Planned Start of Clinical Program and Clearance of U.S. Food and Drug Admin­istra­tion Inves­ti­ga­tional New Drug Appli­ca­tion Reflect Progress in Strategic Partnership with Legend Bio­tech

Spring House, PA (Press Release) – The Janssen Pharma­ceu­tical Com­panies of John­son & John­son an­nounced to­day the initiation of a Phase 1b/2 clin­i­cal devel­op­ment pro­gram studying JNJ-68284528 (developed based on Legend's LCAR-B38M), a chi­meric an­ti­gen re­cep­tor T cell (CAR-T) ther­apy directed against B cell maturation an­ti­gen (BCMA), in patients with re­lapsed or re­frac­tory mul­ti­ple myeloma. The planned start of the Phase 1b/2 study (68284528MMY2001), scheduled to begin en­roll­ment in the sec­ond half of 2018, follows the U.S. Food and Drug Admin­istra­tion (FDA) clear­ance of the Inves­ti­ga­tional New Drug (IND) appli­ca­tion sub­mitted by Janssen.

As an­nounced in De­cem­ber 2017, Janssen entered into a world­wide col­lab­o­ration and license agree­ment with Legend Bio­tech USA Inc. and Legend Bio­tech Ireland Limited ("Legend"), sub­sid­i­aries of GenScript Bio­tech Corpo­ra­tion. Under the terms of the agree­ment, Legend granted Janssen Bio­tech, Inc. a world­wide license to jointly de­vel­op and com­mer­cial­ize JNJ-68284528 in mul­ti­ple myeloma.

"We are com­mit­ted to rapidly ad­vanc­ing JNJ-68284528, and we are pleased to ini­ti­ate a global clin­i­cal devel­op­ment pro­gram to fur­ther eval­u­ate this cell-based ther­apy," said Peter F. Lebowitz, M.D., Ph.D., Global Thera­peutic Area Head, Oncology, Janssen Re­search & De­vel­op­ment, LLC. "As we strive to elim­i­nate mul­ti­ple myeloma, we are hopeful that this BCMA targeted CAR-T ther­apy will play an im­por­tant role in the treat­ment of this dis­ease."

The Phase 1b/2, open-label, multi­center study will eval­u­ate the safety and ef­fi­cacy of JNJ-68284528 in adults with re­lapsed or re­frac­tory mul­ti­ple myeloma. The pri­mary objective of the Phase 1b portion of the study is to char­ac­ter­ize the safety and estab­lish­ the dose of JNJ-68284528, which was in­formed by the first-in-human study with LCAR-B38M CAR-T cells (Legend-2). The pri­mary objective for the Phase 2 portion of the study is to eval­u­ate the ef­fi­cacy of JNJ-68284528 (primary end­point: over­all re­sponse rate [partial re­sponse or better] as defined by the Inter­na­tional Myeloma Work­ing Group re­sponse criteria).

About CAR-T and BCMA

CAR-T cells are an inno­va­tive ap­proach to eradicating 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 Re­frac­tory can­cer oc­curs when a patient's dis­ease is re­sis­tant to treat­ment or in the case of mul­ti­ple myeloma, patients progress within 60 days of their last ther­apy.^3,4 Re­lapsed can­cer means the dis­ease has re­turned after a period of initial, partial or com­plete remission.⁵ In 2018, it is esti­mated that 30,700 people will be diag­nosed and 12,770 will die from the dis­ease in the United States.⁶ 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.⁷

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/JanssenGlobal and www.twitter.com/JanssenUS. Janssen Bio­tech, Inc. and Janssen Re­search & De­vel­op­ment, LLC are part of the Janssen Pharma­ceu­tical Com­panies of John­son & John­son.

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 clin­i­cal devel­op­ment pro­gram studying LCAR-B38M, in patients with re­lapsed or re­frac­tory mul­ti­ple myeloma. 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., Janssen Re­search & De­vel­op­ment, LLC, any of the other Janssen Pharma­ceu­tical Com­panies 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. Leukemia. 2012 Jan; 26(1):149-57.
2. American Cancer Society. "What Is Multiple Myeloma?." Available at: http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-what-is-multiple-myeloma. Accessed March 2018.
3. National Cancer Institute. "NCI Dictionary of Cancer Terms: Refractory." Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=350245. Accessed March 2018.
4. Richardson, et al. "The Treatment of Relapsed and Refractory Multiple Myeloma." ASH Education Book. January 1, 2007 vol. 2007 no. 1 317-323.
5. National Cancer Institute. "NCI Dictionary of Cancer Terms: Relapsed." Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=45866. Accessed March 2018.
6. American Cancer Society. "Key Statistics for Multiple Myeloma." Available at: https://www.cancer.org/cancer/multiple-myeloma/about/key-statistics.html. Accessed March 2018.
7. American Cancer Society. "Diagnosing Multiple Myeloma From Test Results." Available at: http://www.cancer.org/cancer/multiplemyeloma/detailedguide/multiple-myeloma-diagnosis. Accessed March 2018.

Source: Janssen Pharma­ceu­ticals.