Business Wire

CARVYKTI ® (ciltacabtagene autoleucel) Granted Conditional Approval by the European Commission for the Treatment of Patients with Relapsed and Refractory Multiple Myeloma

Share

Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global biotechnology company developing, manufacturing and commercializing novel therapies to treat life-threatening diseases, today announced that the European Commission (EC) has granted conditional marketing authorization of CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) for the treatment of adults with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD) and an anti-CD38 antibody, and have demonstrated disease progression on the last therapy. Legend Biotech entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize cilta-cel in December 2017.

CARVYKTI® is a chimeric antigen receptor T-cell (CAR-T) therapy featuring two B-cell maturation antigen (BCMA)-targeting single domain antibodies.1 CAR-T therapy is specifically developed for each individual patient, and it is administered as a single infusion.1,2

“The approval of CARVYKTI® by the European Commission marks Legend’s first approval in the region and is a significant milestone as we advance our commitment to bring innovative cell therapies to patients with the highest unmet need,” said Ying Huang, Ph.D., Chief Executive Officer of Legend Biotech. “The CARTITUDE-1 data showed that CARVYKTI® provides the potential for long, treatment-free intervals for heavily pre-treated patients, and is a valuable treatment option for patients with multiple myeloma. We look forward to working with Janssen to bring this new option to patients across Europe.”

This approval was supported by the pivotal CARTITUDE-1 study, including patients who had received a median of six prior treatment regimens (range, 3-18), and had previously received an IMiD, PI and anti-CD38 monoclonal antibody.3 Findings showed that at a median duration of 18 months follow-up (range, 1.5-30.5), a one-time treatment with cilta-cel resulted in deep and durable responses, with 98 percent (95 percent confidence interval [CI], 92.7-99.7) of patients with RRMM responding to therapy (98 percent overall response rate [ORR] (N=97)).3,4 Notably, 80 percent of the patients achieved stringent complete response (sCR), a measure in which a physician is unable to observe any signs or symptoms of disease via imaging or other tests after treatment.3

The safety of cilta-cel was evaluated in 179 adult patients across two open-label clinical trials (MMY2001 and MMY2003). The most common adverse reactions (≥20 percent) were neutropenia (91 percent), cytokine release syndrome (CRS) (88 percent), pyrexia (88 percent), thrombocytopenia (73 percent), anemia (72 percent), leukopenia (54 percent), lymphopenia (45 percent), musculoskeletal pain (43 percent), hypotension (41 percent), fatigue (40 percent), transaminase elevation (37 percent), upper respiratory tract infection (32 percent), diarrhea (28 percent), hypocalcemia (27 percent), hypophosphatemia (26 percent), nausea (26 percent), headache (25 percent), cough (25 percent), tachycardia (23 percent), chills (23 percent), encephalopathy (22 percent), decreased appetite (22 percent), oedema (22 percent), and hypokalemia (20 percent).4

“There continues to be an unmet need for patients with relapsed or refractory multiple myeloma who have exhausted existing treatment options. Data from the CARTITUDE-1 trial have shown that cilta-cel provides deep and durable responses for heavily pre-treated patients. Today’s approval by the European Commission reinforces the potential of this new treatment option for multiple myeloma patients in Europe,” said Hermann Einsele, Full Professor of Internal Medicine and Director of the Medizinische Klinik und Poliklinik II of the Julius Maximilian University, Würzburg, Germany.*

As a highly personalized medicine, where a patient’s own T-cells are reprogrammed to target and kill cancer cells, administration of CAR-T therapyrequires extensive training, preparation, and certification to ensure the highest quality product and experience for patients.2 Through a phased approach, Legend Biotech’s strategic partner, Janssen, will work diligently to activate a limited network of certified treatment centers and will aim to increase availability of cilta-cel across Europe, in an effort to provide oncologists and patients with treatment in a reliable manner.

This EC Marketing Authorization follows the approval of CARVYKTI® by the U.S. Food and Drug Administration (FDA) on February 28, 2022.

*Hermann Einsele, M.D., FRCP has not been paid for contributing to this press release.

