Alnylam Pharmaceuticals Inc
NASDAQ:ALNY
Alnylam Pharmaceuticals Inc
In the heart of the biotechnology revolution, Alnylam Pharmaceuticals Inc. has been a pioneering force, transforming the landscape of medicine through its innovative use of RNA interference (RNAi) technology. Founded in 2002 and based in Cambridge, Massachusetts, the company set out on a bold mission to silence disease-causing genes by targeting RNA, the intermediary that carries genetic instructions necessary for protein synthesis. This groundbreaking approach diverges from traditional methods that focus on targeting proteins directly and instead intervenes earlier in the disease process to eliminate or reduce the proteins before they can wreak havoc on the body. Fueled by extensive research and a robust pipeline, Alnylam has successfully brought several RNAi-based therapies to market, such as Onpattro and Givlaari, which address rare genetic disorders with high unmet medical needs.
Alnylam's business model is centered around the commercial success of its novel therapies, partnerships with other pharmaceutical giants, and strategic collaborations for global distribution. By securing alliances with industry leaders like Sanofi and Regeneron, Alnylam has been able to expand its research capabilities and broaden its market reach. Revenue for the company is primarily driven by the sales of its approved products, which continue to grow as they gain acceptance within the medical community. Additionally, Alnylam's extensive patent portfolio and its strategic licensing agreements provide an additional revenue stream, safeguarding its innovations and ensuring it retains a competitive edge in this rapidly advancing field. Through its relentless pursuit of innovation, Alnylam continues to position itself as a trailblazer in turning the once theoretical promise of RNAi into a clinical reality.
In the heart of the biotechnology revolution, Alnylam Pharmaceuticals Inc. has been a pioneering force, transforming the landscape of medicine through its innovative use of RNA interference (RNAi) technology. Founded in 2002 and based in Cambridge, Massachusetts, the company set out on a bold mission to silence disease-causing genes by targeting RNA, the intermediary that carries genetic instructions necessary for protein synthesis. This groundbreaking approach diverges from traditional methods that focus on targeting proteins directly and instead intervenes earlier in the disease process to eliminate or reduce the proteins before they can wreak havoc on the body. Fueled by extensive research and a robust pipeline, Alnylam has successfully brought several RNAi-based therapies to market, such as Onpattro and Givlaari, which address rare genetic disorders with high unmet medical needs.
Alnylam's business model is centered around the commercial success of its novel therapies, partnerships with other pharmaceutical giants, and strategic collaborations for global distribution. By securing alliances with industry leaders like Sanofi and Regeneron, Alnylam has been able to expand its research capabilities and broaden its market reach. Revenue for the company is primarily driven by the sales of its approved products, which continue to grow as they gain acceptance within the medical community. Additionally, Alnylam's extensive patent portfolio and its strategic licensing agreements provide an additional revenue stream, safeguarding its innovations and ensuring it retains a competitive edge in this rapidly advancing field. Through its relentless pursuit of innovation, Alnylam continues to position itself as a trailblazer in turning the once theoretical promise of RNAi into a clinical reality.
Blockbuster Growth: Alnylam reported nearly $3 billion in 2025 net product revenue, an increase of 81% over 2024, driven mainly by the AMVUTTRA launch for ATTR cardiomyopathy.
Profitability Achieved: The company achieved GAAP profitability for the full year 2025 and expects to sustain profitability going forward.
Major Revenue Beat: 2025 product sales exceeded guidance by more than $800 million, highlighting stronger-than-expected ATTR-CM demand.
TTR Franchise Momentum: The TTR franchise saw 151% year-over-year growth in Q4, with AMVUTTRA quickly gaining share as a new treatment start.
Strong 2026 Guidance: 2026 combined net product revenue expected to reach $4.9–$5.3 billion, implying over 70% growth at the midpoint.
Expanding Pipeline: Three Phase III studies were initiated in 2025; pipeline now has over 25 active clinical programs.
Margin Expansion Outlook: Mid-single-digit net price decreases expected in 2026, but margin outlook remains strong, especially post-2030 with potential nucresiran launch.
