help guide targeted, in­di­vid­ualized use of our ther­a­pies; as well as safe and ef­fec­tive identi­fi­ca­tion and treat­ment of early changes in the tumor micro­en­vi­ron­ment. Please visit www.janssen.com/oncology.

About the Janssen Pharma­ceu­tical Com­panies

At the Janssen Pharma­ceu­tical Com­panies of John­son & John­son, we are work­ing to create a world without dis­ease. Transforming lives by finding new and better ways to prevent, intercept, treat and cure dis­ease in­spires us. We bring to­geth­er the best minds and pur­sue the most promising science. We are Janssen. We col­lab­o­rate with the world for the health of everyone in it. Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenUS and www.twitter.com/JanssenGlobal.

About John­son & John­son

Caring for the world, one person at a time, in­spires and unites the people of John­son & John­son. We embrace re­search and science - bringing inno­va­tive ideas, prod­ucts and services to ad­vance the health and well-being of people. Our approx­i­mately 134,100 em­ploy­ees at more than 250 John­son & John­son oper­ating com­pa­nies work with part­ners in health care to touch the lives of over a billion people every day, through­out the world.

Cautions Concerning Forward-Looking State­ments

This press re­lease con­tains "forward-looking state­ments" as defined in the Private Se­cu­ri­ties Lit­i­ga­tion Reform Act of 1995 re­lated to a new col­lab­o­ration and prod­uct devel­op­ment. The reader is cautioned not to rely on these for­ward-looking state­ments. These state­ments are based on cur­rent ex­pec­ta­tions of future events. If under­lying assump­tions prove inaccurate or known or un­known risks or un­cer­tain­ties ma­teri­alize, actual re­­sults could vary ma­teri­ally from the ex­pec­ta­tions and pro­jec­tions of Janssen Bio­tech, Inc. and/or John­son & John­son. Risks and un­cer­tain­ties in­clude, but are not lim­ited to: the poten­tial that the ex­pec­ted ben­e­fits and oppor­tu­ni­ties re­lated to the col­lab­o­ration may not be realized or may take longer to realize than ex­pec­ted; chal­lenges in­her­ent in new prod­uct devel­op­ment, in­­clud­ing the un­cer­tainty of clin­i­cal success and obtaining regu­la­tory ap­prov­als; com­pe­ti­tion, in­­clud­ing tech­no­log­i­cal ad­vances, new prod­ucts and pat­ents attained by com­pet­i­tors; un­cer­tainty of com­mer­cial success for new prod­ucts; the ability of the com­pany and Legend to suc­cess­fully execute stra­te­gic plans; im­pact of business com­bi­na­tions and divestitures; chal­lenges to pat­ents; changes in be­havior and spending pat­terns or fi­nan­cial distress of pur­chasers of health care prod­ucts and services; and global health care reforms and trends to­ward health care cost con­tainment. A fur­ther list and descriptions of these risks, un­cer­tain­ties and other factors can be found in John­son & John­son's Annual Report on Form 10-K for the fiscal year ended Jan­u­ary 1, 2017, in­­clud­ing under “Item 1A. Risk Factors,” its most recently filed Quar­ter­ly Report on Form 10-Q, in­­clud­ing under the cap­tion “Cautionary Note Regarding Forward-Looking State­ments,” and the com­pany's sub­se­quent filings with the Se­cu­ri­ties and Ex­change Com­mis­sion. Copies of these filings are avail­able online at www.sec.gov, www.jnj.com or on re­quest from John­son & John­son. The Janssen Pharma­ceu­tical Com­panies of John­son & John­son and John­son & John­son do not under­take to up­date any for­ward-looking state­ment as a re­­sult of new in­­for­ma­tion or future events or devel­op­ments.

References

1. Kumar, SK et al. Risk of progression and survival in multiple myeloma relapsing after therapy with IMiDs and bortezomib: a multicenter international myeloma working group study. Leukemia. 2012;26(1):149-157. https://www.ncbi.nlm.nih.gov/pubmed/21799510.
2. American Cancer Society. "Multiple Myeloma Overview." Available at: http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma. Accessed December 2017.
3. American Cancer Society. "How is Multiple Myeloma Diagnosed?" Available at: http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-diagnosis. Accessed December 2017.
4. Borrello, I. Can we change the disease biology of multiple myeloma? Leukemia Research. 2012;36(0 1):S3-12. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3698609/.
5. GLOBOCAN 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide: Number of New Cancers in 2015. Available at: http://globocan.iarc.fr/old/burden.asp?selection_pop=224900&Text-p=World&selection_cancer=17270&Text-c=Multiple+myeloma&pYear=3&type=0&window=1&submit=%C2%A0Execute. Accessed December 2017.
6. GLOBOCAN 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide: Number of Cancer Deaths in 2015. Available at: http://globocan.iarc.fr/old/burden.asp?selection_pop=224900&Text-p=World&selection_cancer=17270&Text-c=Multiple+myeloma&pYear=3&type=1&window=1&submit=%C2%A0Execute. Accessed December 2017.

Source: Janssen.