PART I. FINANCIAL INFORMATION [Financial Statements (Unaudited)](index=6&type=section&id=ITEM%201.%20FINANCIAL%20STATEMENTS%20%28UNAUDITED%29) 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](index=11&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) 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 considered[107](index=107&type=chunk)[111](index=111&type=chunk)[112](index=112&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations (MD&A)](index=27&type=section&id=ITEM%202.%20MANAGEMENT%27S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) 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](index=27&type=section&id=Overview%20and%20Pipeline) 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 performance**[126](index=126&type=chunk) - 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 markets[127](index=127&type=chunk) - 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 initiated[134](index=134&type=chunk)[135](index=135&type=chunk) [Results of Operations](index=33&type=section&id=Results%20of%20Operations) 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 cemdisiran[164](index=164&type=chunk)[168](index=168&type=chunk) 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](index=38&type=section&id=Liquidity%20and%20Capital%20Resources) 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 date[186](index=186&type=chunk)[216](index=216&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=38&type=section&id=ITEM%203.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) 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, 2024[187](index=187&type=chunk) [Controls and Procedures](index=38&type=section&id=ITEM%204.%20CONTROLS%20AND%20PROCEDURES) 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 level**[189](index=189&type=chunk) - **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 controls[190](index=190&type=chunk) PART II. OTHER INFORMATION [Legal Proceedings](index=40&type=section&id=ITEM%201.%20LEGAL%20PROCEEDINGS) 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 section[192](index=192&type=chunk) [Risk Factors](index=40&type=section&id=ITEM%201A.%20RISK%20FACTORS) This section outlines significant risks including commercial success, financial losses, future funding needs, third-party reliance, and drug development uncertainties [Financial and Commercial Risks](index=41&type=section&id=Financial%20and%20Commercial%20Risks) 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 profitability**[201](index=201&type=chunk) - 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 profitability[204](index=204&type=chunk) - **Substantial funds are required** to continue research, development, and commercialization activities, and **additional funding may be necessary** but might not be available on acceptable terms[206](index=206&type=chunk)[208](index=208&type=chunk) [Risks Related to Dependence on Third Parties](index=45&type=section&id=Risks%20Related%20to%20Dependence%20on%20Third%20Parties) 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 products[227](index=227&type=chunk) - 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 2025[229](index=229&type=chunk) - 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 supply**[233](index=233&type=chunk)[234](index=234&type=chunk) [Risks Related to Development, Clinical Testing and Regulatory Approval](index=51&type=section&id=Risks%20Related%20to%20Development%2C%20Clinical%20Testing%20and%20Regulatory%20Approval) 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 rate**[257](index=257&type=chunk) - **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-approval[268](index=268&type=chunk) - 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 penalties[286](index=286&type=chunk) [Risks Related to Competition](index=71&type=section&id=Risks%20Related%20to%20Competition) 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 conditions[371](index=371&type=chunk) - **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 success[372](index=372&type=chunk) - 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)[378](index=378&type=chunk)[379](index=379&type=chunk) [Other Information](index=76&type=section&id=ITEM%205.%20OTHER%20INFORMATION) 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)[405](index=405&type=chunk)[406](index=406&type=chunk)[407](index=407&type=chunk)[408](index=408&type=chunk) [Exhibits](index=77&type=section&id=ITEM%206.%20EXHIBITS) 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 files**[409](index=409&type=chunk)

