Alnylam Pharmaceuticals(US:ALNY)2025-07-31 12:11PART I. FINANCIAL INFORMATION Financial Statements (Unaudited) This section presents unaudited condensed consolidated financial statements for Q2 2025, including balance sheets, statements of operations, and cash flows, reporting a net loss of $66.3 million Balance Sheet Summary (in thousands) | Financial Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :--- | :--- | :--- | | Total Assets | $4,566,019 | $4,239,983 | | Total Liabilities | $4,315,428 | $4,172,895 | | Total Stockholders' Equity | $250,591 | $67,088 | Condensed Consolidated Statements of Operations (in thousands) | Income Statement Item | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | | :--- | :--- | :--- | | Total Revenues | $773,689 | $659,825 | | Total Operating Costs and Expenses | $789,888 | $611,211 | | Net Loss | $(66,277) | $(16,889) | | Net Loss Per Share (basic and diluted) | $(0.51) | $(0.13) | Notes to Condensed Consolidated Financial Statements Detailed disclosures support financial statements, covering accounting policies, revenue breakdowns, collaboration agreements, and legal contingencies, highlighting strong AMVUTTRA product revenue growth Net Product Revenues (in thousands) | Product Revenue (in thousands) | Q2 2025 | Q2 2024 | YTD 2025 | YTD 2024 | | :--- | :--- | :--- | :--- | :--- | | AMVUTTRA | $491,953 | $230,109 | $801,945 | $425,350 | | ONPATTRO | $52,538 | $77,244 | $102,027 | $146,461 | | GIVLAARI | $80,849 | $62,127 | $147,817 | $120,183 | | OXLUMO | $46,872 | $40,608 | $88,961 | $83,257 | | Total Net Product Revenues | $672,212 | $410,088 | $1,140,750 | $775,251 | Collaboration Revenues (in thousands) | Collaboration Revenue (in thousands) | Q2 2025 | Q2 2024 | YTD 2025 | YTD 2024 | | :--- | :--- | :--- | :--- | :--- | | Roche | $18,267 | $16,506 | $35,323 | $91,186 | | Regeneron Pharmaceuticals | $32,542 | $207,429 | $83,581 | $234,193 | | Novartis AG | $0 | $2,304 | $0 | $16,820 | | Other | $10,687 | $1,099 | $41,777 | $3,687 | | Total | $61,496 | $227,338 | $160,681 | $345,886 | - The company is involved in patent infringement lawsuits filed in March 2022 against Moderna and Pfizer concerning their mRNA COVID-19 vaccines, with court rulings generally unfavorable to Alnylam and judgments of non-infringement entered, though appeals are being considered107111112 Management's Discussion and Analysis of Financial Condition and Results of Operations (MD&A) Management discusses strong AMVUTTRA product revenue growth, offset by decreased collaboration revenue, detailing the Alnylam P5x25 strategy, pipeline updates, and operating expense analysis Overview and Pipeline Alnylam, a commercial-stage biopharmaceutical company, executes its 'Alnylam P5x25' strategy, driven by AMVUTTRA's expanded approval and a robust late-stage pipeline - The company is pursuing its "Alnylam P5x25" strategy with the goal of becoming a top-tier biotech company by the end of 2025, focusing on sustainable innovation and strong financial performance126 - A major growth driver is the recent approval of AMVUTTRA for the treatment of cardiomyopathy of ATTR amyloidosis in the U.S. (March 2025), EU (June 2025), and other key global markets127 - Key late-stage pipeline programs include: - Zilebesiran (Hypertension): Partnered with Roche, with a Phase 3 trial expected to start in H2 2025 - Nucresiran (ATTR Amyloidosis): Phase 3 trial (TRITON-CM) initiated - Mivelsiran (Alzheimer's & CAA): Phase 2 trial in AD expected in H2 2025; Phase 2 in CAA initiated134135 Results of Operations Q2 2025 total revenues increased 17% to $773.7 million, driven by 64% growth in net product revenues from AMVUTTRA, despite a 73% decrease in collaboration revenue Total Revenues (in thousands) | Revenue Line Item (in thousands) | Q2 2025 | Q2 2024 | % Change | | :--- | :--- | :--- | :--- | | Net product revenues | $672,212 | $410,088 | 64% | | Net revenues from collaborations | $61,496 | $227,338 | (73)% | | Royalty revenue | $39,981 | $22,399 | 78% | | Total revenues | $773,689 | $659,825 | 17% | - The sharp decrease in collaboration revenue in Q2 2025 compared to Q2 2024 was primarily driven by the recognition of $185.0 million in revenue from Regeneron in June 2024 related to a license for cemdisiran164168 Operating Costs and Expenses (in thousands) | Operating Costs (in thousands) | Q2 2025 | Q2 2024 | % Change | | :--- | :--- | :--- | :--- | | Cost of goods sold | $142,029 | $67,271 | 111% | | Research and development | $323,621 | $294,142 | 10% | | Selling, general and administrative | $323,314 | $248,397 | 30% | | Total operating costs | $789,888 | $611,211 | 29% | Liquidity and Capital Resources As of June 30, 2025, the company held $1.11 billion in cash and $1.74 billion in marketable securities, sufficient to fund operations for at least 12 months Condensed Consolidated Statements of Cash Flows (in thousands) | Cash Flow Activity (in thousands) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Net cash provided by operating activities | $35,416 | $42,643 | | Net cash used in investing activities | $(27,612) | $(33,680) | | Net cash provided by financing activities | $97,841 | $160,407 | - The company believes its cash, cash equivalents, and marketable securities of $2.86 billion as of June 30, 2025, are sufficient to fund operations for at least the next 12 months from the filing date186216 Quantitative and Qualitative Disclosures About Market Risk No significant changes to financial market risks, primarily interest rate-related, were reported