Alnylam Pharmaceuticals NASDAQ: ALNY stock price action has been volatile but is uptrending and has gained 100% in the last two years. The uptrend is driven by increasing expectations for market-disrupting heart treatment and could double the price again. The latest action has the market up more than 65% in the last two months and heading to the $350 to $415 range based on the technicals. Alnylam Pharmaceuticals TodayALNYAlnylam Pharmaceuticals$261.91 +5.70 (+2.22%) 52-Week Range$141.98▼$287.55Price Target$ ...

Core Viewpoint - Alnylam Pharmaceuticals' shares fell 8.5% following the announcement of detailed data from the phase III HELIOS-B study for its investigational RNAi therapeutic, Amvuttra, aimed at treating transthyretin-mediated amyloidosis with cardiomyopathy [1][2]. Group 1: Study Results - The HELIOS-B study met its primary endpoint, showing a statistically significant reduction in all-cause mortality and recurrent cardiovascular events in both the overall and monotherapy populations [1]. - Treatment with vutrisiran resulted in a 30% reduction in mortality in the monotherapy population during the double-blind period, but this reduction was deemed non-significant by management [1]. Group 2: Market Performance - Alnylam's shares have increased by 37.3% year-to-date, significantly outperforming the industry growth of 0.6% [4]. - The company reported sales of $425.4 million for Amvuttra in the first half of 2024, reflecting an 82% year-over-year increase [6]. Group 3: Regulatory and Future Prospects - Alnylam plans to submit a supplemental new drug application for vutrisiran to the FDA using a Priority Review Voucher later in 2024 [5]. - If approved for the new indication, vutrisiran is expected to become the new standard of care for treating ATTR amyloidosis with cardiomyopathy, potentially increasing sales and expanding the patient population [6].

Alnylam Pharmaceuticals (ALNY) , a commercial-stage company, focuses on developing novel therapeutics based on RNAi. The company’s pipeline of experimental RNAi therapeutics is focused across three strategic therapeutic areas — genetic medicines, cardio-metabolic disease and hepatic infectious disease. Alnylam currently markets four drugs — Amvuttra (vutrisiran), Givlaari (givosiran), Oxlumo (lumasiran) and Onpattro (patisiran).Portfolio of Marketed Drugs Boosts RevenuesAmvuttra, the company’s lead drug, is ...

Alnylam Pharmaceuticals (ALNY) reported second-quarter 2024 adjusted earnings of 56 cents per share against the Zacks Consensus Estimate of a loss of 74 cents. The improvement was primarily driven by the recognition of a payment of approximately $185 million, which was previously deferred, upon modifying Alnylam’s ongoing collaboration with Regeneron (REGN) . The company had incurred a loss of $1.62 per share in the year-ago quarter.The adjusted figure excluded items like stock-based compensation expenses a ...

