Alnylam Pharmaceuticals — Third Quarter 2026 Earnings Summary

Financial Performance:

• Total revenues for Q3 2026 were $1,458.1 million, a 17% increase compared to $1,249.0 million in Q3 2025.
• Total net product revenues were $1,291.9 million, representing a 52% increase compared to $851.1 million in Q3 2025 (53% at constant exchange rates).
• Total TTR net product revenues (AMVUTTRA and ONPATTRO) were $1,150.0 million, a 59% increase compared to Q3 2025 (60% at constant exchange rates).
• AMVUTTRA net product revenues were $1,130.4 million, up 65% year-over-year (66% at constant exchange rates).
• ONPATTRO net product revenues were $19.6 million, down 50% year-over-year.
• Total Rare net product revenues (GIVLAARI and OXLUMO) were $142.0 million, up 12% compared to Q3 2025 (13% at constant exchange rates).
• GIVLAARI net product revenues were $89.5 million, up 21% year-over-year.
• OXLUMO net product revenues were $52.5 million, down 1% year-over-year.
• Net revenues from collaborations were $71.5 million, down 80% compared to $351.7 million in Q3 2025, primarily due to a $300.0 million milestone revenue recognition from Roche in September 2025.
• Royalty revenue was $94.7 million, up 105% compared to $46.2 million in Q3 2025, driven by increased volume and rates from Novartis' Leqvio sales.
• GAAP income from operations was $345.8 million, down 6% compared to $368.0 million in Q3 2025.
• Non-GAAP income from operations was $424.9 million, down 11% compared to $476.2 million in Q3 2025.
• GAAP net income was $2,167.3 million compared to $292.7 million in Q3 2025.
• Non-GAAP net income was $2,241.9 million, up 412% compared to $437.8 million in Q3 2025.
• GAAP net income per diluted share was $15.72 compared to $2.14 in Q3 2025.
• Non-GAAP net income per diluted share was $16.26 compared to $3.19 in Q3 2025.
• Cost of goods sold was $361.8 million (28.0% of net product revenues) compared to $197.2 million (23.2% of net product revenues) in Q3 2025.
• GAAP R&D expenses were $408.5 million, up 14% compared to $358.8 million in Q3 2025.
• Non-GAAP R&D expenses were $375.7 million, up 21% compared to $310.1 million in Q3 2025.
• GAAP SG&A expenses were $342.0 million, up 6% compared to $322.1 million in Q3 2025.
• Non-GAAP SG&A expenses were $295.7 million, up 13% compared to $262.6 million in Q3 2025.
• Interest expense was $84.4 million, including $58.0 million related to the sale of future Leqvio royalties and $23.0 million related to vutrisiran and zilebesiran development funding liabilities.
• Benefit from income taxes was $1.9 billion, primarily due to the release of valuation allowances on U.S. federal and state deferred tax assets.
• Cash, cash equivalents, and marketable securities were $3.6 billion as of September 30, 2026, compared to $2.9 billion as of December 31, 2025.

Guidance and Future Outlook:

• The company reiterated its full-year 2026 financial guidance.
• Full-year 2026 Total TTR net product revenues guidance is $4,200 million to $4,500 million.
• Full-year 2026 Total Rare net product revenues guidance is $500 million to $600 million.
• Full-year 2026 Total net product revenues guidance is $4,700 million to $5,100 million.
• Full-year 2026 net product revenues growth vs. 2025 is expected to be 57% to 71% at actual currency exchange rates and 57% to 70% at constant exchange rates.
• Full-year 2026 net revenues from collaborations and royalties guidance is $575 million to $625 million.
• Full-year 2026 Non-GAAP R&D and SG&A expenses guidance is $2,700 million to $2,800 million (excluding $330 million to $380 million of stock-based compensation).
• The company expects to achieve its 2026 financial guidance.
• Nucresiran is expected to launch in ATTR-CM by 2030, assuming positive data and regulatory approval.
• A pivotal study for brezosiran is expected to initiate in 2027.
• Clinical data from the Phase 2 trial of ALN-6400 in HHT and the Phase 1 trial of ALN-2232 in obesity are expected to be announced in conjunction with the Q4 and full-year 2026 earnings call in February 2027.
• The PDUFA target action date for cemdisiran is November 2026.

