SAN DIEGO, Dec. 6, 2016 /PRNewswire/ -- Viking
Therapeutics, Inc. ("Viking") (NASDAQ: VKTX), a clinical-stage
biopharmaceutical company focused on the development of novel
therapies for metabolic and endocrine disorders, today announced
that the U.S. Food and Drug Administration (FDA) has granted orphan
drug designation to VK0214 for the treatment of X-linked
adrenoleukodystrophy (X-ALD). VK0214 is a novel, orally
available thyroid receptor beta (TRβ) agonist that selectively
regulates the expression of genes believed to be relevant to the
manifestation of X-ALD.
VK0214 is currently being evaluated in preclinical models of
X-ALD under a sponsored research agreement with the Kennedy Krieger
Institute. Data to date have demonstrated promising in
vivo activity, including significant reductions in plasma very
long chain fatty acids (VLCFA), important biochemical markers of
disease.
"Viking is committed to evaluating the potential of VK0214 for
the treatment of X-ALD, a devastating and progressively
debilitating disease for which there is no approved therapy.
Our research indicates that VK0214 positively impacts key
genes and biomarkers that may affect the onset of X-ALD, which
suggests potential therapeutic benefits in this setting," said
Brian Lian, Ph.D., chief executive
officer of Viking Therapeutics. "The Orphan Drug designation
underscores the importance of new therapies in areas of high unmet
medical need, such as X-ALD, and we look forward to the continued
advancement of VK0214 for this underserved disease."
X-ALD is a rare and often fatal metabolic disorder characterized
by a breakdown in the protective barriers surrounding brain and
nerve cells; a process known as demyelination. The disease,
for which there is no approved treatment, is caused by mutations in
a peroxisomal transporter of VLCFAs, known as ABCD1. As a
result, transporter function is impaired and patients are unable to
efficiently metabolize VLCFA. The resulting accumulation can
trigger a rapid, inflammatory demyelination, which leads to
cognitive impairment, motor skill deterioration, and even
death. X-ALD is estimated to occur in approximately 1 in
17,000 births.
FDA's Orphan Drug Designation program provides certain
incentives for companies developing therapeutics to treat rare
diseases or conditions, defined as those affecting less than
200,000 individuals in the U.S. A drug candidate and its
sponsor must meet several key criteria in order to qualify for, and
obtain, orphan drug status. Once a drug has received orphan
drug designation, the developer qualifies for a range of benefits,
including federal grants, tax credits, reduction in certain
regulatory fees, and the potential for seven years of market
exclusivity for the drug following FDA marketing approval.
About VK0214
VK0214 is a novel, orally available
thyroid receptor beta (TRβ) agonist that selectively modulates
lipoprotein and triglyceride levels in liver tissue. This
mechanism has been demonstrated to affect the expression of the
genes that are relevant to the manifestation of X-ALD. In
X-ALD, mutations in the ABCD1 gene lead to the accumulation of very
long-chain fatty acids (VLCFAs) which is believed to be a
fundamental cause of the disease. Research has shown that
increasing the expression of the ABCD2 gene can counteract this
process and lead to normalization of VLCFA levels. In
preclinical studies, VK0214 has been shown to induce expression of
ABCD2 by increasing TRβ activity, leading to the belief that it may
provide therapeutic benefit to X-ALD patients.
About X-ALD
X-ALD is a rare and often fatal metabolic
disorder characterized by a breakdown in the protective barriers
surrounding brain and nerve cells; a process known as
demyelination. The disease, for which there is no approved
treatment, is caused by mutations in a peroxisomal transporter of
VLCFAs, known as ABCD1. As a result, transporter function is
impaired and patients are unable to efficiently metabolize
VLCFA. The resulting accumulation can trigger a rapid,
inflammatory demyelination, which leads to cognitive impairment,
motor skill deterioration, and even death. X-ALD is estimated
to occur in approximately 1 in 17,000 births.
The thyroid beta receptor is known to regulate expression of an
alternative VLCFA transporter, known as ABCD2. Various
preclinical models have demonstrated that increased expression of
ABCD2 can lead to normalization of VLCFA metabolism.
About Viking Therapeutics, Inc.
Viking Therapeutics,
Inc. is a clinical-stage biopharmaceutical company focused on the
development of novel, first-in-class or best-in-class therapies for
metabolic and endocrine disorders. The company's research and
development activities leverage its expertise in metabolism to
develop innovative therapeutics designed to improve patients'
lives. Viking has exclusive worldwide rights to a portfolio
of five therapeutic programs in clinical trials or preclinical
studies, which are based on small molecules licensed from Ligand
Pharmaceuticals Incorporated. The company's clinical programs
include VK5211, an orally available, non-steroidal selective
androgen receptor modulator, or SARM, in Phase 2 development for
the treatment and prevention of lean body mass loss in patients who
have undergone hip fracture surgery, VK2809, a small molecule
thyroid beta agonist in Phase 2 development for
hypercholesterolemia and fatty liver disease, and VK0612, a
first-in-class, orally available drug candidate in Phase 2
development for type 2 diabetes. Viking is also developing
novel and selective agonists of the thyroid beta receptor for
adrenoleukodystrophy, as well as two earlier-stage programs
targeting metabolic diseases and anemia.
Follow Viking on Twitter @Viking_VKTX.
Forward-Looking Statements
This press release
contains forward-looking statements regarding Viking Therapeutics,
including statements about Viking's expectations regarding its
development activities, timelines and milestones, as well as the
company's goals and plans regarding VK0214. Forward-looking
statements are subject to risks and uncertainties that could cause
actual results to differ materially and reported results should not
be considered as an indication of future performance. These risks
and uncertainties include, but are not limited to: risks associated
with the success, cost and timing of Viking's product candidate
development activities and clinical trials; and risks regarding
regulatory requirements, among others. These forward-looking
statements speak only as of the date hereof. Viking disclaims
any obligation to update these forward-looking statements.
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SOURCE Viking Therapeutics, Inc.