Alnylam Pharmaceuticals (ALNY) Bernstein Insights: Healthcare Leaders and Disruptors - 2nd Annual Healthcare Forum summary

Opening remarks and company overview

• Pioneered RNAi therapeutics with over 20 years of development, now commercializing six products, four directly.

• Focused on launching into the cardiomyopathy segment of the ATTR market, a major growth area.

• Pipeline includes about 20 clinical-stage medicines leveraging the same platform technology.

• Nearing profitability, a goal set five years ago, driven by top-line growth.

ATTR franchise performance and launch dynamics

• Q2 global TTR revenue reached $544 million, up 77% year-over-year, with $383 million from the U.S.

• Cardiomyopathy launch drove a step-change in growth, with an estimated 1,400 patients on therapy by quarter-end.

• Rapid provider setup and formulary access at major U.S. health systems accelerated revenue ahead of expectations.

• Achieved broad payer coverage, especially in Medicare, with most policies enabling first-line access.

• Balanced demand between first and second line, with initial uptake skewed to second line but shifting toward first line.

Market opportunity and competitive landscape

• Cardiomyopathy segment is about 10 times larger than hereditary polyneuropathy, with significant untapped potential.

• Only about 20% of eligible patients are currently treated, with 18,000 new patients presenting annually.

• Competitive market with new entrants; majority of first-line patients still start on tafamidis, but interest in new mechanisms is high.

• Second-line growth driven by patients progressing on existing therapies, with most switching to the new product's mechanism.