[Executive Summary](index=1&type=section&id=Executive%20Summary) Alnylam reported strong Q2 2025 results, with **64%** total net product revenue growth, driven by TTR, and revised full-year guidance upwards [Q2 2025 Performance Overview](index=1&type=section&id=Q2%202025%20Performance%20Overview) Alnylam Pharmaceuticals reported strong Q2 2025 financial results, with total net product revenues growing **64%** year-over-year, driven by the TTR franchise, leading to an upward revision of full-year 2025 revenue guidance - Total Net Product Revenues for Q2 2025 reached **$672 Million**, marking a **64% growth** compared to Q2 2024[1](index=1&type=chunk) - Total TTR Revenues were **$544 Million**, showing a **77% growth** compared to Q2 2024, largely due to AMVUTTRA sales in ATTR-CM[1](index=1&type=chunk)[2](index=2&type=chunk) - Approximately **1,400 ATTR-CM patients** were on AMVUTTRA as of June 30, 2025[1](index=1&type=chunk) - AMVUTTRA received approvals for ATTR-CM treatment in the European Union, Brazil, the United Kingdom, and Japan[1](index=1&type=chunk) - The company initiated the TRITON-CM Phase 3 Trial of Nucresiran in patients with wild-type or hereditary ATTR-CM[1](index=1&type=chunk) FY 2025 Guidance Update | Metric | Prior FY 2025 Guidance | Updated FY 2025 Guidance | Change at Midpoint | | :--------------------------------------- | :--------------------- | :----------------------- | :----------------- | | Total TTR net product revenues | $1,600M - $1,725M | $2,175M - $2,275M | +$575M / 27% | | Total net product revenues | $2,050M - $2,250M | $2,650M - $2,800M | +$575M / 27% | [Second Quarter 2025 and Recent Significant Business Highlights](index=2&type=section&id=Second%20Quarter%202025%20and%20Recent%20Significant%20Business%20Highlights) This section details Alnylam's product performance, pipeline advancements, and key upcoming events for Q2 2025 [Product Performance Highlights](index=2&type=section&id=Product%20Performance%20Highlights) Alnylam's product portfolio demonstrated strong growth in Q2 2025, particularly the TTR franchise driven by AMVUTTRA, while the Rare franchise also contributed positively. ONPATTRO experienced a decline due to patient migration to AMVUTTRA [Total TTR: AMVUTTRA (vutrisiran) & ONPATTRO (patisiran)](index=2&type=section&id=Total%20TTR%3A%20AMVUTTRA%20(vutrisiran)%20%26%20ONPATTRO%20(patisiran)) The TTR franchise saw substantial growth, primarily fueled by AMVUTTRA's strong uptake in ATTR-CM patients and global regulatory approvals. ONPATTRO's revenue decreased as patients transitioned to AMVUTTRA TTR Net Product Revenues | Product | Q2 2025 Net Product Revenues | Q2 2024 Net Product Revenues | % Change | | :-------- | :--------------------------- | :--------------------------- | :------- | | AMVUTTRA | $492 million | $230 million | 114% | | ONPATTRO | $53 million | $77 million | (32)% | | Total TTR | $544 million | $307 million | 77% | - Approximately **1,400 ATTR-CM patients** were on AMVUTTRA as of June 30, 2025[7](index=7&type=chunk) - AMVUTTRA received regulatory approvals for ATTR-CM in Brazil, the European Commission, UK MHRA, and Japanese PMDA[7](index=7&type=chunk) - Additional 42-month data from the HELIOS-B Phase 3 trial reinforced vutrisiran's effect on all-cause mortality (**36% reduction**) and CV mortality (**33% reduction**) in the overall population[7](index=7&type=chunk) [Total Rare: GIVLAARI (givosiran) & OXLUMO (lumasiran)](index=2&type=section&id=Total%20Rare%3A%20GIVLAARI%20(givosiran)%20%26%20OXLUMO%20(lumasiran)) The Total Rare franchise, comprising GIVLAARI and OXLUMO, achieved solid revenue growth in Q2 2025 Rare Net Product Revenues | Product | Q2 2025 Net Product Revenues | Q2 2024 Net Product Revenues | % Change | | :---------- | :--------------------------- | :--------------------------- | :------- | | GIVLAARI | $81 million | $62 million | 30% | | OXLUMO | $47 million | $41 million | 15% | | Total Rare | $128 million | $103 million | 24% | [Pipeline & Clinical Development Updates](index=2&type=section&id=Pipeline%20%26%20Clinical%20Development%20Updates) Alnylam made significant strides in its pipeline, initiating a new Phase 3 trial for nucresiran in ATTR-CM, presenting positive Phase 1 data for mivelsiran in Alzheimer's, and advancing other programs - Initiated the TRITON-CM Phase 3 trial of nucresiran in patients with ATTR-CM, an event-driven cardiovascular outcomes trial targeting **1,200 patients**[7](index=7&type=chunk) - Nucresiran received Fast Track Designation from the FDA for the treatment of ATTR-CM to reduce mortality, cardiovascular hospitalizations, and urgent heart failure visits[7](index=7&type=chunk) - Presented Phase 1 data for mivelsiran in Alzheimer's disease, showing robust, durable, dose-dependent reductions of soluble amyloid precursor protein beta (sAPPβ) in CSF[8](index=8&type=chunk) - Initiated a Phase 1 clinical trial for ALN-4324, an insulin sensitizer targeting GRB14 for type 2 diabetes[8](index=8&type=chunk) - Partner Agios Pharmaceuticals received IND clearance for AG-236 (ALN-TMP) for polycythemia vera (PV) and initiated a Phase 1 study[8](index=8&type=chunk) [Key Upcoming Events](index=3&type=section&id=Key%20Upcoming%20Events) Alnylam has several important clinical data presentations and trial initiations planned for the second half of 2025, including results for zilebesiran and nucresiran, and a new Phase 2 trial for mivelsiran - Results from the KARDIA-3 Phase 2 trial of zilebesiran in hypertension will be presented at the European Society of Cardiology (ESC) Congress on August 30, 2025[10](index=10&type=chunk) - Expected to initiate a Phase 3 cardiovascular outcomes trial of zilebesiran in collaboration with Roche in H2 2025[13](index=13&type=chunk) - Expected to initiate the TRITON-PN Phase 3 trial of nucresiran in hATTR-PN in H2 2025[13](index=13&type=chunk) - Expected to initiate a Phase 2 trial of mivelsiran in Alzheimer's disease in H2 2025[13](index=13&type=chunk) - Partner Regeneron Pharmaceuticals plans to share results from the Phase 3 trial of