Analyst Conference Summary

biotechnology

conference date: April 30, 2026 @ 5:30 AM Pacific Time
for quarter ending: March 31, 2026 (first quarter, Q1)

Forward-looking statements

Overview: Great quarter, fantastic growth rate.

Basic data (GAAP):

Revenue was $1.17 billion, up 6% sequentially from $1.10 billion, and up 96% from $594 million year-earlier.

Net income was $206 million, up 86% sequentially from $112 million, and up from negative $18 million year-earlier.

Diluted EPS was $1.51, up 84% sequentially from $0.82, and up from negative $0.14 year-earlier.

Guidance:

2026 Net Product Revenue $4.90 to $5,30 billion.

Conference Highlights:

Yvonne Greenstreet, CEO of Alnylam, said: "2026 is off to a strong start for Alnylam with the end of the first quarter marking one year since the U.S. launch of Amvuttra for ATTR-CM, and also representing a significant financial milestone with the achievement of over $1 billion in quarterly product revenues for the first time in our history. At the same time, we continued to advance our deep and high-value pipeline of RNAi therapeutics, progressing three ongoing Phase 3 trials, and initiating a Phase 1 study for our first adipose-targeted program for obesity and weight management. In addition, multiple programs across our pipeline are advancing toward key clinical readouts this year. Together, this represents important initial progress toward our Alnylam 2030 goals of achieving global TTR leadership, driving growth through innovation, and scaling with financial discipline as we work to deliver substantial patient impact and create long-term value." Reviewed Analyam 2030 program to gain global TTR leadership. First quarter ever reaching over $1 billion in revenue.

In Q1 2026 Alnylam established collaborations with Viz.ai and the American Heart Association focused on earlier diagnosis, coordinated care and long-term impact in ATTR-CM. Believes 80% of ATTR patients are still untreated. Recently launched Amvuttra in Austria, UK, Switzerland and Italy.

In Q1 2026 in nucresiran, a next-generation TTR silencer, decided to utilize a pre-specified protocol option to expand target enrollment from 1,250 to approximately 1,750 patients. Given the rapid pace of enrollment, Alnylam still expects to launch nucresiran, assuming positive data and regulatory approval, in ATTR-CM by 2030.

In Q1 2026 released additional positive zilebesiran Phase 2 data for cardiovascular risks.

In Q1 2026 partner Regeneron submitted an NDA for cemdisiran for myasthenia gravis.

In Q4 2025 Alnylam began a Phase 2 clinical trial of ALN-4324, an RNAi therapeutic targeting GRB14, for type 2 diabetes mellitus.

In Q1 2026 Alnylam began a Phase 1 clinical trial of ALN-2232, an RNAi therapeutic targeting ACVR1C in adipose tissue for obesity and weight management.

In Q1 2026 Alnylam entered into a research collaboration with Tenaya Therapeutics to discover novel human genetic targets for disease-modifying treatments for cardiovascular diseases.

In Q1 2026 Alnylam entered a new multi-year agreement with Helix to drive precision medicine development. Alnylam will gain access to Helix's deeply phenotyped GenoSphereTM cohorts consisting of comprehensive genomic and longitudinal clinical data, medical, and pharmacy claims that cover a wide range of disease areas.

In Q4 2025 Alnylam announced the planned expansion of its manufacturing facility in Norton, Massachusetts. Plans to invest $250 million to develop the industry's first fully dedicated, proprietary siRNA enzymatic-ligation manufacturing facility.

In the first half of 2026 Alnylam expects to complete enrollment in the Phase 2 mivelsiran trial for cerebral amyloid angiopathy. Plans to start a Phase trial of mivelsiran for Alzheimer's. Also plans to start a phase 2 trial for Alzheimer's.

Alnylam in Q4 2025 initiated the TRITON-CM Phase 3 trial of nucresiran in patients with ATTR-CM.

Net revenue from collaborations was $82 million, down from $99 million year-earlier. Royalty revenue was $49 million, up from $26 million year-earlier.

Non-GAAP net income $273 million, up 61% sequentially from $170 million, and up from $38 million year-earlier. EPS diluted $1.99, up 59% sequentially from $1.25, and up from $0.29 year-earlier.

Cash and equivalents balance at the end of the quarter was $3.01 billion, up sequentially from $2.91 billion. Net cash provided by operating activities was $na million.

See also Alnylam pipeline. In early 2026 had 25 clinical programs including many in late stages.

Operating expenses of $899 million consisted of: $208 million for cost of goods sold; $365 million for research and development; and $323 million for general and administrative expense; $4 million cost of collaboration. Operating income $269 million. Interest & other expense $47 million. $16 million income tax.

Q&A Selective Summary:

First v. second line use of Amvuttra? High confidence in fundamentals. Natural evolution of second line dynamics. Balance between first and second when physician begins with patients who are failing on stabilizers. With experience incrased first line use. Currently modestly more first line. That should support longer utilization. Continuing to expand the prescriber base.

Demand growth for Amvuttra in US? Q1 generally in line with expectations, some inventory impact, but offset by pricing trend. Sequential monthly improvements in quarter. We work with one wholesaler that covers about 80% of our sales, some slight effect from once-weekly shipping dates lower in quarter. For Q2 there will be 13 Wednesdays, over the year 52.

New silencer competition, new data expected? Looking to see cardio-transform (IONS) results, we expect it to be positive. We already know silencer helpful when added onto a stabilizer. We believe we are well-positioned for combinations. We have a robust and durable market share, well positioned against our late-to-market competition.

Amvuttra ex-US pricing? We are pleased with out launches outside the US. Pricing is usually adjusted for ex-US markets. Germany had the most significant impact, but volume should overcome price cut over time. So revenue growth should resume in Q2, ramp rest of year.

We do not believe that the availability of a generic stabilizer will have a significant effect on our growth trajectory.

ATTR-PN market? We are confident. Payors are supportive.

Cemdisiran for MG? The latest data from Regeneron is encouraging.

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