Alnylam Pharmaceuticals, Inc. (ALNY) Q2 2025 Earnings Call Transcript

Access earnings results, analyst expectations, report, slides, earnings call, and transcript.

Overview

The earnings call summary indicates strong financial performance and optimistic guidance, with a focus on sustainable growth and robust uptake of AMVUTTRA. The Q&A reveals management's confidence in their strategy, with minimal barriers to access and positive trends in patient uptake. Despite some lack of specificity in responses, the overall sentiment is positive, supported by steady growth expectations and a strong cash position. The positive outlook on AMVUTTRA and TTR franchise growth suggests a favorable stock price reaction in the short term.

Key Financial Performance

TTR franchise revenues $544 million, 77% year-over-year growth. Growth largely attributable to the AMVUTTRA CM launch.

Total net product revenues $672 million, 64% growth year-over-year. Driven by strong performance in the TTR franchise and the AMVUTTRA launch.

Rare franchise revenues (GIVLAARI and OXLUMO) $128 million, 24% growth year-over-year. Growth was largely demand-driven with favorable GIVLAARI gross-to-net adjustments in the U.S.

U.S. TTR franchise revenues $383 million, 80% quarter-over-quarter growth. Growth driven by AMVUTTRA's ATTR-CM launch and faster-than-anticipated access across payers and providers.

International TTR franchise revenues 18% year-over-year growth. Driven by continued strength in the hATTR-PN business. No ATTR-CM revenue recognized internationally yet.

Gross margin on product sales 79% for the quarter, compared to 84% in the second quarter of 2024. Decrease driven by increased royalties on AMVUTTRA due to higher revenues.

Non-GAAP R&D expenses $274 million, 11% increase year-over-year. Increase due to start-up clinical trial expenses for zilebesiran and TRITON-CM Phase III study for nucresiran.

Non-GAAP SG&A expenses $261 million, 26% increase year-over-year. Increase driven by headcount and investments in support of the AMVUTTRA-ATTR cardiomyopathy launch in the U.S.

Non-GAAP operating income $95 million, $42 million decrease year-over-year. Decrease driven by the recognition of collaboration revenue related to the modification of the Regeneron agreement in Q2 2024.

Cash, cash equivalents, and marketable securities $2.9 billion, compared to $2.7 billion as of December 31, 2024. Increase driven by cash from operations and net proceeds from employee stock option exercises.

You have reached the limit Upgrade to access full content

Get started

Operating Highlights

AMVUTTRA and ATTR-CM: The launch of AMVUTTRA for ATTR-CM has been highly successful, with $544 million in TTR franchise revenues, marking a 77% year-over-year growth. Approximately 1,400 cardiomyopathy patients are now receiving AMVUTTRA in the U.S., contributing $150 million in revenue. International markets are expected to contribute in the second half of the year.

Pipeline advancements: Initiated the TRITON-CM Phase III study for nucresiran, which received FDA Fast Track Designation for ATTR-CM. Encouraging Phase I data for mivelsiran in Alzheimer's disease was shared, and a Phase I study for ALN-4324 in type 2 diabetes was initiated.

Global expansion: Regulatory approvals for AMVUTTRA in Europe, Japan, and Brazil have been secured, with launches in Germany and Japan underway. International markets are expected to contribute to revenue growth in the second half of 2025.

Revenue growth: Total net product revenues reached $672 million, a 64% year-over-year growth. Guidance for 2025 net product revenues has been increased to $2.65 billion to $2.8 billion, reflecting a $575 million increase at the midpoint.

Operational efficiency: Achieved broad provider account setup for AMVUTTRA faster than anticipated, with 90% of U.S. patients able to access treatment within 10 miles of their residence. Payer adoption and physician engagement have been robust.

TTR leadership: Focused on establishing long-term leadership in TTR amyloidosis with AMVUTTRA and pipeline advancements like nucresiran.

Innovation-driven growth: Emphasis on advancing RNAi therapeutics pipeline, including potential blockbuster opportunities in Alzheimer's disease and type 2 diabetes.

You have reached the limit Upgrade to access full content

Get started

Risk or Challenges

Market Conditions: The company faces challenges in international markets as it has yet to recognize any ATTR-CM revenue internationally. Launches in Germany and Japan are expected to contribute in the third quarter, but delays or underperformance in these markets could impact revenue growth.

Regulatory Hurdles: While the FDA has granted Fast Track Designation for nucresiran, the approval process for new drugs like TRITON-CM and mivelsiran remains lengthy and uncertain, potentially delaying market entry and revenue generation.

