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CAMBRIDGE, Mass. - Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), a leader in RNA interference (RNAi) therapies with a market capitalization of $32.75 billion, will showcase new data from its transthyretin amyloidosis (TTR) programs at the Heart Failure 2025 Congress in Belgrade, Serbia, from May 17-20. According to InvestingPro data, the company has demonstrated strong momentum with a 66.91% return over the past year, supported by robust revenue growth of 17.21%. This includes results from the HELIOS-B Phase 3 study on vutrisiran, a treatment for ATTR amyloidosis with cardiomyopathy (ATTR-CM), which has recently gained approvals in the U.S. and Brazil and a positive opinion from the European Medicines Agency (EMA).
The HELIOS-B study data, which will be presented as a late-breaking abstract, details the impact of vutrisiran on cardiovascular hospitalizations and urgent heart failure visits over a period up to 42 months. Vutrisiran, marketed as AMVUTTRA®, is an RNAi therapeutic designed to reduce the production of transthyretin (TTR) protein, which is implicated in the development of ATTR amyloidosis, a condition characterized by the accumulation of amyloid proteins in organs such as the heart and nerves. InvestingPro analysis shows six analysts have recently revised their earnings upward for the upcoming period, reflecting growing confidence in the company’s therapeutic pipeline.
In addition to the HELIOS-B findings, Alnylam will discuss the design and rationale behind the TRITON-CM Phase 3 study of nucresiran (ALN-TTRsc04), an investigational next-generation TTR silencer for patients with ATTR-CM. Further insights will come from an analysis of vutrisiran in patients who showed disease progression while on tafamidis, another medication for ATTR-CM.
ATTR amyloidosis is a rare and often fatal disease, with hereditary (hATTR) and wild-type (wtATTR) forms affecting approximately 50,000 and 200,000-300,000 people worldwide, respectively. RNAi therapeutics like vutrisiran offer a novel approach to treatment by interfering with the genetic mechanisms that produce disease-causing proteins. With an impressive gross profit margin of 86% and a strong current ratio of 3.04, InvestingPro research indicates Alnylam maintains a solid financial foundation to support its therapeutic development programs. Subscribers can access 8 additional ProTips and comprehensive financial analysis through the Pro Research Report.
Alnylam, founded in 2002, has been at the forefront of RNAi therapeutic development, a field that has seen significant growth since the award of the 2006 Nobel Prize for Physiology or Medicine for the discovery of RNAi. The company continues to expand its portfolio of investigational medicines, aiming to address the unmet needs of patients with both rare and common diseases.
This report is based on a press release statement from Alnylam Pharmaceuticals, Inc.
In other recent news, Alnylam Pharmaceuticals reported its first quarter 2025 earnings, surpassing expectations with an earnings per share (EPS) of -0.01 compared to the forecast of -0.93. The company’s revenue also exceeded projections, reaching $594.19 million against an anticipated $588.75 million, marking a 28% year-over-year growth. The TTR franchise was a significant contributor, with revenues increasing by 36% to $359 million. H.C. Wainwright maintained a Buy rating on Alnylam, setting a price target of $500, driven by the robust performance of its lead product, AMVUTTRA, and positive feedback from physicians and payers.
Alnylam reaffirmed its 2025 financial guidance, projecting product revenues between $2.05 billion and $2.25 billion, aligning with analyst estimates. The company expressed confidence in achieving sustainable non-GAAP operating profitability within the year, an important milestone in its strategic plan. Alnylam is also preparing to initiate several clinical programs, including the Phase 3 TRITON-CM trial for nucresiran in ATTR amyloidosis and a cardiovascular outcomes trial for zilebesiran in hypertension. Additionally, the European Medicines Agency’s Committee for Medicinal Products for Human Use has adopted a positive opinion recommending approval of vutrisiran in ATTR cardiomyopathy, potentially enabling European Commission approval by Q3 2025.
Despite these positive developments, Alnylam’s stock fell by 3.89% in pre-market trading, possibly reflecting broader market trends or investor caution. The company continues to focus on its launch of AMVUTTRA in ATTR cardiomyopathy, with expanding formulary access expected to contribute to a strong trajectory in the second half of 2025. The company is committed to advancing its pipeline and delivering innovative RNAi therapeutics across a range of diseases.
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