Madrigal Pharmaceuticals $MDGL is touting a batch of positive mid-stage data from their 12-week LDL/C-lowering trial in treatment-resistant patients with heterozygous familial hypercholesterolemia (HeFH), an intensely competitive field with a roster of players looking to make their mark.
In the study, investigators say that an “optimal” dose of MGL-3196 spurred cuts of 18.8% and 21%. The company adds:
LDL-C lowering was 28.5% in MGL-3196 treated compared to placebo in a prespecified group of patients who did not tolerate high intensity statin doses.
That, they add, could have a significant role in lowering cardio risks in this genetically defined group of patients — though they are a long way from proving that. The drug is also being developed for NASH.
There’s an intense rivalry for the best data here, as Gemphire $GEMP learned to its peril last summer when their drug gemcabene, in-licensed from Pfizer, was linked to a 17% reduction in bad LDL levels, significantly less than the 23% to 28% reductions that Pfizer had been tracking before outlicensing the drug to the Livonia, MI-based company. The Medicines Company $MDCO is also at work on a big Phase III with inclisiran, inlicensed from Alnylam, after seeing dramatic reductions in Phase II. And Amgen has been just as bullish after posting big cuts to LDL-C for Repatha, which is already on the market.
Underscoring just how hard it is to please investors on this front, Madrigal’s shares slid initially during pre-market trading, after the data was posted.
“These Phase II results confirm the potential of MGL-3196 to safely lower LDL cholesterol in high-risk HeFH patients whose disease is not well-controlled, despite the use of maximally tolerated lipid-lowering therapies,” says principal investigator John Kastelein, a professor at the University of Amsterdam. “There is clearly an unmet need for additional therapeutic options for these patients; based on the results of this study, MGL-3196 has the potential to offer one such option.”
The best place to read Endpoints News? In your inbox.
Comprehensive daily news report for those who discover, develop, and market drugs. Join 31,500+ biopharma pros who read Endpoints News by email every day.Free Subscription