LianBio (Nasdaq: LIAN), a biotechnology company dedicated to
bringing innovative medicines to patients in China and other major
Asian markets, today announced that China’s National Medical
Products Administration (NMPA) has accepted with priority review
the New Drug Application (NDA) for mavacamten for the treatment of
symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in
adults.
“We are one step closer to bringing mavacamten to patients with
obstructive HCM in China as a first-in-class, clinically validated
treatment option,” said Yizhe Wang, Ph.D., Chief Executive Officer
of LianBio. “Gaining alignment with the NMPA to file the NDA prior
to the completion of our ongoing Phase 3 trial in China speaks to
the strength of the clinical data that has been generated and to
our commitment to accelerate patient access to critical new
treatments. This is a pivotal moment for LianBio as we prepare to
transform into a commercial-stage company, and we believe is a
great testament to our ability to navigate evolving regulatory and
clinical development pathways in China.”
The NDA is based on data from the global pivotal Phase 3
EXPLORER-HCM trial, which evaluated the safety and efficacy of
mavacamten in patients with symptomatic oHCM compared to placebo.
Results from the EXPLORER-HCM trial showed that mavacamten
demonstrated robust treatment effect, with clinically meaningful
improvements in exercise capacity, functional status, and
patient-reported outcomes, as well as the ability to relieve left
ventricular outflow tract obstruction. The EXPLORER-HCM trial met
all primary and secondary endpoints with statistical significance.
More information about the EXPLORER-HCM trial can be found on
ClinicalTrials.gov (NCT03470545).
The NDA also includes clinical data from LianBio’s Phase 1
pharmacokinetic study of mavacamten in healthy Chinese volunteers.
This study demonstrated a safety and tolerability profile, and a
pharmacokinetic profile comparable to those observed in the Phase 1
pharmacokinetic studies of mavacamten conducted in the United
States. Blinded preliminary safety data from LianBio’s ongoing
Phase 3 EXPLORER-CN trial of mavacamten in Chinese patients with
symptomatic oHCM also supported the early NDA filing. Topline data
from this study are expected mid-2023. More information about the
pharmacokinetic study of mavacamten and the EXPLORER-CN trial can
be found on ClinicalTrials.gov (NCT05135871 and NCT05174416,
respectively).
“HCM significantly impacts patients’ quality of life. Currently,
there is a lack of effective treatment options and innovative drugs
are urgently needed for clinical use,” said Shuyang Zhang, M.D.,
Ph.D., Professor of Cardiology and President, Peking Union Medical
College Hospital, and Principal Investigator, EXPLORER-CN. “As the
first therapy that targets the pathophysiology of disease,
mavacamten holds the potential to improve treatment of oHCM in
China and positively impact patients’ health and ability to perform
tasks of daily living.”
About Camzyos
(mavacamten)Camzyos™ (mavacamten) is the first and
only cardiac myosin inhibitor approved by the U.S. FDA indicated
for the treatment of adults with symptomatic New York Heart
Association (NYHA) class II-III obstructive HCM to improve
functional capacity and symptoms. It has also received regulatory
approvals in Australia, Canada and Brazil. Camzyos is an allosteric
and reversible inhibitor selective for cardiac myosin. Camzyos
modulates the number of myosin heads that can enter “on actin”
(power generating) states, thus reducing the probability of force
producing (systolic) and residual (diastolic) cross-bridge
formation. Excess myosin actin cross bridge formation and
dysregulation of the super relaxed state are mechanistic hallmarks
of HCM. Camzyos shifts the overall myosin population towards an
energy sparing, recruitable, super relaxed state. In HCM patients,
myosin inhibition with Camzyos reduces dynamic left ventricular
outflow tract (LVOT) obstruction and improves cardiac filling
pressures.
LianBio licensed rights from MyoKardia, now a wholly owned
subsidiary of Bristol Myers Squibb, in August 2020 for the
development and commercialization of mavacamten in Mainland China,
Hong Kong, Macau, Taiwan, Thailand and Singapore. Mavacamten was
granted Breakthrough Therapy Designation in China for the treatment
of patients with oHCM in February 2022.
About EXPLORER-HCMThe Phase 3 EXPLORER-HCM
trial enrolled a total of 251 patients with symptomatic (NYHA Class
II or III), obstructive hypertrophic cardiomyopathy. All
participants had measurable left ventricular outflow tract (LVOT)
gradient (resting and/or provoked) >50 mmHg during
screening.
The primary endpoint for EXPLORER-HCM was a composite functional
analysis designed to capture mavacamten’s effect on both symptoms
and function. Secondary endpoints were changes from baseline to
week 30 in postexercise LVOT gradient, pVO2, proportion of
participants with at least one NYHA class improvement, and measures
of patient-reported outcomes. Additional endpoints included changes
from baseline to Week 30 in echocardiographic indices, circulating
biomarkers, cardiac rhythm patterns and accelerometry.