Financial Data and Key Metrics Changes - In Q2 2024, Alnylam Pharmaceuticals achieved a 34% year-over-year growth in global net product revenues, totaling $410 million, primarily driven by a 37% increase in the TTR business [7][11][25] - The company raised its revenue guidance for the year by 11% at the midpoint, reflecting strong commercial performance [7][30] - Total product revenue for Q2 was $410 million, a 34% increase compared to Q2 2023, with TTR and rare franchises growing by 37% and 25%, respectively [25][26] Business Line Data and Key Metrics Changes - The TTR franchise generated $307 million in global net product revenues, marking a 16% increase from Q1 2024 and a 37% increase from Q2 2023 [11][12] - The rare disease franchise, including GIVLAARI and OXLUMO, reported $13 million in global revenues, with a 25% year-over-year growth [16] - GIVLAARI sales increased by 7% year-over-year, while OXLUMO saw a robust 68% growth [16] Market Data and Key Metrics Changes - In the U.S., combined sales of ONPATTRO and AMVUTTRA increased by 16% from Q1 and 40% year-over-year, driven by strong demand for AMVUTTRA [12] - Internationally, TTR franchise growth also increased by 16% from Q1 and 35% year-over-year, primarily due to AMVUTTRA demand [13] Company Strategy and Development Direction - Alnylam aims to become a top-tier biotech company by developing transformative medicines for rare and prevalent diseases, supported by a high-yielding pipeline [9] - The company is preparing for a potential launch of vutrisiran in ATTR cardiomyopathy, which is expected to significantly enhance its commercial portfolio [8][15] - Alnylam is focused on establishing vutrisiran as the standard of care in ATTR-CM, leveraging its strong relationships with TTR centers and integrated customer-facing teams [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving financial goals, including a compounded annual growth rate of over 40% and sustainable non-GAAP operating income [30] - The positive results from the HELIOS-B study are expected to position vutrisiran as a first-line treatment option, addressing significant unmet medical needs [19][38] Other Important Information - Alnylam plans to host a TTR Investor Day on October 9, 2024, to discuss its leadership in ATTR amyloidosis [9][31] - The company is advancing its pipeline with multiple clinical studies, including a supplemental NDA filing for vutrisiran in late 2024 [20][32] Q&A Session Summary Question: Can you discuss the reimbursement landscape in ATTR cardiomyopathy? - Management highlighted the importance of seamless patient access and minimizing co-pay burdens, leveraging the physician-administered nature of AMVUTTRA [33][35] Question: What feedback have you received from physicians regarding vutrisiran? - Physicians are enthusiastic about the potential of vutrisiran to become the standard of care, especially given the impressive HELIOS-B results [37][39] Question: Will you report KM curves and time to events kinetics at the ESC? - Management confirmed plans to provide detailed results, including baseline characteristics and primary endpoint data, at the upcoming ESC Congress [43][46] Question: Can you provide updates on the ALN-APP program? - The ALN-APP program is progressing well, with ongoing studies in both Alzheimer's disease and cerebral amyloid angiopathy [50][60] Question: How has the nature of discussions with physicians changed since Eplontersen's launch? - Discussions have evolved positively, with increased clarity on product differentiation and the potential for market growth [62][64] Question: What is the strategy for growing the market beyond taking share from tafamidis? - Management emphasized the importance of demonstrating the efficacy of vutrisiran and its unique mechanism of action to access a broader patient population [78][80]

For the quarter ended June 2024, Alnylam Pharmaceuticals (ALNY) reported revenue of $659.83 million, up 107% over the same period last year. EPS came in at $0.56, compared to -$2.21 in the year-ago quarter.The reported revenue compares to the Zacks Consensus Estimate of $448.44 million, representing a surprise of +47.14%. The company delivered an EPS surprise of +175.68%, with the consensus EPS estimate being -$0.74.While investors closely watch year-over-year changes in headline numbers -- revenue and earn ...

Alnylam Pharmaceuticals (ALNY) came out with quarterly earnings of $0.56 per share, beating the Zacks Consensus Estimate of a loss of $0.74 per share. This compares to loss of $2.21 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 175.68%. A quarter ago, it was expected that this RNA interference drug developer would post a loss of $0.75 per share when it actually produced a loss of $0.16, delivering a surprise of 78.67%.Over t ...

