Alnylam Pharmaceuticals, Inc.

What they do

Alnylam develops and commercializes RNAi-based medicines that silence disease-causing genes. The company has six approved products, including AMVUTTRA, ONPATTRO, GIVLAARI, and OXLUMO, with two commercialized by collaborators. Its research focuses on genetically validated targets and delivery across multiple tissue types, including liver, CNS, and eye.

Revenue drivers

• AMVUTTRA (vutrisiran) — Approved for ATTR amyloidosis with polyneuropathy and cardiomyopathy; generated $1,012 million in Q2 2026 global net product revenues, the largest contributor to total TTR revenues of $1,030 million.
• ONPATTRO (patisiran) — Approved for hATTR amyloidosis polyneuropathy; contributed $18 million in Q2 2026 global net product revenues, a small but ongoing legacy TTR product.
• Collaborator products (Leqvio, Qfitlia) — Commercialized by partners, generating royalties; Leqvio (inclisiran) and Qfitlia (fitusiran) are part of the portfolio, with Qfitlia mentioned in liquidity discussions.

Recent performance

Q2 2026 global net product revenues were $1,172 million, up 74% from Q2 2025. Total TTR revenues (AMVUTTRA plus ONPATTRO) were $1,030 million, up 89% year-over-year. Full-year 2025 revenue was $3.71 billion with net income of $313.7 million, the first profitable year. Q2 2026 revenue was $1.29 billion per the 10-Q. Cash and equivalents stood at $1.71 billion as of June 30, 2026.

Strategy

The company's Alnylam 2030 strategy targets 25%+ compound annual revenue growth through 2030 and a ~30% non-GAAP operating margin. It aims to build a durable TTR franchise, launch next-generation silencer nucresiran in ATTR amyloidosis by 2028-2030, and deliver 2+ new blockbuster medicines beyond TTR. It plans to expand to 10 tissue types and over 40 clinical programs, reinvesting ~30% of revenues in R&D. Recent moves include AI collaborations for discovery and commercial intelligence, and an exclusive agreement with BeOne Medicines for AMVUTTRA in China.

Risks

• AMVUTTRA revenue concentration — Substantial reliance on AMVUTTRA for growth; if sales underperform, business and stock price could be materially harmed.
• ATTR-CM launch dynamics — Management reduced 2026 TTR guidance due to normalized second-line volume after pent-up demand, indicating potential market growth uncertainty.
• Collaborator dependence — Leqvio and Qfitlia are commercialized by partners; if partners fail to perform, related revenues could be delayed or reduced.
• History of losses — While profitable in 2025, the company had significant losses in prior years and may not sustain profitability given heavy R&D investment.

Outlook

Management revised full-year 2026 TTR net product revenue guidance down from $4,400-$4,700 million to $4,200-$4,500 million, representing about 75% growth at the midpoint. The company expects continued ATTR-CM launch growth, with new pipeline data expected later in 2026. Cash is expected to be sufficient for at least 12 months from the 10-Q filing date.