cemdisiran in myasthenia gravis in H2 2025[11](index=11&type=chunk) [Financial Results for the Quarter Ended June 30, 2025](index=3&type=section&id=Financial%20Results%20for%20the%20Quarter%20Ended%20June%2030%2C%202025) This section provides a detailed analysis of Alnylam's Q2 2025 financial performance, including revenues, operating expenses, and other financial items [Consolidated Financial Results Overview](index=3&type=section&id=Consolidated%20Financial%20Results%20Overview) Alnylam reported a **17%** increase in total revenues for Q2 2025 compared to Q2 2024, but experienced a GAAP operating loss and a significant increase in GAAP net loss per common share, primarily due to increased operating expenses and a decrease in non-GAAP operating income Consolidated Financial Performance | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | % Change | | :-------------------------------------- | :--------------------- | :--------------------- | :------- | | Total revenues | $773,689 | $659,825 | 17% | | GAAP (Loss) income from operations | $(16,199) | $48,614 | (133)% | | Non-GAAP Income from operations | $95,481 | $137,902 | (31)% | | GAAP Net loss per common share – basic | $(0.51) | $(0.13) | 292% | | Non-GAAP Net income per common share – basic | $0.34 | $0.58 | (41)% | [Revenue Analysis](index=4&type=section&id=Revenue%20Analysis) Total net product revenues surged by **64%** year-over-year, driven by AMVUTTRA's strong performance, partially offset by ONPATTRO's decline. Net revenues from collaborations significantly decreased due to a prior-year licensing deal with Regeneron, while royalty revenue saw substantial growth Revenue Breakdown by Product and Type | Revenue Type | Q2 2025 (in thousands) | Q2 2024 (in thousands) | % Change | % Change at CER | | :---------------------------- | :--------------------- | :--------------------- | :------- | :-------------- | | AMVUTTRA | $491,953 | $230,109 | 114% | 112% | | ONPATTRO | $52,538 | $77,244 | (32)% | (33)% | | Total TTR net product revenues| $544,491 | $307,353 | 77% | 75% | | GIVLAARI | $80,849 | $62,127 | 30% | 29% | | OXLUMO | $46,872 | $40,608 | 15% | 13% | | Total Rare net product revenues| $127,721 | $102,735 | 24% | 23% | | Total net product revenues | $672,212 | $410,088 | 64% | 62% | | Net revenues from collaborations| $61,496 | $227,338 | (73)% | (73)% | | Royalty revenue | $39,981 | $22,399 | 78% | 78% | | Total revenues | $773,689 | $659,825 | 17% | 16% | - Total net product revenues increased **64% (62% at CER)** due to AMVUTTRA growth from increased patient demand, mainly in ATTR-CM in the U.S., partially offset by ONPATTRO patient switches, and growth from GIVLAARI and OXLUMO[15](index=15&type=chunk) - Net revenues from collaborations decreased **73%** primarily due to a **$185.0 million** revenue recognition in Q2 2024 from a collaboration modification with Regeneron Pharmaceuticals[16](index=16&type=chunk) [Operating Expenses Analysis](index=4&type=section&id=Operating%20Expenses%20Analysis) Operating expenses increased across the board in Q2 2025. Cost of goods sold rose significantly due to higher AMVUTTRA sales and associated royalties. R&D expenses increased due to clinical trial startup activities for zilebesiran and nucresiran, while SG&A expenses grew due to higher employee compensation and marketing investments for AMVUTTRA's launch Operating Expenses | Expense Type | Q2 2025 (in thousands) | Q2 2024 (in thousands) | % Change | | :----------------------------------------- | :--------------------- | :--------------------- | :------- | | Cost of goods sold | $142,029 | $67,271 | 111% | | % of net product revenues | 21.1% | 16.4% | | | Cost of collaborations and royalties | $924 | $1,401 | (34)% | | GAAP Research and development expenses | $323,621 | $294,142 | 10% | | Non-GAAP Research and development expenses | $274,069 | $246,027 | 11% | | GAAP Selling, general and administrative expenses | $323,314 | $248,397 | 30% | | Non-GAAP Selling, general and administrative expenses | $261,186 | $207,224 | 26% | - Cost of goods sold increased **111%** primarily due to increased AMVUTTRA sales and associated royalties[18](index=18&type=chunk) - GAAP and non-GAAP R&D expenses increased **10%** and **11%** respectively, driven by clinical trial startup activities for zilebesiran (Phase 3 cardiovascular outcomes) and nucresiran (TRITON-CM Phase 3)[19](index=19&type=chunk) - GAAP and non-GAAP SG&A expenses increased **30%** and **26%** respectively, due to higher employee compensation costs and increased marketing investment for AMVUTTRA's ATTR-CM launch[20](index=20&type=chunk) [Other Financial Items](index=5&type=section&id=Other%20Financial%20Items) Interest expense included a significant portion related to the sale of future Leqvio royalties. Total other expense, net, was impacted by a charge from the change in fair value of the development derivative liability. The company recorded an income tax provision primarily due to income generated in Switzerland - Interest expense for Q2 2025 was **$40.2 million**, with **$36.5 million** attributed to the liability related to the sale of future Leqvio royalties[21](index=21&type=chunk) - Total other expense, net, was **$19.2 million**, including a **$15.3 million** charge from the change in fair value