Alnylam Pharmaceuticals (NASDAQ: ALNY) presented Saturday August 29 and Sunday August 30, 2026 late-breaking prespecified subgroup analyses from the Phase 3 HELIOS-B trial of vutrisiran (AMVUTTRA, an RNAi therapeutic targeting transthyretin for transthyretin amyloidosis with cardiomyopathy, ATTR-CM) at ESC Congress 2026 Munich. Vutrisiran demonstrated consistent clinical benefit across all-cause mortality and recurrent cardiovascular events in patients with or without background tafamidis (the transthyretin tetramer stabilizer marketed as Vyndaqel/Vyndamax by Pfizer). The HELIOS-B trial had documented 28.2% reduction in all-cause mortality and 32.8% reduction in cardiovascular events with vutrisiran plus stabilizer. Additional analyses examined healthy aging, functional capacity, safety, and outcomes by sex. Alnylam also presented Amylo'ExTTRa, a large real-world analysis from the French National Health Data System characterizing the multisystem burden of ATTR-CM beyond cardiac manifestations. Vutrisiran anchors Alnylam's ATTR franchise alongside inclisiran (Leqvio, licensed to Novartis for hypercholesterolemia) and zilebesiran (Phase 3 ZENITH trial in hypertension).
Alnylam Pharmaceuticals (NASDAQ: ALNY) shares extended a roughly 30% selloff since the Thursday July 30, 2026 Q2 2026 earnings report, losing approximately $12 billion in market capitalization over the intervening days. The selloff reflects two consecutive negative signals for the RNAi therapeutics leader: the July 30 Amvuttra (vutrisiran, the TTR-directed siRNA that crossed $1 billion in quarterly revenue for the first time at $1.012 billion) full-year 2026 TTR product sales guidance cut to $4.2-4.5 billion (down from prior higher expectations) to reflect normalized second-line volume after the initial pent-up demand from patients waiting for a new therapy, combined with the July AstraZeneca-Ionis eplontersen (Wainua, antisense oligonucleotide) Phase 3 CARDIO-TTRansform study setback that complicates the broader ATTR-CM competitive landscape. The Amvuttra franchise has been positioned as the primary siRNA alternative to Pfizer's Vyndaqel/Vyndamax (tafamidis, small-molecule TTR stabilizer) and BridgeBio's Attruby (acoramidis, next-generation TTR stabilizer). Alnylam presents full eplontersen study data at a late-August medical meeting; analysts flagged the timing as a continued overhang until the presentation clarifies the ATTR-CM segment dynamics. Analyst commentary described the combination as a 'one-two punch' for the company.
Alnylam Pharmaceuticals (NASDAQ: ALNY) reported Q2 2026 earnings Thursday July 30, 2026 before market open. Amvuttra (vutrisiran) quarterly revenue crossed $1 billion for the first time at $1.012 billion (approximately a $4 billion annualized run rate approximately 15 months after the ATTR-CM launch). Total Q2 revenue reached $1.29 billion (+67% year-over-year) but fell short of the $1.31 billion analyst consensus. Net product revenues were $1.17 billion (+74% YoY) versus the $1.22 billion estimate. GAAP diluted EPS was $1.21 versus the $1.49 estimate. Full-year 2026 TTR product sales guidance was revised down to $4.2-4.5 billion to reflect normalized second-line volume after the initial pent-up demand from patients waiting for a new therapy. Pipeline updates: Alnylam expects to announce clinical data from Phase 1 and Phase 2 trials of ALN-6400 in healthy volunteers and patients with hereditary hemorrhagic telangiectasia (HHT) in second-half 2026, plus results from the Phase 1 trial of ALN-2232 (Alnylam's second obesity-track siRNA) in the same second-half window. The ALN-6222 obesity siRNA Phase 1 trial (NCT07624071) opened enrollment in July 2026 with a first-in-human single-ascending-dose design in adults with BMI 30-40 kg/m² at a single site in Mount Royal, Canada through December 2027.
AstraZeneca and Ionis Pharmaceuticals announced Thursday July 9, 2026 that the Phase 3 CARDIO-TTRansform trial of Wainua (eplontersen) did not meet its primary efficacy endpoint in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). The trial enrolled over 1,400 patients and was the largest Phase 3 study conducted in the ATTR-CM population to date; the primary endpoint was a composite of cardiovascular mortality and recurrent CV clinical events through 140 weeks compared with placebo. Prespecified subgroup analyses showed nominally significant reductions in composite events with eplontersen monotherapy versus placebo, while patients also receiving a baseline TTR stabilizer showed no incremental effect. Eplontersen is an antisense oligonucleotide (a nucleic-acid therapeutic modality distinct from peptides but adjacent in the metabolic-and-cardiovascular therapeutic territory this site tracks); the drug is already FDA-approved as Wainua for hereditary transthyretin amyloidosis polyneuropathy. Full CARDIO-TTRansform results will be presented at the European Society of Cardiology (ESC) Congress in August 2026. AstraZeneca and Ionis shares fell on the readout.