The FDA is calling the study a phase II trial and says the company must run a rodent genotoxicity study before it can continue.
The U.S. Food and Drug Administration has put the brakes on a clinical study being conducted by Viking Therapeutics. The agency has placed a clinical hold on Viking’s early-stage study of VK0214, the company’s candidate to treat patients with X-linked adrenoleukodystrophy (X-ALD), a rare and often fatal metabolic disorder.
The regulatory body is requesting another preclinical study be done before Viking moves forward.
VIking was under the impression it was running a phase Ib clinical trial, but the FDA is calling it a phase II trial and says the company must run a rodent genotoxicity study before it can continue. The FDA request was not due to any findings from Viking’s ongoing or previous studies.
VIking had planned to do the study prior to phase II, but will move this up due to the FDA’s request. The company expects a short-term delay, but not a long-term impact on the development timeline for the drug. Viking plans to give the FDA the results in Q2, 2022.
X-ALD is characterized by a breakdown in the protective barriers surrounding brain and nerve cells, a process known as demyelination. There is no approved treatment for the disease, which occurs in approximately 1 in 17,000 births. VK0214 is a novel, orally available small molecule TRβ agonist. It enjoys orphan drug status in the United States for the treatment of X-ALD.
“We look forward to providing the FDA with the requested information in a timely fashion. The current request is in keeping with industry guidance for Phase II studies and is not based on data from previously submitted or ongoing studies. We are confident in the overall safety and potential efficacy profile of VK0214 and expect to submit a response with a goal to resume dosing in the study later this year,” said Viking Chief Executive Officer Brian Lian, Ph.D.
Results from a successful Phase 1 dosing study in healthy individuals showed that VK0214 demonstrated promising safety and tolerability, as well as a predictable mechanism of action. Individuals who received VK0214 saw reductions in low-density lipoprotein cholesterol, triglycerides and apolipoprotein B after 14 days of treatment.
Another drug in Viking’s treatment pipeline is VK2809, a novel, orally available small molecule selective thyroid hormone receptor beta agonist being developed for the treatment of lipid and metabolic disorders. It is currently being evaluated in a study for the treatment of nonalcoholic steatohepatitis and fibrosis (NASH). This is a serious condition in which fat replaces healthy liver tissue in people who consume little or no alcohol.
Earlier this month, Viking announced the start of the first in-human clinical trial for VK2375, a novel therapeutic in development for the potential treatment of various metabolic disorders, and a third candidate in the treatment pipeline.
The results of in vivo studies were recently presented at ObesityWeek 2021, highlighting the encouraging effects observed after treatment with VK2735 as well as other compounds from the series. Compared with control cohorts, data showed improvements in the metabolic profile of diet-induced obese mice treated with Viking’s compounds.
Following this announcement, Lian stated, “The initiation of clinical development with VK2375 is an important milestone for Viking, as it represents our first internally developed program to reach the clinic and serves to further enhance our position as a leader in the development of novel therapeutics for metabolic disorders.”
