Viking Therapeutics is relaunching the longship for a phase 1b metabolic disorder study six months after the FDA sidelined the vessel with a clinical hold.
The X-linked adrenoleukodystrophy, or X-ALD, therapy called VK0214 was placed on a clinical hold in January, with the FDA requesting some preclinical work before advancing the drug into a mid-stage trial. Viking at the time said the dispute stemmed from a mix up in the trial phase. The biotech insisted it was a phase 1b, while the FDA saw it as a phase 2. The difference is that Viking would have had to complete additional tests to have the drug in the more advanced phase.
Viking now says that the work is done and the hold has been lifted. In the second quarter, the company submitted results from an in vivo genotoxicity study to the FDA. These preclinical studies are used to show that a drug substance does not damage genetic material directly or indirectly. The results were clear, showing that the oral therapy VK0214 did not do any harm to genetic material, according to Viking.
The biotech insists that there will be no change to the development timeline for VK0214 despite the six-month gap between the hold being placed and now lifted. Viking had always planned to do the genotoxicity study prior to entering phase 2, but the lab work was accelerated after the FDA’s request.
Patient enrollment is expected to get back underway “in the coming weeks,” according to a Tuesday morning statement. VK0214 has an orphan drug designation from the FDA for X-ALD.
The early-phase study will enroll adult males with adrenomyeloneuropathy, the most common form of X-ALD. The disease is a rare and often fatal metabolic disorder that leads to the breakdown of the protective barrier in the brain and nerve cells. Patients experience cognitive impairment, motor skill deterioration or death. Viking believes that the small molecule thyroid hormone beta receptor VK0214 can reduce levels of very long chain fatty acids that build up in plasma and are believed to contribute to the onset and progression of the disease.