Alnylam Pharmaceuticals Q1 Earnings Call Highlights

Key Points

• Alnylam reported a strong Q1 with $1.036 billion in combined net product revenues (up 121% YoY), led by the TTR franchise which generated $910 million (up 153% YoY) and benefited from increasing U.S. uptake and high adherence.
• On the pipeline, Alnylam is expanding the TRITON‑CM Phase III enrollment by about 500 patients to ~1,750 total and still targets a potential nucresiran launch by 2030 if trials are positive, alongside multiple planned 2026 Phase I/II readouts.
• Financially the company delivered sustained profitability with $339 million non‑GAAP operating income, ended Q1 with $3.0 billion cash and reiterated 2026 guidance of $4.4–$4.7 billion in TTR product sales.

Alnylam Pharmaceuticals NASDAQ: ALNY reported what executives described as a strong start to 2026, driven by continued momentum in its transthyretin (TTR) amyloidosis franchise and sustained performance in its rare disease portfolio. On the company’s first-quarter earnings call, CEO Yvonne Greenstreet said the quarter “demonstrated continued strength across the business,” highlighted by Alnylam’s first quarter topping $1 billion in combined net product revenues.

Product revenue tops $1 billion as TTR franchise drives growth

Chief Commercial Officer Tolga Tanguler said Alnylam delivered $1.036 billion in combined net product revenues in Q1, up 121% year-over-year and up 4% sequentially from Q4 2025. The company’s TTR franchise remained the primary growth engine, with $910 million in global TTR net revenues in the quarter, up 6% from Q4 and 153% year-over-year, according to Tanguler.

In the U.S., Tanguler said TTR revenues increased 9% versus Q4 and “more than 230% year-over-year,” reflecting continued growth in patient demand. He also noted that the $59 million U.S. revenue increase over Q4 occurred despite “fewer Q1 shipping weeks and customer insurance reauthorization dynamics earlier in the year,” which management had flagged previously.

Outside the U.S., Tanguler said international TTR revenues declined $7 million from Q4, primarily due to “updated pricing in Germany following the ATTR-CM launch,” but grew 35% year-over-year. He added that international performance exceeded expectations shared in February, citing continued strength in Japan and ongoing hereditary ATTR polyneuropathy (PN) performance across international markets.

Alnylam’s rare disease portfolio generated $126 million in net revenue, up 15% year-over-year. Tanguler said growth was driven by increased patient demand, partially offset by “higher gross net deductions across U.S. and international markets.”

U.S. launch dynamics: first-line adoption, prescriber growth, and adherence

Management emphasized physician adoption trends for AMVUTTRA in ATTR cardiomyopathy (ATTR-CM). Tanguler said the launch has been supported by three factors: “physician preference and utilization,” “access and affordability,” and “site of care infrastructure.” He also pointed to expanding prescriber participation, noting that Alnylam has added “more than 1,200 unique new U.S. prescribers since last March.”

In the Q&A, Tanguler elaborated on the evolution of first-line versus second-line use, describing “the natural evolution of second-line dynamics as the launch progresses.” He said early second-line use has been driven by physicians transitioning patients who were progressing on stabilizers, and that second-line volume tends to normalize over time as those patients transition. “Growing physician experience has been leading to increased adoption in the first-line setting,” he said, adding that the business is now “modestly weighed towards first line,” while second line remains an ongoing contributor to growth.

Tanguler also highlighted access metrics, stating that “over 90% patients” are covered with first-line access and “most” face zero out-of-pocket costs. He said adherence “exceeded 90%,” which management argued is an important long-term indicator for sustaining benefit and revenue.

Pipeline updates: real-world data, nucresiran trial expansion, and 2026 milestones

Chief Research and Development Officer Pushkal Garg said Alnylam has “over 25 clinical programs” and highlighted new data presented at the American College of Cardiology (ACC) annual meeting supporting AMVUTTRA’s profile in ATTR-CM.

Garg discussed a retrospective cohort study using approximately four years of real-world data in patients with transthyretin-mediated amyloidosis. He said greater than 93% of patients were adherent to vutrisiran (defined as 80% or more days covered), and over 85% remained on therapy for more than one year. He contrasted these results with “low adherence and persistence” seen with “many oral therapies.”

Garg also described a post-hoc analysis from the HELIOS-B trial examining diastolic dysfunction grade (DDG). He said higher DDG at baseline corresponded with adverse outcomes, vutrisiran was associated with a lower risk of worsening DDG versus placebo, and vutrisiran reduced the risk of all-cause mortality and cardiovascular events during the double-blind period regardless of baseline DDG.

