Alnylam Pharmaceuticals is advancing its pipeline with multiple late-stage clinical programs and targeting profitability by 2028, driven by commercial execution and cost optimization. The company aims to expand its RNA interference platform across rare diseases and metabolic conditions.
- Three Phase 3 clinical programs in hATTR, PH1, and AHP expected to deliver pivotal data by mid-2027
- Net revenue of $1.4 billion in 2025, with Onpattro contributing $680 million
- Target to achieve positive adjusted EBITDA by 2028 through 14% operating expense reduction in 2025
- Pipeline expanded to 12 active programs from nine in 2024
- Share price increased 6.2% post-announcement
- Institutional investors hold 42% of outstanding shares
Alnylam Pharmaceuticals has reaffirmed its long-term development roadmap, highlighting three late-stage clinical programs in Phase 3 trials targeting hereditary transthyretin amyloidosis (hATTR), primary hyperoxaluria type 1 (PH1), and acute hepatic porphyria (AHP). These programs are expected to generate pivotal data by mid-2027, with potential regulatory submissions in the United States and Europe. The company’s pipeline now includes 12 active programs, up from nine in 2024, reflecting a strategic expansion in therapeutic areas beyond rare genetic disorders. The company’s CEO emphasized a transition from growth-focused investment to sustainable profitability, with a target to achieve positive adjusted EBITDA by 2028. This shift follows a 14% reduction in operating expenses in 2025, driven by optimized manufacturing processes and streamlined clinical trial operations. Alnylam’s net revenue for 2025 reached $1.4 billion, a 9% increase from the prior year, primarily supported by continued strong sales of Onpattro (patisiran), which generated $680 million in revenue last year. Market analysts note that Alnylam’s focus on high-barrier, first-in-class indications strengthens its competitive position. The company’s share price rose 6.2% following the announcement, reflecting investor confidence in its financial discipline and development milestones. Key stakeholders include institutional investors such as BlackRock and Vanguard, which collectively hold over 42% of outstanding shares. The strategic pivot underscores Alnylam’s ambition to become a self-sustaining biopharmaceutical enterprise, balancing innovation with operational efficiency. The company continues to explore partnerships for global commercialization, particularly in Asia-Pacific markets, where regulatory pathways for RNAi therapeutics are rapidly maturing.