ALNYLAM PHARMACEUTICALS, INC. (ALNY)

Alnylam Pharmaceuticals is a global commercial-stage biopharmaceutical company that develops therapeutics based on RNA interference (RNAi). Founded in 2002, it has pioneered a class of medicines built on small interfering RNA (siRNA), which silences messenger RNA that encodes disease-implicated proteins and so prevents those proteins from being made. Its strategy is to target genetically validated genes in indications with high unmet need, early biomarkers for assessing clinical activity, and a definable path to approval and commercialization. In early 2026 it launched its Alnylam 2030 strategy, which aims for leadership in transthyretin (TTR) revenue, two or more new medicines beyond TTR, expansion to 10 tissue types and more than 40 clinical programs, 25%+ revenue growth, and about a 30% non-GAAP operating margin.

The company has six approved first-in-class RNAi medicines and more than 20 clinical programs. AMVUTTRA (vutrisiran) is approved for ATTR amyloidosis with polyneuropathy and cardiomyopathy in the U.S., the EU, the UK, Japan and other countries. ONPATTRO (patisiran) is approved for hATTR amyloidosis with polyneuropathy. GIVLAARI (givosiran) treats acute hepatic porphyria. OXLUMO (lumasiran) treats primary hyperoxaluria type 1. Two further products are commercialized by collaborators: Leqvio (inclisiran), from Novartis, for hypercholesterolemia, and Qfitlia (fitusiran), from Sanofi, for hemophilia A or B. The pipeline includes nucresiran, a next-generation TTR silencer in Phase 3; zilebesiran for hypertension, in Phase 3 under a collaboration with Roche; mivelsiran for cerebral amyloid angiopathy and Alzheimer's disease; and cemdisiran, which Regeneron is advancing in Phase 3 trials for complement-mediated diseases. Delivery platforms include GalNAc conjugates and lipid nanoparticles for the liver, and C16 conjugates for the central nervous system and eye. Work is also underway on delivery to heart, muscle and adipose tissue, and on the IKARIA and GEMINI technologies.

Its products serve patients with rare and prevalent diseases across the U.S., Europe, Japan, Brazil, Canada and other markets. The company estimates that ATTR amyloidosis with cardiomyopathy affects more than 300,000 people worldwide and hATTR amyloidosis with polyneuropathy fewer than 30,000. Collaborators include Novartis, Sanofi, Roche, Regeneron and PeptiDream. It also has six genetics collaborations, including with the UK Biobank and Our Future Health, to source genetically validated targets.

On financing, in September 2025 the company issued $661.3 million of 0.00% Convertible Senior Notes due 2028 and used part of the proceeds to repurchase most of its 1.00% convertible notes due 2027, leaving about $362.8 million outstanding. It also entered a $500.0 million revolving credit facility with Bank of America as agent, maturing in September 2030, with no borrowings outstanding at December 31, 2025.