Business Segments and Product Lines:

• TTR Franchise: AMVUTTRA is the first and only silencer approved for ATTR-CM and hATTR-PN. U.S. TTR net product revenues increased $99 million compared to Q2 2026, driven by an $81 million increase in demand, a $17 million inventory benefit, and modest net price increases aided by $10 million in favorable gross-to-net adjustments. International TTR net product revenues increased $20 million compared to Q2 2026, driven by ATTR-CM launches in Japan, Germany, and Italy.
• Rare Franchise: GIVLAARI is approved for acute hepatic porphyria (AHP). OXLUMO is approved for primary hyperoxaluria type 1 (PH1).
• Pipeline:
  • Nucresiran: The TRITON-CM Phase 3 study enrollment target was expanded from 1,750 to approximately 2,250 patients to enrich for early disease patients and ensure adequate study power.
  • Brezosiran (ALN-HTT02): Phase 1 data in Huntington's disease showed robust, dose-dependent reductions of mutant huntingtin (mHTT) protein in CSF (60-64% mean reduction at month 3 in the highest dose group). The program is developed in collaboration with Regeneron.
  • Mivelsiran: Completed enrollment in the cAPPricorn-1 Phase 2 trial for cerebral amyloid angiopathy (CAA); initiated a Phase 2 trial for Down syndrome-associated Alzheimer's disease; shared Phase 1 data for early-onset Alzheimer's disease showing robust reductions in sAPPβ and Aβ42 with no increased risk of ARIA events.
  • ALN-5288: Preclinical data and Phase 1 design details presented for a tau-targeting therapeutic for Alzheimer's disease and tauopathies.
  • ALN-6400: Investigational RNAi therapeutic for bleeding disorders; healthy volunteer data to be presented at ASH 2026.
  • ALN-2232: Investigational RNAi therapeutic for obesity and weight management.
  • Cemdisiran: Investigational RNAi therapeutic for myasthenia gravis, advanced by Regeneron; Alnylam eligible for tiered, double-digit royalties up to 15% on global net sales.

Market and Competitive Landscape:

• Vutrisiran is positioned as a first-line treatment option for ATTR-CM, with new analyses from the HELIOS-B trial reinforcing its clinical profile across patient populations and disease manifestations.
• A pooled analysis across four Phase 3 studies showed consistent treatment effect for vutrisiran and patisiran across sexes.
• A subgroup analysis demonstrated consistent clinical benefit for vutrisiran in patients with or without baseline tafamidis use.
• International expansion continues with ATTR-CM launches in Japan, Germany, and Italy.

Risks and Challenges:

• The press release includes standard forward-looking statement risks regarding the ability to successfully launch and market products, clinical trial results, regulatory approvals, manufacturing supply, intellectual property, and strategic collaborations.
• Specific risks mentioned include dependence on third parties for development and commercialization, the outcome of litigation, and potential changes to the 340B Drug Pricing Program or payor utilization management policies.

Management Commentary and Tone:

• CEO Yvonne Greenstreet stated that Q3 results reflect continued growth of the TTR franchise and advancement of key programs with transformative potential.
• Management emphasized a focus on leveraging commercial investments to expand the prescriber base, support access, and bring AMVUTTRA to more patients.
• The expanded enrollment for TRITON-CM was described as strengthening the long-term position of nucresiran.
• Positive initial data for brezosiran was cited as supporting a pathway to a pivotal study in 2027 and reflecting the potential of the RNAi platform beyond the current portfolio.
• The tone is confident and focused on long-term growth and platform expansion.

Other Key Points:

• Collaborations:
  • Roche: Net revenues from collaborations decreased significantly due to the absence of the $300.0 million milestone recognized in Q3 2025.
  • Regeneron: Net revenues from collaborations increased 43% to $32.3 million.
• Upcoming Events:
  • Presentation of two-year open-label extension results for HELIOS-B (vutrisiran) at AHA Scientific Sessions 2026 (November 6-9).
  • Presentation of indirect treatment comparison of vutrisiran vs. acoramidis at ISA (November 15-18).
  • Presentation of Phase 1 healthy volunteer data for ALN-6400 at ASH 2026 (December 12-15).
• Balance Sheet:
  • Deferred revenue increased to $11.7 million from $4.8 million as of December 31, 2025.
  • Convertible debt decreased to $1.0 billion (current and non-current combined) from $1.0 billion (current portion only) as of December 31, 2025, with current portion at $361.5 million and non-current at $651.1 million.
  • Accumulated deficit improved to $(4,164.7) million from $(6,702.5) million as of December 31, 2025.
  • Total stockholders' equity increased to $3,603.9 million from $789.2 million as of December 31, 2025.
• Conference Call: Management will discuss results on October 29, 2026, at 8:30 am ET.