of the development derivative liability, driven by updates to estimated royalties due to Blackstone on AMVUTTRA sales[22](index=22&type=chunk) - Provision for income taxes was **$30.9 million**, mainly due to income in Switzerland and state income taxes in the U.S[23](index=23&type=chunk) [Financial Position & Cash Flow](index=5&type=section&id=Financial%20Position%20%26%20Cash%20Flow) Alnylam's cash, cash equivalents, and marketable securities increased to **$2.86 billion** as of June 30, 2025, primarily driven by net cash inflows from operating activities and proceeds from employee stock option exercises - Cash, cash equivalents and marketable securities were **$2.86 billion** as of June 30, 2025, up from **$2.63 billion** as of March 31, 2025[26](index=26&type=chunk) - The increase in cash was primarily driven by net cash inflows from operating activities and proceeds from the exercise of employee stock options[26](index=26&type=chunk) - Net cash provided by operating activities included **$14.4 million** of payments associated with the Leqvio royalties liability[26](index=26&type=chunk) - Net cash provided by financing activities included **$27.5 million** of payments to Blackstone for development and regulatory approval milestones and AMVUTTRA sales royalties[26](index=26&type=chunk) [2025 Financial Guidance](index=6&type=section&id=2025%20Financial%20Guidance) Alnylam has raised its full-year 2025 financial guidance for total TTR net product revenues and total net product revenues, reflecting strong performance, particularly from the TTR franchise. Guidance for Total Rare net product revenues, net revenues from collaborations and royalties, and non-GAAP operating income was reiterated Updated FY 2025 Financial Guidance | Item | Prior FY 2025 Guidance | Updated FY 2025 Guidance | | :---------------------------------------- | :--------------------- | :----------------------- | | Total TTR net product revenues (PN & CM) | $1,600M - $1,725M | $2,175M - $2,275M | | Total Rare net product revenues | $450M - $525M | $475M - $525M | | Total net product revenues | $2,050M - $2,250M | $2,650M - $2,800M | | Net product revenues growth vs. 2024 (actual) | 25% to 37% | 61% to 70% | | Net product revenues growth vs. 2024 (CER) | 26% to 39% | 59% to 68% | | Net revenues from collaborations and royalties | $650M - $750M | Reiterate FY guidance | | GAAP R&D and SG&A expenses | $2,370M - $2,530M | $2,445M - $2,575M | | Non-GAAP R&D and SG&A expenses | $2,100M - $2,200M | Reiterate FY guidance | | Non-GAAP Operating income | Achieve profitability | Reiterate FY guidance | [Use of Non-GAAP Financial Measures](index=6&type=section&id=Use%20of%20Non-GAAP%20Financial%20Measures) Non-GAAP measures provide insights into Alnylam's operations by excluding non-cash and non-recurring items for clearer performance evaluation - Non-GAAP measures exclude **stock-based compensation expenses** and **realized/unrealized gains or losses on marketable equity securities**[29](index=29&type=chunk) - The company believes non-GAAP measures provide useful information to management and investors for evaluating performance, allocating resources, and planning[31](index=31&type=chunk) - Percentage changes in revenue growth at Constant Exchange Rates (CER) are presented to show underlying business performance without foreign currency fluctuations[30](index=30&type=chunk) [Company and Product Information](index=7&type=section&id=Company%20and%20Product%20Information) This section provides detailed information about Alnylam Pharmaceuticals and its key commercial products [About AMVUTTRA (vutrisiran)](index=7&type=section&id=About%20AMVUTTRA%20(vutrisiran)) AMVUTTRA is an RNAi therapeutic for transthyretin (ATTR) amyloidosis, administered quarterly via subcutaneous injection. It is approved in over 15 countries for hATTR-PN and in the U.S., EU, Brazil, UK, and Japan for ATTR-CM to reduce cardiovascular mortality and hospitalizations - AMVUTTRA is an RNAi therapeutic that rapidly knocks down transthyretin (TTR), addressing the underlying cause of ATTR amyloidosis[34](index=34&type=chunk) - Administered quarterly via subcutaneous injection by a healthcare professional[34](index=34&type=chunk) - Approved in over 15 countries for hATTR-PN and in the U.S., Brazil, EU, UK, and Japan for ATTR-CM to reduce cardiovascular mortality, hospitalizations, and urgent heart failure visits[34](index=34&type=chunk) [About ONPATTRO (patisiran)](index=7&type=section&id=About%20ONPATTRO%20(patisiran)) ONPATTRO is an intravenously administered RNAi therapeutic approved in the U.S., Canada, EU, Switzerland, Brazil, and Japan for the treatment of hATTR amyloidosis with polyneuropathy. It works by silencing TTR messenger RNA to reduce pathogenic protein production - ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR)[35](index=35&type=chunk) - Approved in the U.S., Canada, EU, Switzerland, Brazil, and Japan for hATTR amyloidosis with polyneuropathy[35](index=35&type=chunk) - Designed to silence TTR messenger RNA, reducing TTR protein production and amyloid deposits[35](index=35&type=chunk) [About GIVLAARI (givosiran)](index=7&type=section&id=About%20GIVLAARI%20(givosiran)) GIVLAARI is an RNAi therapeutic targeting ALAS1, approved in the U.S., Brazil, and EU for acute hepatic