- Program to include one trial to confirm the safety and
therapeutic effect on fibrosis in NASH patients with cirrhosis, and
a smaller trial to confirm the safety and therapeutic effect on
NASH patients with advanced fibrosis
- Both studies to begin in the second quarter of
2015
- Company to submit a Special Protocol Assessment to the
FDA by the end of the month
Galectin Therapeutics (Nasdaq:GALT), the leading developer of
therapeutics that target galectin proteins to treat fibrosis and
cancer, announces details of the design of its Phase 2 program with
GR-MD-02 in patients with advanced fatty liver disease, or
nonalcoholic steatohepatitis (NASH) with cirrhosis.
The program includes two clinical studies. The first is a
multicenter, randomized, placebo-controlled, double-blind,
parallel-group Phase 2 trial to evaluate the safety and efficacy of
GR-MD-02 for the treatment of liver fibrosis and resultant portal
hypertension in patients with NASH cirrhosis (the NASH-CX trial).
This trial is expected to commence in the second quarter of 2015
with data readout expected in the fourth quarter of 2017. In
addition, the Company will conduct a smaller trial of shorter
duration in NASH patients with advanced fibrosis (the NASH-FX
trial).
The NASH-CX trial was designed with guidance from the U.S. Food
and Drug Administration (FDA) received during an End-of-Phase 1
meeting. The NASH-CX trial will include 45 sites (and up to 60
sites, if necessary) in the U.S. and Canada and will enroll a total
of 156 patients. It will be comprised of three parallel treatment
arms of 52 patients each, with one arm receiving 8 mg/kg of
GR-MD-02, the expected therapeutic dose, one arm receiving 2 mg/kg
of GR-MD-02 and a third arm receiving placebo. Patients will
receive a total of 26 infusions every other week for one year and
will be evaluated to determine the change in the hepatic venous
pressure gradient (HVPG) as compared with placebo.
The FDA has indicated that HVPG may serve as a surrogate primary
endpoint for NASH cirrhosis. HVPG will be correlated with
secondary endpoints of fibrosis on liver biopsy as well as with
measurement of liver stiffness (FibroScan®) and assessment of liver
metabolism (13C-methacetin breath test, Exalenz), which are
non-invasive measures of the liver that may be used in future
studies.
Galectin is finalizing a submission to the FDA by the end of
February for a Special Protocol Assessment (SPA) to accept this
trial, if positive, as one of the required trials to support
approval of the drug candidate. The FDA has previously agreed to
review this study protocol for acceptance under an SPA.
Peter G. Traber, M.D., Galectin's chief executive officer,
president and chief medical officer, said, "We are very excited to
begin testing our drug candidate in a larger patient population,
and we look forward to working with PPD, our contract research
organization. There are no approved drugs to treat NASH, and fatty
liver disease and NASH are stealth diseases that advance from early
stages through to advanced disease without symptoms. When
symptoms of any significance do occur, the disease is already
full-blown cirrhosis of the liver and the damage is currently
considered to be irreversible and the treatment is liver
transplant. We are hopeful that GR-MD-02 will prove to be a
solution to this significant health problem. As many as 28
million Americans are afflicted with NASH, of which up to 6 million
have advanced fibrosis."
Dr. Traber added, "Our successful Phase 1 program showed
GR-MD-02 to be safe and well-tolerated, and reached the targeted
therapeutic range in the 8 mg/kg dose cohort. Patients in this
group showed encouraging effects on a relevant biomarker of
fibrotic liver disease and a potential signal indicating a
reduction in liver stiffness. These findings are even more notable
in light of the short, four-dose treatment regimen tested."
The NASH-FX trial is a 30-patient, randomized,
placebo-controlled, blinded study to be conducted at Brooke Army
Medical Center with enrollment expected to begin in
mid-2015. Patients with NASH with advanced fibrosis will be
randomized with 20 receiving 8 mg/kg GR-MD-02 and 10 receiving
placebo every other week for 16 weeks, for a total of nine doses.
Following the treatment period, the safety and the efficacy of
GR-MD-02 on liver stiffness will be evaluated as assessed by
magnetic resonance-elastography and FibroScan score, and on imaging
of liver fibrosis using multi-parametric magnetic resonance imaging
(LiverMultiScan®, Perspectum Diagnostics). Top-line data is
expected to be available in mid-2016.
"We believe that this smaller study will provide important
information that could inform future larger trials that ultimately
may widen the target market for GR-MD-02," continued Dr.
Traber. "This study will evaluate three promising non-invasive
tests for assessment of liver fibrosis, while offering shorter
treatment in a population of patients with advanced fibrosis, but
not necessarily cirrhosis."
More details on the clinical trials can be found in Galectin's
Corporate Presentation on our website,
www.galectintherapeutics.com. Galectin anticipates that the cost of
completing both studies will be approximately $20 million over the
two and one-half year duration of the trials. As of December
31, 2014, Galectin had cash and cash equivalents of $29.1
million.
About GR-MD-02
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
scaring 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 will in
the U.S. have 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 Therapeutics
Galectin Therapeutics is developing promising carbohydrate-based
therapies for the treatment of fibrotic liver disease and cancer
based on the Company's unique understanding of galectin proteins,
which are key mediators of biologic function. Galectin seeks to
leverage extensive scientific and development expertise as well as
established relationships with external sources to achieve
cost-effective and efficient development. The Company is pursuing a
development pathway to clinical enhancement and commercialization
for its lead compounds in liver fibrosis and cancer. 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 GR-MD-02
will lead to the first therapy for the treatment of fatty liver
disease with cirrhosis. Factors that could cause actual performance
to differ materially from those discussed in the forward-looking
statements include, among others, that Galectin may not be
successful in developing effective treatments and/or obtaining the
requisite approvals for the use of GR-MD-02 or any of its other
drugs in development. The Company's current clinical trial and any
future clinical studies may not produce positive results in a
timely fashion, if at all, and could prove 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. There is no certainty that FDA and Company
will agree on a SPA or that a SPA would ultimately be acceptable to
FDA nor result in approval of GR-MD-02. 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 further develop and/or fund any
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. 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, 2013, 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.
CONTACT: Galectin Therapeutics Inc.
Peter G. Traber, M.D.
President, CEO & CMO
(678) 620-3186
ir@galectintherapeutics.com
LHA
Kim Golodetz
(212) 838-3777
kgolodetz@lhai.com
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