SAN DIEGO, Sept. 9, 2020 /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
the initiation of a Phase 1 single ascending dose (SAD) and
multiple ascending dose (MAD) clinical trial of VK0214, a novel,
orally available thyroid receptor beta (TRβ) agonist in development
as a potential treatment for X-linked adrenoleukodystrophy (X-ALD).
Enrollment in this study is underway following clearance of the
company's Investigational New Drug (IND) application by the United
States Food and Drug Administration (FDA).
Activation of the thyroid beta receptor has been shown to affect
the expression of genes that are relevant to the manifestation of
X-ALD. In X-ALD, mutations in the ABCD1 gene lead to dysfunction of
the adrenoleukodystrophy protein (ALDP), an important peroxisomal
transporter. In patients, this leads to an accumulation of very
long chain fatty acids (VLCFAs), which is believed to contribute to
the onset and progression of the disease. Research in disease
models has shown that increasing the expression of a related gene
called ABCD2, which encodes a compensatory transporter called the
adrenoleukodystrophy related protein (ADLRP), can result in
normalization of VLCFA levels.
In preclinical studies, VK0214 has been shown to potently
activate the thyroid beta receptor, leading to increased expression
of ABCD2. Data from in vivo studies have
demonstrated that administration of VK0214 produces a significant
reduction of VLCFAs in both plasma and tissue, potentially leading
to a therapeutic benefit. VK0214 has been granted orphan drug
designation by the FDA for the treatment of X-ALD.
The Phase 1 trial is a randomized, double-blind,
placebo-controlled, SAD and MAD study in healthy subjects. The
primary objectives of the study include evaluation of the safety
and tolerability of single and multiple oral doses of VK0214, as
well as the identification of VK0214 doses for further clinical
development in the setting of X-ALD. Study investigators will also
assess pharmacokinetics of single and multiple oral doses of
VK0214. Upon successful completion of the SAD/MAD study, the
company plans to initiate a Phase 1b
study in patients with X-ALD.
"We are excited to advance VK0214 into clinical development to
evaluate as a potential treatment for X-ALD, a progressive,
debilitating disease for which there is no approved therapy. The
safety, tolerability and pharmacokinetic data from this study will
provide key insights for future trials of VK0214 in patients with
X-ALD," said Brian Lian, Ph.D.,
chief executive officer of Viking Therapeutics. "Our prior results
in this area indicate that the thyroid receptor beta is an
important regulator of VLCFA metabolism, and we look forward to
demonstrating the therapeutic potential of VK0214 in combatting
this disease."
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 VLCFAs.
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 is a clinical-stage biopharmaceutical
company focused on the development of novel, orally available,
first-in-class or best-in-class therapies for the treatment of
metabolic and endocrine disorders. Viking's research and
development activities leverage its expertise in metabolism to
develop innovative therapeutics designed to improve patients'
lives. The company's clinical programs include VK2809, a
novel, orally available, small molecule selective thyroid hormone
receptor beta agonist for the treatment of lipid and metabolic
disorders, which is currently being evaluated in a
Phase 2b study for the treatment of biopsy-confirmed
non-alcoholic steatohepatitis (NASH) and fibrosis. In a Phase
2 trial for the treatment of non-alcoholic fatty liver disease
(NAFLD) and elevated LDL-C, patients who received VK2809
demonstrated statistically significant reductions in LDL-C and
liver fat content compared with patients who received
placebo. The company is also developing VK0214, a novel,
orally available, small molecule selective thyroid hormone receptor
beta agonist currently in clinical development for the treatment of
X-linked adrenoleukodystrophy (X-ALD).
The company holds exclusive worldwide rights to a portfolio of
five therapeutic programs, including those noted above, which are
based on small molecules licensed from Ligand Pharmaceuticals
Incorporated.
For more information about Viking Therapeutics, please visit
www.vikingtherapeutics.com. Follow Viking on Twitter
@Viking_VKTX.
Forward-Looking Statements
This press release contains forward-looking statements
regarding Viking Therapeutics, Inc., under the safe harbor
provisions of the U.S. Private Securities Litigation Reform Act of
1995, including statements about Viking's expectations regarding
its development activities and plans regarding VK0214 and its
prospects. Forward-looking statements are subject to risks and
uncertainties that could cause actual results to differ materially
and adversely 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, including those for VK2809 and
VK0214; risks that prior clinical and preclinical results may not
be replicated; risks regarding regulatory requirements; risks
related to the COVID-19 pandemic; and other risks that are
described in Viking's most recent periodic reports filed with the
Securities and Exchange Commission, including Viking's Annual
Report on Form 10-K for the year ended December 31, 2019, and
subsequent Quarterly Reports on Form 10-Q, including the risk
factors set forth in those filings. These forward-looking
statements speak only as of the date hereof. Viking disclaims
any obligation to update these forward-looking statements except as
required by law.
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SOURCE Viking Therapeutics, Inc.