Madrigal Pharmaceuticals Inc. said its drug MGL-3196 reduced liver fat and resolved disease symptoms in patients with a certain liver disease in a mid-stage clinical trial.
The phase 2 study enrolled 125 adults with confirmed nonalcoholic steatohepatitis, or NASH, with patients randomized to receive either the drug or placebo.
NASH is a common liver disease that does not have any U.S. Food and Drug Administration-approved treatment. It is characterized by a build-up of fat in the liver, inflammation, damage of hepatocytes and increasing scarring, and it can lead to permanent damage in a high percentage of patients.
Between weeks 12 and 36, four placebo patients discontinued treatment and four patients on the drug dropped the therapy, while one patient had a repeat liver biopsy that was inadequate at week 36.
At week 36, out of the 73 NASH patients receiving the treatment, 56% saw a decrease in nonalcoholic fatty liver disease, or NAFLD, activity and 27% had symptoms of their disease resolved. NAFLD can refer to a group of conditions characterized by the accumulation of excess fat in the liver of people who drink little or no alcohol.
For the 34 patients on placebo, 32% showed reduced NAFLD activity and only 6% saw a resolution of NASH symptoms.
Out of 46 patients on the drug who had a fat reduction of 30% or more at week 12, 39% saw a resolution of NASH symptoms at week 36 and 70% of the patients showed a decrease in NAFLD activity.
On the secondary endpoints, the drug demonstrated statistically significant reductions in bad cholesterol, liver enzymes, and thickening and scarring of connective tissues in the liver. On liver biopsy, fibrosis or scarring was reduced by at least one point in 23% of placebo and 29% of MGL-3196 treated patients.
"The degree of NASH resolution, an approvable FDA endpoint, in patients who received MGL-3196 for 9 months we believe suggests a high likelihood of success in a larger trial with a somewhat longer treatment period in a phase 3 study designed similarly to this phase 2 study, pending regulatory agreement with such a design," said CEO Paul Friedman.
Friedman added that the drug has a potential to resolve NASH in as little as nine months in 30% to 40% of patients receiving only MGL-3196.
Madrigal's stock price was up 146.07% to $266.82 as of 3 p.m. ET on May 31 following the news.