PART I. FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=6&type=section&id=ITEM%201.%20FINANCIAL%20STATEMENTS%20%28Unaudited%29) Alnylam Pharmaceuticals' unaudited financial statements for Q2 2024 show increased assets and revenues, a narrowed net loss, and positive operating cash flow [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) The balance sheet as of June 30, 2024, reflects an increase in total assets and a significant improvement in stockholders' deficit Balance Sheet Items (In thousands) | Balance Sheet Items (In thousands) | June 30, 2024 | December 31, 2023 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $968,492 | $812,688 | | Total current assets | $3,172,856 | $2,982,697 | | **Total assets** | **$4,009,634** | **$3,829,880** | | **Liabilities & Stockholders' Deficit** | | | | Total current liabilities | $1,055,251 | $967,786 | | **Total liabilities** | **$4,012,701** | **$4,050,524** | | **Total stockholders' deficit** | **$(3,067)** | **$(220,644)** | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) The statements of operations show a substantial increase in revenues and a significant reduction in net loss for both the quarter and six-month periods ended June 30, 2024 Statement of Operations (In thousands) | Statement of Operations (In thousands) | Three Months Ended June 30, 2024 | Three Months Ended June 30, 2023 | Six Months Ended June 30, 2024 | Six Months Ended June 30, 2023 | | :--- | :--- | :--- | :--- | :--- | | **Total revenues** | **$659,825** | **$318,754** | **$1,154,158** | **$638,044** | | Net product revenues | $410,088 | $305,705 | $775,251 | $582,033 | | Net revenues from collaborations | $227,338 | $5,844 | $345,886 | $42,306 | | Income (loss) from operations | $48,614 | $(229,831) | $5,179 | $(379,638) | | **Net loss** | **$(16,889)** | **$(276,024)** | **$(82,824)** | **$(450,125)** | | Net loss per common share | $(0.13) | $(2.21) | $(0.66) | $(3.62) | [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Cash flow statements for the first half of 2024 indicate a positive shift in operating cash flow and an overall net increase in cash and equivalents Cash Flow Activities (In thousands) | Cash Flow Activities (In thousands) | Six Months Ended June 30, 2024 | Six Months Ended June 30, 2023 | | :--- | :--- | :--- | | Net cash provided by (used in) operating activities | $42,643 | $(225,461) | | Net cash used in investing activities | $(33,680) | $(84,930) | | Net cash provided by financing activities | $160,407 | $99,765 | | **Net increase (decrease) in cash** | **$156,309** | **$(208,580)** | | Cash, cash equivalents and restricted cash, end of period | $971,193 | $659,976 | [Notes to Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes provide detailed insights into revenue drivers, significant liabilities, and ongoing patent infringement litigation - The company's strategy, **Alnylam Px25**, aims to transition it to a **top-tier biotech company** by the end of 2025 by delivering transformative medicines and achieving **strong financial performance**[29](index=29&type=chunk) Net Product Revenues (Six Months Ended June 30, In thousands) | Net Product Revenues (Six Months Ended June 30, In thousands) | 2024 | 2023 | | :--- | :--- | :--- | | ONPATTRO | $146,461 | $193,951 | | AMVUTTRA | $425,350 | $233,904 | | GIVLAARI | $120,183 | $105,805 | | OXLUMO | $83,257 | $48,373 | | **Total** | **$775,251** | **$582,033** | Net Revenues from Collaborations (Six Months Ended June 30, In thousands) | Net Revenues from Collaborations (Six Months Ended June 30, In thousands) | 2024 | 2023 | | :--- | :--- | :--- | | Roche | $91,186 | $— | | Regeneron Pharmaceuticals | $234,193 | $17,153 | | Novartis AG | $16,820 | $23,560 | | **Total** | **$345,886** | **$42,306** | - In June 2024, the company amended its agreement with Regeneron, granting a worldwide license for cemdisiran as a monotherapy, which resulted in the immediate recognition of **$191.5 million** in revenue[56](index=56&type=chunk)[61](index=61&type=chunk) - The company has filed **patent infringement lawsuits** against Pfizer/BioNTech and Moderna related to their mRNA COVID-19 vaccines, seeking damages, and these cases are ongoing[105](index=105&type=chunk)[106](index=106&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=28&type=section&id=ITEM%202.