porphyria (AHP) in adults and adolescents. It significantly reduces porphyria attacks and is administered monthly via subcutaneous injection - GIVLAARI is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1)[36](index=36&type=chunk) - Approved in the U.S., Brazil, and EU for the treatment of adults and adolescents (12+) with acute hepatic porphyria (AHP)[36](index=36&type=chunk) - Significantly reduces the rate of porphyria attacks requiring hospitalization, urgent healthcare visits, or IV hemin[36](index=36&type=chunk) - Administered once monthly via subcutaneous injection, utilizing Alnylam's Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology[36](index=36&type=chunk) [About OXLUMO (lumasiran)](index=7&type=section&id=About%20OXLUMO%20(lumasiran)) OXLUMO is an RNAi therapeutic targeting HAO1, approved by the FDA and EMA for primary hyperoxaluria type 1 (PH1) in all age groups. It lowers urinary and plasma oxalate levels by inhibiting glycolate oxidase production, with injection site reactions being the most common adverse reaction - OXLUMO is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1), which encodes glycolate oxidase (GO)[37](index=37&type=chunk) - Approved by the FDA and EMA for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients[37](index=37&type=chunk) - Utilizes Alnylam's ESC-GalNAc-conjugate technology for subcutaneous dosing[37](index=37&type=chunk) - Demonstrated significant reductions in urinary oxalate in pivotal trials, with injection site reactions as the most common drug-related adverse reaction[37](index=37&type=chunk) [About Alnylam Pharmaceuticals](index=8&type=section&id=About%20Alnylam%20Pharmaceuticals) Alnylam is a leading RNAi therapeutics company, translating Nobel Prize-winning science into innovative medicines for rare and prevalent diseases. The company is executing its 'Alnylam P x25' strategy to deliver transformative medicines, achieve sustainable profitability, and solidify its position as a top-tier biotech company - Alnylam is a leading RNAi therapeutics company, founded in 2002, focused on translating RNA interference into innovative medicines[40](index=40&type=chunk) - Commercial RNAi therapeutic products include AMVUTTRA, ONPATTRO, GIVLAARI, and OXLUMO, with Leqvio and Qfitlia commercialized by partners[40](index=40&type=chunk) - Executing on its 'Alnylam P x25' strategy to deliver transformative medicines, achieve sustainable profitability, and become a leading biotech company[40](index=40&type=chunk) [Alnylam Forward Looking Statements](index=8&type=section&id=Alnylam%20Forward%20Looking%20Statements) This section outlines Alnylam's forward-looking statements, including commercial and pipeline expectations, and associated risks - Statements regarding Alnylam's ability to deliver and enable commercial access to medicines, advance its pipeline, and achieve P5x25 goals are forward-looking[41](index=41&type=chunk) - Actual results may differ materially due to risks and uncertainties including successful execution of strategy, efficacy and safety of product candidates, regulatory actions, manufacturing, intellectual property, and dependence on third parties[41](index=41&type=chunk)[42](index=42&type=chunk) - The company explicitly disclaims any obligation to update forward-looking statements, except as required by law[42](index=42&type=chunk) [Condensed Consolidated Financial Statements](index=10&type=section&id=Condensed%20Consolidated%20Financial%20Statements) This section presents Alnylam's condensed consolidated financial statements, including balance sheets, statements of operations, and reconciliations of GAAP to non-GAAP measures [Condensed Consolidated Balance Sheets](index=10&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Alnylam's balance sheet as of June 30, 2025, shows an increase in total assets, driven by higher cash and accounts receivable. Total liabilities also increased, with a notable rise in accrued expenses and development derivative liability, while total stockholders' equity significantly improved Condensed Consolidated Balance Sheets | Item | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :---------------------------------------- | :--------------------------- | :------------------------------- | | Cash and cash equivalents | $1,113,685 | $966,428 | | Marketable debt securities | $1,743,900 | $1,719,920 | | Accounts receivable, net | $567,112 | $405,308 | | Total current assets | $3,643,699 | $3,295,285 | | Total assets | $4,566,019 | $4,239,983 | | Accrued expenses | $918,966 | $793,692 | | Development derivative liability (current) | $120,788 | $93,780 | | Total current liabilities | $1,300,847 | $1,186,272 | | Convertible debt | $1,026,522 | $1,024,621 | | Total liabilities | $4,315,428 | $4,172,895 | | Total stockholders' equity | $250,591 | $67,088 | [Condensed Consolidated Statements of Operations](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) For Q2 2025, Alnylam reported a **17%** increase in total revenues but a significant GAAP net loss of **$66.3 million**, compared to a net loss of **$16.9 million** in Q2 2024. This was driven by higher operating costs, particularly R&D and SG&A, and increased provision for income taxes, despite strong product revenue growth Condensed Consolidated Statements of