Alnylam Pharmaceuticals, Inc., the global leader in RNA interference (RNAi) therapeutics, has announced the launch of its new five-year corporate strategy, “Alnylam 2030,” outlining an ambitious roadmap to scale operations, strengthen leadership in transthyretin (ATTR) amyloidosis, advance a diversified pipeline of innovative medicines, and deliver sustained financial performance. Alongside the strategic update, the company reported preliminary fourth-quarter and full-year 2025 global net product revenues for its marketed therapies AMVUTTRA®, ONPATTRO®, GIVLAARI®, and OXLUMO®.

Introducing Alnylam 2030: Building on a Proven Track Record

Alnylam 2030 represents the continuation of a long-standing tradition of setting and executing against bold five-year objectives. Over the past 15 years, the company has consistently established multi-year strategies and successfully delivered on them, most recently through its P5x25 plan. According to the company, Alnylam met or exceeded all of its previous five-year goals, creating a strong foundation for the next phase of growth.

The new strategy is designed to support the continued scaling of Alnylam’s commercial, research, and operational capabilities while reinforcing its position as the pioneer and market leader in RNAi therapeutics. By the end of 2030, the company aims to deliver leadership across its core franchises, expand into new therapeutic areas, and achieve best-in-class financial performance with discipline and agility.

Strategic Pillars of Alnylam 2030

Achieving Global Leadership in TTR Amyloidosis

At the core of Alnylam 2030 is the company’s ambition to build a durable and industry-leading TTR franchise. Alnylam plans to lead the global TTR market in revenue by 2030, both on an annual basis and cumulatively over the five-year period. Central to this effort is the continued growth of AMVUTTRA and the advancement of next-generation RNAi therapies.

A key milestone under this pillar is the planned launch of nucresiran, Alnylam’s next-generation TTR silencer. The company expects to introduce nucresiran for patients with polyneuropathy by 2028, followed by cardiomyopathy indications by 2030, positioning it as a best-in-class therapy within the ATTR treatment landscape.

Driving Growth Through Sustainable Innovation

Beyond TTR amyloidosis, Alnylam is focused on expanding its pipeline to deliver therapies that can prevent, halt, or potentially reverse disease. The company aims to deliver at least two new transformative medicines beyond TTR with blockbuster revenue potential. To support this objective, Alnylam plans to broaden its RNAi platform to address 10 tissue types and advance more than 40 clinical programs over the next several years.

Investment in research and development remains a priority, with the company committing approximately 30% of revenues to non-GAAP R&D, including selective external innovation opportunities. This disciplined investment approach is intended to generate a high-yield pipeline while maintaining financial sustainability.

Scaling with Financial Discipline and Agility

The third pillar of Alnylam 2030 focuses on delivering sustained, profitable growth. The company is targeting a total revenue compound annual growth rate (CAGR) of more than 25% through the end of 2030, alongside a non-GAAP operating margin of approximately 30%. These financial goals reflect Alnylam’s confidence in its expanding commercial portfolio and its ability to scale efficiently.

Leadership Perspective

Commenting on the announcement, Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam, highlighted the company’s transformation over the past five years. She noted that Alnylam successfully expanded its commercial product portfolio, advanced a robust pipeline of RNAi therapeutics, and achieved strong financial performance leading to sustainable profitability.

Dr. Greenstreet emphasized that the early launch momentum of AMVUTTRA in ATTR cardiomyopathy has established the company’s first flagship commercial franchise. Looking ahead, she described Alnylam 2030 as a clear strategic framework to reinforce TTR leadership, reinvest growing revenues into high-impact innovation, and maintain financial discipline as the organization scales. She underscored Alnylam’s unique position as the pioneer of RNAi therapeutics with six approved products and expressed confidence in the company’s ability to drive the next stage of growth.

Preliminary 2025 Financial Performance

For the fourth quarter of 2025, preliminary global net product revenues for AMVUTTRA were approximately $827 million, while ONPATTRO generated about $32 million. Combined, these results represent total TTR growth of 151% compared to the same period in 2024. For the full year 2025, AMVUTTRA and ONPATTRO generated approximately $2.31 billion and $173 million, respectively, reflecting 103% year-over-year growth for the TTR franchise.

Within Alnylam’s rare disease portfolio, fourth-quarter revenues for GIVLAARI and OXLUMO were approximately $87 million and $50 million, respectively, representing 26% growth compared to the prior year. Full-year 2025 revenues for these products totaled approximately $308 million for GIVLAARI and $191 million for OXLUMO, an 18% increase year over year.

2026 Revenue Outlook

Looking ahead, Alnylam provided combined net product revenue guidance for 2026 of $4.9 billion to $5.3 billion, representing 71% growth at the midpoint compared to 2025. The TTR franchise is expected to generate $4.4 billion to $4.7 billion, while the rare disease portfolio is projected to deliver $500 million to $600 million in revenues. Additional guidance related to collaboration revenue, royalties, and operating expenses will be provided with the company’s full earnings release.

Advancing a Broad and Deep Pipeline

Alnylam also outlined several key pipeline objectives for 2026, including the advancement of Phase 3 trials for nucresiran in both ATTR cardiomyopathy and polyneuropathy, progress in the ZENITH Phase 3 outcomes trial for zilebesiran in hypertension, and expanded development of mivelsiran in cerebral amyloid angiopathy and Alzheimer’s disease. Additional programs across bleeding disorders, type 2 diabetes, Huntington’s disease, and obesity are expected to reach important clinical milestones, alongside the filing of three to four new IND applications by the end of 2026.

Collectively, these initiatives underscore Alnylam’s commitment to long-term growth, innovation, and leadership in RNAi therapeutics under its Alnylam 2030 strategy.

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