Business Wire

Janssen Announces CAR-T Therapy Ciltacabtagene Autoleucel (Cilta-cel) Accepted for Accelerated Assessment in Europe for the Treatment of Patients with Heavily Pretreated Multiple Myeloma

Share

The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) will perform an accelerated assessment of the Marketing Authorisation Application (MAA) for the B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor T-cell (CAR-T) therapy ciltacabtagene autoleucel (cilta-cel). Accelerated assessment is granted by the CHMP when a medicinal product is expected to be of major public health interest and therapeutic innovation and can significantly reduce the review timelines to evaluate an MAA.1

Cilta-cel is an investigational BCMA-directed CAR-T therapy in development for the treatment of adults with relapsed and/or refractory multiple myeloma.2 CAR‑T therapy is a highly personalised technology where a patient’s own T-cells are re-programmed to target and eradicate cancer.3

“Multiple myeloma is a rare, incurable cancer, and has long been an area of focus for Janssen,” said Sen Zhuang, M.D., Ph.D., Vice President, Clinical Research Development, Janssen Research & Development, LLC. “We are deeply committed to improving outcomes for patients with multiple myeloma, with a goal of delivering innovations that have the potential to expand current remission periods and improve quality of life.”

The cilta-cel MAA, which is targeted for submission in the first half of 2021, is supported by the positive results from the Phase 1b/2 CARTITUDE-1 study.2 The latest results from the CARTITUDE-1 study were presented at the American Society of Hematology (ASH) 2020 Annual Meeting.2

“We are excited that the potential clinical benefit of cilta-cel is being recognised and now look forward to working with the EMA to bring this highly innovative treatment to patients in need,” said Saskia De Haes, Vice President, EMEA Regulatory Affairs, Janssen R&D BE.

This accelerated approval milestone in Europe follows the December 2020 announcement of a rolling submission of the Biologics License Application (BLA) for cilta-cel to the U.S. Food and Drug Administration (FDA).4

#ENDS#

About CARTITUDE-1
CARTITUDE-1 (NCT03548207) is an ongoing Phase 1b/2, open-label, multicentre study evaluating the safety and efficacy of cilta-cel in adults with relapsed and/or refractory multiple myeloma, 99 percent of whom were refractory to the last line of treatment; 88 percent of whom were triple-class refractory, meaning their cancer did not, or no longer responds to an immunomodulatory agent (IMiD), a proteasome inhibitor (PI) and an anti-CD38 antibody.2,5

The primary objective of the CARTITUDE-1 study is to characterise the safety and confirm the dose of cilta-cel, informed by the first-in-human study with LCAR-B38M CAR-T cells (LEGEND-2) and to evaluate the efficacy of cilta-cel with overall response as the primary endpoint.2,5

About Ciltacabtagene Autoleucel (cilta-cel)
Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy for the treatment of patients with multiple myeloma. The design comprises a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies.2 CAR-T cells are an innovative approach to eradicating cancer cells by harnessing the power of a patient’s own immune system.6 BCMA is a protein that is highly expressed on myeloma cells.7

In December 2017, Janssen Biotech, Inc. (Janssen) entered into an exclusive worldwide license and collaboration agreement with Legend Biotech to develop and commercialise cilta-cel.8 In May 2018, Janssen initiated a Phase 1b/2 CARTITUDE-1 trial (NCT03548207) to evaluate the efficacy and safety of cilta-cel in adults with relapsed and/or refractory multiple myeloma, informed by the LEGEND-2 study results.2,9

In April 2019, cilta-cel was granted PRIME (PRIority MEdicines) designation by the European Medicines Agency (EMA).10 PRIME offers enhanced interaction and early dialogue with developers of promising medicines, to optimise drug development plans and speed up evaluation of cutting-edge, scientific advances that target a high unmet medical need.11 In February 2020, the European Commission granted orphan designation for cilta-cel.12

About Multiple Myeloma
Multiple myeloma (MM) is an incurable blood cancer that starts in the bone marrow and is characterised by an excessive proliferation of plasma cells.13 In Europe, more than 48,200 people were diagnosed with MM in 2018, and more than 30,800 patients died.14 Around 50 percent of newly diagnosed patients do not reach five-year survival,15,16 and almost 29 percent of patients with multiple myeloma will die within one year of diagnosis.17