Supply Chain and Operational Risks: The company’s rapid expansion and launch activities, particularly for AMVUTTRA, require robust supply chain and operational execution. Any disruptions could impact product availability and revenue.

Economic Uncertainties: Foreign exchange rate fluctuations have already contributed to a $60 million adjustment in revenue guidance. Continued volatility in FX rates could impact financial performance.

Strategic Execution Risks: The company’s ambitious pipeline and global expansion plans require significant investment and operational focus. Failure to execute effectively on these fronts could hinder long-term growth.

You have reached the limit Upgrade to access full content

Get started

Guidance & Outlook

Revenue Guidance: Alnylam has increased its total net product revenues guidance for 2025 from a range of $2.05 billion to $2.25 billion to a revised range of $2.65 billion to $2.8 billion, representing a $575 million or 27% increase at the midpoint. This reflects confidence in the ATTR-CM launch and other commercial products.

TTR Franchise Growth: The TTR franchise guidance has been increased from $1.6 billion to $1.725 billion to a revised range of $2.175 billion to $2.275 billion, representing a 34% increase or more than $550 million at the midpoint. This growth is driven by the strong early launch performance of AMVUTTRA in ATTR cardiomyopathy.

Rare Franchise Guidance: The Rare franchise guidance has been modestly increased by raising the bottom end of the prior guidance range by $25 million, resulting in revised Rare product sales guidance of $475 million to $525 million.

Global Expansion: Regulatory approvals have been secured in Europe, Japan, and Brazil for AMVUTTRA, with launches in Germany and Japan underway, unlocking access to more patients worldwide.

Pipeline Development: The company plans to initiate pivotal studies for nucresiran in hereditary ATTR-PN by the end of 2025, with a potential launch several years ahead of cardiomyopathy. Additionally, Phase II studies for mivelsiran in Alzheimer's disease and ALN-6400 in a bleeding disorder are expected to start later this year.

Long-Term Growth: Alnylam is targeting the launch of nucresiran in ATTR cardiomyopathy around 2030, with the TRITON-CM Phase III study underway. The company is also advancing its CNS platform and other therapeutic areas, including type 2 diabetes and bleeding disorders.

You have reached the limit Upgrade to access full content

Get started

Shareholder Return Plan

The selected topic was not discussed during the call.

You have reached the limit Upgrade to access full content

Get started

Key Q&A

Q:Could you give some details here on the patient profiles for AMVUTTRA with regard to the frontline patients and whether you're seeing mostly mixed phenotype patients or more of a broader population mix?

A:Management highlighted that it is early days but expressed satisfaction with the broad and robust uptake of AMVUTTRA. They noted usage across first-line patients and those progressing on stabilizers, as well as among community and academic physicians. Initial uptake was faster among stabilizer progressors, but a healthy share of first-line patients is now being achieved.

Q:How are you thinking about net price for AMVUTTRA and gross-to-net moving forward?

A:Management reiterated their expectation of a modest and gradual reduction in net price over time, with a mid-single-digit reduction expected in 2025 relative to 2024. They also highlighted that payer dynamics are moving as expected, with minimal out-of-pocket costs for patients.

Q:Was there any bolus effect in cardiomyopathy scripts in the second quarter? Could you clarify the extent to which stocking contributed to the growth of U.S. AMVUTTRA revenue in the second quarter?

A:Management stated that the results are not a 'flash in the pan' and expect continued sustainable growth. They noted a $25 million inventory benefit in Q2, driven by demand rather than increased days on hand. Gross-to-net had an opposite movement due to increased Part D rebates and higher 340B utilization, resulting in a slight net price decrease.

Q:Can you speak in more detail to the assumptions underpinning the updated TTR franchise guidance and provide assurance that the new guidance is not aggressive?

A:Management expects steady growth in both first-line and second-line patient flow, with encouraging trends in first-line use. They emphasized that the guidance reflects early but strong validation of their clinical and commercial strategy. They also noted confidence in achieving the guidance, which implies significant year-over-year growth.

Q:As it relates to stabilizer progressors, can you talk about the criteria physicians are using? What percent of patients on a stabilizer would be considered progressors and good candidates for AMVUTTRA?

A:Management estimated that 33-50% of patients on stabilizers progress and become candidates for AMVUTTRA. They noted that physicians use a variety of clinical factors to determine progression, and over time, more guidelines may develop.

Q:How should we think about the rate of new patient starts per quarter from here? How do you expect the mix of first-line and stabilizer progressor patients to evolve over time?