since fiscal year 2024 - There have been no significant changes to the company's financial market risk exposures, which are mainly related to interest rates, as of June 30, 2025, compared to December 31, 2024187 Controls and Procedures Management concluded disclosure controls and procedures were effective as of June 30, 2025, with no material changes in internal control over financial reporting - The CEO and CFO concluded that as of June 30, 2025, the company's disclosure controls and procedures were effective at the reasonable assurance level189 - No changes in internal control over financial reporting occurred during the quarter ended June 30, 2025, that have materially affected, or are reasonably likely to materially affect, these controls190 PART II. OTHER INFORMATION Legal Proceedings This section refers readers to Note 12 of the condensed consolidated financial statements for material pending legal proceedings - For a discussion of material pending legal proceedings, the report refers to Note 12, "Commitments and Contingencies," in the financial statements section192 Risk Factors This section outlines significant risks including commercial success, financial losses, future funding needs, third-party reliance, and drug development uncertainties Financial and Commercial Risks The company faces significant financial and commercial risks, including AMVUTTRA's success, an accumulated deficit of $7.41 billion, and securing substantial future funding - The commercial success of approved products, especially AMVUTTRA for ATTR amyloidosis with cardiomyopathy, is uncertain and critical to achieving profitability201 - The company has a history of significant operating losses, with an accumulated deficit of $7.41 billion as of June 30, 2025, and may not achieve or sustain profitability204 - Substantial funds are required to continue research, development, and commercialization activities, and additional funding may be necessary but might not be available on acceptable terms206208 Risks Related to Dependence on Third Parties Alnylam's business heavily relies on third-party collaborators and CMOs for development, commercialization, and supply, posing risks of partnership termination or manufacturing disruptions - The company's business could be adversely affected if collaborators like Roche, Regeneron, or Novartis terminate their agreements or fail to perform their obligations, potentially delaying or halting the development and commercialization of key products227 - There is a risk that Roche could terminate the zilebesiran collaboration before the Phase 3 trial begins, which would result in the loss of a significant milestone payment and could impact the company's ability to achieve operating profitability in 2025229 - The company relies on a limited number of third-party Contract Manufacturing Organizations (CMOs) for drug substance and all drug product requirements, where the loss of a supplier or their inability to provide sufficient supply could delay clinical trials and harm commercial supply233234 Risks Related to Development, Clinical Testing and Regulatory Approval Product candidates face high failure risks in development due to expensive, lengthy, and uncertain clinical trials, with potential for undesirable side effects and extensive ongoing regulatory oversight - Product candidates may fail in development or experience significant delays, as nonclinical and clinical testing is expensive, lengthy, and has a high historical failure rate257 - Undesirable side effects or unexpected adverse properties in products or product candidates could delay or prevent regulatory approval, limit commercial potential, or result in significant negative consequences post-approval268 - Marketed products and approved candidates are subject to extensive and ongoing regulatory oversight, and failure to comply with continuing U.S. and foreign requirements could result in limited or withdrawn approvals and other penalties286 Risks Related to Competition The company faces intense competition from large pharmaceutical companies and existing treatments, with AMVUTTRA competing against lower-priced oral drugs and alternative technologies - The company faces intense competition from large pharmaceutical companies with greater resources and from existing approved drugs for the same conditions371 - AMVUTTRA for ATTR-CM competes with orally administered drugs VYNDAQEL/VYNDAMAX (Pfizer) and ATTRUBY (BridgeBio), which have lower list prices in the U.S., potentially impacting AMVUTTRA's commercial success372 - The company faces direct competition from other companies developing RNAi therapeutics (e.g., Arrowhead, Roche, Silence Therapeutics) and from alternative platforms like antisense technology (e.g., Ionis)378379 Other Information This section discloses that during Q2 2025, certain officers and directors entered into Rule 10b5-1 trading plans for the future sale of company securities - During the quarter ended June 30, 2025, certain officers and directors adopted Rule 10b5-1 trading plans for the sale of company securities, including: - Dennis A. Ausiello, M.D. (Director) - Colleen F. Reitan (Director) - Tolga Tanguler (Chief Commercial Officer)405406407408 Exhibits This section lists exhibits filed with the Form 10-Q, primarily consisting of certifications from the principal executive and financial officers and Inline XBRL documents - The exhibits filed with this report include certifications by the CEO and CFO pursuant to SEC rules, as well as Inline XBRL data files409