Although treatment may result in remission, unfortunately, patients will most likely relapse as there is currently no cure.18 Refractory MM is when a patient’s disease progresses within 60 days of their last therapy.19 Relapsed cancer is when the disease has returned after a period of initial, partial or complete remission.15 While some patients with MM have no symptoms at all, others are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.20 Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and require new therapies for continued disease control.21

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 Pharmaceutica NV, Janssen Research & Development, LLC, Janssen R&D BE, and Janssen Biotech, Inc. 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 ciltacabtagene autoleucel. 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 Pharmaceutica NV and/or any of the other Janssen Pharmaceutical Companies 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 behavior 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 29, 2019, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in the company’s most recently filed Quarterly Report on Form 10-Q, 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. 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 EMA. Accelerated Assessment. Available at: https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/accelerated-assessment. Last accessed January 2021.
2 Madduri, D et al. Cartitude-1: Phase 1b/2 Study of Ciltacabtagene Autoleucel, a B-Cell Maturation Antigen–Directed Chimeric Antigen Receptor T Cell Therapy, in Relapsed/Refractory Multiple Myeloma. Oral Presentation. Presented at 2020 American Society of Hematology Annual Meeting.
3 NHS. CAR-T Therapy. Available at: https://www.england.nhs.uk/cancer/cdf/car-t-therapy/. Last accessed January 2021.
4 JnJ.com. Janssen Initiates Rolling Submission of a Biologics License Application to U.S. FDA for BCMA CAR-T Therapy Ciltacabtagene Autoleucel (cilta-cel) for the Treatment of Relapsed and/or Refractory Multiple Myeloma. Available at: https://www.jnj.com/janssen-initiates-rolling-submission-of-a-biologics-license-application-to-u-s-fda-for-bcma-car-t-therapy-ciltacabtagene-autoleucel-cilta-cel-for-the-treatment-of-relapsed-and-or-refractory-multiple-myeloma. Last accessed January 2021.
5 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 (CARTITUDE-1). Available at: https://clinicaltrials.gov/ct2/show/NCT03548207 Last accessed January 2021.
6 Hay A, Cheung M. CAR T-cells: costs, comparisons, and commentary. J Med Econ. 2019: 22(7): 613-615, DOI: 10.1080/13696998.2019.1582059
7 Cho SF, Anderson KC, Tai YT. Targeting B-cell maturation antigen (BCMA) in multiple myeloma: potential uses of BCMA-based immunotherapy. Front Immunol. 2018;9:18-21.
8 JnJ.com Janssen Enters Worldwide Collaboration and License Agreement with Chinese Company Legend Biotech to Develop Investigational CAR-T Anti-Cancer Therapy. Available at: https://www.jnj.com/media-center/press-releases/janssen-enters-worldwide-collaboration-and-license-agreement-with-chinese-company-legend-biotech-to-develop-investigational-car-t-anti-cancer-therapy Last accessed January 2021.
9 Chen L, Xu J, Fu Sr W, Jin S, Yang S, Yan S, Wu W, Liu Y, Zhang W, Weng X, Wang Y. Updated phase 1 results of a first-in-human open-label study of Lcar-B38M, a structurally differentiated chimeric antigen receptor T (CAR-T) cell therapy targeting B-cell maturation antigen (Bcma).
10 JnJ.com. Janssen Announces Investigational CAR-T Therapy JNJ-68284528 Granted PRIME Designation by the European Medicines Agency. Available at: https://www.jnj.com/janssen-announces-investigational-car-t-therapy-jnj-68284528-granted-prime-designation-by-the-european-medicines-agency Last accessed January 2021.
11 European Medicines Agency. PRIME Factsheet. Available at: https://www.ema.europa.eu/en/human-regulatory/research-development/prime-priority-medicines Last accessed January 2021.
12 European Medicines Agency (EMA). Public summary of opinion on orphan designation Available at: https://www.ema.europa.eu/en/documents/orphan-designation/eu/3/20/2252-public-summary-positive-opinion-orphan-designation-autologous-human-t-cells-genetically_en.pdf. Last accessed January 2021.
13 American Society of Clinical Oncology. Multiple myeloma: introduction. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/introduction Last accessed January 2021.
14 GLOBOCAN 2018. Cancer Today Population Factsheets: Europe Region. Available at: https://gco.iarc.fr/today/data/factsheets/populations/908-europe-fact-sheets.pdf. Last accessed January 2021.
15 American Society of Clinical Oncology. Multiple Myeloma: Statistics. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/statistics. Last accessed January 2021.
16 Cancer Research UK. Myeloma Statistics. Available at: https://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/myeloma#heading-Two Last accessed January 2021.
17 Costa LJ, Gonsalves WI, Kumar SK. Early mortality in multiple myeloma. Leukemia. 2015;29:16168.
18 Abdi J, Chen G, Chang H, et al. Drug resistance in multiple myeloma: latest findings and new concepts on molecular mechanisms. Oncotarget. 2013;4:2186–2207.
19 Richardson P, Mitsiades C, Schlossman R, et al. The treatment of relapsed and refractory multiple myeloma. Hematology Am Soc Hematol Educ Program. 2007:317-23.
20 American Cancer Society. Multiple Myeloma: Symptoms and Signs. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/symptoms-and-signs. Last accessed January 2021.
21 Kumar SK, Lee JH, Lahuerta JJ, 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;2621:149-57.

