Alnylam Pharmaceuticals (Nasdaq: ALNY) presented encouraging Phase 1 results for nucresiran (formerly ALN-TTRsc04), a next-generation RNAi therapeutic for transthyretin (ATTR) amyloidosis, at the American Heart Association Scientific Sessions 2024 in Chicago.
The data revealed that a single dose of nucresiran at 300 mg or higher led to rapid and sustained reductions in serum TTR levels. At Day 15, mean TTR reductions exceeded 90%, peaking at over 96% by Day 29, and remained above 90% through Day 180. By Day 360, the 300 mg dose showed a mean TTR reduction of over 70%, with data from higher doses (600 mg and 900 mg) still pending. All doses demonstrated low variability between patients and were well tolerated, with no serious side effects or safety signals reported.
Dr. Pushkal Garg, Alnylam’s Chief Medical Officer, highlighted the potential of nucresiran to enable biannual or annual dosing, calling it a “new paradigm” in ATTR amyloidosis treatment.
Phase 1 Study Overview:
The trial evaluated safety, tolerability, and pharmacokinetics in 48 healthy adults, randomized to receive doses ranging from 5 mg to 900 mg or placebo. The drug’s safety profile was excellent, with most adverse events mild and unrelated to treatment.
Alnylam plans to announce Phase 3 development plans in early 2025.
