CAMBRIDGE, Mass. - Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) disclosed detailed results from its HELIOS-B Phase 3 study, showing significant benefits of its investigational drug vutrisiran for patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study met all primary and secondary endpoints with statistical significance, suggesting potential as a standard of care for this fatal condition.
Vutrisiran, an RNAi therapeutic, demonstrated a 28% reduction in all-cause mortality and recurrent cardiovascular events in the overall population, with a 31% reduction during the 33-36-month double-blind period and 36% up to month 42. In the monotherapy population, the drug reduced the primary composite endpoint by 33% and all-cause mortality by 35% up to month 42.
The study enrolled patients predominantly with New York Heart Association (NYHA) Class I or II with wild-type disease, reflecting the contemporary ATTR-CM patient population. The treatment also showed benefits across multiple clinical measures of disease progression, including the 6-Minute Walk Test, Kansas City Cardiovascular Questionnaire, and NYHA Class.
Subgroup analyses indicated consistent benefits across all key patient segments, with trends toward greater efficacy in patients with earlier disease stages. The safety and tolerability profile of vutrisiran was consistent with previous findings, with adverse event rates comparable to placebo.
The results, presented at the European Society of Cardiology Congress 2024 and published in The New England Journal of Medicine, are based on a press release statement from Alnylam. The company plans to file these data with global regulatory authorities, including a supplemental New Drug Application with the U.S. Food and Drug Administration using a Priority Review Voucher.
Vutrisiran is currently approved in over 15 countries for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. ATTR amyloidosis is a rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin proteins that accumulate as amyloid deposits in various body parts. The condition affects approximately 50,000 people worldwide with hereditary forms and an estimated 200,000-300,000 with wild-type forms.
Alnylam, a leader in RNAi therapeutics, continues to innovate in this field, aiming to transform the lives of patients with rare and prevalent diseases. The company's pipeline includes multiple late-stage development candidates, and it is executing its strategy to deliver transformative medicines and achieve a leading biotech profile.
In other recent news, Alnylam Pharmaceuticals has been the focus of several analyst firms following promising top-line data from the HELIOS-B study evaluating the efficacy of the drug Amvuttra. BMO Capital and RBC Capital have maintained an Outperform rating on Alnylam, while Goldman Sachs (NYSE:GS) upgraded the stock from Neutral to Buy. These ratings reflect the firms' confidence in the potential market success of Amvuttra, with BMO Capital estimating peak sales could reach around $8 billion.
Alnylam's second-quarter earnings report surpassed expectations in both revenue and profit, largely due to the growth of its TTR franchise and a milestone payment from a licensing agreement with Regeneron (NASDAQ:REGN). As a result, the company updated its 2024 revenue guidance, now expecting product revenues to reach between $1.575 billion and $1.65 billion.
Canaccord Genuity maintained a Buy rating and raised the share target for Alnylam, following the company's impressive second-quarter earnings report. These recent developments continue to paint a promising picture for Alnylam's ongoing initiatives and future growth.
InvestingPro Insights
Alnylam Pharmaceuticals (NASDAQ:ALNY) has shown a remarkable performance in the market, with InvestingPro data highlighting a significant 91.3% three-month price total return and a near 52-week high, priced at 99.81% of its peak. The company's market capitalization stands robust at $36.85 billion, reflecting investor confidence in its potential for growth and innovation in the field of RNAi therapeutics.
The financial health of Alnylam is underscored by a gross profit margin of 87.0% over the last twelve months as of Q2 2024, indicating efficient operations and strong control over costs. Despite this, analysts do not anticipate the company will be profitable this year, as reflected in a negative P/E ratio of -495.2. This is not uncommon for biotech firms that are heavily invested in research and development, and Alnylam's pipeline of late-stage development candidates could signal future profitability.
InvestingPro Tips for Alnylam Pharmaceuticals reveal that analysts have adjusted their earnings expectations upwards for the upcoming period, a testament to the company's promising prospects following the successful results of the HELIOS-B Phase 3 study. Additionally, the company's liquid assets exceed its short-term obligations, providing financial stability and the ability to navigate through the costly phases of drug development and regulatory approval processes.
To explore more about Alnylam Pharmaceuticals' financials and market performance, InvestingPro offers additional insights and tips, with 14 more tips available for investors seeking detailed analysis (https://www.investing.com/pro/ALNY).
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.