(Bloomberg) — Pfizer Inc. said that results from a midstage, six-week study of its drug that prevents the body from metabolizing fructose, a natural sugar found in fruits and vegetables, showed a statistically significant improvement in liver fat.
The medication, an inhibitor of the fructokinase enzyme, could reverse or prevent the progression of the liver disease nonalcoholic steatohepatitis, known as NASH, and perhaps even treat diabetes, according to the company.
“We were pleasantly surprised by the results,” said Pfizer Senior Vice President Morris Birnbaum, who leads the New York company’s internal medicine research division. “Beyond improving liver fat, we think we’re working with a mechanism that can be used more broadly to treat metabolic abnormalities.”
Fructose, used to sweeten sodas and other foods and drinks, has long been the bane of doctors and public-health officials seeking to curb the growing epidemics of obesity, diabetes and liver disease. Equipped with the new data, Pfizer will explore treating those conditions by targeting how the body processes the sugar.
Pfizer’s trial looked at 53 patients who had high levels of fat in their livers, of whom nearly a third had mild diabetes. Those who got the highest dose of the drug saw an average 26.5 percent reduction in liver fat. Patients who got a placebo saw liver fat decline by 7.8 percent.
Results from a lower dose, which didn’t entirely inhibit the metabolism of fructose, weren’t statistically significant. Pfizer said there weren’t any meaningful side effects. Pfizer plans to present the data at a medical meeting next month.
NASH, which affects an estimated 3 percent to 12 percent of all Americans, doesn’t currently have pharmaceutical treatments on the market. Current treatments for the disease, which is difficult to diagnose, include weight loss, diet and exercise. GlobalData Plc, a research firm, has predicted that the market for the drugs could reach $18 billion by 2026.
Unlike many competitors who are seeking to get into the NASH market by treating fibrosis in the liver, Pfizer is aiming at how the body processes nutrients.
“Our underlying hypothesis is that if we address liver fat in the NASH population, it will have a positive effect in improving their fibrosis,” said Birnbaum.
Last October, Pfizer and Novartis AG announced a clinical collaboration to evaluate combination therapies for NASH. Based on Wednesday’s data, Birnbaum said the fructokinase inhibitor could potentially be combined with one of Novartis’ antifibrotic drugs in a future study.
Birnbaum said the study also found a surprising, statistically significant reduction in insulin resistance. The company will study whether the drug could be used for NASH, diabetes, or perhaps both diseases.
“We’re hopeful that it’s going to be unusual and affect multiple components related to the metabolic system,” Birnbaum said.
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