Operations | Item | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | | :------------------------------------ | :---------------------------------------------- | :---------------------------------------------- | | Net product revenues | $672,212 | $410,088 | | Net revenues from collaborations | $61,496 | $227,338 | | Royalty revenue | $39,981 | $22,399 | | Total revenues | $773,689 | $659,825 | | Cost of goods sold | $142,029 | $67,271 | | Research and development | $323,621 | $294,142 | | Selling, general and administrative | $323,314 | $248,397 | | Total operating costs and expenses | $789,888 | $611,211 | | (Loss) income from operations | $(16,199) | $48,614 | | Total other expense, net | $(19,159) | $(59,781) | | Provision for income taxes | $(30,919) | $(5,722) | | Net loss | $(66,277) | $(16,889) | | Net loss per common share — basic and diluted | $(0.51) | $(0.13) | [Reconciliation of GAAP to Non-GAAP Measures](index=12&type=section&id=Reconciliation%20of%20GAAP%20to%20Non-GAAP%20Measures) This section provides a reconciliation of GAAP to non-GAAP financial measures, primarily adjusting for stock-based compensation expenses and realized/unrealized gains/losses on marketable equity securities. These adjustments convert GAAP net loss to non-GAAP net income, offering an alternative view of the company's operational profitability GAAP to Non-GAAP Reconciliation | Item | Three Months Ended June 30, 2025 (in thousands) | Three Months Ended June 30, 2024 (in thousands) | | :---------------------------------------- | :---------------------------------------------- | :---------------------------------------------- | | GAAP Research and development expenses | $323,621 | $294,142 | | Less: Stock-based compensation expenses | $(49,552) | $(48,115) | | Non-GAAP Research and development expenses| $274,069 | $246,027 | | GAAP Selling, general and administrative expenses | $323,314 | $248,397 | | Less: Stock-based compensation expenses | $(62,128) | $(41,173) | | Non-GAAP Selling, general and administrative expenses | $261,186 | $207,224 | | GAAP (Loss) income from operations | $(16,199) | $48,614 | | Add: Stock-based compensation expenses | $111,680 | $89,288 | | Non-GAAP Operating income | $95,481 | $137,902 | | GAAP Net loss | $(66,277) | $(16,889) | | Add: Stock-based compensation expenses | $111,680 | $89,288 | | Add: Realized and unrealized loss on marketable equity securities | $1,350 | $1,367 | | Less: Income tax effect of GAAP to non-GAAP reconciling items | $(2,631) | — | | Non-GAAP Net income | $44,122 | $73,766 | | Non-GAAP Net income per common share – basic | $0.34 | $0.58 | | Non-GAAP Net income per common share – diluted | $0.32 | $0.56 | [Reconciliation of GAAP to Non-GAAP Product Revenue Growth at Constant Currency](index=13&type=section&id=Reconciliation%20of%20GAAP%20to%20Non-GAAP%20Product%20Revenue%20Growth%20at%20Constant%20Currency) This reconciliation adjusts reported product revenue growth rates for the impact of foreign currency translation, providing a 'constant currency' view to better reflect underlying business performance. For Q2 2025, total net product revenue growth at constant currency was **62%**, slightly lower than the reported **64%** Product Revenue Growth at Constant Currency | Product/Category | Reported Growth | Impact of Foreign Currency Translation | Growth at Constant Currency | | :-------------------------------- | :-------------- | :------------------------------------- | :-------------------------- | | AMVUTTRA net product revenue | 114% | (2)% | 112% | | ONPATTRO net product revenue | (32)% | (1)% | (33)% | | Total TTR net product revenue | 77% | (2)% | 75% | | GIVLAARI net product revenue | 30% | (1)% | 29% | | OXLUMO net product revenue | 15% | (2)% | 13% | | Total Rare net product revenue | 24% | (1)% | 23% | | Total net product revenue | 64% | (2)% | 62% | | Total revenue | 17% | (1)% | 16% |

Core Viewpoint - The market anticipates Alnylam Pharmaceuticals (ALNY) to report a year-over-year decline in earnings despite an increase in revenues for the quarter ended June 2025, with actual results being crucial for stock price movement [1][2]. Financial Expectations - Alnylam is expected to report a quarterly loss of $0.03 per share, reflecting a year-over-year change of -105.4% [3]. - Revenues are projected to be $671.11 million, which is a 1.7% increase from the same quarter last year [3]. Estimate Revisions - The consensus EPS estimate has been revised 62.96% higher in the last 30 days, indicating a reassessment by analysts [4]. - A positive Earnings ESP of +831.25% suggests that analysts have recently become more optimistic about Alnylam's earnings prospects [12]. Earnings Surprise History - In the last reported quarter, Alnylam was expected to post a loss of $0.56 per share but actually reported a loss of -$0.01, resulting in a surprise of +98.21% [13]. - Over the past four quarters, Alnylam has beaten consensus EPS estimates three times [14]. Industry Comparison - Incyte (INCY), another player in the Zacks Medical - Biomedical and Genetics industry, is expected to post earnings of $1.4 per share for the same quarter, indicating a year-over-year change of +176.9% [18]. - Incyte's revenues are expected to reach $1.15 billion, up 10.3% from the previous year [18].

