LianBio (Nasdaq: LIAN), a biotechnology company dedicated to
bringing innovative medicines to patients in China and other major
Asian markets, today announced positive topline results from the
Phase 3 EXPLORER-CN trial investigating mavacamten for the
treatment of Chinese patients with symptomatic obstructive
hypertrophic cardiomyopathy (oHCM).
EXPLORER-CN met its primary endpoint, demonstrating a
statistically significant and clinically meaningful improvement in
Valsalva left ventricular outflow tract (LVOT) gradient from
baseline to week 30 compared to placebo (p<0.001). Additionally,
mavacamten demonstrated improvement for all secondary endpoints,
including change from baseline to week 30 in resting LVOT peak
gradient, proportion of participants achieving a Valsalva LVOT peak
gradient <30 mmHg at week 30, proportion of participants
achieving a Valsalva LVOT peak gradient <50 mmHg at week 30,
proportion of participants with at least one NYHA class improvement
from baseline to week 30, change from baseline to week 30 in Kansas
City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score
(CSS), and change from baseline to week 30 in left ventricular mass
index evaluated by cardiac magnetic resonance imaging.
Safety results in the trial were consistent with previous
studies of mavacamten in symptomatic oHCM, and no new safety
signals were reported. No participants in the study experienced
decreases in left ventricular ejection fraction (LVEF) <50 %
that required dose interruption.
The Phase 3 EXPLORER-CN trial enrolled a total of 81 patients in
China with symptomatic (NYHA Class II or III) oHCM. All
participants had one measurable LVOT gradient (resting or Valsalva)
>50 mmHg during screening.
“For decades, physicians have been utilizing medicines to try to
manage only the symptoms of hypertrophic cardiomyopathy,” said
Shuyang Zhang, M.D., Ph.D., Professor of Cardiology and President,
Peking Union Medical College Hospital and Principal Investigator of
EXPLORER-CN. “Before mavacamten, oHCM patients had no therapeutic
option that targeted the underlying pathophysiology of the disease.
The topline results from EXPLORER-CN add to the body of evidence
demonstrating mavacamten is an effective treatment option for
patients worldwide who are living with this chronic and
debilitating condition.”
In April 2023, the China National Medical Products
Administration (NMPA) accepted with Priority Review a New Drug
Application (NDA) for mavacamten for the treatment of adults with
symptomatic oHCM. 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 a 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.
The NDA also includes clinical data from LianBio’s Phase 1
pharmacokinetic study of mavacamten in healthy Chinese volunteers
that demonstrated a favorable safety and tolerability profile, and
a pharmacokinetic profile comparable to that observed in the Phase
1 pharmacokinetic studies of mavacamten conducted in the United
States. Blinded preliminary safety data from EXPLORER-CN also
supported the early NDA filing.
“We believe the speed and quality with which the EXPLORER-CN
trial was conducted is a testament to LianBio’s ability to
accelerate patient access to innovative medicines,” said Yizhe
Wang, Ph.D., Chief Executive Officer of LianBio. “I would like to
acknowledge our team’s hard work and commitment to the oHCM
community and thank the EXPLORER-CN investigators and patients for
their participation in this important study. With positive pivotal
data now in hand and the NDA on file, we are optimistic about
bringing mavacamten to market in China next year.”
LianBio will complete a full evaluation of the EXPLORER-CN data
and expects to work with investigators to present detailed results
at an upcoming medical meeting.
Analyst and Investor Conference Call and Live
WebcastLianBio will host a virtual event for analysts and
investors to review the topline data from EXPLORER-CN and an
overview of the China market opportunity for mavacamten, pending
regulatory approval. The live webcast will begin at 8:00 a.m. EDT /
8:00 p.m. CST on Monday, May 1. Listeners can access a live audio
webcast of the conference call on the investor section of LianBio’s
website at http://investors.Lianbio.com.
Research analysts who would like to participate in the live
Q&A can register to obtain dial-in and passcode details.
Participants may pre-register at any time, including up to and
after the call start time.
A replay of the webcast and accompanying slides will be
available on the LianBio website for 90 days following the
call.
About MavacamtenCamzyos® (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, Brazil and Switzerland. 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 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 EXPLORER-HCM Phase 3
trial enrolled a total of 251 patients with symptomatic (NYHA Class
II or III) oHCM. All participants had one measurable LVOT gradient
(resting 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) oHCM. All participants had one measurable LVOT
gradient (resting or Valsalva) >50 mmHg during screening.
Patients were randomized 2:1 to mavacamten or placebo.
The primary endpoint for EXPLORER-CN is the change from baseline
to week 30 in Valsalva LVOT gradient. Secondary endpoints include
change from baseline to week 30 in resting LVOT peak gradient,
proportion of participants achieving a Valsalva LVOT peak gradient
<30 mmHg at week 30, proportion of participants achieving a
Valsalva LVOT peak gradient <50 mmHg at week 30, proportion of
participants with at least one NYHA class improvement from baseline
to week 30, change from baseline to week 30 in Kansas City
Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score (CSS),
change from baseline to week 30 in N-terminal pro B-type
Natriuretic Peptide (NT-proBNP), change from baseline to week 30 in
cardiac troponin, and change from baseline to week 30 in left
ventricular mass index evaluated by cardiac magnetic resonance
imaging.
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
patients with both obstructive and non-obstructive HCM, 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, there are an estimated 1.1 million to 2.8 million
patients with 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 and inflammatory disease
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,” “believe,”
“continue,” “estimate,” “potential,” “will” 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 LianBio’s beliefs
regarding LianBio’s ability to accelerate patient access to
innovative medicines and LianBio’s strong clinical development
capabilities in China; LianBio’s plans to use the data from
EXPLORER-CN and its pharmacokinetics study to support registration
of mavacamten in China, including its plans to complete a full
evaluation of the EXPLORER-CN data and its expectation that it will
work with investigators to present detailed results at an upcoming
medical meeting; and LianBio’s expectations regarding its ability
to and the timeframe within which it expects to bring mavacamten to
market in China. Actual results may differ materially from those
indicated by such forward-looking statements as a result of various
important factors, including: LianBio’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; LianBio’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; LianBio’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.
In addition, topline and interim data from clinical trials may
not be indicative of final results, and the results of early
clinical trials may not be indicative of the results of later
clinical trials. Moreover, nonclinical and clinical data are often
susceptible to varying interpretations and analyses, and many
companies that have believed their product candidates performed
satisfactorily in nonclinical and clinical trials have nonetheless
failed to obtain marketing approval of their products. There is a
risk that additional nonclinical and/or clinical safety studies
will be required by the NMPA or similar regulatory authorities in
other jurisdictions, or that subsequent studies will not match
results seen in prior studies. As a result, topline data should be
viewed with caution until the final data are available.
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
LianBio (NASDAQ:LIAN)
Historical Stock Chart
From Jun 2024 to Jul 2024
LianBio (NASDAQ:LIAN)
Historical Stock Chart
From Jul 2023 to Jul 2024