01—Abstract
Summary
Summary of the 8-K filed May 1, 2025. Cited figures are marked; the original filing is the authoritative source.
Alnylam Pharmaceuticals reported first quarter 2025 results on May 1, 2025, led by strong commercial momentum in its transthyretin (TTR) franchise.
Global net product revenues reached $469 milliona, up 28% year over year (30% at constant exchange rates), driven by AMVUTTRA, which generated $310 million, up from prior periods on increased patient demand. Total revenues were $594 million, up 20%. Total TTR net product revenues rose 36% to $359 million, while Total Rare revenues grew 8% to $109 million.
The company also reported its first quarter GAAP income from operations of $18 millionb, compared with a loss of $43 million a year earlier, and non-GAAP operating income of $75 million. GAAP net loss narrowed to $57 million, or $0.44 per share, from $66 million, or $0.52 per share. The quarter was marked by U.S. FDA approval of AMVUTTRA for ATTR-CM (cardiomyopathy of transthyretin-mediated amyloidosis), an important expansion of the franchise, and by Sanofi's FDA approval of Qfitlia (fitusiran), the sixth Alnylam-discovered RNAi therapeutic approved in the U.S. Management reiterated full-year 2025 guidance of $2.05 billion to $2.25 billion in combined net product revenues and expects to achieve non-GAAP operating profitability for the year.
Collaboration revenue declined 16% to $99 millionc because a $65 million Roche milestone recognized in the prior-year period did not recur, partially offset by Regeneron growth and a $30 million Vir Biotechnology payment. The balance sheet remains substantial but carries significant obligations. Cash, cash equivalents and marketable securities totaled $2.63 billion at March 31, 2025, down from $2.69 billion at year-end 2024, primarily due to operating cash use.
Other expense included a $58.9 milliond non-cash charge from the fair value change of the development derivative liability, which management attributed to the ATTR-CM approval. Total stockholders' equity improved to $115 million from $67 million, and the company continues to carry large liabilities tied to the Leqvio royalty sale and its development derivative.
02—Key points
Key Highlights
- Global net product revenues of $469M grew 28% YoY (30% at constant currency), with AMVUTTRA contributing $310M and ONPATTRO $49M.
- Total TTR net product revenues rose 36% to $359M, while Total Rare revenues grew 8% to $109M led by GIVLAARI ($67M) and OXLUMO ($42M).
- GAAP operating income of $18M swung from a $43M loss a year earlier; non-GAAP operating income was $75M versus $2M.
- FDA approved AMVUTTRA for ATTR-CM, and Sanofi received FDA approval for Qfitlia, the sixth Alnylam-discovered RNAi therapeutic approved in the U.S.
- Full-year 2025 guidance reiterated: total net product revenues of $2.05B–$2.25B and collaboration/royalty revenues of $650M–$750M, with non-GAAP operating profitability targeted.
- Collaboration revenue fell 16% to $99M as a prior-year $65M Roche milestone did not recur, partly offset by Regeneron growth and a $30M Vir payment.
- Cash and marketable securities were $2.63B, down from $2.69B at year-end, while a $58.9M non-cash charge on the development derivative liability weighed on other expense.
03—Questions
Frequently Asked Questions
Did Alnylam achieve profitability in the first quarter of 2025?
On a GAAP basis, Alnylam reported income from operations of $18.1 million, a sharp improvement from a $43.4 million loss in Q1 2024. However, after interest expense, a $58.9 million non-cash fair value charge on the development derivative liability, and taxes, the company still posted a GAAP net loss of $57.5 million, or $0.44 per share. On a non-GAAP basis, operating income was $74.8 million and the net loss was $1.3 million, or $0.01 per share.
What is driving revenue growth?
Growth is concentrated in the TTR franchise. AMVUTTRA revenue reached $310 million, up on increased patient demand, while ONPATTRO declined as patients switched to AMVUTTRA. GIVLAARI also grew, and total net product revenues rose 28%. Collaboration revenue declined because a $65 million Roche milestone from the prior year did not recur.
Does the company expect to meet its 2025 financial targets?
Management reiterated 2025 guidance, including total net product revenues of $2.05 billion to $2.25 billion, with TTR revenues of $1.6 billion to $1.725 billion. It also expects collaboration and royalty revenues of $650 million to $750 million and non-GAAP operating income in positive territory, which is consistent with its stated goal of achieving profitability. Guidance assumes the AMVUTTRA ATTR-CM launches in Germany and Japan in the second half of 2025, and the $300 million Roche milestone for initiating a zilebesiran Phase 3 outcomes trial.
How strong is the company's liquidity position?
Alnylam ended the quarter with $2.63 billion in cash, cash equivalents and marketable securities, down from $2.69 billion at year-end 2024, mainly due to operating cash use. Operating cash outflow was $118.3 million, which included $61.7 million of payments allocated to interest on the Leqvio royalty sale liability. The company also carries substantial obligations, including about $1.03 billion of convertible debt and roughly $1.42 billion in long-term royalty sale liabilities.
What upcoming catalysts should investors watch?
Key catalysts include anticipated approvals of AMVUTTRA for ATTR-CM in Japan in Q2 2025 and the European Union in Q3 2025, initiation of the TRITON-CM Phase 3 trial of nucresiran in the first half of 2025, and KARDIA-3 Phase 2 data for zilebesiran in the second half of 2025. Those results are expected to inform a Phase 3 cardiovascular outcomes trial for zilebesiran planned for the second half of 2025.
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