Alnylam Pharmaceuticals develops and commercializes RNA interference (RNAi) therapeutics, with news centered on its approved medicines, TTR franchise, clinical pipeline and financial performance. Company updates commonly address AMVUTTRA/vutrisiran and ONPATTRO/patisiran for transthyretin-mediated amyloidosis, GIVLAARI/givosiran for acute hepatic porphyria, OXLUMO/lumasiran for primary hyperoxaluria type 1, and partner-commercialized RNAi medicines.
Recurring developments include quarterly product revenue reports, guidance, clinical and real-world data for ATTR-CM and other cardiovascular programs, pipeline progress across cardio-metabolic disease, neuroscience and hematology, disease-awareness collaborations, and investor conference presentations. Alnylam also reports on its Alnylam 2030 strategy and commercialization of RNAi medicines in global markets.
Alnylam Pharmaceuticals (ALNY), a leader in RNAi therapeutics, has announced its upcoming presentation at the BofA Securities 2025 Health Care Conference. The company's management will deliver a corporate overview on Wednesday, May 14, 2025, at 11:20 AM PT (2:20 PM ET). Investors and interested parties can access a live audio webcast of the presentation through the Investors section of Alnylam's website at www.alnylam.com/events. A replay of the presentation will be made available on the company's website within 48 hours after the event.
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Alnylam Pharmaceuticals (ALNY) has received a positive CHMP opinion recommending approval of vutrisiran for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM) in the EU. This recommendation is based on the successful HELIOS-B Phase 3 study, which met all 10 pre-specified endpoints.
Vutrisiran, marketed as AMVUTTRA®, demonstrated significant reductions in mortality and cardiovascular events while maintaining functional status and quality of life. The treatment offers quarterly subcutaneous dosing and has already received approvals in the U.S. and Brazil in March 2025.
The study showed consistent benefits across all patient subgroups, including those on TTR stabilizers. Safety profile was comparable to placebo, with main reactions including injection site reactions and increases in blood alkaline phosphatase. European Commission's final decision is expected in June 2025.
Alnylam Pharmaceuticals (Nasdaq: ALNY), a leader in RNAi therapeutics, has scheduled its first quarter 2025 financial results announcement for Thursday, May 1, 2025, before U.S. markets open. The company will host a conference call at 8:30 am ET the same day, where management will discuss Q1 2025 performance and future outlook. A live audio webcast will be accessible through the Investors section of Alnylam's website, with an archived version available approximately two hours after the event.
Alnylam Pharmaceuticals (Nasdaq: ALNY), a leader in RNAi therapeutics, has announced its upcoming participation in the Needham 24th Annual Virtual Healthcare Conference. The company's management will deliver a corporate overview presentation on Tuesday, April 8, 2025, at 11:45 am ET.
Interested parties can access a live audio webcast of the presentation through the Investors section of Alnylam's website at www.alnylam.com/events. A replay of the presentation will be made available on the company's website within 48 hours following the event.
Alnylam Pharmaceuticals (ALNY) presented new data from the HELIOS-B Phase 3 study of vutrisiran (AMVUTTRA®) for ATTR amyloidosis with cardiomyopathy at ACC.25. The data follows the recent FDA approval of AMVUTTRA as the first RNAi therapeutic to reduce cardiovascular death, hospitalizations, and urgent heart failure visits in ATTR-CM patients.
Key findings include:
- Improved echocardiographic systolic and diastolic cardiac function compared to placebo
- Maintained or improved functional capacity, health status, and quality of life
- Greatest benefits observed in patients with early-stage disease
- Significant improvements in diastolic function and attenuation of left and right ventricular systolic function declines at Month 18
The study demonstrated vutrisiran's efficacy across various baseline heart failure severities, with results published in JACC. The treatment showed beneficial effects on cardiac structure and function, emphasizing the importance of early intervention in this progressive disease.
Alnylam Pharmaceuticals (Nasdaq: ALNY) announces FDA approval of Qfitlia™ (fitusiran), marking a significant breakthrough as the first siRNA therapeutic for hemophilia A or B treatment. This represents Alnylam's sixth approved RNAi therapeutic in the US.
Qfitlia works by lowering antithrombin (AT), a protein that inhibits blood clotting, and is approved for routine prophylaxis in patients 12 years and older with hemophilia A or B, with or without inhibitors. Clinical studies demonstrated a 90% reduction in annualized bleeding rates through subcutaneous injections administered every two months.
Under a collaboration agreement with Sanofi, who holds global development and commercialization rights, Alnylam is eligible to receive tiered royalties of 15-30% on global net sales. The treatment has potential to benefit an estimated one million people worldwide living with hemophilia A and B. Regulatory submissions have also been completed in China and Brazil.
Alnylam Pharmaceuticals (ALNY) has received FDA approval for AMVUTTRA® (vutrisiran) to treat cardiomyopathy in adults with ATTR amyloidosis (ATTR-CM). The approval expands AMVUTTRA's indication, making it the first therapeutic approved for both ATTR-CM and polyneuropathy of hereditary ATTR amyloidosis.
The approval is based on the HELIOS-B Phase 3 trial results, where AMVUTTRA demonstrated:
- 28% reduction in all-cause mortality and cardiovascular events
- 36% mortality reduction through 42 months
- 33% risk reduction in mortality and cardiovascular events in monotherapy patients
ATTR-CM affects approximately 150,000 people in the U.S. and 300,000 worldwide. AMVUTTRA works by reducing TTR production through RNAi therapy, requiring only four subcutaneous doses annually. The treatment is expected to have broad insurance coverage with most patients paying $0 out-of-pocket costs.
Alnylam Pharmaceuticals (Nasdaq: ALNY), a leader in RNAi therapeutics, has announced its upcoming participation in the Stifel 2025 Virtual CNS Forum. The company's management will deliver a corporate overview presentation on Tuesday, March 18, 2025, at 3:00 pm ET.
Interested parties can access a live audio webcast of the presentation through the Investors section of Alnylam's website at www.alnylam.com/events. A replay of the presentation will be made available on the company's website within 48 hours following the event.