About CARVYKTI® (ciltacabtagene autoleucel; cilta-cel)

CARVYKTI® is a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient’s own T-cells with a transgene encoding a chimeric antigen receptor (CAR) that identifies and eliminates cells that express BCMA.4 BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells.4 The CARVYKTI® CAR protein features two BCMA-targeting single domain antibodies designed to confer high avidity against human BCMA.4 Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.4

In December 2017, Legend Biotech Corporation entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize cilta-cel.5

In April 2021, Legend announced the submission of a Marketing Authorisation Application to the European Medicines Agency seeking approval of cilta-cel for the treatment of patients with relapsed or refractory multiple myeloma. In addition to U.S. Breakthrough Therapy Designation granted in December 2019, cilta-cel also received Breakthrough Therapy Designation in China in August 2020. In February 2019, cilta-cel received Orphan Drug Designation from the U.S. FDA from the European Commission in February 2020, and from the Pharmaceuticals and Medicinal Devices Agency (PMDA) in Japan in June 2020. In May 2022, the Committee for Orphan Medicinal Products recommended by consensus that the orphan designation for cilta-cel be maintained, on the basis of clinical data demonstrating improved and sustained complete response rates following treatment.6 Cilta-cel was approved the U.S. Food and Drug Administration (FDA) in February 2022.5

About CARTITUDE-1

CARTITUDE-1 (NCT03548207)7 is an ongoing Phase 1b/2, open-label, single arm, multi-center trial evaluating cilta-cel for the treatment of adult patients with relapsed or refractory multiple myeloma, who previously received at least three prior lines of therapy including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD) and an anti-CD38 monoclonal antibody. Of the 97 patients enrolled in the trial, 99 percent were refractory to the last line of treatment and 88 percent were triple-class refractory, meaning their cancer did not respond, or no longer responds, to an IMiD, a PI and an anti-CD38 monoclonal antibody.4

About Multiple Myeloma

Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells.8 In Europe, it is estimated that more than 50,900 people were diagnosed with multiple myeloma in 2020, and approximately 32,500 patients died.9 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.10 Although treatment may result in remission, unfortunately, patients will most likely relapse.11 Patients who relapse after treatment with standard therapies, including protease inhibitors, immunomodulatory agents, and an anti-CD38 monoclonal antibody, have poor prognoses and few treatment options available.12,13

About Legend Biotech

Legend Biotech is a global biotechnology company dedicated to treating, and one day curing, life-threatening diseases. Headquartered in Somerset, New Jersey, we are developing advanced cell therapies across a diverse array of technology platforms, including autologous and allogenic chimeric antigen receptor T-cell, T-cell receptor (TCR-T), and natural killer (NK) cell-based immunotherapy. From our three R&D sites around the world, we apply these innovative technologies to pursue the discovery of safe, efficacious and cutting-edge therapeutics for patients worldwide.

Learn more at www.legendbiotech.com and follow us on Twitter and LinkedIn.

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to Legend Biotech’s strategies and objectives; statements relating to CARVYKTI™, including Legend Biotech’s expectations for CARVYKTI™, such as Legend Biotech’s manufacturing and commercialization expectations for CARVYKTI™ and the potential effect of treatment with CARVYKTI™; statements about submissions for cilta-cel to, and the progress of such submissions with, the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), the Chinese Center for Drug Evaluation of National Medical Products Administration (CDE) and other regulatory authorities; the anticipated timing of, and ability to progress, clinical trials, including patient enrollment; the submission of Investigational New Drug (IND) applications to, and maintenance of such applications with, regulatory authorities; the ability to generate, analyze and present data from clinical trials; and the potential benefits of Legend Biotech’s product candidates. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors. Legend Biotech’s expectations could be affected by, among other things, uncertainties involved in the development of new pharmaceutical products; unexpected clinical trial results, including as a result of additional analysis of existing clinical data or unexpected new clinical data; unexpected regulatory actions or delays, including requests for additional safety and/or efficacy data or analysis of data, or government regulation generally; unexpected delays as a result of actions undertaken, or failures to act, by our third party partners; uncertainties arising from challenges to Legend Biotech’s patent or other proprietary intellectual property protection, including the uncertainties involved in the U.S. litigation process; competition in general; government, industry, and general public pricing and other political pressures; the duration and severity of the COVID-19 pandemic and governmental and regulatory measures implemented in response to the evolving situation; as well as the other factors discussed in the “Risk Factors” section of the Legend Biotech’s Annual Report filed with the Securities and Exchange Commission on March 31, 2022. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those described in this press release as anticipated, believed, estimated or expected.​ Any forward-looking statements contained in this press release speak only as of the date of this press release. Legend Biotech specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

