Alnylam/Regeneron say early-stage data support Phase 2 trial for NASH therapy
Benito Vega

Alnylam (ALNY) and Regeneron (NASDAQ:REGN) announced Thursday that early Phase 1 data for ALN-HSD, an experimental therapy for liver disease nonalcoholic steatohepatitis (NASH), supported their plans to begin a Phase 2 trial for the candidate later this year.
The companies have completed the Part A of the Phase 1 study, which involved healthy adult volunteers.
In Part B of the trial involving adult NASH patients, the first two cohorts have completed at least six months on the study. The remaining cohorts are under investigation for a lower dose or a later biopsy time point.
The companies said that in the trial, the main goal of which was the frequency of adverse events, ALN-HSD demonstrated an encouraging safety and tolerability profile so far.
In 44 healthy adults who received the therapy, five patients reported the commonest treatment-emergent adverse event of mild injection site reaction. There were no treatment-related serious adverse events.
In the first two Part B cohorts, the patients who received ALN-HSD (N=20) were found to have a more robust target knockdown, lower liver enzymes, and biopsy-derived nonalcoholic fatty liver disease (NAFLD) Activity Score compared to those who received placebo (N=4).
ALN-HSD is a subcutaneously delivered RNAi therapeutic developed under a collaboration ALNY and REGN announced in 2018 to identify treatments for NASH and related disorders.