Alnylam Pharmaceuticals (ALNY) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Key clinical data and study outcomes

• HELIOS-B study for patisiran showed significant results, hitting primary and all secondary endpoints, with a 35%-36% reduction in all-cause mortality in TTR cardiomyopathy patients.

• Benefits were observed even in patients already on background therapies like tafamidis and SGLT2 inhibitors, with additive effects noted.

• Quality of life and functional status improved rapidly, with consistent results across all subgroups, including those on tafamidis.

• Vutrisiran demonstrated a 41% reduction in all-cause mortality when combined with tafamidis, and up to 65% reduction in patients with lower NT-proBNP.

• Early separation of benefit curves was seen for key endpoints, attributed to the rapid action of RNAi therapy.

Market positioning and differentiation

• Patisiran is positioned as a potential first-line monotherapy and as a combination or switch therapy for TTR cardiomyopathy.

• Differentiation is driven by all-cause mortality reduction, rapid onset of effect, and quality of life improvements.

• The new mechanism of action offers an orthogonal approach compared to existing therapies, appealing to physicians.

• Cross-study comparisons are limited due to differences in patient populations, but the efficacy data is compelling.

• The product is expected to become the next standard of care, with strong physician enthusiasm.

Commercial strategy and market opportunity

• TTR cardiomyopathy is a large and rapidly growing market, with 80% of patients currently untreated and diagnosis rates rising.

• The company has a decade of experience in TTR, robust commercial infrastructure, and a proven track record in polyneuropathy.

• Plans include scaling up for a larger market, leveraging established relationships with key centers and opinion leaders.

• Early adopters are expected to be newly diagnosed patients and those progressing on stabilizers, with combination use growing post-tafamidis genericization (expected 2028).

• Over 90% of patients currently have seamless access, and 95% continue treatment, supporting future growth.