Alnylam Pharmaceuticals Inc. (ALNY) has unveiled new insights from its Phase 1 clinical trial of nucresiran, formerly termed ALN-TTRsc04, a next-generation RNA interference (RNAi) therapy targeted at treating transthyretin (ATTR) amyloidosis. The findings were disclosed during an oral session at the American Heart Association Scientific Sessions 2024 in Chicago.
The results revealed that administering a single dose of nucresiran at 300 mg or higher resulted in rapid and consistent knockdown of serum transthyretin (TTR) levels across patients. On average, there was a reduction exceeding 90% from the baseline by Day 15, which was sustained through at least Day 180. At these dosage levels, maximum mean reductions in TTR levels surpassed 96% by Day 29.
Moreover, by Day 360, serum TTR levels remained significantly reduced, with over 70% mean reduction following a single 300 mg dose. Data for the 600 mg and 900 mg dose groups on Day 360 are pending. Thus far, all doses of nucresiran have been well-tolerated.
The ongoing Phase 1 study aims to determine the safety, pharmacodynamics, and pharmacokinetics of single doses of nucresiran in a population of healthy participants.