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Dive into the Biopharma Space

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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


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