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 (Nasdaq: ALNY) announced that co-founder Dr. Phillip A. Sharp will retire from the Board of Directors effective May 8, 2025. Dr. Sharp, a Nobel Prize laureate who helped pioneer the RNAi revolution, has served as a key advisor since co-founding the company in 2002.
Dr. Sharp will continue as a member of Alnylam's Scientific Advisory Board. His distinguished career includes winning the 1993 Nobel Prize in Physiology or Medicine for RNA splicing discovery, the 2004 National Medal of Science, and the 2020 AACR Award for Lifetime Achievement in Cancer Research. He serves as Institute Professor Emeritus at MIT's Koch Institute for Integrative Cancer Research and was Founding Director of the McGovern Institute for Brain Research.
Alnylam Pharmaceuticals highlighted significant progress in its R&D pipeline at its R&D Day event. Key updates include:
- Phase 3 TRITON program for nucresiran in ATTR-CM and hATTR-PN, aiming for over 95% knockdown with twice-annual dosing.
- Phase 3 trial for zilebesiran to transform hypertension treatment, enrolling up to 11,000 patients globally.
- New programs for Huntington’s disease, bleeding disorders, and type 2 diabetes.
- Platform advances for improved delivery solutions and manufacturing innovations.
Data from the HELIOS-B study showed vutrisiran reduced all-cause mortality by 36% in patients with ATTR amyloidosis with cardiomyopathy. Vutrisiran is under FDA review with a target action date of March 23, 2025.
Alnylam is committed to expanding its RNAi therapeutics, targeting all major tissue types by 2030. The company will file IND applications for nine new programs by 2025, including two in new tissues, two in the CNS, and five in the liver.
The event was webcast live and a replay will be available on Alnylam's website.
Alnylam Pharmaceuticals reported strong financial results for Q4 and full year 2024, achieving global net product revenues of $451M (Q4) and $1.65B (FY), representing 30% and 33% growth compared to 2023. The company's TTR franchise, including ONPATTRO and AMVUTTRA, generated combined revenues of $343M in Q4 and $1.22B for the full year, showing 35% and 34% growth respectively.
Key developments include FDA acceptance of vutrisiran's supplemental NDA for ATTR amyloidosis with cardiomyopathy, with a PDUFA date of March 23, 2025. The Rare disease portfolio (GIVLAARI and OXLUMO) achieved combined revenues of $108M in Q4 and $423M for the full year.
For 2025, Alnylam projects total net product revenues between $2.05B-$2.25B, representing 25-37% growth. The company expects to achieve profitability on a non-GAAP basis and forecasts collaboration and royalty revenues of $650M-$750M.
Alnylam Pharmaceuticals (Nasdaq: ALNY) has announced it will report its fourth quarter and full year 2024 financial results on Thursday, February 13, 2025, before U.S. markets open. The company's management will host a conference call at 8:30 am ET on the same day to discuss the results and provide updates on the company's operations and future outlook.
A live audio webcast will be available on the Investors section of Alnylam's website, with an archived version accessible approximately two hours after the event.
Alnylam Pharmaceuticals announced preliminary fourth quarter and full year 2024 global net product revenues, reporting strong performance with total net product revenues of $1,646 million, representing 33% annual growth. The company's TTR franchise, including ONPATTRO and AMVUTTRA, generated Q4 revenues of $56 million and $287 million respectively, while GIVLAARI and OXLUMO contributed $65 million and $44 million.
For 2025, Alnylam provided combined net product revenue guidance of $2,050-$2,250 million, projecting 31% growth at the mid-point. The company expects to achieve non-GAAP operating income profitability in 2025. Key pipeline goals include FDA approval for vutrisiran in ATTR cardiomyopathy by March 23, 2025, initiating Phase 3 studies for nucresiran and zilebesiran, and advancing multiple clinical programs including mivelsiran for Alzheimer's disease.
Alnylam Pharmaceuticals (Nasdaq: ALNY), a leader in RNAi therapeutics, has announced its participation in the 43rd Annual J.P. Morgan Healthcare Conference.
The event will take place on Monday, January 13, 2025, at 9:45 am PT (12:45 pm ET) at The Westin St. Francis in San Francisco. Management will present a company overview and engage in a Q&A session. During the presentation, Alnylam will discuss its unaudited fourth quarter and full year 2024 global net product revenues.
A live audio webcast of the presentation and Q&A session will be accessible on the Investors section of the Company’s website, www.alnylam.com/events. A replay will be made available on the website within 48 hours post-event.
Alnylam Pharmaceuticals (Nasdaq: ALNY), a leader in RNAi therapeutics, has announced its upcoming presentation at the Piper Sandler 36th Annual Healthcare Conference. The company overview will be presented on December 5, 2024, at 11:00 am ET at the Lotte New York Palace Hotel in New York City. The presentation will be accessible via live audio webcast in the Investors section of Alnylam's website, with a replay available within 48 hours after the event.
Alnylam Pharmaceuticals (ALNY) announced FDA acceptance of their supplemental New Drug Application (sNDA) for vutrisiran to treat ATTR amyloidosis with cardiomyopathy (ATTR-CM). The FDA set a PDUFA date of March 23, 2025. The application is based on positive results from the HELIOS-B Phase 3 study, which showed favorable effects on cardiovascular outcomes, survival, and quality of life in ATTR-CM patients. If approved, vutrisiran would become the first treatment approved in the U.S. for both polyneuropathy manifestations of hATTR and cardiomyopathy manifestations of ATTR amyloidosis.
Alnylam Pharmaceuticals presented new Phase 1 study results for nucresiran, a next-generation RNAi therapeutic for ATTR amyloidosis treatment. A single dose of ≥300mg achieved rapid TTR knockdown of >90% by Day 15, sustained through Day 180. Peak reductions of >96% were reached by Day 29. At 300mg, TTR reduction remained >70% at Day 360. The drug showed encouraging safety profile with low inter-patient variability. The 600mg dose achieved 95.0% TTR reduction at Day 15 and 96.0% at Day 180, while the 900mg dose showed 91.7% reduction at Day 15 and 94.2% at Day 180. The data supports potential biannual or annual dosing regimens.
Alnylam Pharmaceuticals (Nasdaq: ALNY) has announced its upcoming presentations at two major investor conferences in November 2024. The company will present at the UBS Global Healthcare Conference on November 12 at 8:45 am PT in Ranchos Palos Verdes, CA, and at the Jefferies London Healthcare Conference on November 20 at 9:30 am GMT in London.
Live audio webcasts will be accessible through the Investors section of Alnylam's website, with replays available within 48 hours after each presentation.