Alnylam Pharmaceuticals: A High-Conviction Buy Amid Record Revenue Growth and Strategic Pipeline Momentum

Alnylam Pharmaceuticals (ALNY) has emerged as a standout performer in the biotech sector, driven by its groundbreaking RNAi therapeutics and relentless focus on unmet medical needs. In Q2 2025, the company delivered a stunning 64% year-over-year revenue increase, with total net product revenues hitting $672 million. This outperformance was fueled by its Total TTR (transthyretin-related) franchise, which generated $544 million in net product revenues—a 77% surge compared to Q2 2024 [1]. The TTR franchise’s dominance is anchored by AMVUTTRA® (vutrisiran), a gene-silencing therapy that has become the gold standard for treating ATTR-CM (transthyretin amyloid cardiomyopathy). By June 30, 2025, AMVUTTRA had reached 1,400 patients globally, with 42-month data from the HELIOS-B trial showing a 36% reduction in all-cause mortality and a 33% reduction in cardiovascular mortality [1]. These results not only validate the drug’s efficacy but also underscore Alnylam’s ability to transform patient outcomes in rare diseases.

The company’s strategic pipeline momentum further cements its long-term value proposition. Alnylam has initiated the TRITON-CM Phase 3 trial of nucresiran, a next-generation TTR silencer with potential for >95% knockdown and twice-annual dosing [1]. This trial, designed to evaluate nucresiran’s impact on all-cause and cardiovascular mortality in wild-type or hereditary ATTR-CM patients, builds on the success of AMVUTTRA and positions Alnylam to dominate the expanding ATTR-CM market. Additionally, the company is advancing zilebesiran, an RNAi therapy for hypertension, into a large Phase 3 cardiovascular outcomes trial in collaboration with Roche. This trial, expected to enroll 11,000 patients across 30+ countries, could unlock a blockbuster opportunity in a $100+ billion market [2].

Alnylam’s financial strength and operational execution reinforce its appeal as a compounder. The company raised its 2025 guidance to $2.65–$2.8 billion in total net product revenues and $2.175–$2.275 billion in TTR franchise revenues, reflecting confidence in global adoption of AMVUTTRA and regulatory approvals in key markets like the EU, UK, Japan, and Brazil [1]. With $2.9 billion in cash as of Q2 2025 [3], Alnylam is well-positioned to fund its pipeline while transitioning to a cash-flow-positive model. Analysts project the ATTR-CM market to grow to $3.5 billion by 2033, a trajectory Alnylam is uniquely poised to capture through its proprietary GalNAc conjugation platform and first-mover advantage [4].

Alnylam’s competitive moat is rooted in its RNAi platform innovation. The company’s ability to silence genes with precision—whether targeting TTR, angiotensinogen, or huntingtin—demonstrates the versatility of its technology. Recent advancements, such as delivery systems for crossing the blood-brain barrier and targeting muscle and kidney tissues, expand the platform’s applicability to diseases like Alzheimer’s and hypertension [2]. These innovations, combined with strategic partnerships (e.g., Roche, Sanofi), create a durable advantage in a rapidly evolving therapeutic landscape.

For investors seeking exposure to next-generation RNA therapeutics, Alnylam offers a compelling combination of near-term revenue growth, transformative pipeline assets, and a robust balance sheet. The company’s raised guidance, coupled with its leadership inATTR-CM and hypertension, positions it as a high-conviction buy for those willing to capitalize on the RNA revolution.

Source:
[1] Alnylam Pharmaceuticals Press Release | Jul 31, 2025 [https://investors.alnylam.com/press-release?id=29136]
[2] Alnylam Pharmaceuticals Press Release | Feb 25, 2025 [https://investors.alnylam.com/press-release?id=28716]
[3] Alnylam Announces Preliminary* Fourth Quarter and Full Year ... [https://investors.alnylam.com/press-release?id=28611]
[4] Alnylam's Amvuttra: A Game-Changer in ATTR-CM and ... [https://www.ainvest.com/news/alnylam-amvuttra-game-changer-attr-cm-catalyst-biotech-growth-2508/]