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Alnylam Pharmaceuticals, Inc. (ALNY): Business Model Canvas [Dec-2025 Updated] |
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Alnylam Pharmaceuticals, Inc. (ALNY) Bundle
You're looking to understand the engine driving Alnylam Pharmaceuticals, Inc.'s push for non-GAAP profitability in 2025, right? As someone who's spent two decades dissecting pharma models, I can tell you their Business Model Canvas is built on proprietary RNAi tech and anchored by the TTR franchise, aiming for Net Product Sales between $2.95 billion and $3.05 billion this year, plus another $650 million to $750 million from collaborations. This structure clearly shows how they manage high R&D expenses against the massive commercial upside from products like AMVUTTRA. Honestly, it's a masterclass in translating complex science into a clear, multi-billion dollar revenue stream.
Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Key Partnerships
You're looking at the backbone of Alnylam Pharmaceuticals, Inc.'s external growth engine. These alliances are critical because they bring in non-product revenue-cash infusions and future royalties-that help fund the rest of the pipeline. Honestly, the numbers coming from these collaborations in 2025 are what really move the needle on the profitability story.
The Key Partnerships block shows how Alnylam leverages its RNAi platform through licensing and co-development deals, which is smart capital deployment. Here's the breakdown of the major players and the associated financial data as of late 2025, primarily reflecting Q3 2025 performance.
Strategic Collaboration Financials and Milestones
The value generated from these partnerships is clear in the recent quarterly filings. For instance, the collaboration revenue for the three months ended September 30, 2025, was $352 million, a significant jump from the prior year period.
Here's a look at the specific agreements:
- The agreement with Roche for zilebesiran resulted in the recognition of $300 million of milestone revenue in September 2025, triggered by dosing the first patient in the ZENITH Phase 3 clinical trial. The total potential deal value remains up to $2.8 billion. Development costs for the first indication are shared 40% Alnylam and 60% Roche.
- The Novartis collaboration on LEQVIO (inclisiran) generated royalty revenue that doubled to $46 million in Q3 2025, representing a 98% increase compared to Q3 2024.
- The Sanofi collaboration on Qfitlia (fitusiran), which received US FDA approval on March 28, 2025, makes Alnylam eligible for tiered royalties ranging from 15% to 30% on worldwide net sales. Forecasts suggest Qfitlia could reach peak sales of roughly $1.3 billion globally by 2030, with an annual cost to patients around $642,000.
- The amended deal with Regeneron for cemdisiran provided Alnylam with a $10 million upfront payment. For cemdisiran as a monotherapy, Alnylam is entitled to tiered, double-digit royalties, up to 15% on net sales. For combination use, milestones of up to $325.0 million are tied to performance.
- Regarding Vir Biotechnology and elebsiran, Alnylam elected not to opt-in to its profit-sharing option for the CHB and CHD indications following an amendment in Q1 2025. This leaves Vir solely responsible for development, manufacturing, and commercialization.
You can see the structure and potential upside clearly when you map out the key financial terms:
| Partner | Asset | Key Financial Trigger/Term (Late 2025) | Alnylam Financial Upside/Share |
| Roche | Zilebesiran | $300 million milestone achieved in Q3 2025 | Equal profit share in U.S.; Low double-digit royalties outside U.S. |
| Novartis | LEQVIO (inclisiran) | Royalty revenue increased 98% in Q3 2025 vs Q3 2024 | Royalty revenue of $46 million in Q3 2025 |
| Sanofi | Qfitlia (fitusiran) | FDA approval March 28, 2025 | 15% to 30% tiered royalties on global net sales |
| Regeneron | Cemdisiran | $10 million upfront payment received in amended deal | Tiered royalties up to 15% (monotherapy); Milestones up to $325.0 million (combination) |
| Vir Biotechnology | Elebsiran | Alnylam elected not to opt-in to profit-sharing in Q1 2025 | Vir solely responsible for commercialization |
The immediate financial impact is substantial; the $300 million Roche milestone in Q3 2025 was a primary driver for the 149% year-over-year increase in Total net revenues from collaborations, which reached $351,742 thousand for the quarter.
Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Key Activities
You're looking at the core engine driving Alnylam Pharmaceuticals, Inc.'s current financial performance and future value, which centers on translating its RNAi platform into approved, commercialized medicines. This means heavy investment in discovery, rigorous clinical testing, and aggressive market penetration for new launches.
Research and Development (R&D) of RNAi Therapeutics
The R&D activity is substantial, focusing on expanding the number of approved medicines and advancing late-stage assets. Alnylam Pharmaceuticals reports having 6+ marketed products globally and a pipeline featuring over 20 clinical programs; 10+ in late stages. The company is committed to delivering sustainable innovation, targeting 4+ Investigational New Drug (IND) applications per year. For the twelve months ending September 30, 2025, research and development expenses reached $1.248B, marking a 13.62% increase year-over-year. This spend directly supports pipeline advancement, including clinical trial expenses for zilebesiran and nucresiran. A new focus indication announced for ALN-6400 is hereditary hemorrhagic telangiectasia (HHT).
Global Commercialization and Launch of AMVUTTRA for ATTR Cardiomyopathy (ATTR-CM)
The commercial activity is currently dominated by the launch of AMVUTTRA (vutrisiran) for ATTR-CM. This launch is a major driver of the company's financial outlook. By the end of Q2 2025, approximately 1,400 ATTR-CM patients were on AMVUTTRA. The momentum continued through Q3 2025, where patient demand for AMVUTTRA ATTR-CM roughly doubled quarter-over-quarter. This success led Alnylam Pharmaceuticals to raise its full-year 2025 total net product revenue guidance to a range of $2.95 billion to $3.05 billion.
Here's a snapshot of the commercial revenue performance as of the third quarter of 2025:
| Metric | Q3 2025 Amount | Year-over-Year Growth |
| AMVUTTRA Net Product Revenue | $685 million | 165% |
| ONPATTRO Net Product Revenue | $39 million | N/A (Decreased due to switches to AMVUTTRA) |
| Total TTR Franchise Net Product Revenues | $724 million | 135% |
| Total Net Product Revenues (All Products) | $851 million | 103% |
Initiating Phase 3 Cardiovascular Outcomes Trial (CVOT) for Zilebesiran (Hypertension)
Advancing the zilebesiran program is a key activity, especially the initiation of its Phase 3 cardiovascular outcomes trial (CVOT), named ZENITH. The initiation of this trial triggered a significant financial event: Alnylam Pharmaceuticals recognized a $300 million milestone payment from its collaborator, Roche, in Q3 2025, tied to the dosing of the first patient in the ZENITH study. This trial is evaluating zilebesiran for hypertension and improving cardiovascular outcomes.
Manufacturing and Supply Chain Management for Complex, High-Value Therapeutics
Managing the supply chain for complex RNAi therapeutics involves maintaining high quality while balancing cost of goods sold (COGS) against high selling prices. For Q3 2025, the gross margin on product sales was 77%. The COGS as a percentage of net product revenue for that quarter stood at 23.2%. The CFO noted that the gross margin was expected to be lower in Q4 2025 due to higher royalty rates.
Securing Broad Payer Coverage and Formulary Access for Commercial Products
Unlocking access is critical for realizing revenue from new launches like AMVUTTRA. The company's commercial success is explicitly linked to achieving broad payer coverage. For the AMVUTTRA ATTR-CM launch in the U.S., management reported that most patients are accessing the drug with $0 Out-of-Pocket (OOP) costs. This suggests effective gross-to-net management and favorable coverage terms being secured, helping drive the strong patient uptake seen in the U.S. TTR net product revenue, which reached $543 million in Q3 2025.
Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Key Resources
You're looking at the core assets Alnylam Pharmaceuticals, Inc. uses to run its business as of late 2025. These aren't just ideas; they are the tangible and intangible things that make the whole operation work.