References
_______________________________________

1 Martin, T. Updated Results From CARTITUDE-1: Phase 1b/2 Study of Ciltacabtagene Autoleucel, a B-cell Maturation Antigen–Directed Chimeric Antigen Receptor T Cell Therapy, in Patients with Relapsed/Refractory Multiple Myeloma. Abstract #549 [Oral]. Presented at the 2021 American Society of Hematology (ASH) Annual Meeting & Exposition Annual Meeting.

2 NHS. CAR-T Therapy. https://www.england.nhs.uk/cancer/cdf/car-t-therapy/. Accessed May 2022.

3 Usmani, S. Ciltacabtagene autoleucel, a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor T-cell (CAR-T) therapy, in relapsed/refractory multiple myeloma (R/R MM): Updated results from CARTITUDE-1. Abstract #8005 [Oral]. Presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.

4 European Medicines Agency. CARVYKTI (Ciltacabtagene Autoleucel) Summary of Product Characteristics. May 2022.

5 CARVYKTI™ (ciltacabtagene autoleucel), BCMA-Directed CAR-T Therapy, Receives U.S. FDA Approval for the Treatment of Adult Patients with Relapsed or Refractory Multiple Myeloma. Available at: https://legendbiotech.com/wp-content/uploads/2022/02/CARVYKTI%E2%84%A2-ciltacabtagene-autoleucel-BCMA-Directed-CAR-T-Therapy-Receives-U.S.-FDA-Approval-2.pdf. Accessed May 2022.

6 European Commission. Community Register of Orphan Medicinal Products. Available at: https://ec.europa.eu/health/documents/community-register/html/o2252.htm. Accessed May 2022.

7 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. Accessed May 2022.

8 American Society of Clinical Oncology. Multiple myeloma: introduction. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/introduction. Accessed May 2022.

9 GLOBOCAN 2020. Cancer Today Population Factsheets: Europe Region. Available at: https://gco.iarc.fr/today/data/factsheets/populations/908-europe-fact-sheets.pdf. Accessed May 2022.

10 American Cancer Society. Multiple myeloma: early detection, diagnosis and staging. Available at: https://www.cancer.org/content/dam/CRC/PDF/Public/8740.00.pdf. Accessed May 2022.

11 Rajkumar SV. Multiple myeloma: 2020 update on diagnosis, risk-stratification and management. Am J Hematol. 2020;95(5),548-567. doi:10.1002/ajh.25791.

12 Kumar SK, Dimopoulos MA, Kastritis E, et al. Natural history of relapsed myeloma, refractory to immunomodulatory drugs and proteasome inhibitors: a multicenter IMWG study. Leukemia. 2017;31(11):2443- 2448.

13 Gandhi UH, Cornell RF, Lakshman A, et al. Outcomes of patients with multiple myeloma refractory to CD38- targeted monoclonal antibody therapy. Leukemia. 2019;33(9):2266-2275.

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Investor Contacts:
Joanne Choi, Senior Manager of Investor Relations, Legend Biotech
joanne.choi@legendbiotech.com

Crystal Chen, Manager of Investor Relations, Legend Biotech
crystal.chen@legendbiotech.com

Press Contact:
Tina Carter, Corporate Communications Lead, Legend Biotech
tina.carter@legendbiotech.com
(908) 331-5025

About Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

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

Napster Outlines New Web3 Music Ecosystem29.6.2022 18:15:00 CEST | Press release

Napster Music Inc., the music streaming service, today announced the publication of its Litepaper V1 which outlines its plans to apply Web3 technology to its existing business and millions of users in order to improve how music makers, rights holders and fans can interact. A new entity, Napster Innovation Foundation, will be issuing $NAPSTER tokens using the Algorand blockchain protocol to streamline its existing platform functionality as well as open up new options for creating value around streaming music. "We’re entering the streaming-plus era of music, which is one of the few scaled business sectors where adoption of blockchain tech actually makes immediate sense for everyone,” said Emmy Lovell, Interim CEO of Napster. “People who make music, people who listen to it and those who own its IP all already depend heavily on technology. Web3 offers a chance to deepen, extend and improve the music ecosystem.” “Napster is a strong, self-sustaining business and an iconic brand,” said Matt

izzi Taps Verimatrix XTD and Streamkeeper to Protect Its Popular Media Apps, Endpoint29.6.2022 17:45:00 CEST | Press release