%20MANAGEMENT%27S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) Management discusses the significant revenue growth, controlled expense increases, narrowed net loss, and strong liquidity position for the first half of 2024 [Overview](index=28&type=section&id=Overview) This section provides an overview of Alnylam's focus on RNAi therapeutics, its strategic goals, commercialized products, and recent positive clinical trial results - The company's **Alnylam Px25 strategy**, launched in 2021, focuses on transitioning to a **top-tier biotech company** by the end of 2025 through sustainable innovation and strong financial performance[110](index=110&type=chunk) - **Positive topline results** from the **HELIOS-B trial** of **vutrisiran** in patients with **ATTR amyloidosis with cardiomyopathy** were reported in June 2024, with plans for global regulatory filings later in the year[110](index=110&type=chunk) [Our Product Pipeline](index=30&type=section&id=Our%20Product%20Pipeline) The company maintains a robust product pipeline spanning commercial products and numerous clinical programs, with recent positive data readouts and regulatory advancements - The pipeline features **five commercial products** and a **broad range of clinical programs** across rare, specialty, and prevalent diseases[115](index=115&type=chunk) - Recent **positive data readouts** include the **HELIOS-B Phase 3 study** for vutrisiran and the **KARDIA-2 Phase 2 study** for zilebesiran[122](index=122&type=chunk) - Collaboration partner **Sanofi** has submitted **regulatory filings** for **fitusiran** in China, Brazil, and the U.S., with a U.S. FDA target action date of March 28, 2025[122](index=122&type=chunk) [Results of Operations](index=34&type=section&id=Results%20of%20Operations) Operating results for the first half of 2024 show substantial revenue growth, particularly from collaborations, alongside increased operating expenses and a significantly reduced net loss Financial Performance (Six Months Ended June 30, in thousands) | Financial Performance (Six Months Ended June 30) | 2024 (in thousands) | 2023 (in thousands) | % Change | | :--- | :--- | :--- | :--- | | **Total revenues** | **$1,154,158** | **$638,044** | **81%** | | Net product revenues | $775,251 | $582,033 | 33% | | Net revenues from collaborations | $345,886 | $42,306 | >500% | | **Total operating costs and expenses** | **$1,148,979** | **$1,017,682** | **13%** | | Research and development | $555,137 | $479,095 | 16% | | Selling, general and administrative | $459,194 | $398,348 | 15% | | **Net loss** | **$(82,824)** | **$(450,125)** | **(82)%** | - The increase in collaboration revenue was primarily driven by the modification of the Regeneron agreement, which resulted in the immediate recognition of **$191.5 million** for the cemdisiran monotherapy license[134](index=134&type=chunk) - **R&D expenses increased** due to costs for preclinical activities, clinical trials for zilebesiran (KARDIA-3) and mivelsiran (cAPPRicorn-1), and expenses related to the HELIOS-B trial data readout[141](index=141&type=chunk) [Liquidity and Capital Resources](index=38&type=section&id=Liquidity%20and%20Capital%20Resources) The company's liquidity position is strong, with sufficient cash to fund operations and achieve self-sustainability without future equity financing, supported by positive operating cash flow - The company believes its current cash position will be sufficient to achieve a **self-sustainable profile** without the need for **future equity financing**[151](index=151&type=chunk) Cash Flow Summary (Six Months Ended June 30, In thousands) | Cash Flow Summary (Six Months Ended June 30, In thousands) | 2024 | 2023 | | :--- | :--- | :--- | | Net cash provided by (used in) operating activities | $42,643 | $(225,461) | | Net cash used in investing activities | $(33,680) | $(84,930) | | Net cash provided by financing activities | $160,407 | $99,765 | [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=39&type=section&id=ITEM%203.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) There have been no significant changes to the company's financial market risk exposures, primarily related to interest rates, since the prior fiscal year-end - There have been **no significant changes** to the company's financial market risk exposures, which are mainly related to **interest rates**, since December 31, 2023[152](index=152&type=chunk) [Item 4. Controls and Procedures](index=39&type=section&id=ITEM%204.%20CONTROLS%20AND%20PROCEDURES) Management concluded that disclosure controls and procedures were effective as of June 30, 2024, with no material changes to internal control over financial reporting - As of June 30, 2024, the CEO and CFO concluded that the company's disclosure controls and procedures were **effective**[153](index=153&type=chunk) - **No material changes** were made to the internal control over financial reporting during the quarter ended June 30, 2024[154](index=154&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=41&type=section&id=ITEM%201.