Results of another drug trial for PSC have now been published following preliminary results announced last year
These results are particularly interesting because although the NGM282 PSC drug did not improve alkaline phosphatase(ALP), it did improve markers of fibrosis turnover and hepatic inflammation.
Traditionally, PSC drug trials have tended to use a reduction in ALP as an indication of effectiveness.
We hope that future trials in PSC continue to measure alternative ways to demonstrate effectiveness and that they embrace emerging non-invasive technologies to do so.
"We present for the first time, the clinical and laboratory effects of a first-in-class, engineered analogue of the endocrine hormone FGF19 in patients with PSC. By incorporating non-invasive markers of fibrosis, beyond standard liver injury markers, we show that NGM282 impacted on fibrosis turnover and hepatic inflammation without changing alkaline phosphatase. Our findings demonstrate the complexities of using highly potent rational agents in PSC, and furthermore challenge the dogma about what the appropriate endpoints should be for trials in PSC."