Madrigal Claims To Solve Genetic Cholesterol Disorders With Drug, NASH


Madrigal Pharma is very excited about the release of their new drug, MGL-3196. It performed well in a phase 2 trail in the second of its two target signs: heterozygous familial hypercholesterolemia (HeFH).

The share price of this biotech has tripled within the last three months, and this is due to the anticipated success of MGL-3196. MGL-3196 was in lead indication nonalcoholic steatohepatitis (NASH). Its stock continued to increase another three points and it is now up to $146 in after hours trading on the Nasdaq.

NASH is said to be the bigger opportunity, but MGL-3196 is a first-in-class liver-directed thyroid hormone receptor beta-selective agonist. Originally it had been developed by Roche, and hitting the goal in HeFH could allow for the beginning of another market for the once-daily pill.

A recent trial showed that Madrigal’s drug achieved a massive reduction in LDL cholesterol compared to those who took the placebo pill after about a three month treatment period. There has recently been improvements with other biomarkers including
triglyceride levels, apolipoprotein B and lipoprotein(a).

The experiment called for 116 Europeans that had HeFH and were taking some sort of current treatment that wasn’t working for them. Madrigal’s drug was successful in having 18.8% LDL cholesterol declines compared to those that took the placebo. Patients that were on the optimal dose saw an even greater reduction of 21%.

Some of the patients were unable to tolerate the high-dose statins. This caused an even greater different between the MGL-3196 and the placebo. It was recorded at almost 29%.

The CEO of Madrigal, Paul Friedman, said that he was very pleased with how safe and efficient the MGL-3196 in HeFH was. The data also showed the positive role of MGL-3196 in NASH.

Analysts were told that the company now has “multiple shots on goal” with the drug, but the company isn’t ready to get on route to market due to the fact that Madrigal were positive in their trails for NASH and HeFH.

HeFH is a very common diseases and it can be inherited, it is a bit more rare considering that its present in 1 out of 200 people, but creating the drug for other cholesterol disorders could allow for an even greater market.


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