Alnylam Pharmaceuticals

Alnylam Pharmaceuticals' Phase I trial of nucresiran, an RNAi therapeutic for ATTR amyloidosis, showed significant serum TTR level reductions after a single dose, with sustained effects over time and low inter-patient variability. The study evaluated safety, pharmacokinetics, and pharmacodynamics in healthy individuals, with most adverse events being mild and unrelated to the treatment. Nucresiran is part of the IKARIA platform, aiming for deeper and more durable TTR knockdown, potentially allowing for less frequent dosing. Phase III development plans are anticipated for Q1 2025.

Nucresiran, an RNAi therapeutic for ATTR amyloidosis, showed rapid TTR knockdown of >90% by Day 15 and >96% by Day 29, with potential for biannual or annual dosing. The drug was well-tolerated, with plans for Phase 3 development in Q1 2025.

Alnylam's Phase 1 data for nucresiran shows single doses ≥300mg achieve >90% TTR reduction by Day 15, sustained through Day 180, with peak reductions >96% by Day 29. At 300mg, TTR reduction remains >70% at Day 360, supporting potential biannual or annual dosing.

MARCH Phase 2 study shows 39% and 46% HBsAg loss in low baseline HBsAg participants with tobevibart + elebsiran and tobevibart + elebsiran + PEG-IFNα, respectively. No new safety concerns; TEAEs mild to moderate. Functional cure data expected Q2 2025.

Timmerman Report celebrates its 10th anniversary with events in Boston (Mar. 6) and Seattle (Mar. 13), featuring biotech leaders discussing future trends in biopharma.

FDA places clinical hold on Rapport Therapeutics' phase 2a trial for RAP-219 in diabetic peripheral neuropathic pain; Alnylam Pharmaceuticals to assess mivelsiran in cAPPricorn-1 for cerebral amyloid angiopathy; FDA accepts PTC Therapeutics' resubmitted NDA for ataluren in nonsense mutation Duchenne muscular dystrophy.

Trump's presidency may bring changes to federal health agencies, with potential influence from Robert F. Kennedy Jr. on FDA policies. The drug industry is concerned about Kennedy's role, while biotech leaders are optimistic about reduced scrutiny of mergers and acquisitions under the FTC. Trump's potential policies on drug pricing and U.S. firms' ties to China are also under scrutiny.