top of page

NEWS & INSIGHTS

Dive into the Biopharma Space

Stay up-to-date on major biopharma news, deeper dives into specific companies and biopharma themes, and how it all relates to biopharma partners.

Recent Advances in NASH Provide Opportunity for CROs and Clinical Sites

  • 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


Comentários


bottom of page