Madrigal Pharmaceuticals today released strongly positive results for its NASH drug Resmetirom which are far superior to what almost anyone expected.
Their strongly positive results were across the board for the pivotal MAESTRO-NASH study in patients with fibrotic NASH. Resmetirom achieved highly statistically significant results in two key primary endpoints with both daily oral doses, 80 mg and 100 mg, relative to placebo. Specifically:
- NASH resolution (ballooning of 0, inflammation of 0-1) and ≥2-point NAS reduction with no worsening of fibrosis
- Fibrosis improvement by at least one stage with no worsening of NAS at 80 and 100 mg, respectively)
These endpoints are the targets established by FDA for approval of drugs to treat NASH. Importantly, a drug need only meet one of these two choices to be considered for approval but Madrigal met both which makes them a superior candidate for registration.
If you would like to read a longer description of the results, here is a link to an article about the results as reported by STAT.
Resmetirom also demonstrated potentially clinically meaningful LDL-lowering, a key secondary endpoint. We believe this morning's results represent not only a stunning surprise, but also makes MAESTRO-NASH a landmark study for the whole field of NASH and the new bar for success. We believe resmetirom is poised to be the first foundational therapy approved for NASH, as a safe, once-daily oral therapy that addresses the pathogenic drivers of disease.
In other news, for those who may have missed it, we gave a demo of The Wellness League which will be the main patient outreach platform for us in the future. It is a short, 30 minute, description of our strategy for delivering patient support. Click the link below to watch it.
Patient care and therapies are changing fast and keeping you informed is an important part of our job.