Galectin Therapeutics (NASDAQ: GALT), the leading developer of
therapeutics that target galectin proteins, announced today that it
has enrolled its first patients in the NASH-RX trial. NASH-RX is an
international, seamless, adaptively-designed Phase 2b/3 trial of
its galectin-3 inhibitor belapectin (GR-MD-02), the company's lead
compound, in nonalcoholic steatohepatitis (NASH) cirrhosis patients
who have clinical signs of portal hypertension and are at risk of
developing esophageal varices. Belapectin had previously been shown
that it could prevent the development of new varices in this
patient population in the Phase 2 NASH-CX clinical trial
(Gastroenterology 2020;158:1334–1345 or
https://doi.org/10.1053/j.gastro.2019.11.296).
NASH-RX is expected to enroll approximately 315 NASH patients in
the Phase 2b part of the trial at approximately 130 sites in 12
countries in North America, Europe, Asia and Australia. During the
Phase 2b part of the trial, two belapectin doses, 2 mg/kg of lean
body mass (LBM) and 4 mg/kg LBM, will be compared to placebo. Prior
trials have demonstrated the good tolerance profile and apparent
safety of belapectin with doses of up to 8 mg/kg LBM, notably for
up to 52 weeks of treatment in patients with NASH cirrhosis (Phase
2b NASH-CX Study).
The study design provides for a prespecified interim analysis
(IA) of efficacy and safety data conducted after all planned
subjects in the Phase 2b component have completed at least 78 weeks
(18 months) of treatment and a gastro-esophageal endoscopic
assessment. This adaptive design allows for patients to seamlessly
transition from the Phase 2b component into the Phase 3 stage, as
well as helps determine the optimal dose, bolsters the efficacy
signal, and re-evaluates the sample size and statistical power for
the Phase 3 stage of the trial. These adaptations are designed to
increase the statistical power for detecting a successful outcome.
The IA also provides for adjustment in the randomization ratio,
refinement of inclusion/exclusion criteria and the potential
termination of the study for overwhelming efficacy or for
futility.
“The unmet medical need for an effective treatment for patients
with NASH cirrhosis remains a compelling motivation to vigorously
pursue our therapy,” commented Harold H. Shlevin, Ph.D., President
and Chief Executive Officer of Galectin Therapeutics. “Moreover, if
the results of the NASH-RX trial are compelling, there could be the
potential for accelerated FDA approval and/or partnership
opportunities.”
Unlike most other clinical trials focused primarily on earlier
stages of NASH, the NASH-RX study population will comprise patients
with compensated liver cirrhosis. Based on the results of the
NASH-CX trial, NASH-RX is focused on patients who have not yet
developed esophageal varices but are at increased risk of
developing these potentially life-threatening complications.
Consequently, patient selection for both Phase 2b and Phase 3 will
be based on clinical signs of portal hypertension such as a
depressed platelet count (thrombocytopenia), an enlargement of the
spleen (splenomegaly) and/or evidence of collateral vessels.
The primary endpoint of the trial is to assess the effect of
belapectin on the incidence of new varices. A centralized review
system of video recording of esophagogastroduodenoscopy (EGD) has
been put in place, and the primary endpoint will be adjudicated by
expert EGD readers. Key secondary endpoints will assess the type of
varices (sizes and/or bleeding) and other clinical events, such as
ascites, hepatic encephalopathy, listing for liver transplantation
or death.
NASH-RX was designed in accordance with advice from the U.S.
Food and Drug Administration (FDA) and with key contributions from
our NASH-RX co-primary study investigators, Dr. Naga Chalasani and
Dr. Stephen Harrison, both widely recognized expert
hepatologists for NASH, biostatistical experts and numerous other
collaborators at Covance, the CRO for the study. The design of the
NASH-RX trial optimizes patient enrollment and retention and
minimizes the need for invasive tests.
Pol Boudes, M.D., Chief Medical Officer for Galectin commented,
“Once liver fibrosis has progressed to cirrhosis, NASH patients can
no longer expect significant improvements from changes in their
lifestyle. These patients are in dire need of new options, and this
trial may prove pivotal in improving their condition. The study’s
seamless and adaptive design is very innovative, and the primary
endpoint minimizes the inconvenience for patients while being
relevant to real life medical practice. We are extremely excited to
start the study and give thanks in advance to all the patients who
will participate and the medical teams that will support them.”
Details on the trial, entitled “A Seamless Adaptive Phase 2b/3,
Double-Blind, Randomized, Placebo-controlled Multicenter,
International Study Evaluating the Efficacy and Safety of
Belapectin (GR-MD-02) for the Prevention of Esophageal Varices in
NASH Cirrhosis,” are posted
on www.clinicaltrials.gov (NCT04365868) and WHO’s trial
registry platform.
