Combined Phase 1b/2 CARTITUDE-1 study presented
at ASH 2020 Annual Meeting show 97 percent overall response rate
and 12-month progression free survival rate of 77 percent at median
follow up of 12.4 months
Legend Biotech Corporation (NASDAQ: LEGN) (“Legend Biotech”), a
global clinical-stage biopharmaceutical company engaged in the
discovery and development of novel cell therapies for oncology and
other indications, today announced the latest data results from the
combined Phase 1b/2 CARTITUDE-1 study (NCT03548207) of
ciltacabtagene autoleucel (cilta-cel), an investigational B-cell
maturation antigen (BCMA) directed chimeric antigen receptor T cell
(CAR-T) therapy, for the treatment of patients with relapsed or
refractory multiple myeloma (RRMM), sponsored by Janssen Research
& Development, LLC. The data continued to show a very high
overall response rate (ORR) that deepened over time, with 97
percent of patients achieving a response and 67 percent of patients
achieving a stringent complete response (sCR) at a median follow-up
of 12.4 months.1 The data were presented at the 62nd American
Society of Hematology (ASH) Annual Meeting and Exposition (Abstract
#177) as an oral presentation.
“A 97 percent response rate is extraordinary when you consider
the patient population who, prior to cilta-cel, have generally
experienced low response rates,” said Deepu Madduri, M.D.,
Assistant Professor of Medicine, Hematology and Medical Oncology,
Tisch Cancer Institute at Mount Sinai, New York, and principal
CARTITUDE-1 study investigator. “Considering the early, deep and
durable responses that we’ve seen at low-dose infusion of cilta-cel
and its manageable safety profile, we look forward to further
studying outpatient administration of cilta-cel in patients with
multiple myeloma in earlier settings.”
The trial included 97 patients treated with cilta-cel who
received a median of six (range, 3-18) prior lines of therapy; 88
percent (n=85) were triple-refractory, 42 percent (n=41) were
penta-refractory and 99 percent (n=96) were refractory to the last
line of therapy.1 The median administered dose was 0.71x106 CAR+
viable T cells/kg and manufacturing of cilta-cel was successful for
all patients. ORR per independent review was 97 percent, which
included a sCR rate of 67 percent, very good partial response rate
(VGPR) of 26 percent (VGPR or better, 93 percent) and partial
response rate of 4 percent. Median time to first response was 1
month (range, 0.9-8.5) and responses were ongoing in 72 percent
(n=70) of patients. Of 57 minimal residual disease (MRD) evaluable
patients, 93 percent (n=53) were MRD negative at 10-5.1 Median
progression-free survival (PFS) was not reached at median follow-up
of 12.4 months (range, 1.5-24.9). The 12-month PFS rate was 77
percent (95 percent confidence interval [CI], 66-84) and the
12-month OS rate was 89 percent (95 percent CI, 80-94).1
The study also demonstrated a manageable safety profile for
cilta-cel at the recommended Phase 2 dose.1 In the combined
results, the most common hematologic adverse events (AEs) observed
in the CARTITUDE-1 study were neutropenia (96 percent); anemia (81
percent); thrombocytopenia (79 percent); leukopenia (62 percent);
and lymphopenia (53 percent).1 Cytokine release syndrome (CRS) of
any grade was observed in 95 percent of patients, with a median
duration of four days (range, 1-97), and 99 percent of which
resolved within 14 days of onset. Of the 92 patients with CRS, most
were Grade 1/2 (95 percent, n=87), 3 percent were Grade 3 (n=3), 1
percent was Grade 4 (n=1) and 1 percent was Grade 5 (n=1).1 The
median onset of CRS was seven days (range, 1-12) post-infusion,
with 89 percent (n=82) of patients experiencing CRS onset at day
four or later, which is supportive of potential outpatient
administration for cilta-cel. Total CAR-T cell neurotoxicity of any
grade was observed in 21 percent (n=20) of patients, with Grade ≥3
neurotoxicity observed in 10 percent (n=10) of patients.1 Of these,
Immune effector Cell-Associated Neurotoxicity Syndrome (ICANS) was
observed in 16 patients and generally occurred concurrently with
CRS; other neurotoxicities were observed in 12 patients and
generally occurred after resolution of CRS and/or ICANS (eight
patients experienced both ICANS and other neurotoxicities).1 ICANS
events were resolved in all patients with a median time to recovery
of four days (range, 1-12).1 Other neurotoxicities were resolved in
six patients at a median time of 75 days (range, 2-160) and were
not resolved in six patients (one with ongoing toxicity, one died
from neurotoxicity and four died due to other causes).1 Fourteen
deaths were reported during the study: five due to disease
progression, three due to adverse events unrelated to treatment
(acute myelogenous leukemia [n=2], pneumonia [n=1]) and six due to
adverse events related to treatment (sepsis and/or septic shock
[n=2], CRS/HLH [n=1], neurotoxicity [n=1], respiratory failure
[n=1], and lung abscess [n=1]).1
“We are encouraged by the see strong results from the
CARTITUDE-1 study showing the potential of our lead product
candidate cilta-cel to be a transformative treatment option for
patients living with relapsed or refractory multiple myeloma,” said
Dr. Ying Huang, Chief Executive Officer and Chief Financial Officer
of Legend Biotech. “We look forward to advancing this potentially
life-saving treatment approach for patients in need.”
