A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH)

Phase 1 Terminated 6 enrolled Alnylam Pharmaceuticals

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The purpose of this study is to evaluate the safety and tolerability of single ascending doses of ALN-HSD in healthy participants (Part A) and multiple doses of ALN-HSD in patients with NASH (Parts B and C).

Timeline

Start: 2020-10-09
Primary completion: 2023-01-06
Completion: 2023-12-21

Outcome

Results reported · no primary-endpoint met/missed verdict (typical of a dose-finding / safety study)

Stopped (Business): “Study ALN-HSD-001 was terminated due to business reasons and changes in the development strategy of the product. No safety issues were found.”

release “The study was not powered to achieve statistical significance on these endpoints, and the primary outcome measure is frequency of adverse events.” regeneron.com ↗

Publications

Badri P, Kolachana K, Duong A, Lasko M, Nandi T, Mehrotra N, Robbie GJ. Platform Assessment of Concentration-QTc Relationship Across GalNAc-siRNA Molecules. Clin Pharmacokinet. 2026 Feb;65(2):293-311. doi: 10.1007/s40262-025-01606-0. Epub 2025 Dec 12.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	RAPIROSIRAN	Small interfering RNA (siRNA)	—	Subcutaneous