Regulatory News: Verimatrix, (Euronext Paris: VMX) (Paris:VMX), the leader in powering the modern connected world with people-centered security, today announced that izzi , a mexican telecommunications firm that offers the country’s popular izzi tv and izzi go services, is set to build upon its existing use of Verimatrix content protection and anti-piracy solutions with the addition of Streamkeeper as well as Extended Threat Defense (XTD) technologies to protect many of their fast-growing iOS, Android and web streaming services plus its Android set-top box services. With an ever-increasing customer base with millions of users throughout Mexico and a vast array of premium entertainment delivery platforms, izzi decided to expand its use of Verimatrix following success with its current VCAS and Multi-DRM deployments. Verimatrix XTD will soon be used to protect code within all of the company’s PayTV apps throughout all izzi platforms, while the company will also benefit from comprehensive

EIG Launches Chilean Renewables Platform, Grupo Cerro29.6.2022 17:00:00 CEST | Press release

EIG, an institutional investor to the global energy sector and one of the world’s leading infrastructure investors, today launched Grupo Cerro, a new renewable energy platform in Chile. The newly formed entity includes EIG-owned Cerro Dominador plants and the recently acquired ANPAC, the owner of a portfolio of 11 small and medium run-of-river hydroelectric power plants in the O’Higgins, El Maule, Bio Bio and La Araucania regions. The 110 MW ANPAC portfolio includes operating plants and projects at various stages of construction and development in both the spot and stabilized-tariff markets. Following the acquisition, Grupo Cerro now manages over 280 MW of installed capacity in Chile, including the Cerro Dominador Photovoltaic (PV) and Concentrated Solar Power (CSP) plants. The group’s portfolio also includes several projects in advanced phases of construction and development, including the Likana Solar complex, one of the largest CSP projects in the world with a capacity of 690 MW, Pa

Trackforce Valiant Acquires TrackTik Software, Creates the World’s Largest Security Workforce Management Company29.6.2022 16:00:00 CEST | Press release

Trackforce Valiant, a leading provider of security workforce management solutions, today announced the acquisition of TrackTik Software, one of the most innovative and fastest growing cloud-based security workforce management providers. The combination of these two companies has created a new dominant market leader in providing visibility and control for the physical security workforce. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220629005064/en/ “We are very excited to welcome TrackTik into the Trackforce Valiant family,” said Trevor Campion, CEO of Trackforce Valiant. “Now more than ever, we believe in the importance of providing the right security solutions for companies of all sizes and business needs. The addition of TrackTik enables us to provide a wide range of technology options that are purpose-built for the unique needs of physical security operations.” TrackTik was founded in 2013 with the mission of providing

Hy Stor Energy Strategic Partnership with Key Gulf Coast Port Becomes First to Deliver Renewable Hydrogen Access for Manufacturing, Industrial Applications, Port Operations and Long Duration Energy Storage29.6.2022 15:00:00 CEST | Press release

Hy Stor Energy LP (Hy Stor Energy), a company pioneering renewably produced green hydrogen and energy storage at scale in Mississippi, announced today a strategic partnership with the Hancock County Port and Harbor Commission (HCPHC) to provide zero-carbon, zero-methane hydrogen to Port Bienville Industrial Park and Stennis International Airport, accelerating the decarbonization of land, air, sea, and space. Through this partnership, Port Bienville will be the first port in the Gulf Region to integrate renewable hydrogen as a fuel into its operations. During the first phase of the partnership, the hydrogen hub is expected to produce an estimated 350 tons/day (320,000 kg/day) of renewable hydrogen and store more than 71,000 tons (69 million kg) of hydrogen in underground salt caverns. The existing Port Bienville infrastructure will expand to better enable decarbonization efforts and will be in a premium location to allow for new manufacturing of circular hydrogen infrastructure – the fi