About CARTITUDE-1
CARTITUDE-1 (NCT03548207) is an ongoing Phase 1b/2, open-label,
multicenter study evaluating the safety and efficacy of
ciltacabtagene autoleucel in adults with relapsed or refractory
multiple myeloma, 99 percent of whom were refractory to the last
line of treatment; 88 percent of whom were triple-class
refractory(to at least 1 immunomodulatory drug [IMiD], 1 proteasome
inhibitor [PI] and 1 anti-CD38 antibody).
The primary objective of the Phase 1b portion of the study,
involving 29 patients, was to characterize the safety and confirm
the dose of ciltacabtagene autoleucel, informed by the
first-in-human study with LCAR-B38M CAR-T cells (LEGEND-2). The
Phase 2 portion of the study, involving 68 additional patients, is
evaluating the efficacy of ciltacabtagene autoleucel with overall
response rate as the primary endpoint.
About Cilta-cel
Cilta-cel is an investigational chimeric antigen receptor T
(CAR-T) cell therapy, formerly identified as JNJ-4528 outside of
China and LCAR-B38M CAR-T cells in China, that is being studied in
a comprehensive clinical development program for the treatment of
patients with relapsed or refractory multiple myeloma and in
earlier lines of treatment. The design consists of a structurally
differentiated CAR-T with two BCMA-targeting single domain
antibodies. In December 2017, Legend Biotech, Inc. entered into an
exclusive worldwide license and collaboration agreement with
Janssen Biotech, Inc. (Janssen) to develop and commercialize
cilta-cel. In addition to a Breakthrough Therapy Designation (BTD)
granted in the U.S. in December 2019, cilta-cel received a PRIority
MEdicines (PRiME) designation from the European Commission in April
2019 and BTD in China in August 2020. In addition, Orphan Drug
Designation was granted for cilta-cel by the U.S. FDA in February
2019, and by the European Commission in February 2020.
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.2 Although treatment may result in remission,
unfortunately, patients will most likely relapse. 3 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.4 Refractory multiple myeloma is when a patient’s
disease is non-responsive or progresses within 60 days of their
last therapy.5,6 While some patients with multiple myeloma have no
symptoms until later stages of the disease, most patients are
diagnosed due to symptoms that can include bone problems, low blood
counts, calcium elevation, kidney problems or infections.7 Patients
who relapse after treatment with standard therapies, including
protease inhibitors and immunomodulatory agents, have poor
prognoses and few treatment options.8
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 800
employees across the United States, China and Europe, along with
our differentiated technology, global development, and
manufacturing strategies and expertise, provide us with the strong
potential to discover, develop, and manufacture cutting-edge cell
therapies for patients in need. We are engaged in a strategic
collaboration to develop and commercialize our lead product
candidate, ciltacabtagene autoleucel, an investigational BCMA
targeted CAR-T cell therapy for patients 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
Statements in this press release about future expectations,
plans and prospects, as well as any other statements regarding
matters that are not historical facts, may 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
clinical efforts, its collaboration to develop and commercialize
cilta-cel, and the data relating to the CARTITUDE-1 study. 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, including the factors discussed in the “Risk
Factors” section of the 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. Readers should not rely
upon the information on this page as current or accurate after its
publication date.
The safety and efficacy of the product candidates and/or uses
under investigation have not been established. There is no
guarantee that the product candidates will receive health authority
approval or become commercially available in any country for the
uses being investigated.
________________________________ 1 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. Abstract #177.
Oral Presentation at 2020 American Society of Hematology Annual
Meeting. 2 American Society of Clinical Oncology. Multiple myeloma:
introduction. Available at:
https://www.cancer.net/cancer-types/multiple-myeloma/introduction.
Accessed November 2020. 3 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. 4 National
Cancer Institute. NCI dictionary of cancer terms: relapsed.
Available at:
https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=45866.
Accessed November 2020. 5 National Cancer Institute. NCI dictionary
of cancer terms: refractory. Available at:
https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=350245.
Accessed November 2020. 6 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. 7 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 November 2020. 8 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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For Media and Investor Relations inquiries, please
contact: Jessie Yeung, Head of Corporate Finance and Investor
Relations, Legend Biotech jessie.yeung@legendbiotech.com or
investor@legendbiotech.com Surabhi Verma, Manager of Investor
Relations and Corporate Communications, Legend Biotech
Surabhi.verma@legendbiotech.com or media@legendbiotech.com For
Medical Affairs inquiries, please contact: Tonia Nesheiwat,
Executive Director, Medical Affairs, Legend Biotech
tonia.nesheiwat@legendbiotech.com or
medicalinformation@legendbiotech.com
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