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Alnylam Pharmaceuticals
Alnylam Pharmaceuticals (ALNY) investor relations material
Alnylam Pharmaceuticals 44th Annual J.P. Morgan Healthcare Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.44th Annual J.P. Morgan Healthcare Conference summary12 Jan, 2026
Strategic achievements and financial performance
Achieved six approved RNAi medicines, treating 500,000 patients globally and generating $2,987 million in 2025 net product revenue, with 81% year-over-year growth and a 50% revenue CAGR since 2015.
Amvuttra's launch for ATTR cardiomyopathy drove TTR franchise revenue to $2.5 billion, doubling from the prior year and exceeding guidance by $800 million, with 103% YoY growth and 53% CAGR from 2020 to 2025.
2026 guidance projects $4.9–$5.3 billion in product sales, with TTR franchise expected to reach $4.4–$4.7 billion and a 71% YoY increase at midpoint.
On track for non-GAAP profitability, with formal year-end results to be announced in February.
Non-GAAP financial measures used for internal evaluation and investor communication.
2030 vision and strategic pillars
New five-year goals (Alnylam 2030) focus on global TTR leadership, sustainable innovation, and disciplined scaling.
Aim to lead TTR revenue by 2030, launch Nucresiran in polyneuropathy by 2028 and cardiomyopathy by 2030.
Plan to deliver two or more new transformative medicines beyond TTR, expand to 10 tissue types, and build a pipeline of over 40 clinical programs.
Targeting 25%+ total revenue CAGR and ~30% non-GAAP operating margin through 2030.
Aspiring to generate over $10 billion in annual revenue by 2030, driven by multiple blockbuster products.
Product and pipeline highlights
Amvuttra rapidly gained parity with tafamidis in new patient starts, with strong first-line use and broad access; 90% of patients can receive treatment within 10 miles of home.
Nucresiran, a next-generation TTR silencer, targets 95% TTR knockdown with biannual dosing, aiming for best-in-class status and durable franchise growth.
Pipeline includes Zilebesiran for hypertension, ALN-4324 and ALN-2232 for metabolic disease, Mivelsiran for cerebral amyloid angiopathy and Alzheimer's, ALN-HTT02 for Huntington's, and ALN-6400 for bleeding disorders.
Four clinical data readouts, three phase III studies, three phase II initiations, and three to four new INDs expected in 2026.
Focus on expanding RNAi delivery to 10+ tissue types, leveraging biobank data, and pursuing multiplexing for complex diseases.
What drives 30% operating margin before nucresiran?
What defines success for new tissue type expansion?
ALN-HTT02: What defines success in Phase I data?
Partial view of Summaries dataset, powered by Quartr API
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Next Alnylam Pharmaceuticals earnings date
Q4 202520 Feb, 2026
Alnylam Pharmaceuticals
Next Alnylam Pharmaceuticals earnings date
Q4 202520 Feb, 2026
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Frequently asked questions
RNAi Pioneers
Alnylam Pharmaceuticals is a biopharmaceutical company at the forefront of RNA interference (RNAi) therapeutics. Founded in 2002, the company is headquartered in Cambridge, Massachusetts. Alnylam's primary focus lies in harnessing the potential of RNAi, a naturally occurring biological pathway within cells, to target and silence specific genes that cause diseases. Their innovative approach has led to the development of a range of treatments for rare genetic, cardio-metabolic, hepatic infectious, and central nervous system diseases. Alnylam conducts research in its field, striving to develop new treatment options for patients who have limited or inadequate alternatives.
Built on RNAi
RNA stands for ribonucleic acid, a nucleic acid present in all living cells. There are two different forms of RNA used in RNAi-processes: Small interfering RNA (siRNA) and microRNA (miRNA). RNA interference (RNAi) is like a security system for cells. Small RNA molecules latch onto a complex called RISC. They then target specific messages, known as mRNAs, stopping them from making proteins. This helps control which proteins are made and when. RNAi also gets rid of unneeded mRNAs. Besides controlling protein production, RNAi helps defend cells against viruses by targeting and destroying their RNA, preventing the virus from making harmful proteins.
A Relatively Young Company
Founded in 2002, Alnylam Pharmaceuticals was established with the clear goal of utilizing RNA interference to develop and eventually market various treatments. Recognizing the potential of RNAi to silence disease-causing genes, the company aimed to pioneer its therapeutic applications. In its early years, Alnylam focused on foundational research, gradually evolving its drug pipeline. Over its existence, Alnylam has formed partnerships and made strategies with various pharmaceutical entities, further solidifying its position as a leader in the RNAi therapeutics field. Being publicly traded is nothing unique in the pharmaceutical industry, and a list of notable companies in the same field includes Eli Lilly, Pfizer, Astra Zeneca, and many others.
FDA Approval
Even though RNAi had plenty of doubters in the early stages, faith remained high at Alnyam. The company ran at a loss for several years, but by 2018, the company achieved a significant milestone by securing FDA approval for its first RNAi therapeutic, Patisiran. This marked the beginning of a road to profitability for the company, as they were able to scale their operations with an approved product.
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