Proprietary RNA interference (RNAi) technology platform (GalNAc conjugate delivery)
Alnylam Pharmaceuticals, Inc. stands on its Nobel Prize-winning science, translating RNA interference (RNAi) into a new class of medicines. The core technology involves Small interfering RNA (siRNA) molecules that silence messenger RNA (mRNA) upstream, preventing disease-causing proteins from being made. The delivery aspect is key; for instance, Vutrisiran uses the Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, which is designed for increased potency and high metabolic stability, potentially allowing for infrequent subcutaneous injections. The company also highlights platform advances for delivery to adipose, muscle, heart, and kidney, and for crossing the blood-brain barrier.
Commercial Product Portfolio: AMVUTTRA, ONPATTRO, GIVLAARI, and OXLUMO
The commercial success is driven by a growing portfolio of approved RNAi therapeutics. As of the third quarter of 2025, Alnylam Pharmaceuticals, Inc. had 6+ marketed products globally, with over 0.5 million patients on their RNAi therapeutics worldwide. The TTR franchise, led by AMVUTTRA, is the primary growth engine.
| Product | Indication Area | Q3 2025 Net Product Revenue (Millions USD) | Year-over-Year Growth (Approximate) |
| AMVUTTRA (Vutrisiran) | TTR Franchise | $685.3 (Combined with ONPATTRO, AMVUTTRA was $685M) | 165% (AMVUTTRA alone) |
| ONPATTRO (Patisiran) | TTR Franchise | $39 | Decreasing due to switches to AMVUTTRA |
| GIVLAARI | Rare Disease | $74 | Growth |
| OXLUMO | Rare Disease | $53 | Growth |
The company raised its full-year 2025 Total Net Product Revenue Guidance to a range of $2.95 billion to $3.05 billion, up from a previous range of $2.65 billion to $2.8 billion. The TTR Franchise guidance was specifically increased to $2.475 billion to $2.525 billion for the full year 2025.
Extensive Intellectual Property (IP) portfolio protecting RNAi mechanisms and delivery
Alnylam Pharmaceuticals, Inc. protects its innovations with a broad intellectual property estate. This estate includes fundamental, chemistry, delivery, and target patents and patent applications. The patent portfolio covers oligonucleotides, including synthetic RNA molecules, optimized for various delivery modalities like lipid- and conjugate-based systems. This IP is protected across the world's major pharmaceutical markets, specifically the United States, European Union, and Japan, among others. This strong IP position has validated itself through collaborations with major pharmaceutical companies, including Roche, Regeneron, Sanofi, and Novartis.
Specialized R&D and global commercial infrastructure
The infrastructure supports both the current commercial products and a deep pipeline. As of late 2025, the pipeline includes over 20 clinical programs, with 10+ in late stages. The company is executing on its P5x25 strategy, aiming to file 4+ Investigational New Drug (IND) applications per year by the end of 2025. The R&D investment is significant; Non-GAAP R&D Expenses for Q3 2025 were $310 million. The commercial build-out is reflected in Non-GAAP SG&A Expenses of $263 million for Q3 2025, driven by investments supporting the AMVUTTRA launch in the U.S. Key infrastructure advancements include initiating the ZENITH Phase 3 trial for zilebesiran in hypertension and advancing the TRITON-PN study for nucresiran.
- Pipeline goal by YE 2025: File 4+ INDs per year.
- Late-stage clinical programs: 10+.
- Q3 2025 Non-GAAP R&D Expense: $310 million.
- Q3 2025 Non-GAAP SG&A Expense: $263 million.
Cash, cash equivalents, and marketable securities to fund operations
The company maintains a substantial liquidity position to fund its operations and growth initiatives. As of September 30, 2025, cash, cash equivalents, and marketable securities stood at $2.7 billion, down from $2.9 billion on June 30, 2025, due to refinancing activities. These activities included using cash to repurchase a large portion of 1.00% convertible senior notes due 2027, partially offset by net proceeds from new convertible notes due 2028 and net cash inflows from operations. The company reported Non-GAAP Operating Income of $476 million for Q3 2025. The accumulated deficit as of March 31, 2025, was $7.35 billion.
| Financial Metric | Amount as of September 30, 2025 |
| Cash, Cash Equivalents, and Marketable Securities | $2.7 billion |
| Cash, Cash Equivalents, and Marketable Securities (June 30, 2025) | $2.9 billion |
| Non-GAAP Operating Income (Q3 2025) | $476 million |
| Accumulated Deficit (as of March 31, 2025) | $7.35 billion |
Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Value Propositions
You're looking at the core reasons why Alnylam Pharmaceuticals, Inc. is capturing market share and achieving profitability milestones as we close out 2025. The value proposition centers on delivering truly novel, mechanism-based medicines, moving beyond incremental improvements.
