Madrigal Pharmaceuticals

Neurocrine Biosciences, Madrigal Pharmaceuticals, and Vanda Therapeutics provided investors with key updates on their recent drug launches during Q1 earnings calls.

Madrigal Pharmaceuticals' resmetirom demonstrated significant efficacy in the Phase 3 MAESTRO-NASH trial, achieving both primary endpoints of NASH resolution and liver fibrosis improvement.

• French biotech Inventiva announces major restructuring, planning to lay off 50% of employees and discontinue all preclinical research except for its lead MASH candidate lanifibranor. • Company's Phase III trial for lanifibranor in MASH has achieved over 95% patient randomization, with top-line results expected in second half of 2026. • Strategic financial moves, including structured financing and milestone payments, combined with cost-cutting measures, expected to extend operations through Q3 2026.

Madrigal Pharmaceuticals reports Rezdiffra (resmetirom) demonstrates potential benefits in patients with compensated MASH cirrhosis (F4c).

Madrigal Pharmaceuticals has begun a rolling submission to the FDA for resmetirom, a potential treatment for NASH with liver fibrosis.

The FDA approved Madrigal's Rezdiffra, the first treatment for metabolic dysfunction-associated steatohepatitis (MASH), addressing a significant unmet need in liver disease.

• The FDA approved Rezdiffra (resmetirom) as the first specific treatment for nonalcoholic steatohepatitis (NASH), addressing a critical unmet need for patients with liver damage. • Duvyzat (givinostat) gained approval as the first nonsteroidal treatment for all genetic variants of Duchenne muscular dystrophy (DMD), offering a new option for patients aged 6 and older. • Cobenfy (xanomeline and trospium) was approved for schizophrenia, representing the first new class of antipsychotic medication in 35 years, with a novel mechanism of action. • Itovebi (inavolisib) received approval in combination with palbociclib and fulvestrant for certain types of breast cancer, demonstrating a 57% reduction in disease progression or death.

• Madrigal Pharmaceuticals' Rezdiffra, the first FDA-approved treatment for MASH, is experiencing rapid sales growth, with over 6,800 patients on treatment in Q3 2024. • Madrigal is actively engaging with specialist physicians and expanding its reach in the U.S. and anticipates potential EU approval for Rezdiffra by mid-2025. • A new clinical trial is underway to investigate Rezdiffra's efficacy in MASH patients with compensated cirrhosis, potentially opening up a new market segment.

• The non-alcoholic steatohepatitis (NASH) market is poised for substantial growth, projected to expand significantly by 2032 across the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan. • This growth is fueled by the introduction of novel therapies targeting NASH, with several drugs in Phase II and Phase III clinical trials showing promise for improving treatment outcomes. • Key players such as Madrigal Pharmaceuticals, Intercept Pharmaceuticals, and Novo Nordisk are actively developing innovative treatments, contributing to a dynamic and competitive market landscape. • The increasing prevalence of NASH and the growing understanding of its pathophysiology are driving the demand for effective therapies, creating opportunities for pharmaceutical companies.

The FDA has granted accelerated approval to Rezdiffra (resmetirom) for treating noncirrhotic NASH with moderate to advanced liver fibrosis.