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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.
About this update from Viking Therapeutics, Inc.
[{"type":"text","content":"First-in-human clinical trial of novel, selective thyroid receptor ß agonist for X-ALD\n\n\nSAN 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).\n\n \n \n \n \n \n \n\n \nActivation 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. \nIn 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. \nThe 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/MA...