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CAMBRIDGE, Mass. - Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), a biotechnology company with a market capitalization of $39.2 billion and impressive revenue growth of 17.2% over the last twelve months, announced today that the European Commission (EC) has granted approval for its RNAi therapeutic, AMVUTTRA (vutrisiran), to treat adult patients with wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This marks the first EC approval of an RNAi therapeutic for this condition, which affects an estimated 100,000 people across Europe.According to InvestingPro analysis, Alnylam maintains a strong gross profit margin of 86%, indicating efficient cost management in its drug development programs. The company’s stock has delivered an impressive 96.9% return over the past year.
The approval is based on results from the HELIOS-B Phase 3 study, which demonstrated up to a 36% reduction in all-cause mortality and improvements in functional capacity and quality of life with just four quarterly doses per year. The study also showed a 28% reduction in the primary composite of all-cause mortality and recurrent cardiovascular events compared to placebo.InvestingPro data reveals that analysts expect Alnylam to achieve profitability this year, with multiple additional ProTips available for subscribers looking to deep-dive into the company’s financials. Get access to the comprehensive Pro Research Report, part of InvestingPro’s coverage of 1,400+ top US stocks.
ATTR-CM is characterized by the deposition of misfolded transthyretin (TTR) proteins in the heart, leading to heart failure and reduced life expectancy. AMVUTTRA works by silencing messenger RNA to prevent the production of TTR, addressing the root cause of the disease. The treatment is administered subcutaneously once every three months, which can be done by healthcare professionals or by patients and their caregivers at home.
The HELIOS-B trial enrolled a diverse group of patients, including those already receiving standard-of-care therapies, which makes the results particularly relevant to real-world clinical practice. The safety profile of AMVUTTRA was comparable to placebo, with the most common adverse reactions being injection site reactions and increases in blood alkaline phosphatase and alanine transaminase.
This approval follows previous authorizations in the U.S. and Brazil, with Alnylam seeking to expand global access to vutrisiran. In May 2025, the European Medicines Agency’s Committee for Orphan Medicinal Products endorsed maintaining the EU Orphan Designation for AMVUTTRA in ATTR amyloidosis, reflecting its potential as a significant treatment advancement.
The company continues to focus on securing access to AMVUTTRA for eligible patients across the EU. Trading near its 52-week high of $310.22, Alnylam’s stock appears slightly overvalued according to InvestingPro’s Fair Value analysis. The information reported here is based on a press release statement and InvestingPro data.
In other recent news, Alnylam Pharmaceuticals has garnered attention with several developments. H.C. Wainwright reaffirmed a Buy rating and a $500 price target, citing promising results from the launch of Amvuttra for treating ATTR amyloidosis with cardiomyopathy. RBC Capital and JPMorgan both maintained their price targets at $330, with RBC Capital expressing optimism about Alnylam’s market performance and potential profitability within the year. JPMorgan’s analysis of recent sales data suggests a strong start for Amvuttra, potentially leading to a positive adjustment in near-term estimates. Stifel also maintained a Buy rating with a $345 target, highlighting Amvuttra’s potential as a first-line therapy for cardiomyopathy. The company is reportedly expanding its site-of-care network to enhance treatment uptake, with Amvuttra already included in formularies at major institutions. Alnylam’s pipeline continues to advance with upcoming trials for nucresiran, a next-generation TTR silencer. Analysts express confidence in Alnylam’s capacity for sustained revenue growth and market share expansion.
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