CAMBRIDGE, Mass. - Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) today announced successful outcomes from its HELIOS-B Phase 3 trial for vutrisiran, an investigational therapeutic for ATTR amyloidosis with cardiomyopathy. The study met its primary endpoint, showing a significant reduction in mortality and recurrent cardiovascular events in patients.
The trial, which involved 654 participants, indicated a 28% reduction in the composite of all-cause mortality and recurrent cardiovascular incidents in the overall population, and a 33% reduction in the monotherapy group. Additionally, a 36% reduction in all-cause mortality was observed in the overall population, with a similar 35% decrease in the monotherapy group, up to Month 42.
Secondary endpoints also showed promising results, with improvements in the 6-minute walk test, Kansas City Cardiomyopathy Questionnaire, and New York Heart Association Class—key measures of disease progression. These findings suggest potential benefits to patients' survival, function, and quality of life.
Chief Medical Officer of Alnylam, Pushkal Garg, M.D., expressed optimism about the data, indicating the company's intention to file for regulatory approval to make vutrisiran available to patients globally. The safety profile of vutrisiran was consistent with previous studies, exhibiting no significant increase in adverse events compared to the placebo.
The company plans to submit detailed results from the HELIOS-B study to the European Society of Cardiology and to file a supplemental New Drug Application with the U.S. Food and Drug Administration using a Priority Review Voucher later this year.
ATTR amyloidosis is a fatal condition caused by misfolded transthyretin proteins accumulating in the body. Vutrisiran, designed to target the underlying cause of the disease, is already approved in over 15 countries for hereditary transthyretin-mediated amyloidosis polyneuropathy in adults.
These findings are based on a press release statement.
InvestingPro Insights
As Alnylam Pharmaceuticals (NASDAQ:ALNY) makes strides in the development of treatments for ATTR amyloidosis, the company's financial health and market performance provide additional context for investors. According to InvestingPro data, Alnylam holds a market capitalization of 20.96 billion USD, reflecting its substantial presence in the biopharmaceutical industry. Despite not being profitable in the last twelve months, the company has shown a robust revenue growth of 75.2% during the same period, highlighting the potential for future profitability.
Investors might also take note of the company's solid gross profit margin of 83.95%, which suggests that Alnylam is effective in controlling its cost of goods sold relative to its sales. This is an important metric for assessing the company's operational efficiency. Moreover, the company's liquid assets exceed short-term obligations, as per InvestingPro Tips, which indicates a strong liquidity position that can support ongoing research and development efforts.
While analysts do not anticipate Alnylam to be profitable this year, the company's stock has shown a strong return over the last five years, which could be appealing to long-term investors. Additionally, Alnylam operates with a moderate level of debt, which may provide some comfort regarding the company's financial leverage and ability to manage its financial obligations.
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