About Belapectin (GR-MD-02)
Belapectin (GR-MD-02) is a complex carbohydrate drug that
targets galectin-3, a critical protein in the pathogenesis of fatty
liver disease and fibrosis. Galectin-3 plays a major role in
diseases that involve scarring of organs including fibrotic
disorders of the liver, lung, kidney, heart and vascular system.
The drug binds to galectin proteins and disrupts their function.
Preclinical data in animals have shown that GR-MD-02 has robust
treatment effects in reversing liver fibrosis and cirrhosis.
About Fatty Liver Disease with Advanced Fibrosis and
Cirrhosis
Non-alcoholic steatohepatitis (NASH), also known as fatty liver
disease, has become a common disease of the liver with the rise in
obesity rates. NASH is estimated to affect up to 28 million people
in the U.S. Fatty liver disease is characterized by the presence of
fat in the liver along with inflammation and damage in people who
consume little or no alcohol. Over time, patients with fatty liver
disease can develop fibrosis, or scarring of the liver, and it is
estimated that as many as 1-2 million individuals in the U.S. will
develop cirrhosis, a severe liver disease for which liver
transplantation is the only treatment available. Approximately
6,300 liver transplants are performed annually in the U.S. There
are no drug therapies approved for the treatment of liver
fibrosis.
About Galectin TherapeuticsGalectin
Therapeutics is dedicated to developing novel therapies to improve
the lives of patients with chronic liver disease and cancer.
Galectin’s lead drug belapectin (formerly known as GR-MD-02) is a
carbohydrate-based drug that inhibits the galectin-3 protein, which
is directly involved in multiple inflammatory, fibrotic, and
malignant diseases, for which it has Fast Track designation by the
U.S. Food and Drug Administration. The lead development program is
in non-alcoholic steatohepatitis (NASH) with cirrhosis, the most
advanced form of NASH-related fibrosis. This is the most common
liver disease and one of the largest drug development opportunities
available today. Additional development programs are in treatment
of combination immunotherapy for advanced melanoma and other
malignancies. Advancement of these additional clinical programs is
largely dependent on finding a suitable partner. Galectin seeks to
leverage extensive scientific and development expertise as well as
established relationships with external sources to achieve
cost-effective and efficient development. Additional information is
available at www.galectintherapeutics.com.
Forward Looking StatementsThis press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. These statements
relate to future events or future financial performance, and use
words such as “may,” “estimate,” “could,” “expect” and others. They
are based on management’s current expectations and are subject to
factors and uncertainties that could cause actual results to differ
materially from those described in the statements. These statements
include those regarding the hope that Galectin’s development
program for belapectin will lead to the first therapy for the
treatment of fatty liver disease with cirrhosis and those regarding
the hope that our lead compounds will be successful in cancer
immunotherapy and in other therapeutic indications. Factors that
could cause actual performance to differ materially from those
discussed in the forward-looking statements include, among others,
that trial endpoints may not be achieved; Galectin may not be
successful in developing effective treatments and/or obtaining the
requisite approvals for the use of belapectin or any of its other
drugs in development; the Company may not be successful in scaling
up manufacturing and meeting requirements related to chemistry,
manufacturing and control matters; the Company’s NASH-RX trial and
any future clinical studies may not produce positive results in a
timely fashion, if at all, and could require larger and longer
trials, which would be time consuming and costly; plans regarding
development, approval and marketing of any of Galectin’s drugs are
subject to change at any time based on the changing needs of the
Company as determined by management and regulatory agencies;
regardless of the results of any of its development programs,
Galectin may be unsuccessful in developing partnerships with other
companies or raising additional capital that would allow it to
complete the NASH-RX trial or further develop and/or fund
additional studies or trials. Galectin has incurred operating
losses since inception, and its ability to successfully develop and
market drugs may be impacted by its ability to manage costs and
finance continuing operations. Global factors such as coronavirus
may limit access to NASH patient populations around the globe and
slow trial enrollment and prolong the duration of the trial and
significantly impact associated costs. For a discussion of
additional factors impacting Galectin’s business, see the Company’s
Annual Report on Form 10-K for the year ended December 31, 2019,
and subsequent filings with the SEC. You should not place undue
reliance on forward-looking statements. Although subsequent events
may cause its views to change, management disclaims any obligation
to update forward-looking statements.
Company Contact:Jack Callicutt, Chief Financial
Officer(678) 620-3186ir@galectintherapeutics.com.
Media Contact:Gregory FCALexi Burchmore,
Account Supervisor(215) 297-3607lexib@gregoryfca.com
Galectin Therapeutics and its associated logo is a registered
trademark of Galectin Therapeutics Inc.
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