A:Management reported 1,400 paid patients in the first quarter, with a balanced mix of first-line and stabilizer progressor patients. They expect both categories to grow but did not provide specific quarterly projections.

Q:Can you comment on how common stabilizer use requirements are across different plans and what percentage of patients these plans cover?

A:Management stated that access is not a barrier, with broad coverage across Medicare and commercial payers. They noted that step edits requiring stabilizer use are minimal and in the single digits, with effective tools in place to manage these cases.

Q:Out of the 1,400 patients treated so far, what percentage are first-line patients, and how many received free drug?

A:Management described the uptake as broad and balanced between first-line and stabilizer progressor patients but did not provide specific percentages. They noted minimal use of the Quick Start Program for free drugs.

Q:Have you started to see any combination use of AMVUTTRA with other treatments early in the launch?

A:Management observed a small portion of combination use, which they expect to grow as tafamidis goes generic. However, they did not provide specific data on combination use.

Q:What trends are you seeing in the V122I variant population in first-line access? Are step edits the same as prior authorizations?

A:Management noted that AMVUTTRA works equally well in wild-type and V122I patients, with no specific hereditary or V122I indication trends. They clarified that step edits are minimal and distinct from prior authorizations, primarily requiring stabilizer use first.

Q:Review of Unclear Management Responses

A:Management avoided providing specific percentages or detailed breakdowns for certain questions, such as the exact proportion of first-line patients among the 1,400 treated or precise quarterly projections for new patient starts. They also used general language when discussing payer dynamics and combination use, without offering detailed data or clarity.

You have reached the limit Upgrade to access full content

Get started

Earnings Word Cloud

The most frequently occurring keywords in this quarter's earning call

ALNY Transcript

Alnylam Pharmaceuticals, Inc. (ALNY) Presents at Jefferies London Healthcare Conference 2025 Transcript

Neutral11-19

Alnylam Pharmaceuticals, Inc. (ALNY) Presents at UBS Global Healthcare Conference 2025 Transcript

Neutral11-10

Alnylam Pharmaceuticals, Inc. (ALNY) Q3 2025 Earnings Call Transcript

Positive10-30

The earnings call summary and Q&A indicate strong financial performance and optimistic guidance, particularly with increased revenue guidance and global expansion. The management's confidence in product launches and pipeline development, alongside positive reception from payers and no significant barriers, suggest a positive outlook. Although some uncertainties exist, such as CMS reimbursement changes, the overall sentiment remains positive with growth prospects in multiple areas.

Alnylam Pharmaceuticals, Inc. (ALNY) Presents At Morgan Stanley 23rd Annual Global Healthcare Conference Transcript

Neutral9-10

ALNY Slides

Alnylam Q2 2025 slides: ATTR-CM launch drives 64% revenue growth, guidance raised

2025-07-31

ALNY Report

ALNYLAM PHARMACEUTICALS, INC. 10-K

10-K

2025-02-13

ALNYLAM PHARMACEUTICALS, INC. 10-Q

10-Q

2024-10-31

ALNYLAM PHARMACEUTICALS, INC. 10-Q

10-Q

2024-08-01

ALNYLAM PHARMACEUTICALS, INC. 10-Q

10-Q

2024-05-02

Frequently Asked Questions

Where does this earnings call transcript come from?

All transcripts are sourced directly from the official live webcast or the company’s official investor relations website. We use the exact words spoken during the call with no paraphrasing of the core discussion.

How soon is the transcript available after the earnings call ends?

Full verbatim transcripts are typically published within 4–12 hours after the call ends. Same-day availability is guaranteed for all S&P 500 and most mid-cap companies.

Is the transcript edited or altered in any way?

No material content is ever changed or summarized in the “Full Transcript” section. We only correct obvious spoken typos (e.g., “um”, “ah”, repeated 10 times”, or clear misspoken ticker symbols) and add speaker names/titles for readability. Every substantive sentence remains 100% as spoken.

Why do some answers appear as “Unclear” or “Inaudible”?

When audio quality is poor or multiple speakers talk over each other, we mark the section instead of guessing. This ensures complete accuracy rather than introducing potential errors.

Who creates the AI Summary and Key Q&A highlights shown above the transcript?

They are generated by a specialized financial-language model trained exclusively on 15+ years of earnings transcripts. The model extracts financial figures, guidance, and tone with 97%+ accuracy and is regularly validated against human analysts. The full raw transcript always remains available for verification.