On Alnylam’s next-generation TTR silencer nucresiran, Garg said the TRITON-CM Phase III study is enrolling faster than expected. He added that patients enrolling are “somewhat milder” on average than those in HELIOS-B due to greater awareness and earlier diagnosis, and emphasized that TRITON-CM is event-driven. Garg announced Alnylam will use a pre-specified protocol option to expand enrollment by approximately 500 patients, increasing the study from 1,250 to about 1,750 total participants. He said the company still projects a launch by 2030, assuming positive data and regulatory approval, and noted that TRITON-PN is ongoing with potential to support approval in hereditary ATTR polyneuropathy by 2028 if successful.

Garg also outlined 2026 milestones, including completing enrollment in the cAPPricorn-1 Phase II trial of mivelsiran in cerebral amyloid angiopathy in the first half of the year, initiating a Phase II trial of mivelsiran in Alzheimer’s disease, and initiating a Phase II trial of ALN-6400 in a second bleeding disorder. In the second half of 2026, he said Alnylam expects to share:

• Phase I healthy-volunteer data for ALN-6400 and Phase II results in hereditary hemorrhagic telangiectasia
• Phase I data for ALN-HTT02 in patients with Huntington’s disease
• Phase I data for ALN-2232 in development for obesity and weight management

Financial results: profitability continues; guidance reiterated

Chief Financial Officer Jeff Poulton said total global net product revenues were “more than $1 billion” in Q1, driven by AMVUTTRA uptake in ATTR-CM. He reported $82 million in collaboration revenue, down 17% year-over-year, primarily due to a $30 million milestone payment from Vir in Q1 2025. Royalty revenue was $49 million, up 85% year-over-year, driven by higher global AMVUTTRA sales.

Gross margin on product sales was 80%, down 5% from Q1 2025. Poulton attributed the decline to increased royalties on AMVUTTRA as higher revenues raised the average royalty rate payable to Sanofi, noting that the royalty rate “resets each calendar year” and that average rates are expected to increase over the course of the year as sales rise.

On expenses, Poulton said non-GAAP R&D was $335 million, up 39% year-over-year, driven by costs for three Phase III studies, including the ZENITH cardiovascular outcomes trial for zilebesiran and the TRITON-CM and TRITON-PN studies for nucresiran. Non-GAAP SG&A was $283 million, up 36%, driven by investments supporting the AMVUTTRA ATTR-CM launch in the U.S. and key international markets, along with compensation and scaling investments.

Alnylam reported non-GAAP operating income of $339 million, which Poulton said was “more than four times” the prior-year level. He said the company delivered profitability on both GAAP and non-GAAP net income bases for the third consecutive quarter. Cash, equivalents, and marketable securities ended Q1 at $3.0 billion, up from $2.9 billion at the end of 2025, which Poulton attributed primarily to strong operating performance.

Alnylam reiterated 2026 guidance, including $4.4 billion to $4.7 billion in TTR product sales. Poulton said the guidance “continues to reflect an assumption of significantly higher quarter-on-quarter revenue growth for the balance of the year.”

During Q&A, Poulton provided color on U.S. quarterly dynamics, saying Q1 U.S. growth was “primarily demand-driven,” with some positive inventory impacts offset by pricing trends and insurance reauthorizations. He also noted that U.S. ordering and revenue recognition are affected by the number of Wednesdays in a quarter, with 14 Wednesdays in Q4 2025 and 12 in Q1 2026. Looking ahead, he said Q2 will have 13 Wednesdays, Q3 14, and Q4 13.

In closing remarks, Greenstreet said the company is “off to a good start in 2026,” citing continued momentum in the AMVUTTRA ATTR-CM launch and advancement across Alnylam’s pipeline of RNAi therapeutics.

About Alnylam Pharmaceuticals NASDAQ: ALNY

Alnylam Pharmaceuticals, Inc NASDAQ: ALNY is a biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics. Founded to translate the scientific discovery of RNAi into new medicines, Alnylam applies small interfering RNA (siRNA) technology to silence disease-causing genes. The company develops therapies designed to provide durable disease modification by targeting underlying genetic drivers across a range of rare and more prevalent conditions.

Alnylam has advanced multiple siRNA-based products into commercialization, initially using lipid nanoparticle delivery and more recently employing GalNAc-conjugate chemistry to enable targeted delivery to the liver with subcutaneous dosing.

This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to contact@marketbeat.com.