Non-alcohol related fatty liver disease (NAFLD) is a global health challenge
In the U.S. alone there are ~94 million cases
NASH is a highly prevalent advanced form of NAFLD
Over 26M cases in U.S. alone
No approved treatments for NASH in U.S.
But recent progress (over past year) has reignited area
After years of failures, 3 mechanisms of action (MOA) appear be effective
Based on surrogate markers
FXR agonists
But might have too many AEs
ICPT, TERN
THR-beta agonists
Look promising
MDGL, VKTX, TERN
FGF-21 analogs
Look promising
AKRO, ETNB
NASH trials are difficult
Difficult to recruit
Many NASH trials require liver biopsies
Difficult procedure with risks
Keeps many NASH patients out of NASH trials
Viking (VKTG) delayed 2B readout and stopped 2 cohorts due to recruitment issues
Many trials are very large and take years
Intercept (ICPT) announced it won't continue to clinical outcomes if FDA rejects accelerated approval again
But recent promising results & huge market opportunity will drive many more clinical trials
Many large (global), lengthy clincal trials need to be run
E.g., sponsors include publicly traded mid and small caps such as TERN, VKTX, AKRO, and ETNB
Likely more ex-US sites will be needed
MDGL-Resmiterom may get accelerated approval by U.S. FDA
Possible for late 2023/early 2024
May make it more difficult to recruit NASH patients into U.S. clinical trials
Non-invasive testing (NIT) can be used for some NASH trials
Thus, no need for liver biopsy
Can be used for large safety trials that are needed
As 2' trials to help establish efficacy
See Madgrigal's (MDGL) MAESTRO-NAFLD-1 for safety database
NIT only
>1200 patient, 52 wk global trial for safety database
>700 patients in addtnl 52 wk extension trial
This article is not legal, tax, or investment advice. Originally published 5/20/23 EJV
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