Viking Therapeutics Announces Initiation of Phase 1 Clinical Trial of VK0214

First-in-human clinical trial of novel, selective thyroid receptor ß agonist for X-ALD

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.


Company Codes: NASDAQ-SMALL:VKTX

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