Transformative, disease-modifying treatments for rare and prevalent diseases define the offering. The commercial success is clear: Alnylam Pharmaceuticals, Inc. posted Total Net Product Revenues of $851 Million for the third quarter of 2025, which was a 103% Growth compared to Q3 2024. This momentum led to raising the full-year 2025 total net product revenue guidance to a range of $2.95 Billion to $3.05 Billion. The company achieved a net income of $251.1 Million in Q3 2025, a 325% Increase from a loss in Q3 2024, marking its first profitable quarter in over two decades.
For the TTR franchise specifically, which includes AMVUTTRA, the net product revenue hit $724 Million in Q3 2025, representing 135% Growth year-over-year. This financial performance is the direct result of the value delivered by their lead product.
AMVUTTRA: Convenient quarterly subcutaneous administration for ATTR-CM is a major differentiator. The drug, vutrisiran, is approved for the cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM). The uptake has been rapid, particularly in the U.S., where ATTR-CM patient demand roughly doubled quarter-over-quarter from Q2 to Q3 2025. The convenience of a quarterly subcutaneous dose contrasts sharply with older, more frequent dosing regimens, helping drive adoption. Globally, AMVUTTRA has accumulated more than 8,000 patient-years of experience.
The scientific foundation is the first-in-class mechanism of action (gene silencing) for previously untreatable conditions. This RNA interference (RNAi) approach addresses the disease at its source by knocking down the problematic protein, transthyretin (TTR). This mechanism is what underpins the potential for long-term disease modification.
The potential for long-term cardiovascular benefit in ATTR-CM is strongly supported by the latest HELIOS-B data presented in late 2025. Analyses through up to 48 months showed that vutrisiran reduced the risk of the composite endpoint of all-cause mortality (ACM) or first cardiovascular (CV) event by 37% in the overall population. For the monotherapy group, this risk reduction was 42%. Furthermore, post hoc analyses showed that treatment was associated with a reduction in mean Days Lost to Death and/or Hospitalization (DLDH) of more than one month versus placebo over a three-year period. Imaging data also suggested structural benefit, with Cardiac Magnetic Resonance (CMR) showing amyloid regression in 22% of treated patients, compared to no regression in the placebo group.
Alnylam Pharmaceuticals, Inc. is also extending this RNAi platform into common diseases, evidenced by the pipeline expansion into common diseases like hypertension (zilebesiran). The company, in collaboration with Roche, is advancing zilebesiran into a global Phase 3 Cardiovascular Outcomes Trial (CVOT) called ZENITH, which is expected to initiate by the end of 2025. This trial is massive, planning to enroll approximately 11,000 patients. The Phase 2 KARDIA-3 study supported this move by showing clinically meaningful reductions in office systolic blood pressure at month three with continuous control through month six using a 300 mg every six months dosing regimen. This advancement immediately generated value, as Alnylam recognized $300 Million in milestone revenue from Roche in September 2025 upon the first patient dose in ZENITH.
Here's a quick look at the key product and pipeline metrics as of late 2025:
| Metric | Product/Program | Value/Data Point |
| Q3 2025 Net Product Revenue | Total (All Products) | $851 Million |
| FY 2025 Net Product Revenue Guidance (Midpoint) | Total | $3.00 Billion |
| Q3 2025 TTR Franchise Revenue | AMVUTTRA/ONPATTRO | $724 Million |
| HELIOS-B CV Event Risk Reduction (Overall Pop.) | AMVUTTRA (Vutrisiran) | 37% |
| ZENITH Phase 3 Enrollment Target | Zilebesiran | Approximately 11,000 patients |
| Q3 2025 Collaboration Revenue | Roche Milestone (ZENITH) | $300 Million |
The value proposition is built on delivering superior clinical outcomes with convenient administration, which is translating directly into top-line growth and profitability.
Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Customer Relationships
Alnylam Pharmaceuticals, Inc. focuses its customer relationships on ensuring access and providing deep support for specialized patient populations, driven by its Patient Access Philosophy established in 2017.
High-touch patient support programs for rare disease communities.
The in-house ecosystem, Alnylam Assist®, is primarily staffed by dedicated Alnylam employees to support patients and providers throughout the treatment journey. This includes expert management of treatment initiation, access, reimbursement, and appeals. Financial assistance options are available through this program for eligible patients. The Patient Assistance Program (PAP) provides access to treatment at no cost for eligible patients, primarily those who are uninsured. The Copay Program covers certain out-of-pocket costs for eligible patients with commercial insurance. As of June 30, 2025, approximately 1,400 ATTR-CM patients were on AMVUTTRA.
The commercial performance supporting these rare disease communities as of the third quarter ended September 30, 2025, is detailed below:
| Franchise/Product | Q3 2025 Net Product Revenue | Year-over-Year Growth (vs Q3 2024) |
| TTR Franchise (AMVUTTRA & ONPATTRO) | $724 million | 135% |
| Rare Franchise (GIVLAARI & OXLUMO) | $127 million | 14% |
The full year 2025 total net product revenue guidance is set between $2.95 billion and $3.05 billion.
Direct engagement with specialist physicians (cardiologists, neurologists, hepatologists).
Engagement is structured to provide education and support for complex RNAi therapeutics. The company maintains dedicated teams to support healthcare providers throughout the patient journey with an Alnylam therapeutic. Furthermore, the company supports access to diagnostic resources and genetic testing through programs like Alnylam Act® in the U.S., Canada, and Brazil.
Proactive negotiation of value-based agreements with payers for broad access.
Alnylam Pharmaceuticals, Inc. has a history of pursuing innovative contracting, including value-based agreements (VBAs) designed to align access and outcomes with the value the therapies deliver. Agreements in principle for OXLUMO were reached with Express Scripts, Harvard Pilgrim, and Highmark. The company's Patient Access Philosophy commits to pursuing value-based agreements. Broad payer coverage is cited as a key driver for the uptake of AMVUTTRA.
Dedicated field medical and commercial teams for complex product education.
Field engagement is led by a senior structure, such as the Executive Director overseeing the U.S. Field Medical team, which includes Medical Science Liaison (MSL) and Health Systems Medical Outcomes Liaison (H-MOSL) teams. These teams are responsible for executing a unified U.S. KOL (Key Opinion Leader) and Health Systems Engagement Plan. The company also invests in its personnel, having launched the Catalyst Program for Leadership Development in its International Commercial and Medical Field teams in 2024, with goals for 2025 employee engagement.
- The company expects to achieve non-GAAP profitability in 2025 if it meets its net product revenue guidance.
- The TTR franchise revenue guidance for 2025 is $2.475 billion to $2.525 billion.
- The company's commitment to expanding market access ensures patients can access therapies in approximately 70 countries via direct or distributor infrastructure.
Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Channels
You're looking at how Alnylam Pharmaceuticals, Inc. gets its transformative RNAi medicines to patients and partners as of late 2025. It's a mix of building out their own commercial muscle while relying on established relationships for global reach and specific indications.
Direct Sales Force in the U.S. and Key Global Markets for Owned Products
Alnylam Pharmaceuticals, Inc. is driving its own commercial execution, especially for the TTR franchise products like AMVUTTRA. The U.S. market is clearly a primary focus for this direct channel, evidenced by the strong revenue figures coming from that region for the ATTR-CM indication.
The company's focus on patient access efforts supports this direct channel strategy. For instance, as of June 30, 2025, Alnylam Pharmaceuticals, Inc. reported approximately 1,400 ATTR-CM patients on AMVUTTRA.
