Key Takeaways Amvuttra drives Alnylam's top line through expanded label and as patients switch from Onpattro.Givlaari, Oxlumo, and royalties from Leqvio add incremental revenues and global growth potential.Rare disease drugs delivered $236.8M in H1 2025 revenues, up 16% year over year.Alnylam Pharmaceuticals’ (ALNY) primary top-line driver is its newest drug, Amvuttra (vutrisiran), which is approved in the United States and the EU for treating the polyneuropathy of hereditary transthyretin-mediated (hATTR) ...

Core Viewpoint - Novartis has received FDA approval for an updated label for Leqvio® (inclisiran), allowing its use as a monotherapy to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, alongside diet and exercise [1][2][3] Group 1: Product Information - Leqvio is now indicated for use as a standalone treatment, removing the previous requirement for combination with statin therapy [3] - The updated label specifies "hypercholesterolemia" instead of "primary hyperlipidemia," focusing on LDL-C reduction [3] - Leqvio is administered twice a year by healthcare providers, which may enhance patient adherence and long-term management of LDL-C levels [2][8] Group 2: Market Need and Impact - Up to 80% of patients with atherosclerotic cardiovascular disease (ASCVD) in the US do not achieve the recommended LDL-C target of less than 70 mg/dL, highlighting a significant unmet medical need [2][8] - The 2025 ACC/AHA guidelines recommend more aggressive treatment strategies to meet LDL-C targets, further emphasizing the importance of Leqvio's new indication [2][8] Group 3: Company Background - Novartis has global rights to develop, manufacture, and commercialize Leqvio through a collaboration with Alnylam Pharmaceuticals, a leader in RNAi therapeutics [4] - The company aims to address critical challenges in cardiovascular disease management and improve patient outcomes through innovative treatments [10][11]

Core Viewpoint - Alnylam Pharmaceuticals reported better-than-expected financial results for Q1 2025, with adjusted losses narrower than estimates and significant revenue growth driven by strong product sales, particularly for Amvuttra [1][2][16]. Financial Performance - The company reported total revenues of $594.2 million, exceeding the Zacks Consensus Estimate of $588.2 million, and reflecting a 20% year-over-year increase [2]. - Net product revenues reached $468.5 million, up 28% year-over-year, primarily due to increased demand for Amvuttra, Givlaari, and Oxlumo [2][6]. - Net revenues from collaborators were $99.2 million, down 16% from the previous year, largely due to a prior milestone payment from Roche [3]. Product Sales - Amvuttra generated sales of $310 million, a 59% increase year-over-year, and surpassed estimates [6]. - Givlaari recorded sales of $67 million, a 15% increase year-over-year, but slightly missed estimates [7]. - Oxlumo's sales were $42.1 million, remaining flat year-over-year and missing estimates [7]. Expenses and Cash Position - Adjusted R&D expenses were approximately $241.3 million, remaining stable year-over-year [10]. - Adjusted SG&A expenses increased by 12% to $207 million, driven by marketing efforts for Amvuttra [11]. - Cash, cash equivalents, and marketable securities totaled $2.63 billion as of March 31, 2025, down from $2.69 billion at the end of 2024 [11]. Guidance and Collaborations - The company reiterated its 2025 financial guidance, expecting net product revenues between $2.05 billion and $2.25 billion, and collaboration revenues between $650 million and $750 million [12]. - Alnylam is advancing several collaborations, including with Regeneron and Roche, which are expected to enhance its product pipeline and revenue streams [13][14][17]. Market Performance - Year-to-date, Alnylam's stock has gained 8.4%, outperforming the industry, which has seen a decline of 1.8% [8].