Biopsy data confirm Viking MASH drug's benefit

Viking Therapeutics has announced promising results from a Phase 2 trial of their liver disease medication, VK2809, which targets metabolic dysfunction-associated steatohepatitis (MASH). The study evaluated VK2809 over a 52-week period and demonstrated significant benefits compared to a placebo in reducing fibrosis and resolving inflammation among patients.

VK2809's efficacy was initially observed after 12 weeks of treatment, meeting the primary endpoint of the study. The longer-term, 52-week assessment, which was based on liver biopsy results, confirmed these benefits. This comes shortly after the approval of the first MASH drug, Rezdiffra by Madrigal Pharmaceuticals, highlighting the emerging interest and progress in treating this liver condition.

MASH, previously known as non-alcoholic steatohepatitis (NASH), is a liver disease often associated with obesity and metabolic syndrome. The development of effective treatments has been challenging; for example, a drug from Intercept Pharmaceuticals was rejected twice by the FDA due to safety concerns. However, both Madrigal and Viking have focused on targeting the thyroid hormone beta receptor, which plays a crucial role in regulating fat and glucose metabolism.

In the trial, VK2809 was tested at several doses (between 1 and 10 milligrams), administered either daily or every other day. The trial results were impressive: 69% of patients treated with VK2809 saw their NASH symptoms resolve without worsening fibrosis, compared to just 29% in the placebo group. Moreover, 51% of VK2809 recipients showed improvement in fibrosis scores without worsening NASH, and 44% improved in both fibrosis and NASH, compared to 34% and 20% in the placebo group, respectively.

A more conservative analysis, which considered participants with missing data as non-responders, showed fewer participants meeting the improvement criteria, but the overall positive trend remained. Despite some dosage groups not showing statistically significant differences when compared to the placebo, the trial results were consistent with expectations set by analysts.

Regarding safety, side effect rates for VK2809 were comparable to those for the placebo. Additionally, most doses resulted in significant reductions in liver enzyme levels, indicating improved liver function.

Despite the promising results, Viking Therapeutics is still some years away from bringing VK2809 to market. The company is expected to meet with the FDA to discuss the possibility of a Phase 3 trial. Analyst Steven Seedhouse from Raymond James suggested that Viking might seek a licensing partner to help with the costs of the trial, regulatory submissions, and eventual commercialization.

The trial results led to a significant drop in Viking's share price, reflecting investor caution despite the positive data. The company's focus is now divided between VK2809 and another investigational drug aimed at weight loss, which further complicates its path forward.

These developments underscore the importance of innovative therapies for MASH, a condition projected to rise alongside obesity rates. While Madrigal's Rezdiffra is now available, VK2809 represents a potential future option for patients, contingent on further successful trials and regulatory approval.

In summary, Viking Therapeutics' VK2809 has shown significant promise in treating MASH by improving liver fibrosis and resolving inflammation in a majority of patients over a year-long period. The path ahead includes additional trials and potential strategic partnerships to bring this treatment to market.