First investigational product being
recommended for Breakthrough Therapy Designation in China
Legend Biotech Corporation (NASDAQ:LEGN) announced today that
the China Center for Drug Evaluation, National Medical Products
Administration (CDE, NMPA) has recommended Breakthrough Therapy
Designation (BTD) for ciltacabtagene autoleucel (cilta-cel;
LCAR-B38M CAR-T cells), an investigational B-cell maturation
antigen (BCMA) targeted chimeric antigen receptor (CAR) T-cell
therapy being studied for the treatment of adults with relapsed or
refractory multiple myeloma (RRMM).
The BTD for cilta-cel (LCAR-B38M CAR-T cells) is based on the
ongoing Phase 2 CARTIFAN-1 study being conducted in China (MMY2002,
NCT03758417, CTR20181007), the ongoing Phase 1b/2 CARTITUDE-1 study
of cilta-cel (JNJ-4528) being conducted in the US (MMY2001,
NCT03548207) and Japan and the Phase 1, first-in-human LEGEND-2
study conducted in China (NCT03090659). Ciltacabtagene autoleucel
(cilta-cel) refers to both LCAR-B38M CAR-T cells and JNJ-4528.
LCAR-B38M CAR-T cell identifies the investigational product being
studied in China and JNJ-4528 identifies the investigational
product being studied outside of China, both of which are
representative of the same CAR-T cell therapy.
The BTD procedure is part of the recently revised Drug
Registration Regulation which went into effect on July 1, 2020. The
BTD process is designed to expedite the development and review of
therapies that are intended for treatment of serious diseases for
which there is no existing treatment and where preliminary evidence
indicates advantages of the therapy over available treatment
options.1 Cilta-cel is the first product that has been recommended
for BTD in China.2 As per the working procedure for BTD (2020
No.82) issued by NMPA on July 8, 2020, CDE had completed the review
and recommended to grant the BTD on August 4th, and BTD will be
granted after 5 working days of publicity period (August 5 to 12)
on the CDE website.
In December 2017, Legend Biotech entered into a worldwide
collaboration and license agreement with Janssen Biotech, Inc., one
of the Janssen Pharmaceutical Companies of Johnson & Johnson,
to jointly develop and commercialize cilta-cel in patients with
multiple myeloma. Cilta-cel is a structurally differentiated CAR-T
cell therapy containing a 4-1BB co-stimulatory domain and two
BCMA-targeting single domain antibodies designed to confer
avidity.
“Breakthrough designation recommendation by the China CDE of
NMPA represents an important regulatory milestone in the continued
development of cilta-cel in multiple myeloma patients in China,”
said Frank Zhang, PhD, CEO of Legend Biotech. “Legend, in
collaboration with Janssen, will continue to advance this
investigational therapy in China and globally.”
Previously, the following regulatory designations have been
granted to Janssen for cilta-cel:
- In July 2020, the Korea Ministry of Food and Drug Safety
granted orphan drug designation.3
- In June 2020, the Japan Ministry of Health, Labor and Welfare
granted orphan drug designation.4
- In February 2020, the European Commission granted orphan
designation.5
- In December 2019, the U.S. Food and Drug Administration (FDA)
granted Breakthrough Therapy Designation.6
- In April 2019, the European Medicines Agency granted Janssen a
PRIME (PRIority MEdicines) designation.7
- In February 2019, the FDA granted Janssen orphan drug
designation for the treatment of multiple myeloma.8
About the Clinical Development Program
CARTIFAN-1 The Phase 2 CARTIFAN-1 confirmatory trial
(MMY2002, NCT03758417, CTR20181007) is being conducted in China to
further evaluate cilta-cel (LCAR-B38M CAR-T cells) in patients with
RRMM who have received at least 3 prior lines of therapy and have
received a proteasome inhibitor (PI) and an immunomodulatory drug
(IMiD®); and documented disease progression within 12 months of
starting the most recent therapy. 9
CARTITUDE-1 Cilta-cel (JNJ-4528) is currently being
investigated in the Phase 1b/2 CARTITUDE-1 (MMY2001, NCT03548207)
pivotal study conducted in US and Japan for the treatment of
patients with multiple myeloma who have received at least 3 prior
lines of therapy or are double refractory to a PI and IMiD®;
received a PI, an IMiD and anti-CD38 antibody; and documented
disease progression within 12 months of starting the most recent
therapy. 10
CARTITUDE-2 The global, multi-cohort Phase 2 CARTITUDE-2
(MMY2003, NCT04133636) study, cilta-cel (JNJ-4528) is actively
recruiting patients with multiple myeloma in various clinical
settings. This study is being conducted to evaluate the overall
minimal residual disease (MRD) negative rate of participants who
receive JNJ-4528 to further explore efficacy and safety in earlier
patient populations.11
CARTITUDE-4 The global, Phase 3 CARTITUDE-4 (MMY3002,
NCT04181827) study, cilta-cel (JNJ-4528) is actively recruiting
patients with multiple myeloma who have received 1-3 prior lines of
therapy including a PI and IMiD and are refractory to lenalidomide.
