PRIME (PRIority MEdicines) desig­na­tion based on clin­i­cal study re­­sults eval­u­ating safety and ef­fi­cacy of novel CAR-T ther­apy in the treat­ment of patients with ad­vanced re­lapsed or re­frac­tory mul­ti­ple myeloma

Beerse, Belgium (Press Release) - The Janssen Pharma­ceu­tical Com­panies of John­son & John­son to­day an­nounced that the Euro­pean Medicines Agency (EMA) has granted a PRIME (PRIority MEdicines) desig­na­tion for the com­pany’s inves­ti­ga­tional B-cell maturation an­ti­gen (BCMA) chi­meric an­ti­gen re­cep­tor T-cell (CAR-T) ther­apy, JNJ-68284528 (JNJ-4528). PRIME offers en­hanced inter­action and early dialogue to optimise devel­op­ment plans and speed up evaluation of cutting-edge, scientific ad­vances that target a high unmet med­i­cal need.¹

“The PRIME desig­na­tion of this novel BCMA CAR-T ther­apy highlights the value of regu­la­tory inno­va­tion in the Euro­pean Union,” said Sjaak Bot, Vice Pres­i­dent, Head EMEA Regulatory Affairs at Janssen Biologics B.V. “We hope to bring this im­por­tant ad­vance to patients as quickly as pos­si­ble and this PRIME desig­na­tion, the first for Janssen, marks an im­por­tant mile­stone to­wards poten­tial mar­ket ap­prov­al.”

The PRIME desig­na­tion is based on re­­sults from the Phase 1/2 LEGEND-2 study (NCT03090659) eval­u­ating LCAR-B38M CAR-T cells, sponsored by Nanjing Legend Bio­tech Co.,² and the Phase 1b/2 CARTITUDE-1 study (NCT03548207) eval­u­ating JNJ-4528, sponsored by Janssen and being con­ducted in col­lab­o­ration with Legend Bio­tech USA Inc.³ Results from the LEGEND-2 study were pre­sented at the American Society of He­ma­tol­ogy (ASH) 2018 annual meeting.⁴ Results from the CARTITUDE-1 study will be pre­sented in the future.

“CAR-T ther­apy is an ex­cit­ing thera­peutic plat­form that har­nesses the patient’s im­mune sys­tem to attack tumour cells,” said Sen Zhuang, M.D., Ph.D., Vice Pres­i­dent, Oncology Clinical De­vel­op­ment, Janssen Re­search & De­vel­op­ment, LLC. “We con­tinue to ad­vance this novel BCMA targeted CAR-T ther­apy through clin­i­cal stud­ies globally as we strive to bring it to the patients with mul­ti­ple myeloma around the world.”

JNJ-4528 is cur­rently being in­ves­ti­gated for the treat­ment of patients with mul­ti­ple myeloma who have re­ceived at least three prior regi­mens, in­clud­ing a pro­te­a­some in­hib­i­tor (PI), an immuno­modu­la­tory drug (IMiD), and an anti-CD38 anti­body, and have doc­u­mented dis­ease pro­gres­sion within 12 months of start­ing the most recent ther­apy, or are double re­frac­tory to an IMiD and PI.3 These patients have few avail­able treat­ment op­tions and are often faced with poor out­comes.⁵

In De­cem­ber 2017, Janssen entered into a world­wide col­lab­o­ration and licence agree­ment with Legend Bio­tech to jointly de­vel­op and com­mer­cialise LCAR-B38M in mul­ti­ple myeloma.⁶ In China, the Phase 2 CARTIFAN-1 con­firmatory trial (NCT03758417), sponsored by Nanjing Legend Bio­tech Co. Ltd. and registered with the Center for Drug Evaluation (CTR20181007), is actively recruiting to fur­ther eval­u­ate LCAR-B38M in patients with ad­vanced re­lapsed or re­frac­tory mul­ti­ple myeloma.⁷

About LEGEND-2

LEGEND-2 (NCT03090659) is an on­go­ing single-arm, open-label Phase 1/2 study being con­ducted at four participating hos­pi­tals in China eval­u­ating the ef­fi­cacy and safety of LCAR-B38M for the treat­ment of re­lapsed or re­frac­tory mul­ti­ple myeloma.²

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.⁸ By targeting BCMA via this ap­proach, CAR-T ther­a­pies may have the poten­tial to redefine treat­ment for mul­ti­ple myeloma.

