CAMBRIDGE, Mass. - Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY), a leader in RNAi therapeutics, has submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for vutrisiran, aimed at treating ATTR amyloidosis with cardiomyopathy (ATTR-CM). The company announced the news today, leveraging a Priority Review Voucher for an expedited FDA review process.
The sNDA submission is based on positive results from the HELIOS-B Phase 3 study, which demonstrated that vutrisiran reduced all-cause mortality and cardiovascular events in patients with ATTR-CM. The study also showed improvements in functional capacity and quality of life for these patients. Vutrisiran's safety profile was consistent with previous findings, with adverse event rates comparable to placebo.
ATTR amyloidosis is a fatal condition characterized by misfolded transthyretin proteins accumulating in the body, leading to nerve, heart, and gastrointestinal tract damage. Vutrisiran, already approved for the treatment of polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis, is now seeking approval for the broader indication of ATTR-CM, which includes both hereditary and wild-type forms of the disease.
Alnylam is hosting a TTR Investor Day event today in New York City, where the company's management will discuss the potential market leadership of vutrisiran in ATTR-CM treatment. The event will be available via webcast on Alnylam's website, and a replay will be offered shortly after its conclusion.
The HELIOS-B study was a global, multicenter, randomized, double-blind, placebo-controlled trial involving 655 adult patients with ATTR-CM. Participants received either vutrisiran or placebo every three months for up to 36 months, followed by an open-label extension period.
Alnylam, founded in 2002, has been at the forefront of RNAi therapeutic development, a technology recognized with the Nobel Prize in Physiology or Medicine in 2006. The company continues to work on a deep pipeline of investigational medicines, including multiple late-stage development candidates.
The information in this article is based on a press release statement from Alnylam Pharmaceuticals.
In other recent news, Alnylam Pharmaceuticals has made significant progress following positive Phase 3 HELIOS-B trial data for its ATTR-CM treatment. The company's Q2 earnings report exceeded expectations in both revenue and profit, primarily due to the growth of its TTR franchise and a milestone payment from a licensing agreement with Regeneron (NASDAQ:REGN). As a result, Alnylam updated its 2024 revenue guidance, now expecting product revenues to reach between $1.575 billion and $1.65 billion.
Analysts from Goldman Sachs, TD Cowen, BofA Securities, Piper Sandler, and Canaccord Genuity have all maintained their positive ratings on Alnylam. The company is set to submit a supplemental New Drug Application in the United States in the second half of 2024, with launches projected for mid-2025, pending regulatory approvals.
Alnylam revealed new findings from their research, including improvements in cardiac structure and function as measured by echocardiography after administration of their drug vutrisiran. Additionally, they reported early positive changes in NT-proBNP and troponin I levels, as early as six months post-administration.
BridgeBio Pharma (NASDAQ:BBIO), in collaboration with Alnylam, presented a new post hoc analysis of recurrent all-cause mortality and cardiovascular-related hospitalization events. Lastly, Alnylam is planning a significant expansion of its sales force to target an estimated 5,000 cardiologists currently prescribing Vyndamax. These recent developments underscore Alnylam's commitment to addressing unmet medical needs in the treatment of ATTR-CM.
InvestingPro Insights
Alnylam Pharmaceuticals' recent sNDA submission for vutrisiran aligns with the company's strong financial performance and market position. According to InvestingPro data, Alnylam's revenue growth has been impressive, 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 gross profit margin of 87% in the last twelve months ending Q2 2024 underscores its operational efficiency, which is crucial for sustaining its research and development efforts. This aligns with an InvestingPro Tip highlighting Alnylam's impressive gross profit margins.
Despite not being profitable over the last twelve months, Alnylam's market capitalization of $34.82 billion reflects investor confidence in its long-term potential. This is further supported by another InvestingPro Tip indicating that 10 analysts have revised their earnings upwards for the upcoming period, suggesting optimism about the company's future performance.
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