ALN-HTT02

July 30, 2026: Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) reported Q2 2026 results and pipeline/regulatory updates. Q2 global net product revenues were $1.172B (+74% vs Q2 2025), driven by Total TTR revenues of $1.030B (+89%): AMVUTTRA (vutrisiran) $1.012B and ONPATTRO (patisiran) $18M. The company lowered full-year 2026 Total TTR net product revenue guidance to $4.2–$4.5B (from $4.4–$4.7B), citing moderated/normalized second-line demand in the U.S. ATTR-CM launch after initial pent-up demand.

Clinical/data catalysts: New subgroup analyses from the HELIOS-B Phase 3 trial in ATTR-CM were presented at Heart Failure 2026, showing maintained reductions in all-cause mortality and recurrent CV events across key subgroups; a pooled safety analysis (>25,000 patient-years) reported no clinically meaningful ocular effects from vitamin A lowering. Alnylam also shared the design of DemonsTTRate, a prospective real-world ATTR-CM observational study (expected >2,000 patients; up to 5 years follow-up).

Pipeline: Phase 2 initiated for ALN-6400 (plasminogen) in von Willebrand disease with heavy menstrual bleeding; Phase 1 initiated for ALN-6222 (INHBE) in obesity. Mivelsiran (APP) updates included completed enrollment in cAPPricorn-1 Phase 2 in CAA and initiation of a Phase 2 study in Down syndrome-associated Alzheimer’s; additional Phase 1 early-onset AD data were presented at AAIC 2026 (no increased ARIA risk; durable CSF biomarker reductions; up to 30 months exposure). ALN-5288 (MAPT) preclinical data and Phase 1 design were also presented at AAIC 2026.

Regulatory catalyst (partner): Regeneron announced FDA and EMA acceptance of regulatory applications for cemdisiran in anti-AChR+ generalized myasthenia gravis; FDA Priority Review with a November 2026 target action date, and an anticipated European Commission decision in 2H 2027.

Business development/upcoming: Exclusive agreement with BeOne Medicines to commercialize AMVUTTRA in China (subject to marketing authorization). Initial Phase 1 ALN-HTT02 Huntington’s disease results will be presented at EHDN on October 23, 2026; additional 2H 2026 readouts are expected for ALN-6400 (Phase 1/2) and ALN-2232 (Phase 1 obesity/weight management).