About Multiple Myeloma

Multiple myeloma is an incurable blood can­cer that starts in the bone mar­row and is char­ac­ter­ised by an excessive pro­lif­er­a­tion of plasma cells.⁹ In Europe, more than 48,200 people were diag­nosed with mul­ti­ple myeloma in 2018, and more than 30,800 patients died.¹⁰ Almost 40 per­cent of patients with mul­ti­ple myeloma do not reach five-year sur­vival.¹¹

Although treat­ment may re­­sult in remission, unfortunately, patients will most likely relapse as there is cur­rently no cure.¹² Re­frac­tory mul­ti­ple myeloma is when a patient’s dis­ease is non-responsive or progresses within 60 days of their last ther­apy.^13,14 Re­lapsed myeloma is when the dis­ease has re­turned after a period of initial, partial or com­plete remission and does not meet the definition of being re­frac­tory.¹⁵ While some patients with mul­ti­ple myeloma have no symp­toms at all, most patients are diag­nosed due to symp­toms that can in­clude bone prob­lems, low blood counts, cal­cium elevation, kidney prob­lems or in­fec­tions.¹⁶ Patients who relapse after treat­ment with stan­dard ther­a­pies, in­clud­ing PIs and IMiDs, have poor prognoses and few treat­ment op­tions avail­able.¹⁷

About the Janssen Pharma­ceu­tical Com­panies of John­son & John­son

At Janssen, we’re creating a future where dis­ease is a thing of the past. We’re the Pharma­ceu­tical Com­panies of John­son & John­son, work­ing tirelessly to make that future a reality for patients every­where by fighting sickness with science, im­prov­ing access with ingenuity, and heal­ing hope­less­ness with heart. We focus on areas of med­i­cine where we can make the biggest dif­fer­ence: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.

Learn more at www.janssen.com/emea. Follow us at www.twitter.com/janssenEMEA for our latest news. Janssen Biologics B.V. 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 re­gard­ing prod­uct devel­op­ment and the poten­tial ben­e­fits and treat­ment im­pact of LCAR-B38M and JNJ-68284528. 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 ma­teri­alise, actual re­­sults could vary ma­teri­ally from the ex­pec­ta­tions and pro­jec­tions of Janssen Biologics B.V., Janssen Re­search & De­vel­op­ment, LLC, any of the Janssen Pharma­ceu­tical Com­panies of John­son & John­son and/or John­son & John­son. Risks and un­cer­tain­ties in­clude, but are not lim­ited to: chal­lenges and un­cer­tain­ties in­her­ent in prod­uct re­search and devel­op­ment, in­clud­ing the un­cer­tainty of clin­i­cal success and of obtaining regu­la­tory ap­prov­als; un­cer­tainty of com­mer­cial success; manu­fac­tur­ing dif­fi­culties and delays; com­pe­ti­tion, in­clud­ing tech­no­log­i­cal ad­vances, new prod­ucts and pat­ents attained by com­pet­i­tors; chal­lenges to pat­ents; prod­uct ef­fi­cacy or safety con­cerns re­­sult­ing in prod­uct recalls or regu­la­tory action; changes in be­haviour and spending pat­terns of pur­chasers of health care prod­ucts and services; changes to appli­cable laws and reg­u­la­tions, in­clud­ing 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 De­cem­ber 31, 2017, in­clud­ing in the sections cap­tioned “Cautionary Note Regarding Forward-Looking State­ments” and “Item 1A. Risk Factors,” and in the com­pany’s sub­se­quent Quar­ter­ly Reports on Form 10-Q and other 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. None of the Janssen Pharma­ceu­tical Com­panies nor John­son & John­son under­takes to up­date any for­ward-looking state­ment as a re­­sult of new in­for­ma­tion or future events or devel­op­ments.

References

1. European Medicines Agency. PRIME Factsheet. Available at: https://www.ema.europa.eu/en/human-regulatory/research-development/prime-priority-medicines Last accessed March 2019.
2. ClinicalTrials.gov. LCAR-B38M-02 cells in treating re­lapsed / refractory (R/R) multiple myeloma (LEGEND-2). NCT03090659. Available at: https://clinicaltrials.gov/ct2/show/NCT03090659 Last accessed March 2019.
3. ClinicalTrials.gov. A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-Cell Maturation Antigen (BCMA) in Participants With Relapsed or Refractory Multiple Myeloma. NCT03548207. Available at: https://clinicaltrials.gov/ct2/show/NCT03548207 Last accessed March 2019.
4. Zhao WH, Liu J, Wang BY, et al. Updated analysis of a phase 1, open-label Study of LCAR-B38M, a chi­meric an­ti­gen re­cep­tor T-cell ther­apy directed against B-cell maturation an­ti­gen, in patients with re­lapsed / refractory multiple myeloma. Presented at 60th Annual Meeting and Exposition of the American Society of Hematology (ASH), San Diego, CA, USA, 1-4 De­cem­ber 2018: Abstract 955.
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Source: Janssen.