Alnylam Pharmaceuticals Reports Third Quarter 2026 Financial Results and Highlights Recent Period Progress

− Generated Third Quarter 2026 Global Net Product Revenues of $1,292 Million (52% Growth Compared with Q3 2025), Driven Primarily by Total TTR Revenues of $1,150 Million (59% Growth Compared with Q3 2025) –

− Expands Target Enrollment in TRITON-CM Phase 3 Study of Nucresiran to 2,250 Patients; Study Design Continues to Support Expected ATTR-CM Launch by 2030 –

− Presented Positive Phase 1 Data for Brezosiran (ALN-HTT02) Demonstrating Robust Target Engagement and Encouraging Safety and Tolerability Profile in Patients with Huntington's Disease; Expect to Initiate Pivotal Study in 2027 –

− On Track to Achieve 2026 Financial Guidance –

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter ended September 30, 2026, and reviewed recent business highlights.

“Our third quarter results reflect the continued growth of our TTR franchise and advancement of key programs with the potential for transformative patient impact,” said Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam. “As we continue through the final months of the year and plan for 2027, we remain laser focused on leveraging our commercial investments to expand the prescriber base, support access, and bring AMVUTTRA to more patients with ATTR amyloidosis. In line with our goal to build durable leadership in TTR, the expanded enrollment target for TRITON-CM we are announcing today further positions nucresiran for success and strengthens our long-term position. Lastly, we believe the positive initial data for brezosiran in Huntington's disease both support a pathway to initiating a pivotal study in 2027 and reflect the potential of our RNAi platform to create meaningful value beyond our current portfolio.”

Third Quarter 2026 and Recent Significant Business Highlights

Total TTR: AMVUTTRA^® (vutrisiran) & ONPATTRO^® (patisiran)

• Achieved global net product revenues for AMVUTTRA and ONPATTRO for the third quarter of $1,130 million and $20 million, respectively, together representing $1,150 million in total TTR net product revenues and 59% total TTR growth compared to Q3 2025.
  • U.S. TTR net product revenues increased $99 million compared with Q2 2026 with the growth driven by an $81 million increase in demand, a $17 million inventory benefit, and a modest increase in net price as Q3 was aided by approximately $10 million in favorable gross-to-net adjustments.
  • International TTR net product revenues increased $20 million compared with Q2 2026 driven primarily by ongoing ATTR-CM launches in Japan, Germany, and Italy as well as the timing of orders in partner markets.

• Presented new analyses from the HELIOS-B Phase 3 clinical trial of vutrisiran in patients with ATTR-CM at the European Society of Cardiology (ESC) Congress 2026. The findings further reinforce the clinical profile of vutrisiran across ATTR patient populations, treatment settings, and manifestations of disease, and vutrisiran's position as a first-line treatment option for ATTR-CM.
  • A late-breaking prespecified subgroup analysis demonstrated that vutrisiran provided consistent clinical benefit across all-cause mortality and recurrent cardiovascular events in patients with or without tafamidis use at baseline. The results were simultaneously published in the Journal of the American College of Cardiology.
  • Additional post-hoc analyses highlighted the positive impact of vutrisiran in addressing the multisystemic manifestations of ATTR-CM.
  • A pooled analysis across four positive Phase 3 studies of vutrisiran and patisiran showed consistent treatment effect across sexes.

Total Rare: GIVLAARI^® (givosiran) & OXLUMO^® (lumasiran)

• Achieved global net product revenues for GIVLAARI and OXLUMO for the third quarter of $89 million and $53 million, respectively, together representing $142 million in total Rare net product revenues and 12% total Rare growth compared to Q3 2025.

