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Alnylam Pharmaceuticals (ALNY) investor relations material
Alnylam Pharmaceuticals Bernstein Insights: Healthcare Leaders and Disruptors - 2nd Annual Healthcare Forum summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Bernstein Insights: Healthcare Leaders and Disruptors - 2nd Annual Healthcare Forum summary23 Sep, 2025
Opening remarks and company overview
Highlighted leadership in RNAi therapeutics with six commercial products and a robust pipeline of 20 clinical candidates.
Emphasized focus on launching into the cardiomyopathy segment of the ATTR market, a major growth driver.
Stated the company is nearing profitability, a goal set five years ago.
ATTR market expansion and launch performance
Achieved $544 million in global TTR revenue in Q2, a 77% year-over-year increase, with $383 million from the U.S.
Rapid provider setup and formulary access enabled faster-than-expected launch progress.
Balanced demand between first and second line use, with 1,400 cardiomyopathy patients on therapy by quarter-end.
Most payer policies enabled first line access, supporting broad uptake.
Market dynamics and competitive landscape
Initial demand skewed toward second line, but first line use built steadily through the quarter.
First line market remains largely untapped, with only 20% of patients currently treated.
Competitive environment includes established and new entrants; focus remains on differentiating with HELIOS-B data.
Second line growth driven by patients switching from existing therapies to the new product.
TTR growth strategy post-TAF generics?
Next major growth driver beyond TTR?
Capital allocation priorities post-profitability?
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Next Alnylam Pharmaceuticals earnings date
Q3 202530 Oct, 2025
Next Alnylam Pharmaceuticals earnings date
Q3 202530 Oct, 2025
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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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