The Janssen Pharmaceutical Companies of Johnson & Johnson 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.
“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., 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. Results from the LEGEND-2 study were presented at the American Society of Hematology (ASH) 2018 annual meeting. 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.
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. 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.
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. 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. In Europe, more than 48,200 people were diagnosed with multiple myeloma in 2018, and more than 30,800 patients died. Almost 40 percent of patients with multiple myeloma do not reach five-year survival.
Although treatment may result in remission, unfortunately, patients will most likely relapse as there is currently no cure. Refractory multiple myeloma is when a patient’s disease is non-responsive or progresses within 60 days of their last therapy., 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. 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. Patients who relapse after treatment with standard therapies, including PIs and IMiDs, have poor prognoses and few treatment options available.
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.
 European Medicines Agency. PRIME Factsheet. Available at: https://www.ema.europa.eu/en/human-regulatory/research-development/prime-priority-medicines Last accessed: March 2019
 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.
 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.
Mobile: +1 215-620-0111
Mobile: +31 621-385-718
Phone: +1 732-524-2955
Phone: +1 732-524-3922