CP-205852
January 2021

Contact information

Media Enquiries:
Noah Reymond
Mobile: +31 621-385-718
Email: NReymond@ITS.JNJ.com

Investor Relations:

Christopher DelOrefice
Office: +1 732-524-2955

Jennifer McIntyre
Office: +1 732-524-3922

About Business Wire

Business Wire
Business Wire



Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Scoro Raises $16 Million in Series B Funding To Invest In Its End-to-End Work Management Software9.3.2021 11:00:00 CETPress release

Scoro, an award-winning work management software for professional service businesses, has closed a $16.4 million Series B funding round led by Kennet Partners. Additional investors include Columbia Lake Partners, Inventure, Livonia Partners and Tera Ventures. The investment will fuel continued global expansion and is supported by new French and Spanish versions of the software. “Even before Covid-19 hit, most professional service businesses used as many as a dozen different platforms daily,” said Scoro’s Founder and CEO, Fred Krieger. “However, teams now realise that constantly switching between tools harms productivity and results in lost data and duplicated work. In many ways, these tools became weapons of mass distraction. We’re proud to have the support of Kennet Partners and other investors as we help businesses identify and remove these weapons of mass distraction with our end-to-end work management software.” Scoro's most recent product launch has been a modernization of the Gan

1010data Partners with Strategix CFT to Enhance the Value of Retailer Data and Expand European Coverage9.3.2021 11:00:00 CETPress release

1010data, a provider of analytical intelligence and data sharing solutions to the retail, consumer goods and financial services markets, and Strategix CFT, a boutique consulting company that specializes in innovative retail technologies and comprehensive systems integration, announced today that they have entered into a strategic partnership to provide offerings that deliver enhanced customer insights. Targeted at Retailers and Consumer Packaged Goods (CPG) companies across the UK and Europe, the two companies will use their combined resources to develop analytics solutions that deliver the fastest predictive and prescriptive customer insights. The new partnership will provide advanced data analytics technology driving best practices for large retailers to optimize and grow their business in this fast-changing market environment. This will include a single, powerful source of data enabling collaborative planning between retailers and suppliers. A single, integrated analytics platform e

XPRIZE and Cognizant Announce Grand Prize Winners in Pandemic Response Challenge to Restart Economies Around the World9.3.2021 09:31:00 CETPress release

XPRIZE, the world’s leader in designing and operating incentive competitions to solve humanity’s grand challenges, in partnership with Cognizant(Nasdaq: CTSH), one of the world's leading technology and professional services companies, today announced the Grand Prize Winners in the $500K Pandemic Response Challenge. The four-month global competition was designed to harness the power of data and artificial intelligence in equipping policymakers, health officials and business leaders with the insights and guidance necessary to implement public safety measures that help keep local economies open while minimizing virus outbreaks as vaccines become more widely available. These decision-makers are encouraged to use the results, methodologies and technology from the challenge as reference on approaches to minimize potential outbreaks, now and moving forward. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210309005419/en/ Winning fir

