Alnylam Pharmaceuticals

At Muffett Investments, we believe RNA interference (RNAi) therapeutics represent one of the most important advances in medicine since the discovery of penicillin. While penicillin allowed physicians to treat bacterial infections that were once fatal, RNA interference allows physicians to silence disease-causing genes before the harmful proteins they encode are ever produced. It is a shift from treating the consequences of disease to modifying its cause — and we believe it will define the next generation of pharmaceutical innovation.

Alnylam Pharmaceuticals has spent more than two decades building the platform that made this possible. What began as a company proving RNAi in rare genetic diseases has evolved into a diversified business of approved medicines, growing royalty income, and a late-stage pipeline spanning rare disease, cardiovascular disease, hypertension, metabolic disease and autoimmune disease. Unlike most biotechnology companies, which live or die on a single asset, Alnylam's value lies in the platform itself — and in its ability to repeatedly convert scientific discovery into commercial medicine.

Why We Own It

• Global leader in a clinically validated RNA interference platform, twenty years in the making.
• Multiple approved medicines plus a growing, high-margin royalty stream from Leqvio.
• Exposure to both rare disease and some of the largest chronic disease markets in medicine.
• Strategic collaborations with Roche, Novartis and Regeneron.
• Multiple late-stage clinical catalysts and a first full year of GAAP profitability in 2025.

This report sets out that thesis in full: what RNA interference is and why it matters, why Alnylam leads it, where the platform is headed next, what could go wrong, what we believe Alnylam is worth, and our final verdict. We believe the market continues to price Alnylam as a successful rare-disease biotechnology company. We believe the evidence increasingly describes something larger — a platform capable of reshaping cardiovascular and metabolic medicine over the next decade.

“The future belongs to technologies that treat the cause rather than the consequence.”

Muffett Investments

Medicine Has Always Moved Upstream

Throughout history, medicine has evolved by intervening progressively earlier in the biological pathway responsible for disease. The earliest medicines focused on treating symptoms. As scientific understanding improved, therapies evolved to target the biological processes responsible for disease. Today, advances in molecular biology have created the opportunity to intervene even earlier — at the level of gene expression itself.

At Muffett Investments, we believe this progression is not accidental. It represents the natural evolution of modern medicine — and RNA interference is its next stage.

The Full Table of Contents

• 01 Why RNA Interference Represents the Next Revolution in Medicine
• 02 Why Alnylam Has Become the Global Leader in RNA Interference
• 03 Why Cardiometabolic Disease Could Transform Alnylam's Future
• 04 Leqvio: The First Mainstream RNA Interference Success
• 05 AMVUTTRA: The Product That Validated the Platform
• 06 Zilebesiran: Could RNA Interference Redefine the Treatment of Hypertension?
• 07 Cemdisiran: Expanding RNA Interference into Autoimmune Disease
• 08 The Next Generation of RNA Therapeutics
• 09 Financial Analysis: Approaching the Inflection Point
• 10 Risks to the Investment Thesis
• 11 Alnylam in 2035: A Vision of the Future
• 12 Valuation: How Should Investors Value a Platform Company?
• 13 The Muffett Verdict

This report is prepared by Muffett Investments for informational purposes only and does not constitute investment advice or a recommendation to buy or sell any security. Biotechnology investing carries significant risk, including the risk of substantial loss. Past performance is not indicative of future results.