The financial results show the impact of this direct sales effort:
| Metric | Period Ended September 30, 2025 | Period Ended June 30, 2025 |
| U.S. TTR Net Product Revenues | $543 million | N/A (Q2 data not broken out this way) |
| Total TTR Franchise Revenues | $724 million | $544 million |
| Total Net Product Revenues | $851 million | $672 million |
The 2025 total net product revenue guidance, as revised in October 2025, is set between $2.95 billion and $3.05 billion.
Distribution Network via Specialty Pharmacies and Distributors
For its commercial products, Alnylam Pharmaceuticals, Inc. relies on established distribution channels suited for complex, high-cost specialty medications. This involves selecting specific partners to manage the supply chain and patient support.
The use of specialty pharmacies is a known component of the distribution strategy, as seen historically with products like OXLUMO, which utilized Orsini Specialty Pharmacy as a limited distribution partner.
The general landscape for specialty drugs in 2025 shows manufacturers carefully selecting dispensing partners. As of January 2025, 34% of unique specialty drugs tracked utilized exclusive networks (only one pharmacy).
Key aspects of this channel include:
- Limited Distribution Drug (LDD) network utilization.
- Partnering with independent specialty pharmacies.
- Managing complex regimens and patient support needs.
Commercialization Partners (e.g., Novartis, Sanofi) for Ex-U.S. and Co-developed Products
Alnylam Pharmaceuticals, Inc. utilizes strategic collaborations to expand reach for co-developed assets and generate royalty revenue from products commercialized by others.
Key partnerships and associated financial/development milestones as of late 2025 include:
| Partner | Product/Indication | 2025 Channel Activity/Status |
| Novartis | Leqvio (inclisiran) | Royalty revenue increased in Q3 2025 due to higher global net sales volume. |
| Roche | Zilebesiran (Hypertension) | Expected initiation of Phase 3 cardiovascular outcomes trial in the second half of 2025. Generated $300 million in milestone revenue in Q3 2025. |
| Sanofi | Fitusiran (Hemophilia A/B) | Expected FDA approval PDUFA target action date of March 28, 2025. |
| Regeneron | Cemdisiran (Myasthenia Gravis) | Plans to share Phase 3 trial results in the second half of 2025. Q2 2025 collaboration revenue was impacted by a $185.0 million recognition event in Q2 2024. |
Net revenues from collaborations decreased in the three months ended June 30, 2025, compared to the same period in 2024, largely due to that specific recognition event with Regeneron Pharmaceuticals.
Global Regulatory Submissions and Approvals (FDA, EMA, PMDA)
Securing global regulatory approvals is a critical channel for market access, particularly for expanding the indications of flagship products like AMVUTTRA.
Significant regulatory achievements for AMVUTTRA (vutrisiran) for ATTR-CM in 2025 include:
- U.S. FDA supplemental New Drug Application (sNDA) approval in Q1 2025.
- European Commission (EC) regulatory approval.
- UK Medicines and Healthcare Products Regulatory Agency (MHRA) approval.
- Japanese Pharmaceuticals and Medical Devices Agency (PMDA) approval.
- Brazilian Health Regulatory Agency (ANVISA) approval.
The PDUFA target action date for the AMVUTTRA ATTR-CM sNDA was March 23, 2025.
Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Customer Segments
The Customer Segments for Alnylam Pharmaceuticals, Inc. are concentrated in patients with rare, genetically defined diseases, with a strategic expansion into prevalent conditions.
Goal by end of 2025:
- Over 0.5 million patients on Alnylam RNAi therapeutics globally.
