Alnylam Advances RNAi Therapeutics Pipeline, Awaits Key Regulatory Decisions
Key Insights
Alnylam anticipates FDA approval for vutrisiran to treat ATTR amyloidosis with cardiomyopathy (search) by March 23, 2025, and aims for EU and Japan approvals later in the year.
A Phase 3 trial for nucresiran in ATTR amyloidosis with cardiomyopathy (search) is set to begin in the first half of 2025, expanding Alnylam's focus on cardiac amyloidosis.
Alnylam's pipeline progresses with zilebesiran in hypertension (search), mivelsiran in Alzheimer's, and ALN-6400 in bleeding disorders (search), with key data readouts and trial initiations expected in 2025.
Alnylam Pharmaceuticals is making strides in its RNAi therapeutics pipeline, with key regulatory decisions and clinical trial initiations anticipated throughout 2025. The company's focus remains on addressing unmet needs in rare and prevalent diseases through its innovative RNA interference (RNAi) technology.
Vutrisiran's Potential Approval for ATTR Cardiomyopathy
The FDA is expected to decide on the supplemental New Drug Application (sNDA) for vutrisiran for treating ATTR amyloidosis with cardiomyopathy (search) by March 23, 2025. Vutrisiran, already marketed as AMVUTTRA for polyneuropathy, could offer a new treatment option for patients with the cardiac manifestation of ATTR amyloidosis. Parallel regulatory filings have been submitted in major regions, including Europe and Japan, with potential approvals expected in the second half of 2025.
Expanding ATTR Pipeline with Nucresiran
Building on its commitment to addressing ATTR amyloidosis, Alnylam plans to initiate a Phase 3 study of nucresiran in patients with ATTR amyloidosis with cardiomyopathy (search) in the first half of 2025. Nucresiran has already received Orphan Drug Designation from the FDA and has shown positive interim Phase 1 data. This subcutaneous RNAi therapeutic targets transthyretin (TTR) and aims to reduce the production of the TTR protein, which is responsible for amyloid deposits in tissues.
Advancing Programs in Hypertension, Alzheimer's, and Bleeding Disorders
Alnylam is also advancing its pipeline in other therapeutic areas. The company expects to report results from the KARDIA-3 Phase 2 study of zilebesiran for hypertension (search) in the second half of 2025 and initiate a Phase 3 cardiovascular outcomes trial in the same period. In Alzheimer's disease, Alnylam plans to report interim results from Part B of the Phase 1 study of mivelsiran and initiate a Phase 2 study in the second half of 2025. Additionally, a Phase 2 study of ALN-6400 for bleeding disorders (search) is slated to begin in the latter half of the year.
Partnered Program Milestones
Alnylam's partnered programs are also progressing. Sanofi anticipates FDA approval for fitusiran, an RNAi therapeutic for hemophilia A (search) and B, by March 28, 2025. Vir Biotechnology expects to initiate a Phase 3 study of elebsiran for chronic hepatitis delta (search) and report functional cure results from a Phase 2 study in chronic hepatitis B (search) in 2025.
Financial Performance and Guidance
Alnylam reported a 30% increase in net product revenues for the fourth quarter of 2024 compared to the same period in 2023, driven by sales of AMVUTTRA. The company anticipates combined net product revenue for ONPATTRO, AMVUTTRA, GIVLAARI, and OXLUMO to range from $2.05 billion to $2.25 billion in 2025, representing a 31% growth at the midpoint of the guidance range. Alnylam also expects to achieve non-GAAP operating income profitability in 2025.