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Alnylam Pharmaceuticals (ALNY) investor relations material
Alnylam Pharmaceuticals Jefferies London Healthcare Conference 2025 summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Jefferies London Healthcare Conference 2025 summary19 Nov, 2025
Strategic focus and achievements
Achieved rapid growth with six marketed products and a pipeline of over 20 programs, driven by a strong innovation engine.
Focused on TTR leadership, innovation-driven growth, and disciplined financial management to achieve profitability.
Successfully met or exceeded ambitious five-year P5x25 goals, aligning the organization and investors.
On track to achieve sustainable non-GAAP profitability and planning to announce new five-year objectives in early 2025.
Committed to ongoing value creation for patients and investors, with optimism for future RNAi therapeutic advancements.
Amvuttra launch and market dynamics
Amvuttra launch in cardiomyopathy exceeded expectations, rapidly securing access in 170 healthcare systems covering 80% of prescriptions.
Patient volume demand doubled in the last quarter, with broad and balanced utilization across academic and community centers.
No significant access barriers; most patients have zero out-of-pocket costs, supporting strong uptake.
Increased revenue guidance after Q2 and Q3, reflecting launch momentum and sustainable growth.
Broad patient mix includes both first-line and second-line use, with strong physician interest in HELIOS-B data.
Competitive landscape and future opportunities
Modest combination use observed, with significant potential expected post-2028 when generic stabilizers become available.
Confident in maintaining leadership despite upcoming AstraZeneca-Ionis study, leveraging head start and product differentiation.
Real-world evidence and new data, such as GI outcomes, are key to further differentiating Amvuttra.
Underdiagnosis remains a challenge, but improved diagnostic tools and physician education are driving higher diagnosis rates.
Community centers now account for over half of business, supported by 2,000+ contracted infusion centers for broad patient access.
What sustains AMVUTTRA's growth trajectory?
How does Nucresiran secure TTR leadership?
Next blockbuster beyond TTR franchise?
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Next Alnylam Pharmaceuticals earnings date
Q4 202520 Feb, 2026
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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