Key Pipeline Developments

• Today announced an update to the TRITON-CM Phase 3 study of nucresiran, an investigational next-generation RNAi therapeutic, in patients with ATTR-CM. Nucresiran represents a key component of Alnylam's long-term growth strategy, with the potential to reinforce leadership in ATTR amyloidosis and expand the durability of the Company's TTR franchise.
  • The Company is expanding target enrollment from 1,750 to approximately 2,250 patients. This expansion is enriched for patients with early disease who are expected to experience greatest benefit and ensures adequate study power under conservative estimates of outcome event rates.
  • Given the rapid pace of enrollment and the event-driven nature of the study’s primary endpoint, the Company still expects to launch nucresiran, assuming positive data and regulatory approval, in ATTR-CM by 2030.

• Reported initial data at the European Huntington's Disease Network Clinical Research Congress from the Phase 1 clinical trial of brezosiran (ALN-HTT02) in patients with Huntington's disease. Brezosiran has the potential to establish a new growth platform beyond TTR and underscores Alnylam's strategy to broaden the application of RNAi therapeutics to new tissue types and therapeutic areas.
  • Patients treated with single ascending intrathecal doses of brezosiran experienced robust, dose-dependent reductions of mutant huntingtin (mHTT) protein in cerebrospinal fluid (CSF), with target engagement demonstrated using two independent assays. A mean reduction of CSF mHTT of 60-64% from baseline was observed at month 3 in the highest dose group evaluated to date, with a maximum individual reduction of 83%. The observed durability of mHTT-lowering shows the potential for a twice-yearly dosing paradigm.
  • An encouraging safety and tolerability profile has been observed to date, with no dose-dependent increases in CSF inflammatory markers, CSF neurofilament light chain (NfL) or brain ventricular volume.
  • The Company plans to initiate a placebo-controlled pivotal study in 2027, pending regulatory alignment, to evaluate the ability of brezosiran to slow disease progression in adults with Huntington's disease. This program is being developed by Alnylam in collaboration with Regeneron Pharmaceuticals, Inc. (Regeneron).

Additional Pipeline Progress

• Advanced mivelsiran, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) for the treatment of cerebral amyloid angiopathy (CAA) and Alzheimer’s disease.
  • Completed enrollment in the cAPPricorn-1 Phase 2 clinical trial in patients with CAA.
  • Initiated a Phase 2 clinical trial in patients with Down syndrome-associated Alzheimer's disease.
  • Shared additional Phase 1 data in early-onset Alzheimer's disease at the Alzheimer's Association International Conference (AAIC) 2026. An analysis of safety data from single- and multiple-doses of mivelsiran showed no evidence of increased risk of amyloid-related imaging abnormality (ARIA) events. Results also showed robust, durable reductions in CSF soluble amyloid beta precursor protein (sAPPβ) and amyloid beta 42 (Aβ42), with up to 30 months of treatment exposure. The most common adverse events (AEs) were procedural pain and procedural headache, and no serious or severe AEs were deemed related to study drug.

• Presented preclinical data and Phase 1 design details at AAIC 2026 for ALN-5288, an investigational RNAi therapeutic targeting microtubule-associated protein tau (MAPT) for Alzheimer's disease and tauopathies.

Key Upcoming Events

Alnylam announced today that it will present two-year open-label extension results from the HELIOS-B Phase 3 clinical trial of vutrisiran at the American Heart Association (AHA) Scientific Sessions 2026, being held November 6-9 in Chicago.

• The Company will also present results from an indirect treatment comparison of vutrisiran vs. acoramidis at the International Symposium of Amyloidosis (ISA), being held November 15-18 in Montevideo, Uruguay.

Alnylam also announced today that it will share Phase 1 healthy volunteer data for ALN-6400, an investigational RNAi therapeutic in development for bleeding disorders, at the 2026 American Society of Hematology (ASH) Annual Meeting and Exposition, being held December 12-15 in New Orleans.

The Company now expects to announce clinical data from the Phase 2 trial of ALN-6400 in patients with hereditary hemorrhagic telangiectasia (HHT), as well as from the Phase 1 trial of ALN-2232 in obesity and weight management, in conjunction with its fourth quarter and full year 2026 earnings call in February 2027.