%20LEGAL%20PROCEEDINGS) This section refers to Note 13 for details on material pending legal proceedings, primarily patent infringement lawsuits against Pfizer and Moderna - For details on legal proceedings, the report refers to Note 13, which discusses ongoing **patent infringement lawsuits** against Pfizer and Moderna[156](index=156&type=chunk) [Item 1A. Risk Factors](index=41&type=section&id=ITEM%201A.%20RISK%20FACTORS) The company faces diverse risks including financial performance, operational dependencies, clinical and regulatory challenges, intellectual property disputes, and market competition - Key business risks include the unsuccessful marketing of approved products, a history of financial losses (**$7.09 billion accumulated deficit** as of June 30, 2024), and the need for substantial future funding[158](index=158&type=chunk)[169](index=169&type=chunk) - The company is **highly dependent on third parties** for collaborations (e.g., Roche, Regeneron, Novartis), manufacturing, and conducting clinical trials, and failure by these parties could delay or terminate programs[158](index=158&type=chunk)[178](index=178&type=chunk) - Significant industry risks include potential **clinical trial failures**, inability to obtain **regulatory approval** (as exemplified by the FDA's CRL for patisiran for cardiomyopathy), ongoing regulatory oversight, and **unfavorable pricing regulations** or healthcare reforms like the Inflation Reduction Act[159](index=159&type=chunk)[190](index=190&type=chunk)[199](index=199&type=chunk) - **Intellectual property** is a major risk area, with potential for **patent challenges**, **litigation costs** (including ongoing lawsuits against Pfizer and Moderna), and the need to license third-party patents[163](index=163&type=chunk)[221](index=221&type=chunk) - The company faces **intense competition** from existing drugs and new technologies, including other RNAi and antisense therapies, which could render its products obsolete or noncompetitive[164](index=164&type=chunk)[226](index=226&type=chunk) [Item 5. Other Information](index=76&type=section&id=ITEM%205.%20OTHER%20INFORMATION) This section discloses executive Rule 10b5-1 trading plans and corrects the deadlines for stockholder proposals for the 2025 annual meeting - Executive Vice President Kevin Fitzgerald and Director Dennis A. Ausiello entered into **Rule 10b5-1 trading plans** in May 2024 for the potential sale of company stock[239](index=239&type=chunk)[240](index=240&type=chunk)[241](index=241&type=chunk) - The company **corrected the deadlines** for **stockholder proposals** for the 2025 annual meeting: proposals for inclusion in proxy materials are due by December 2, 2024, and other business must be noticed between January 16, 2025, and February 15, 2025[242](index=242&type=chunk)[243](index=243&type=chunk) [Item 6. Exhibits](index=77&type=section&id=ITEM%206.%20EXHIBITS) This section lists the exhibits filed with the Form 10-Q, including stock award agreements and required officer certifications - Exhibits filed include forms of **Performance Stock Unit**, **Restricted Stock Unit**, and **Nonstatutory Stock Option agreements**, as well as required **CEO and CFO certifications**[245](index=245&type=chunk)

Wall Street analysts forecast that Alnylam Pharmaceuticals (ALNY) will report quarterly loss of $0.71 per share in its upcoming release, pointing to a year-over-year increase of 67.9%. It is anticipated that revenues will amount to $452.73 million, exhibiting an increase of 42% compared to the year-ago quarter.The current level reflects an upward revision of 2.4% in the consensus EPS estimate for the quarter over the past 30 days. This demonstrates how the analysts covering the stock have collectively reapp ...

Alnylam Pharmaceuticals (ALNY) is expected to deliver a year-over-year increase in earnings on higher revenues when it reports results for the quarter ended June 2024. This widely-known consensus outlook gives a good sense of the company's earnings picture, but how the actual results compare to these estimates is a powerful factor that could impact its near-term stock price. The stock might move higher if these key numbers top expectations in the upcoming earnings report, which is expected to be released on ...