Core Viewpoint - Alnylam Pharmaceuticals (ALNY) has received a Zacks Rank 2 (Buy) upgrade, indicating a positive outlook on its earnings estimates, which is a significant factor influencing stock prices [1][4]. Earnings Estimates and Stock Performance - The Zacks rating system focuses on a company's changing earnings picture, with the Zacks Consensus Estimate tracking EPS estimates from sell-side analysts [2]. - A strong correlation exists between changes in earnings estimates and near-term stock price movements, with institutional investors using these estimates to assess fair value [5]. Recent Developments for Alnylam - Alnylam's earnings estimates have seen a notable increase, with the Zacks Consensus Estimate for the fiscal year ending December 2025 rising by 52.6% [9]. - The upgrade to Zacks Rank 2 places Alnylam in the top 20% of Zacks-covered stocks, suggesting potential for market-beating returns in the near term [11]. Zacks Rank System - The Zacks Rank system classifies stocks into five groups based on earnings estimates, with a proven track record of Zacks Rank 1 stocks generating an average annual return of +25% since 1988 [8]. - The system maintains a balanced distribution of "buy" and "sell" ratings across over 4,000 stocks, ensuring that only the top 5% receive a "Strong Buy" rating [10].

Core Viewpoint - The case of the Astellas Pharma employee being sentenced for espionage highlights the complexities of Sino-Japanese relations and the implications of China's legal system on foreign nationals [1][2]. Group 1: Legal Proceedings - The Beijing Second Intermediate People's Court sentenced the Astellas employee to 3 years and 6 months in prison for espionage, recognizing his actions of receiving compensation for providing information to a Japanese agency as espionage activities [1][2]. - The Chinese authorities encouraged the employee to confess, indicating that a guilty plea could lead to a lighter sentence, which reflects a judicial transaction in this rare case involving a Japanese national [2]. - The legal basis for the plea deal stems from the revised Criminal Procedure Law implemented in October 2018, which allows for reduced sentences in exchange for confessions [2]. Group 2: Background Information - The detained individual, over 60 years old, had served as a senior executive for Astellas in China for more than 20 years and was previously the vice president of the China Japan Chamber of Commerce [3]. - The employee was detained in March 2023 as he was preparing to return to Japan, indicating a strategic timing by Chinese authorities [3]. - The case is part of ongoing tensions between China and Japan, with the Chinese leadership closely monitoring relations with neighboring countries amid broader geopolitical dynamics [2].

日本经济新闻（中文版：日经中文网）田岛如生 北京报道 安斯泰来 版权声明：日本经济新闻社版权所有，未经授权不得转载或部分复制，违者必究。 日本驻华大使馆的职员正在就旁听审判进行协调。日本政府多次要求中国方面尽早释放。中 方则表示将依法进行处罚，未同意日方要求…… 日经中文网 https://cn.nikkei.com 北京市第二中级人民法院将于7月16日对被起诉间谍罪的日本安斯泰来制药日籍男员工进行公 开宣判。日本驻华大使馆的职员正在就旁听审判进行协调。中日相关人士透露了上述消息。 该法院7月9日向日本大使馆转达了消息。围绕该男子被拘事件，包括日本首相石破茂在内， 日本政府多次要求中国方面尽早释放。中方则表示将依法进行处罚，未同意日方要求。 该男子是日本安斯泰来制药的当地法人高管，2023年3月在北京被逮捕。中国检察机关于 2024年8月以间谍罪起诉该男子，但并未透露具体罪状。该法院于2024年11月进行了首次公 审。 ...

Core Insights - Alnylam Pharmaceuticals (ALNY) is currently in Phase 10 of its 18-phase Adhishthana Cycle, with expectations for a peak formation in Phase 11, scheduled to end in September 2025 [1][5][6] Phase Analysis - The stock experienced a significant gap-up of over 30% on June 24 after positive results from its drug Vutrisiran for treating transthyretin amyloid cardiomyopathy, aligning with the principles of the Adhishthana framework [4][5] - Phase 9 was characterized by powerful structural shifts, leading to the current bullish stance in Phase 10, which is still building without signs of a peak [5][6] - Peak formation in Phase 10 typically occurs between the 18th and 23rd bars, but ALNY has not yet shown signs of this peak [6] Monthly Chart Insights - On the monthly chart, ALNY is in Phase 6, associated with the creation of a Nirvana level, projected to emerge around $240 by September 30, 2025 [9][10] - The stock has been forming its monthly Adhishthana Cakra since Phase 4 and is currently near the upper arc of this structure, indicating potential exhaustion as Phase 10 matures [10] Investor Outlook - Existing investors are advised to hold while monitoring for signs of structural breakdown, which would indicate a descent phase, referred to as the "Wrath of Ganga" [11] - New entrants are cautioned against entering during Phase 10 due to poor risk-reward dynamics, as the stock is trading near the higher arc of the monthly Adhishthana Cakra [12]

Core Insights - Alnylam Pharmaceuticals has received European Commission approval for the label expansion of its RNAi therapeutic Amvuttra to treat wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy [1][9] - Year-to-date, Alnylam's stock has increased by 29.1%, significantly outperforming the industry, which has seen a decline of 0.9% [2] Approval Details - The European Commission's approval was based on positive results from the late-stage HELIOS-B study, where Amvuttra met all 10 pre-specified primary and secondary endpoints [3][9] - The HELIOS-B trial demonstrated statistically significant reductions in all-cause mortality and recurrent cardiovascular events, along with major improvements in functional capacity, health status, and heart failure symptoms [4][9] Competitive Advantage - Amvuttra is administered quarterly via subcutaneous injection, providing an advantage over existing therapies that require daily oral dosing [5] - The drug's novel RNAi mechanism allows for rapid knockdown of transthyretin (TTR), addressing the disease at its source [6] Market Impact - The approval in the EU follows similar approvals in the United States and Brazil, expanding Amvuttra's global footprint [6] - Alnylam reported better-than-expected first-quarter 2025 results, with revenue growth driven by increasing Amvuttra sales due to heightened patient demand [10][11]

