Alnylam/Regeneron-Backed NASH Candidate Shows Encouraging Safety Profile In Healthy Volunteers

  • Alnylam Pharmaceuticals Inc ALNY and Regeneron Pharmaceuticals Inc REGN announced preliminary Phase 1 data supporting the clinical advancement of ALN-HSD for nonalcoholic steatohepatitis (NASH).
  • ALN-HSD is an investigational RNAi therapeutic targeting HSD17B13.
  • After single-dose evaluation in healthy adult volunteers (Part A), multiple doses of ALN-HSD are being studied in adult patients with NASH (Part B). 
  • Patients in the first two Part B cohorts (200 and 400 mg quarterly) have completed at least six months on the study.
  • The remaining cohorts explore a lower dose or a later biopsy time point. 
  • Related: Alnylam Posts Mid-Stage Study Data From Regeneron-Partnered Kidney Disease Drug.
  • In the first two Part B cohorts, ALN-HSD was associated with robust target knockdown and numerically lower liver enzymes and nonalcoholic fatty liver disease Activity Score over six months in patients receiving ALN-HSD (N=20) relative to placebo (N=4). 
  • The study was not powered to achieve statistical significance on these endpoints, and the primary outcome measure is the frequency of adverse events. 
  • ALN-HSD has exhibited an encouraging safety and tolerability profile.
  • The most common treatment-emergent adverse event in healthy subjects treated with ALN-HSD (N=44) was injection site reaction.
  • The companies plan to initiate a Phase 2 study in adult patients with NASH in late 2022.
  • Price Action: REGN shares are down 0.58% at $693.60, and ALNY is down 0.61% at $207.00 on the last check Thursday.
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