CAMBRIDGE, Mass. - Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), a pioneer in RNA interference (RNAi) therapeutics, has submitted a regulatory application to the European Medicines Agency (EMA) for vutrisiran, a drug aimed at treating ATTR amyloidosis with cardiomyopathy (ATTR-CM). The submission is based on the positive outcome of the HELIOS-B Phase 3 study, which showed a significant reduction in the risk of death and cardiovascular events compared to a placebo.
ATTR-CM is a progressive, debilitating disease that can lead to heart failure. Vutrisiran, which is already approved in the EU for treating hereditary transthyretin-mediated (hATTR) amyloidosis in adults with polyneuropathy, works by silencing the TTR gene that causes the disease. The HELIOS-B study's results, which met all primary and secondary endpoints with statistical significance, were published in The New England Journal of Medicine and demonstrated vutrisiran's potential benefits for patients with ATTR-CM.
The drug's safety profile in HELIOS-B was consistent with previous findings for hATTR amyloidosis treatment. Adverse events reported were comparable between the vutrisiran and placebo groups. In addition to the EMA application, Alnylam has submitted a supplemental New Drug Application to the U.S. Food and Drug Administration (FDA) for vutrisiran to treat ATTR-CM, with plans for further global regulatory submissions.
Vutrisiran is designed to address the underlying cause of transthyretin (ATTR) amyloidosis, a fatal disease characterized by misfolded TTR proteins that accumulate as amyloid deposits in the body, affecting the nerves, heart, and gastrointestinal tract. ATTR can manifest as polyneuropathy, cardiomyopathy, or both, and occurs in hereditary (hATTR) and wild-type (wtATTR) forms.
This announcement is based on a press release statement from Alnylam Pharmaceuticals, Inc. The company continues to focus on bringing innovative RNAi therapeutics to patients with significant unmet medical needs.
In other recent news, Alnylam Pharmaceuticals has been making significant strides, particularly with its Q2 earnings report exceeding expectations in both revenue and profit. This success has led to an updated 2024 revenue guidance of between $1.575 billion and $1.65 billion, attributed to the growth of its TTR franchise and a milestone payment from a licensing agreement with Regeneron (NASDAQ:REGN). The company has revised its patient prevalence estimate for ATTR-CM to over 300,000, indicating a larger potential market for its drug, Amvuttra. Alnylam has also submitted a supplemental New Drug Application (sNDA) for Amvuttra and vutrisiran, both treatments for ATTR-CM, to the FDA. Analyst firms including Canaccord Genuity, TD Cowen, BMO Capital, Piper Sandler, and Oppenheimer have maintained positive ratings on Alnylam, reflecting these recent developments. The company's commercial strategy for the launch of Amvuttra in treating ATTR-CM received positive feedback from BofA Securities. Alnylam is also preparing for the potential market entry of vutrisiran by expanding its field team to cover approximately 3,700 healthcare providers, representing nearly 95% of the patient population affected by ATTR-CM. Lastly, in collaboration with BridgeBio Pharma (NASDAQ:BBIO), Alnylam presented a new post hoc analysis of recurrent all-cause mortality and cardiovascular-related hospitalization events.
InvestingPro Insights
Alnylam Pharmaceuticals' (NASDAQ:ALNY) recent regulatory submission for vutrisiran in Europe aligns with the company's strong financial performance and market position. According to InvestingPro data, Alnylam has demonstrated impressive revenue growth, with a 107% increase in quarterly revenue as of Q2 2024. This growth trajectory supports the company's aggressive pursuit of new drug approvals and market expansions.
The company's focus on innovative RNAi therapeutics is reflected in its robust gross profit margin of 87%, as reported by InvestingPro. This high margin suggests that Alnylam's products, including vutrisiran, have the potential to be highly profitable if approved for additional indications like ATTR-CM.
InvestingPro Tips highlight that 10 analysts have revised their earnings upwards for the upcoming period, indicating positive expectations for Alnylam's financial performance. This optimism may be partly due to the potential approval and commercialization of vutrisiran for ATTR-CM in Europe.
Despite these positive indicators, it's worth noting that Alnylam is currently trading at a high EBIT valuation multiple, according to another InvestingPro Tip. This suggests that investors are pricing in significant future growth, potentially based on the success of drugs like vutrisiran.
For investors seeking a more comprehensive analysis, InvestingPro offers 13 additional tips for Alnylam Pharmaceuticals, providing a deeper understanding of the company's financial health and market position.
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