The study is being conducted to evaluate the efficacy of JNJ-4528
compared to standard therapies7 including daratumumab, pomalidomide
and low-dose dexamethasone (DPd) or pomalidomide, bortezomib and
low-dose dexamethasone (PVd).12
LEGEND-2 LEGEND-2 (NCT03090659) is an ongoing, Phase 1,
single-arm, open-label, first-in-human, study of 74 patients being
conducted at four participating hospitals in China evaluating the
efficacy and safety of LCAR-B38M CAR-T cells for the treatment of
patients with relapsed or refractory multiple myeloma.13
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.14
Although treatment may result in remission, unfortunately,
patients will most likely relapse as there is currently no cure.15
Refractory multiple myeloma is when a patient’s disease is
non-responsive or progresses within 60 days of their last
therapy.16,17 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.18 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.19
Patients who relapse after treatment with standard therapies,
including protease inhibitors and immunomodulatory agents, have
poor prognoses and few treatment options available.20
About Legend Biotech Legend Biotech is a global
clinical-stage biopharmaceutical company engaged in the discovery
and development of novel cell therapies for oncology and other
indications. Our team of over 700 employees across the United
States, China and Europe, along with our differentiated technology,
global development and manufacturing strategies and expertise,
provides us with the strong potential to discover, develop and
commercialize best-in-class cell therapies for patients in
need.
We are engaged in a strategic collaboration with Janssen Biotech
to develop and commercialize our lead product candidate, cilta-cel,
an investigational BCMA-targeted CAR-T cell therapy for patients
living with multiple myeloma. This candidate is currently being
studied in registrational clinical trials.
To learn more about Legend Biotech, visit us on LinkedIn, or on
Twitter @LegendBiotech or at www.legendbiotech.com.
Cautions Concerning Forward-Looking Statements This press
release contains “forward-looking statements” within the meaning of
The Private Securities Litigation Reform Act of 1995 relating to
the business of Legend Biotech Corporation, including express or
implied discussions regarding product development, the potential
benefits and treatment impact of LCAR-B38M CAR-T cells/JNJ-4528,
and the regulatory approval process for these product candidates.
Such forward-looking statements reflect the current views of
Legend’s management regarding future events, and involve known and
unknown risks, uncertainties and other factors that may cause
actual results to be materially different from any future results,
performance or achievements expressed or implied by such
statements. Actual results may differ materially from those
indicated by such forward-looking statements as a result of various
important factors, including, among other things, uncertainties
involved in the development of new pharmaceutical products;
unexpected clinical trial results, including additional analysis of
existing clinical data or unexpected new clinical data; unexpected
regulatory actions or delays or government regulation generally;
Legend’s ability to obtain or maintain 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, and the other factors discussed in the “Risk
Factors” section of Legend Biotech’s prospectus filed with the
Securities and Exchange Commission on June 8, 2020. Any
forward-looking statements contained in this press release speak
only as of the date hereof, and Legend Biotech specifically
disclaims any obligation to update any forward-looking statement,
whether as a result of new information, future events or
otherwise.
References
- China Drug Registration Regulation:
http://www.gov.cn/gongbao/content/2020/content_5512563.htm
- Proposed Breakthrough Therapy Type Announcement:
http://www.cde.org.cn/news.do?method=changePage&pageName=service&frameStr=26.
Accessed August 2020
- Korea Ministry of Food and Drug Safety.
https://www.mfds.go.kr/brd/m_76/view.do?seq=14749. Accessed August
2020.
- Japan Ministry of Health, Labor and Welfare.
https://www.mhlw.go.jp/hourei/doc/tsuchi/T200629I0030.pdf. Accessed
August 2020.
- European Medicines Agency. 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.
Accessed August 2020.
- Legendbiotech.com. U.S. Food and Drug Administration Grants
Breakthrough Therapy Designation for JNJ-68284528, an
Investigational BCMA CAR-T Cell Therapy. Available at:
https://www.legendbiotech.com/pdf/JNJ4528_Breakthrough_Therapy_Designation_Release.pdf.
Accessed August 2020.
- Legendbiotech.com. European Medicines Agency Grants Janssen
PRIME Designation for JNJ-68284528 (LCAR-B38M), an Investigational
BCMA CAR-T Therapy Discovered by Legend Biotech. Available at:
https://www.legendbiotech.com/pdf/Legend_LCAR_PRIME_Designation_Press%20Release_4-1-2019.pdf.
Accessed August 2020.
- Accessdata.fda.gov. U.S. Food & Drug Administration.
Available at:
https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=20186721.
Accessed August 2020.
- CARTIFAN-1 (NCT03758417). Available:
https://clinicaltrials.gov/ct2/show/NCT03758417. Accessed August
2020.
- CARTITUDE-1 (NCT03548207). Available:
https://clinicaltrials.gov/ct2/show/NCT03548207. Accessed August
2020.
- CARTITUDE-2 (NCT04133636). Available:
https://clinicaltrials.gov/ct2/show/NCT04133636. Accessed August
2020.
- CARTITUDE-4 (NCT041818270. Available:
https://clinicaltrials.gov/ct2/show/NCT04181827. Accessed August
2020.
- LEGEND-2 (NCT03090659). Available:
https://clinicaltrials.gov/ct2/show/NCT03090659. Accessed August
2020.
- American Society of Clinical Oncology. Multiple myeloma:
introduction. Available at:
https://www.cancer.net/cancer-types/multiple-myeloma/introduction.
Accessed August 2020
- 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.
- National Cancer Institute. NCI dictionary of cancer terms:
refractory. Available:
https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=350245.
Accessed August 2020.
- 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.
- National Cancer Institute. NCI dictionary of cancer terms:
relapsed. Available at:
https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=45866.
Accessed August 2020.
- 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 August 2020.
- 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;26:149-57.
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version on businesswire.com: https://www.businesswire.com/news/home/20200805006079/en/
For Medical Affairs inquiries: Tonia Nesheiwat, Executive
Director, Medical Affairs, Legend Biotech
tonia.nesheiwat@legendbiotech.com or
medicalinformation@legendbiotech.com
For Media and Investor Relations inquiries: Jessie Yeung,
Head of Corporate Finance and Investor Relations, Legend Biotech
jessie.yeung@legendbiotech.com or investor@legendbiotech.com
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