Summary of Alnylam Pharmaceuticals (ALNY) FY Conference Call - June 09, 2025 Company Overview - **Company**: Alnylam Pharmaceuticals (ALNY) - **Industry**: Biotechnology - **Key Product**: Ambutra for TTR cardiomyopathy Core Points and Arguments 1. **European Approval**: Alnylam secured European approval for Ambutra for cardiomyopathy, enhancing its global momentum in the TTR franchise [3][4] 2. **Five-Year Strategy**: The company is on track to achieve its 2021 five-year goals, including sustainable non-GAAP profitability by the end of 2025 [5][6] 3. **Revenue Growth**: Alnylam's revenues are primarily driven by the TTR franchise, with a robust pipeline for future growth [6][7] 4. **Pipeline Programs**: - Zarbisiran for hypertension and a program for Huntington's disease are highlighted as significant growth levers [10][11] - ARN 6400 targeting plasminogen is noted for its potential as a vessel hemolytic agent [12] 5. **Market Dynamics**: The company is experiencing rapid progress in treating patients with TTR cardiomyopathy, with over 50% of priority health systems having Ambutra on formulary [16][17] 6. **Financial Guidance**: Alnylam provided guidance for TTR revenue between $1.6 billion and $1.725 billion, representing approximately 36% year-over-year growth at the midpoint [22] 7. **Polyneuropathy Growth**: The polyneuropathy segment is growing at a rate of 28-34% year-over-year, with expectations for continued growth alongside cardiomyopathy [23][24] 8. **Patient Access**: The company reports broad access across payer types, with the majority of patients paying $0 out of pocket [33][35] 9. **Patient Services**: Alnylam has a comprehensive patient services organization to support access and adherence, with over 95% adherence rates in polyneuropathy [31][38] 10. **Pricing Strategy**: The company anticipates modest adjustments to Ambutra pricing over time, but specifics are not yet available [42] 11. **Global Launch Plans**: Alnylam plans to launch Ambutra in Germany and Japan in the second half of 2025, with additional launches expected throughout 2026 [45] 12. **Clinical Data**: The HELIOS B trial data shows a 36% reduction in all-cause mortality and a 33% reduction in cardiovascular mortality, reinforcing the drug's value proposition [47][48] 13. **Pipeline Expansion**: Alnylam is committed to advancing its pipeline, with multiple programs in various stages of development, including obesity and diabetes treatments [62][64] Additional Important Content - **Regulatory Strategy**: The orphan drug designation for Alnylam's products has positioned the company favorably in negotiations related to pricing and access [14] - **Commercial Strategy**: The company emphasizes a "buy and build" approach for its products, focusing on establishing a strong commercial footprint [20] - **Innovation Engine**: Alnylam aims to deliver 2-4 new INDs per year, expanding its siRNA technology across various tissues [59][60] - **Capital Allocation**: The company plans to focus on progressing its existing pipeline rather than pursuing significant business development opportunities at this time [64]

Core Insights - The recent announcement of former US President Biden's prostate cancer diagnosis has brought attention to prostate cancer, the second most common malignancy among men globally [2] - The incidence and mortality rates of prostate cancer in China are rising, with 134,200 new cases and 47,500 deaths reported in 2022 [2] - The treatment landscape for prostate cancer is evolving, with numerous innovative pharmaceutical companies developing new therapies, enhancing treatment options for patients [2][4] Market Dynamics - The global prostate cancer treatment market was valued at $35.3 billion in 2022 and is projected to grow to $56.4 billion by 2028, with a compound annual growth rate (CAGR) of 8.3% [4] - Major pharmaceutical companies are competing in this lucrative market, with Pfizer and Astellas' enzalutamide generating $5.926 billion in global sales in 2023, ranking sixth among oncology drugs [3][4] - Chinese pharmaceutical companies are transitioning from generic to innovative drug development, with Heng Rui Medicine's new drug, Rivelutamide, set to launch in December 2024 [4] Treatment Advances - Treatment options for prostate cancer have expanded significantly, with survival rates improving from 2-3 years to over 5 years due to advancements in therapies such as new anti-androgens and PARP inhibitors [3] - The introduction of targeted therapies, such as Novartis' Pluvicto, which achieved $271 million in its first year, indicates a shift towards precision medicine in prostate cancer treatment [3][4] Screening Challenges - Early detection of prostate cancer remains a challenge, with many patients diagnosed at advanced stages due to the disease's asymptomatic nature in early stages [5][6] - The five-year survival rate for prostate cancer patients in China is approximately 66.4%, significantly lower than over 95% in developed countries, highlighting the need for improved screening practices [5] - PSA testing is the primary screening method, and initiatives are underway to increase screening coverage in China, aiming for 40% coverage in the next five years [7]