Dive Brief:
- A pill to treat the liver disease MASH developed by Viking Therapeutics maintained its benefit over a year of treatment, reducing fibrosis and resolving inflammation in significantly more study participants than among those given placebo in a Phase 2 trial, the company said Tuesday.
- The assessment confirmed an earlier finding taken after 12 weeks of treatment with the drug, dubbed VK2809, which was the study’s primary endpoint. The 52-week results were based on a liver biopsy, while at 12 weeks investigators measured improvement using magnetic resonance imaging.
- The data come just months after the Food and Drug Administration granted approval for the first drug for the disease, Madrigal Pharmaceuticals’ Rezdiffra. While Madrigal launches that pill, which works the same way as VK2809, Viking is still years away from the market. The company is also now devoting significant resources to a weight loss drug.
Dive Insight:
After years of fitful development, the approval of the first drug for MASH was a milestone for a condition that some experts believe will grow hand-in-hand with obesity prevalence. Short for metabolic dysfunction-associated steatohepatitis, MASH was previously referred to as NASH, or non-alcoholic steatohepatitis.
Another liver disease drug developed by Intercept Pharmaceuticals had been hailed as a potential breakthrough based on the findings of a Phase 2 trial, but the FDA rejected it twice over safety risks.
Madrigal and Viking have homed in on a different target, called the thyroid hormone beta receptor, which when activated, helps regulate fat and glucose metabolism.
Viking tested its pill at four doses between 1 and 10 milligrams, either once daily or every other day. Grouped together, 69% of people who got VK2809 saw their NASH resolve without any change in fibrosis scores, significantly more than the 29% of people who received placebo. Of the participants who got VK2809, 51% had an improvement in fibrosis scores with no worsening of NASH and 44% experienced improvement in both, compared with 34% and 20% of placebo patients, respectively.
Viking also shared a more conservative analysis that classified participants with missing data as “non-responders.” A smaller share of participants met each of the three criteria on this score.
In both cases, some individual dose groups didn't show a statistically significant difference versus the comparator placebo group. The data did, however, meet the expectations set by Jefferies analyst Roger Song in an April note to clients.
Study participants taking VK2809 had similar side effect rates to those taking a placebo and, at most doses, levels of liver enzymes were significantly reduced, Viking said.
Viking shares fell by double digits in morning trading Tuesday.
In a June 2 note to clients, Raymond James analyst Steven Seedhouse wrote that Viking will likely next meet with FDA staff to map out a possible Phase 3 trial. He suggested the company may seek a licensing partner to help pay for that trial along with potential regulatory submissions and commercialization.