Louise Campbell starts this discussion by citing her sources of optimism about NASH drug development: (i) the high likelihood of getting a drug to market over the next couple of years, which will lead to better education of patients and providers alike; (ii) FDA looking for more inclusion and diversity about drug trials, which will provide even more information for historically underserved populations; and (iii) in her observations and work, patients already are becoming more aware about Fatty Liver disease and the importance of treating it.
From there, the conversation turns to consider what drug development has proven already. Jörn Scattenberg notes that even if OCA never receives approval, it has proven that regression in monotherapy is possible. Roger Green expands on this thought, noting that OCA had demonstrated efficacy and resmetirom appears to have demonstrated safety in the liver. More important to him, each drug has the prospect of proving both efficacy and safety within the next year or so. Roger goes on to note that if combination therapy is to become the ultimate strategy in an environment where so many MOAs exist, this will provide continued research funding and energy for the foreseeable future.
Jörn brings Quality of Life into the discussion as an approvable endpoint of extreme interest to patients that will become the “third pillar” of drug assessment.
These comments and the answers to Roger’s closing question confirm that the Surfers believe there are sound reasons to be optimistic about drug development in the mid-term and quite possibly the short-term as well.
This episode and all its conversations are sponsored by Madrigal Pharmaceuticals. Conversations 25.5 and 25.6 are a two-part summary of Madrigal’s disease-focused presentation at the recent CLDF LiverConnect meeting.