01—Abstract
Summary
Summary of the 8-K filed February 13, 2025. Cited figures are marked; the original filing is the authoritative source.
Alnylam Pharmaceuticals reported strong fourth quarter and full year 2024 results, with global net product revenues of $451 milliona in Q4 (up 30% year over year) and $1,646 million for the full year (up 33%).
Growth was driven primarily by AMVUTTRA, whose sales rose to $970 millionb for 2024 from $558 million in 2023, partially offset by an expected decline in ONPATTRO as patients switch to AMVUTTRA. The company reported a full-year GAAP net loss of $278 million, narrowing from $440 million in 2023, and a non-GAAP operating income of $95 million for the year, compared with a non-GAAP operating loss of $60 million in 2023. Q4 non-GAAP operating loss narrowed to $14 million, and the company reiterated its goal of achieving non-GAAP profitability in 2025.
The most important near-term catalyst is the March 23, 2025 PDUFA date for the supplemental NDA for vutrisiran (AMVUTTRA) in ATTR amyloidosis with cardiomyopathy (ATTR-CM), which was filed using a Priority Review Voucher. Management's 2025 guidance assumes approval by that date. Management guided to 2025 total net product revenues of $2.05 billion to $2.25 billionc (25% to 37% growth at currency rates as of December 31, 2024), with collaboration and royalty revenues of $650 million to $750 million.
That guidance includes a $300 milliond milestone from Roche tied to initiation of a Phase 3 cardiovascular outcomes trial for zilebesiran, and royalties from Sanofi's fitusiran, which has a March 28, 2025 PDUFA date. The balance sheet strengthened, with cash, cash equivalents and marketable securities of $2.69 billion at year-end 2024, up from $2.44 billion a year earlier. Total stockholders' equity moved to a positive $67 million from a deficit of $221 million.
Investors should note that 2024 collaboration revenue declined by $36 million year over year because 2023 included a $310 million upfront from Roche, and that Q4 GAAP results were affected by elevated stock-based compensation and higher royalty rates payable on AMVUTTRA sales. The company will also host an R&D Day on February 25, 2025.
02—Key points
Key Highlights
- Q4 2024 net product revenues of $450.8 million, up 30% year over year (29% at constant exchange rates); full-year 2024 net product revenues of $1,646.2 million, up 33%.
- AMVUTTRA full-year 2024 net product revenues reached $970.5 million, up from $557.8 million in 2023, while ONPATTRO declined to $252.9 million as patients switched to AMVUTTRA.
- Full-year 2024 non-GAAP operating income of $95.2 million, a swing from a $60.5 million non-GAAP operating loss in 2023; Q4 2024 non-GAAP operating loss narrowed to $13.5 million from $74.4 million.
- GAAP net loss narrowed to $278.2 million for 2024 from $440.2 million in 2023, and GAAP loss per share improved to $(2.18) from $(3.52).
- Key regulatory catalysts: vutrisiran sNDA for ATTR-CM has a March 23, 2025 PDUFA date (filed with a Priority Review Voucher), and Sanofi's fitusiran has a March 28, 2025 PDUFA date.
- 2025 guidance: total net product revenues of $2.05-$2.25 billion, collaboration and royalty revenues of $650-$750 million, non-GAAP R&D and SG&A of $2.10-$2.20 billion, and a target of achieving non-GAAP operating profitability.
- Cash, cash equivalents and marketable securities rose to $2.69 billion at December 31, 2024, and stockholders' equity turned positive at $67.1 million from a $220.6 million deficit a year earlier.
03—Questions
Frequently Asked Questions
Will Alnylam achieve non-GAAP profitability in 2025?
Management's 2025 guidance includes a goal to achieve non-GAAP operating profitability. This follows a non-GAAP operating income of $95.2 million for full-year 2024, so the company has already reached profitability on that basis for the year. The guidance assumes continued growth in AMVUTTRA, including U.S. approval for ATTR-CM by March 23, 2025, and approvals and launches in Germany and Japan in the second half of 2025.
What is the key regulatory event for investors to watch in 2025?
The most significant near-term event is the March 23, 2025 PDUFA action date for the supplemental NDA for vutrisiran (AMVUTTRA) in ATTR amyloidosis with cardiomyopathy. A positive decision would open a major new indication for the franchise. Management's 2025 revenue guidance assumes approval by that date. Fitusiran, which Sanofi is advancing, has a separate PDUFA date of March 28, 2025, and would generate royalties for Alnylam if approved.
Why did collaboration revenue decline in 2024?
Net revenues from collaborations fell by $36.0 million to $510.2 million in 2024. The decline reflects that 2023 included $310.0 million of revenue from Roche's upfront payment for the zilebesiran collaboration, while 2024 included $185.0 million from the Regeneron collaboration after the June 2024 modification that gave Regeneron an exclusive license to cemdisiran as a monotherapy.
What is driving the change in product revenue mix?
AMVUTTRA is driving growth, with 2024 revenues of $970.5 million, up from $557.8 million in 2023. ONPATTRO revenues declined to $252.9 million from $354.5 million as patients switched to AMVUTTRA. Rare disease products GIVLAARI and OXLUMO grew 29% for the full year to $422.9 million combined. Management expects total 2025 TTR net product revenues of $1.60-$1.725 billion and total Rare net product revenues of $450-$525 million.
What is the impact of higher royalty rates on margins?
Cost of goods sold as a percentage of net product revenues rose to 22.8% in Q4 2024 from 20.8% a year earlier, mainly due to higher royalty rates payable on AMVUTTRA sales. For the full year, cost of goods sold fell to 18.6% of net product revenues from 21.6%, because 2023 included about 5.0 percentage points of one-time charges related to cancelled ONPATTRO manufacturing commitments and inventory impairment that did not recur in 2024.
04—Continue