The current and near-term customer base is segmented as follows:
| Customer Segment | Disease Indication | Relevant Population Data/Metric |
| Patients with Transthyretin (TTR) Amyloidosis | hATTR Amyloidosis with Polyneuropathy (hATTR-PN) | ONPATTRO (patisiran) is approved for this indication. |
| Patients with Transthyretin (TTR) Amyloidosis | ATTR Amyloidosis with Cardiomyopathy (ATTR-CM) | AMVUTTRA (vutrisiran) received U.S. FDA approval for ATTR-CM in March 2025. |
| Patients with Transthyretin (TTR) Amyloidosis | ATTR Amyloidosis (General/Future) | Estimated global population of >300K patients. |
| Patients with Transthyretin (TTR) Amyloidosis | ATTR-CM (Undiagnosed) | Approximately ~80% of ATTR-CM patients are undiagnosed globally. |
| Patients with Transthyretin (TTR) Amyloidosis | ATTR-CM (Treated with Vutrisiran) | Approximately ~1,400 people were on AMVUTTRA therapy at the end of Q2 2025. |
| Patients with Rare Genetic Diseases | Acute Hepatic Porphyria (AHP) | Treated with GIVLAARI; generated $55 million in 2020 revenue. |
| Patients with Rare Genetic Diseases | Primary Hyperoxaluria Type 1 (PH1) | Treated with OXLUMO; generated $300,000 in 2020 revenue. |
| Future: Prevalent Diseases | Hypertension | Over 60 million patients in 7 major markets have uncontrolled hypertension and high CV risk. Phase 3 ZENITH CVOT expected to enroll approximately 11,000 patients. |
| Future: Prevalent Diseases | Cerebral Amyloid Angiopathy (CAA) | CAA is the second leading cause of intracerebral hemorrhage, with approximately 80K cases in the U.S. each year. |
| Future: Prevalent Diseases | Alzheimer's Disease (AD) | Mivelsiran is being evaluated for potential to reduce AD progression. |
| Future: Prevalent Diseases | Huntington's Disease (HD) | Over 100K symptomatic HD patients globally. |
The TTR franchise saw significant growth, with combined net product revenues from AMVUTTRA and ONPATTRO reaching $685 million in the third quarter of 2025.
For the prevalent hypertension segment, the Phase 3 ZENITH trial for zilebesiran is designed to enroll up to 11,000 patients across more than 30 countries.
Alnylam Pharmaceuticals, Inc. is also on track to meet its '2-2-5' pipeline goal, which includes filing Investigational New Drug (IND) applications for nine new Alnylam-led programs by the end of 2025, with two targeting the central nervous system (CNS).
The company reiterated its 2025 combined net product revenue guidance in May 2025 to be between $2,050 million and $2,250 million.
Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Cost Structure
The Cost Structure for Alnylam Pharmaceuticals, Inc. is heavily weighted toward research, development, and commercialization activities necessary to bring complex RNA interference (RNAi) therapeutics to market and support their global rollout. These are significant, ongoing expenditures.
High R&D expenses for clinical trials are a major cost driver. For the three months ended September 30, 2025, non-GAAP Research & Development (R&D) expenses were reported at $310 million. This spending increase, up 23% compared to the prior year period, is directly linked to costs associated with initiating multiple Phase III clinical studies. Specifically, these include the ZENITH Phase III cardiovascular outcomes trial for zilebesiran and the TRITON-CM Phase III study for nucresiran in patients with ATTR amyloidosis with cardiomyopathy.
Commercialization efforts also demand substantial resources, reflected in Significant Selling, General, and Administrative (SG&A) costs. For the same third quarter of 2025, non-GAAP SG&A expenses reached $263 million, marking a 35% increase year-over-year. This rise is attributed to increased headcount and other investments supporting the U.S. launch of AMVUTTRA for ATTR cardiomyopathy.
The overall expected operating expense for the year reflects these investments. Alnylam Pharmaceuticals, Inc. narrowed its full-year 2025 Non-GAAP Combined R&D and SG&A guidance to $2.15 billion to $2.2 billion.
The complexity of producing RNAi drugs translates directly into Manufacturing and supply chain costs, which are captured within the Cost of Goods Sold (COGS). This is evidenced by the gross margin performance on product sales. For the third quarter of 2025, the gross margin on product sales was 77%, a decrease from 80% in the third quarter of 2024.
This margin pressure is directly tied to Royalty payments on product sales. The decrease in gross margin was explicitly driven by increased royalties on AMVUTTRA. In a related transaction, Royalty Pharma acquired a 1% royalty interest in AMVUTTRA for an upfront payment of $310 million, with payments commencing on October 1, 2025, and extending through March 2035. Furthermore, Alnylam Pharmaceuticals, Inc. is eligible to receive tiered royalties ranging from 15 and 30 percent on global net sales of fitusiran, a therapy advanced by its partner Sanofi.