The PDUFA target action date for cemdisiran, an investigational RNAi therapeutic for the treatment of adult patients with generalized myasthenia gravis who are anti-acetylcholine receptor antibody-positive, is in November 2026. Cemdisiran is being advanced by Alnylam's collaborator, Regeneron. Assuming regulatory approval, Alnylam is eligible to receive tiered, double-digit royalties of up to 15 percent on global net sales of cemdisiran.

Third Quarter 2026 Financial Results

For an explanation of our use of non-GAAP financial measures, refer to the “Use of Non-GAAP Financial Measures” section later in this press release and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP measure, see the tables at the end of this press release.

Revenue Summary

Total Net Product Revenues

• Total net product revenues increased 52% and 53% at actual currency and at CER, respectively, during the three months ended September 30, 2026, compared to the same period in 2025. The increase was primarily due to growth from AMVUTTRA revenues driven by increased patient demand, mainly in patients with ATTR-CM in the U.S., and growth from an increased number of patients treated with GIVLAARI, which was partially offset by a decreased number of patients treated with ONPATTRO.

Net Revenues from Collaborations

• Net revenues from collaborations decreased during the three months ended September 30, 2026, as compared to the same period in 2025, primarily due to the recognition of $300.0 million of milestone revenue under our collaboration with Roche in September 2025 associated with the dosing of the first patient in the ZENITH Phase 3 clinical trial of zilebesiran.

Royalty Revenue

• Royalty revenue increased during the three months ended September 30, 2026, as compared to the same period in 2025, due to increased volume and rate of royalties earned from global net sales of Leqvio by Novartis.

Operating Expense Summary

Cost of Goods Sold

• Cost of goods sold as a percentage of net product revenues increased during the three months ended September 30, 2026, as compared to the same period in 2025, primarily as a result of increased sales of AMVUTTRA and an associated increase in the blended royalty rate payable on net sales of AMVUTTRA.

Research & Development (R&D) Expenses

• GAAP and non-GAAP R&D expenses for the three months ended September 30, 2026 increased as compared to the same period in 2025, primarily due to increased clinical trial expenses for the ZENITH Phase 3 clinical trial of zilebesiran in patients with uncontrolled hypertension, the TRITON-CM Phase 3 clinical trial of nucresiran in patients with ATTR-CM, and the TRITON-PN Phase 3 clinical trial of nucresiran in patients with hATTR-PN, along with increased spend on various preclinical programs.

Selling, General & Administrative (SG&A) Expenses

• GAAP and non-GAAP SG&A expenses for the three months ended September 30, 2026 increased as compared to the same period in 2025, primarily due to increased sales and marketing investments associated with the ongoing global commercial launch of AMVUTTRA in ATTR-CM.

Other Financial Highlights

Interest expense

• Interest expense for the three months ended September 30, 2026 of $84 million included interest of $58 million attributed to the liability related to the sale of future Leqvio royalties and $23 million attributed to the liabilities related to vutrisiran and zilebesiran development funding.

Benefit from income taxes

• During the three months ended September 30, 2026, we recorded a benefit from income taxes of $1.9 billion, primarily related to the release of valuation allowances on certain U.S. federal and state deferred tax assets.

Financial position

• Cash, cash equivalents and marketable securities were $3.6 billion as of September 30, 2026, as compared to $2.9 billion as of December 31, 2025, with the increase primarily driven by net cash inflows from operating activities.
• Net cash provided by operating activities for the three months ended September 30, 2026 included $35 million of payments associated with the liability related to the sale of future Leqvio royalties recorded to interest expense, as well as $34 million of payments associated with the liabilities related to vutrisiran and zilebesiran development funding recorded to interest expense.

A reconciliation of our GAAP to non-GAAP financial results is included in the tables at the end of this press release.

2026 Financial Guidance

Full-year 2026 financial guidance is reiterated and consists of the following:

Use of Non-GAAP Financial Measures

This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses and non-recurring gains or losses outside the ordinary course of the Company’s business. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies.