The NAGA Group AG Closes USD 30 Million Financing Framework and Reports Record Growth9.3.2021 09:23:00 CETPress release

The NAGA Group AG (XETRA: N4G, ISIN: DE000A161NR7), provider of the social network for financial market trading NAGA.com, reports on the largest financing arrangement in the company's history to date and further record growth in February 2021. NAGA has signed an agreement with the US Fund Yorkville Advisors securing a growth financing framework of up to USD 30 million. "We are excited about the deal with Yorkville and are setting the course for NAGA to scale even faster. The financing gives us the power and the flexibility to allow the company to be taken to the next level. NAGA is a growth case in a very large and rapidly growing market. This decade will be definitely the breakthrough period for FinTechs", comments NAGA CEO Benjamin Bilski on the closed deal. In addition, the company reports on preliminary results for February which have once again significantly exceeded the record figures from January 2021. In February, NAGA achieved a record user growth with 37,000 new accounts and

Fibocom Announces Multiple Next-Gen 5G NR Modules to 3GPP R16 Standard at MWC Shanghai 20219.3.2021 05:15:00 CETPress release

Fibocom (Stock Code: 300638), a leading provider of cellular embedded wireless module solutions for the Internet of Things (IoT), announced the world-wide launch of the next generation 5G NR modules compliant with the 3GPP R16 standard during Mobile World Congress Shanghai 2021 (MWCS21). The new launch includes FM160 and FG160 5G module series of Sub-6GHz frequency range, as well as FM160W and FG160W series of mmWave range. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210308005973/en/ Fibocom Next-Gen 5G Modules (Photo: Fibocom) Product video on YouTube: https://www.youtube.com/watch?v=Wg0nE1WtoBM The new 5G NR module series is based on the newly-announced Qualcomm® Snapdragon™ X65 and X62 5G Modem-RF Systems and delivers enhanced mobile broadband and high-reliability, low-latency wireless services for IIoT (Industrial IoT), FWA ( Fixed Wireless Access), 4K/8K live streaming, telemedicine, private 5G networks, and other ma

Statement on International Women’s Day 2021 by Julia Simon, Chief Legal and Diversity Officer of Mary Kay Inc.8.3.2021 23:00:00 CETPress release

Below is a statement by Julia Simon, Chief Legal Officer and Chief Diversity Officer, Mary Kay Inc., on International Women’s Day 2021. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210308005888/en/ Julia Simon, Chief Legal Officer and Chief Diversity Officer (Photo: Mary Kay Inc.) Empowered women empower women. This belief serves as the foundation on which all work, internally and externally, rests at Mary Kay Inc. This mantra inspires Mary Kay’s tireless efforts to hire and promote women in leadership positions, support the Women’s Empowerment Principles established by UN Women and UN Global Compact, and develop entrepreneurship and economic opportunities to increase women’s participation and combat gender bias everywhere. If we lift one, we lift all. On International Women’s Day (IWD) 2021 that message rings especially poignant. The theme of this year’s IWD is “Women in leadership: Achieving an equal future in a COVID-19

CGTN: Improving Hong Kong's Electoral System: What's at Stake?8.3.2021 20:06:00 CETPress release

China is mulling improving the electoral system of the Hong Kong Special Administrative Region (HKSAR) – but critics say it's a plan to eliminate opposition forces in the city. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210308005793/en/ Why are improvements imperative? When explaining the draft decision on improving the electoral system of the HKSAR to lawmakers, Wang Chen, vice chairman of the National People's Congress (NPC) Standing Committee, said the move is to prevent anti-China, destabilizing forces from seizing power, citing the months-long riots in 2019. "To remedy the situation, it is important to take necessary steps to improve the electoral system and remove existing institutional deficiencies and risks to ensure the administration of Hong Kong by Hong Kong people with patriots as the main body," Wang said. Alex Fan, founding president of the International Youth Legal Exchange Federation in Hong Kong, told CG

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom