SAN FRANCISCO — Madrigal Pharmaceuticals will be “ready to go” if the FDA grants its nonalcoholic steatohepatitis hopeful an accelerated approval in March, the company’s new CEO Bill Sibold said Wednesday.

Resmetirom would be the first approved treatment in an area that’s seen failure after failure, including Intercept’s closely-watched FXR agonist obeticholic acid, which was rejected for a second time in June following safety concerns. Madrigal completed its submission shortly after Intercept’s CRL, though its candidate relies on a different mechanism of action known as thyroid hormone beta-receptor agonism.

“This is the veritable graveyard of drug development,” Sibold told investors at the JP Morgan Healthcare Conference. “We are planning for success.”

Sibold joined Madrigal in September from Sanofi, where he served as executive vice president of specialty care. Resmetirom falls under the specialty category, Sibold said Wednesday, adding that it will be marketed and priced as such. “This is a high unmet need and the product provides tremendous value,” he noted.

ICER reported in May that resmetirom would be cost-effective between $39,600 and $50,100 per year.

Madrigal will target an estimated 315,000 NASH patients in the US with significant fibrosis who are being seen by hepatologists and gastroenterologists. NASH, also referred to as metabolic dysfunction-associated steatohepatitis (MASH), is caused by fat buildup in the liver that leads to hepatic ballooning and inflammation and then fibrosis.

“One of the requisites of a great launch is having a great team, and that’s something that we’re well on the way to building,” Sibold said. “It’s no small feat when you’re scaling up a company like this, but we think that we’ve brought an experienced team in.”

While he doesn’t expect that a liver biopsy will be required for treatment, Sibold noted that it’s difficult to predict what the label will look like, considering patients have no other options. Experts don’t expect it to be that way for long as excitement builds around a number of potential treatments, including GLP-1s.

Weight loss in NASH patients is thought to be associated with reduced hepatic inflammation. Both Novo Nordisk and Eli Lilly are testing their respective blockbuster GLP-1 drugs, semaglutide and tirzepatide, in NASH. However, Leerink analysts wrote in a note following the annual NASH-TAG conference earlier this month that the class has “not yet demonstrated a convincing effect on improving fibrosis” in NASH patients.

When asked about the potential impact of GLP-1s on resmetirom treatment during a Q&A session, Sibold suggested that early-stage NASH, before there is fibrosis or significant fibrosis, may be “a reasonable place for a GLP-1 to play.”

“We think it’s important that any patient do whatever lifestyle modifications, etc., take what they need to to help them if they are obese, we’re certainly supportive of that,” Sibold said. “But we do believe in the population that we are prioritizing, which is these NASH with significant fibrosis, that they are in need of a liver-directed therapy quickly.”

Source:
https://endpts.com/jpm24-madrigal-says-its-well-on-the-way-to-potential-nash-launch-ahead-of-fda-decision/
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