About EXPLORER-CNThe Phase 3 EXPLORER-CN trial
enrolled a total of 81 patients in China with symptomatic (NYHA
Class II or III), obstructive hypertrophic cardiomyopathy. All
participants had measurable left ventricular outflow tract (LVOT)
gradient (resting and/or provoked) >50 mmHg during
screening.
The primary endpoint for EXPLORER-CN is the change from baseline
to week 30 in Valsalva LVOT gradient. Secondary endpoints include
changes from baseline to week 30 in LVOT obstruction, proportion of
participants with at least one NYHA class improvement, measures of
patient-reported outcomes, cardiac biomarkers, and left ventricular
mass index. Additional endpoints include changes from baseline to
Week 30 in echocardiographic and cardiac magnetic resonance
parameters, proportion of participants achieving NYHA class I and
resting and Valsalva LVOT gradient < 30 mmHg at week 30.
About Hypertrophic CardiomyopathyHypertrophic
cardiomyopathy (HCM) is a chronic, progressive disease in which
excessive contraction of the heart muscle and reduced ability of
the left ventricle to fill can lead to the development of
debilitating symptoms and cardiac dysfunction. HCM is estimated to
affect one in every 500 people globally. The most frequent cause of
HCM is mutations in the heart muscle proteins of the sarcomere. In
both obstructive and non-obstructive HCM patients, exertion can
result in fatigue or shortness of breath, interfering with a
patient’s ability to participate in activities of daily living. HCM
has also been associated with increased risks of atrial
fibrillation, stroke, heart failure and sudden cardiac death.
In China, we estimate there are approximately 1.1 million to 2.8
million patients with HCM, with approximately two-thirds of
patients having oHCM, and one-third of patients having
non-obstructive HCM.
About LianBioLianBio is a cross-border
biotechnology company on a mission to bring transformative
medicines to historically underserved patients in China and other
Asian markets. Through partnerships with highly innovative
biopharmaceutical companies around the world, LianBio is advancing
a diversified portfolio of clinically validated product candidates
with the potential to drive new standards of care across
cardiovascular, oncology, ophthalmology, inflammatory disease and
respiratory indications. LianBio is establishing an international
infrastructure to position the company as a partner of choice with
a platform to provide access to China and other Asian markets. For
more information, please visit www.lianbio.com.
Cautionary Note Regarding Forward-Looking
StatementsStatements 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. The words “expect,” “estimate” and
“potential” and similar expressions are intended to identify
forward-looking statements, although not all forward-looking
statements contain these identifying words. Forward-looking
statements in this press release include, but are not limited to,
statements regarding the Company’s plans and expectations to report
topline results in mid-2023 and to use the data from EXPLORER-CN
and its pharmacokinetic study to support registration of mavacamten
in China; the potential impact of mavacamten on patient lives; the
Company’s beliefs regarding Chinese oHCM patients’ demand for new
therapies, physician and patient enthusiasm for mavacamten, and the
Company’s strong clinical development capabilities in China; the
Company’s ability to navigate complex regulatory environments in
China; the continued growth of the Company as and transformation to
a commercial-stage company; and the Company’s work with respect to
its ability to develop and bring mavacamten to patients in Asia.
Actual results may differ materially from those indicated by such
forward-looking statements as a result of various important
factors, including: the Company’s ability to successfully initiate
and conduct its planned clinical trials and complete such clinical
trials and obtain results on its expected timelines, or at all; the
Company’s plans to leverage data generated in its partners’ global
registrational trials and clinical development programs to obtain
regulatory approval and maximize patient reach for its product
candidates; the Company’s ability to identify new product
candidates and successfully acquire such product candidates from
third parties; competition from other biotechnology and
pharmaceutical companies; general market conditions; the impact of
changing laws and regulations and those risks and uncertainties
described in LianBio’s filings with the U.S. Securities and
Exchange Commission (SEC), including LianBio’s Annual Report on
Form 10-K for the year ended December 31, 2022 and subsequent
filings with the SEC. Any forward-looking statements contained in
this press release speak only as of the date hereof, and LianBio
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 this information as current
or accurate after its publication date.
For investor inquiries, please contact:
Elizabeth Anderson, VP Communications and Investor
Relations E:
elizabeth.anderson@lianbio.com T: +1 646 655
8390
For media inquiries, please contact:
Josh Xu, Director of Communications
E: josh.xu@lianbio.com T: +86 136
6140 8315
Katherine Smith, Evoke Canale
E: katherine.smith@evokegroup.com
T: +1 619 849 5378
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