Alnylam Pharmaceuticals (ALNY) Goldman Sachs 47th Annual Global Healthcare Conference 2026 summary

Strategic goals and financial outlook

• Aims for 25% revenue CAGR and 30% non-GAAP operating margin through 2030, with margin expansion to mid-40% post-2030 as product mix shifts.

• Focuses on TTR leadership, targeting peak revenue franchise status and cumulative growth through 2030.

• Pipeline diversification is a priority, with plans for two or more new blockbuster products beyond TTR and 40 clinical programs by 2030.

• R&D investment set at 30% of revenue, including headroom for business development.

• International expansion ongoing, with launches in major markets and ongoing negotiations in France and Spain.

Product and pipeline updates

• AMVUTTRA is the primary revenue driver through 2030, with nucresiran expected to contribute post-2028 and more significantly after 2030.

• Zilebesiran for hypertension and ACVR1C for obesity/metabolic disease are advancing, with key data readouts expected within the next year.

• Huntington’s disease program (ALN-HTT) aims for >50% knockdown, with promising preclinical and early clinical data.

• Plasminogen knockdown program targets bleeding disorders, with data expected on efficacy and safety later this year.

• Alzheimer’s and cerebral amyloid angiopathy programs are progressing, with phase I and II studies underway and enrollment milestones targeted.

Market dynamics and access

• U.S. TTR market guidance for 2026 is $4.4–$4.7 billion, with Q1 impacted by phasing and price adjustments, but growth expected for the remainder of the year.

• Over 90% of U.S. patients have first-line access to AMVUTTRA, with efforts to expand prescriber base and improve diagnosis via AI partnerships.

• Net price in the U.S. is expected to decline gradually, with a 5% reduction year-over-year.

• Combination use with tafamidis is limited to fee-for-service segments, with broader opportunity expected after tafamidis goes generic in 2031.

• Competitive landscape includes stabilizers, depleters, and gene editing, with silencer class expected to remain foundational.