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Madrigal touts a mid-stage success in HeFH, cutting bad cholesterol in a competitive field

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.

John Kastelein

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.”


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