Here's a look at the key expense and margin figures from the third quarter of 2025 and the full-year guidance:
| Cost/Metric Category | Amount/Value | Period/Context |
| Non-GAAP Combined R&D and SG&A Guidance | $2.15 billion to $2.2 billion | Full Year 2025 Guidance |
| Non-GAAP R&D Expenses | $310 million | Q3 2025 |
| Non-GAAP SG&A Expenses | $263 million | Q3 2025 |
| Gross Margin on Product Sales | 77% | Q3 2025 |
| Royalty Revenue | $46 million | Q3 2025 (Doubled year-over-year) |
| AMVUTTRA Royalty Acquisition Cost | $310 million | Upfront payment for 1% royalty (Royalty Pharma) |
| Fitusiran Royalty Tier | 15% to 30% | Tiered royalties on global net sales |
You can see the direct impact of pipeline progression on the P&L. The $46 million in royalty revenue for the quarter shows that some external revenue streams are material, but the high R&D spend is clearly the engine running the cost structure right now.
- Costs driven by initiation of ZENITH and TRITON-CM Phase III trials.
- SG&A increase driven by AMVUTTRA ATTR-CM launch investments.
- Gross margin reduction due to increased royalties on AMVUTTRA.
- Royalty payments to Royalty Pharma for AMVUTTRA commence October 1, 2025.
Finance: draft 13-week cash view by Friday.
Alnylam Pharmaceuticals, Inc. (ALNY) - Canvas Business Model: Revenue Streams
You're looking at the latest top-line projections for Alnylam Pharmaceuticals, Inc. as of late 2025. The company has definitely been seeing strong uptake, leading to guidance increases.
Net Product Sales
Alnylam Pharmaceuticals, Inc. raised its full-year 2025 guidance for total net product revenues. You should note this reflects confidence in the ongoing commercial performance of the marketed RNAi therapeutics.
The updated full-year 2025 guidance for Net Product Sales is set in the range of $2.95 billion to $3.05 billion.
This total revenue stream is segmented across two primary franchises:
| Franchise Segment | 2025 Full-Year Revenue Guidance Range |
| TTR Franchise (AMVUTTRA, ONPATTRO) | $2.475 billion to $2.525 billion |
| Rare Franchise (GIVLAARI, OXLUMO) | $475 million to $525 million |
The TTR franchise guidance was specifically raised to this range, representing a significant increase from prior expectations.
TTR Franchise Sales (AMVUTTRA, ONPATTRO)
The guidance for the TTR franchise, which includes AMVUTTRA (vutrisiran) and ONPATTRO (patisiran), is projected to fall between $2.475 billion and $2.525 billion for fiscal year 2025.
To give you a sense of the momentum, the third quarter of 2025 saw TTR franchise revenues hit $724 million, marking 135% year-over-year growth.
Rare Franchise Sales (GIVLAARI, OXLUMO)
For the Rare franchise, comprising GIVLAARI (givosiran) and OXLUMO (lumasiran), the expected revenue range for the full year 2025 is $475 million to $525 million.
In the third quarter of 2025, the Rare franchise generated $127 million in total net product revenue.
Collaboration Revenue
Alnylam Pharmaceuticals, Inc. also books revenue from its partnerships. The guidance for FY 2025 net revenues from collaborations and royalties combined is set between $650 million and $750 million.
The third quarter of 2025 provided a strong look at this stream:
- Collaboration revenue for Q3 2025 was $352 million.
- This Q3 figure was driven by $300 million in milestone revenue from the Roche partnership related to the ZENITH Phase 3 trial.
- The prior guidance for total net product revenues in Q1 2025 had included a collaboration+royalty revenue expectation of $650 million to $750 million.
Royalty Revenue
A portion of the non-product revenue comes from royalties on partner-led products. You should track LEQVIO (inclisiran), which Alnylam Pharmaceuticals, Inc. partners on with Novartis, as a key driver here.
In the third quarter of 2025, royalty revenue doubled to $46 million, directly attributed to higher LEQVIO sales.
Finance: draft 13-week cash view by Friday.
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