Galectin Therapeutics, Inc. (NASDAQ: GALT), the leading developer
of therapeutics that target galectin proteins, today announced that
it has submitted to the U.S. Food and Drug Administration (FDA) its
protocol for a seamless adaptively-designed Phase 2b/3 clinical
study, the NASH-RX trial, evaluating the safety and efficacy of its
galectin-3 inhibitor, belapectin (GR-MD-02), for the prevention of
esophageal varices in patients with non-alcoholic steatohepatitis
(NASH) cirrhosis.
The filing anticipates clinical trials will begin in the second
quarter of this year. The major features of the innovative,
seamless adaptively-designed Phase 2b/3 study design are summarized
below and graphically depicted in the figure:
- In patients with NASH cirrhosis and clinical signs of portal
hypertension but without esophageal varices at baseline, this trial
will assess the effect of belapectin on the incidence of new
varices (the primary endpoint) – as well as assessing the effect on
the incidence of long-term, clinically significant
cirrhosis-related “hard” outcomes (a key secondary efficacy
endpoint).
- The design of this trial reflects the unmet medical needs of
the target patient population for belapectin treatment: NASH
patients with compensated cirrhosis who develop esophageal
varices. Bleeding varices are a cause of death in about
one-third of cirrhotic patients. There is no approved treatment for
preventing varices in these patients. The development of new
varices reflects the progression of hepatic cirrhosis and thus
portends the development of other cirrhosis complications and
outcomes such as significant ascites, hepatic encephalopathy, and
ultimately liver failure.
- During the first 18 months of the trial, two belapectin dose
levels (2 mg/kg LBM and 4 mg/kg LBM) will be compared to placebo.
Then, at the interim analysis (IA), one belapectin dose will be
selected based on efficacy and safety, for continued evaluation in
Stage 2 (Phase 3). Prior trials have demonstrated the safety of
belapectin with doses of up to 8 mg/kg for 52 weeks (Phase 2b Study
GT-026).
“The protocol filed today reflects the previous feedback
received from the FDA, as well as key contributions from Dr. Naga
Chalasani and Dr. Stephen Harrison, NASH opinion leaders who
are co-primary investigators for the study, biostatistical experts
and numerous other collaborators at Covance, our CRO,” said Harold
H. Shlevin, Ph.D., President and Chief Executive Officer of
Galectin Therapeutics. “We are very grateful for all of their
efforts, and for the previous feedback and suggestions from the
FDA. The study design has been modified and further refined such
that it provides for a prespecified interim analysis (IA). The IA
of efficacy and safety data will be conducted after all planned
subjects in Phase 2b component have completed at least 78 weeks (18
months) of treatment and a gastro-esophageal endoscopic assessment.
The purpose of the IA is to allow potential seamless adaptive
modifications of the study, including: (1) the selection of the
optimal dose of belapectin for Phase 3; (2) the re-estimation of
the study sample size for Phase 3 portion of the trial;
(3) the re-evaluation of the randomization ratio for the Phase
3 portion of the trial; (4) the refinement of the inclusion and
exclusion criteria for the Phase 3 portion of the trial, including
the CTP status; (5) and/or termination of the study for
overwhelming efficacy or futility.
The new trial design also minimizes invasive testing
requirements, which we believe will enhance the enrollment and
retention of patients. It also provides for a seamless transition
of patients from the phase 2b component into the phase 3 stage, as
well as provides for the potential addition of new patients. The
trial design preserves the surrogate end-point concepts previously
discussed with the FDA.
As previously communicated, this is a uniquely challenging time
to start a new clinical trial, and we are doing everything in our
direct control to prepare for the initiation of the study later
this quarter; however, factors beyond our control, specifically
related to the COVID-19 pandemic, may delay the trial’s
initiation. Notwithstanding that, we remain optimistic in
moving forward. The unmet medical need for effective
treatment for patients with NASH cirrhosis remains an important
motivation.
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” is now posted on www.clinicaltrials.gov
(NCT04365868). Galectin Therapeutics will share more details about
the protocol at the time the clinical trial begins. In addition,
the FDA has received Galectin’s protocol for the hepatic impairment
study which will be conducted in subjects with normal hepatic
function and subjects with varying degrees of hepatic impairment
(NCT04332432). This filing anticipates that this study will also
begin in the second quarter of this year.
About Belapectin (GR-MD-02)
Belapectin 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 belapectin has robust treatment effects in
reversing liver fibrosis and cirrhosis.
About Fatty Liver Disease with Advanced Fibrosis and
Cirrhosis
Non-alcoholic steatohepatitis (NASH) has become a common disease
of the liver with the rise in obesity and other metabolic diseases.
NASH is estimated to affect up to 28 million people in the U.S. It
is characterized by the presence of excess fat in the liver along
with inflammation and hepatocytes damage (ballooning) in people who
consume little or no alcohol. Over time, patients with NASH can
develop excessive fibrosis, or scarring of the liver, and
ultimately liver cirrhosis. It is estimated that as many as 1-2
million individuals in the U.S. will develop cirrhosis as a result
of NASH, for which liver transplantation is the only curative
treatment available. Approximately 8,890 liver transplants are
performed annually in the U.S. There are no drug therapies approved
for the treatment of liver fibrosis or cirrhosis.
About Galectin Therapeutics
Galectin Therapeutics is dedicated to developing novel therapies
to improve the lives of patients with chronic liver disease and
cancer. Galectin’s lead drug belapectin 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 Statements
This 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 established in our clinical trials 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 currently planned clinical trial and any future clinical
studies as modified to meet the requirements of the FDA 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 any other 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. Belapectin is the USAN
assigned name for Galectin Therapeutics’ galectin-3 inhibitor
GR-MD-02
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/535ab060-4a23-4e52-b9d6-527cf591fd12
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