Bullish indicating open at $55-$60, IPO prices at $37
CAMBRIDGE, Mass. - Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), a leader in RNAi therapeutics with a market capitalization of $32.82 billion, announced the FDA approval of AMVUTTRA for treating the cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM) in adults. This approval, based on the HELIOS-B Phase 3 clinical trial results, signifies AMVUTTRA as the first and only FDA-approved therapy to address both the cardiomyopathy and polyneuropathy manifestations of ATTR amyloidosis. According to InvestingPro analysis, Alnylam’s stock has delivered an impressive 71.33% return over the past year, reflecting strong market confidence in its therapeutic pipeline.
ATTR-CM is a fatal condition affecting approximately 150,000 people in the U.S., characterized by the accumulation of misfolded transthyretin (TTR) proteins leading to irreversible cardiovascular damage. AMVUTTRA, an RNAi therapeutic, targets the disease at its source by reducing TTR production, which is responsible for the amyloid buildup. The company’s strong financial position is evident in its impressive 85.62% gross profit margin and healthy current ratio of 2.78, indicating robust operational efficiency and solid liquidity.
Clinical trial data demonstrated AMVUTTRA’s significant impact on reducing mortality and cardiovascular events, with a 28% reduction in all-cause mortality (ACM) and recurrent cardiovascular (CV) events during the trial period. Additionally, patients experienced improved functional capacity and quality of life. The safety profile of AMVUTTRA was confirmed in the HELIOS-A trial for hATTR-PN, noting common adverse reactions such as pain in extremity and arthralgia.
Yvonne Greenstreet, MBChB, CEO of Alnylam, expressed gratitude to all contributors to the clinical trials and emphasized the company’s commitment to further innovation for ATTR amyloidosis patients. Ronald Witteles, M.D., HELIOS-B Investigator, highlighted the trial’s reflection of the real-world patient population and the meaningful clinical benefits observed.
Alnylam offers comprehensive patient support services through Alnylam Assist®, facilitating access to the treatment with the majority of patients expected to pay zero out-of-pocket costs. The company has established a track record of working with payers, including value-based agreements that tie clinical outcomes to financial terms.
Marketing authorization applications using HELIOS-B data are currently under review by health agencies worldwide, with additional regulatory submissions planned for 2025. With revenue growth of 22.97% in the last twelve months and analysts predicting profitability this year, Alnylam appears well-positioned for future growth. For detailed financial analysis and comprehensive insights, including Fair Value estimates and growth projections, visit InvestingPro, where you’ll find exclusive research reports and expert analysis on Alnylam and 1,400+ other top stocks.
The information in this article is based on a press release statement.
In other recent news, Alnylam Pharmaceuticals announced that Dr. Phillip A. Sharp, a co-founder and Nobel laureate, will retire from the company’s Board of Directors in May 2025. Dr. Sharp will continue to contribute to Alnylam’s Scientific Advisory Board. H.C. Wainwright has raised its price target for Alnylam to $500, maintaining a Buy rating, following the company’s recent R&D Day, which reinforced confidence in Alnylam’s RNA interference (RNAi) pipeline. The company reported fourth-quarter earnings that aligned with previous estimates, projecting net product revenues between $2.05 billion and $2.25 billion for 2025.
RBC Capital Markets reiterated its Outperform rating with a $310 target, emphasizing Alnylam’s robust research and development efforts ahead of the upcoming Prescription Drug User Fee Act (PDUFA) date. Piper Sandler also maintained an Overweight rating, with a $304 target, citing the anticipated approval of AMVUTTRA for ATTR-CM, which could significantly boost sales. Alnylam is preparing for Phase 3 TRITON trials for nucresiran and the upcoming PDUFA decision for fitusiran. The company disclosed new targets for obesity and type 2 diabetes during its R&D Day, highlighting its strategic expansion into new therapeutic areas.
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