The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in this press release are stock-based compensation expenses, and loss related to covertible debt. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards. The Company has excluded the loss related to convertible debt because the Company believes the item is a non-recurring transaction outside the ordinary course of the Company’s business.

Percentage changes in revenue growth at CER are presented excluding the impact of changes in foreign currency exchange rates for investors to understand the underlying business performance. The current period’s foreign currency revenue values are converted into U.S. dollars using the average exchange rates from the prior period.

The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between GAAP and non-GAAP measures is provided later in this press release.

Conference Call Information

Management will provide an update on the Company and discuss third quarter 2026 results as well as expectations for the future via conference call on Thursday, October 29, 2026, at 8:30 am ET. A live audio webcast of the call will be available on the Investors section of the Company’s website at www.alnylam.com/events. An archived webcast will be available on the Alnylam website approximately two hours after the event.

About AMVUTTRA^® (vutrisiran)

AMVUTTRA^® (vutrisiran) is a transthyretin (TTR) silencer that delivers rapid knockdown of TTR at the source to address the underlying cause of transthyretin amyloidosis (ATTR). In a clinical study, AMVUTTRA rapidly knocked down TTR in as early as six weeks and decreased TTR levels by 87% with two and a half years of treatment. It is approved as a treatment for the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and for the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in various countries, globally. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only silencer approved for the treatment of ATTR-CM and hATTR-PN. For more information about AMVUTTRA, including the full U.S. Prescribing Information, visit AMVUTTRA.com.

About ONPATTRO^® (patisiran)

ONPATTRO is an RNAi therapeutic that is approved in the United States and Canada for the treatment of adults with hATTR amyloidosis with polyneuropathy. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby reducing the production of TTR protein before it is made. Reducing the pathogenic protein leads to a reduction in amyloid deposits in tissues. For more information about ONPATTRO, including full Prescribing Information, visit ONPATTRO.com.

About GIVLAARI^® (givosiran)

GIVLAARI (givosiran) is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) approved in the United States and Brazil for the treatment of adults with acute hepatic porphyria (AHP). GIVLAARI is also approved in the European Union for the treatment of AHP in adults and adolescents aged 12 years and older. In the pivotal trial, GIVLAARI was shown to significantly reduce the rate of porphyria attacks that required hospitalizations, urgent healthcare visits or intravenous hemin administration at home compared to placebo. GIVLAARI is Alnylam’s first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. GIVLAARI is administered via subcutaneous injection once monthly at a dose based on actual body weight and should be administered by a healthcare professional. GIVLAARI works by specifically reducing elevated levels of ALAS1 messenger RNA (mRNA), leading to reduction of toxins associated with attacks and other disease manifestations of AHP. For more information about GIVLAARI, including the full U.S. Prescribing Information, visit GIVLAARI.com.

About OXLUMO^® (lumasiran)

OXLUMO (lumasiran) is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1). HAO1 encodes glycolate oxidase (GO). Thus, by silencing HAO1 and depleting the GO enzyme, OXLUMO inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. OXLUMO utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. OXLUMO has received regulatory approvals from the U.S. Food and Drug Administration (FDA) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients and from the European Medicines Agency (EMA) for the treatment of PH1 in all age groups. In the pivotal ILLUMINATE-A trial, OXLUMO was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients reaching normal or near-normal levels. In the ILLUMINATE-B pediatric Phase 3 trial, OXLUMO demonstrated an efficacy and safety profile consistent to that observed in ILLUMINATE-A. In the ILLUMINATE-C trial, OXLUMO resulted in substantial reductions in plasma oxalate in patients with advanced PH1. Across all three studies, injection site reactions (ISRs) were the most common drug-related adverse reaction.

Alnylam Pharmaceuticals, Inc.

Bo Piela
(Media)
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Josh Brodsky
(Investors)
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