01—Abstract
Summary
Summary of the 8-K filed February 15, 2024. Cited figures are marked; the original filing is the authoritative source.
Alnylam Pharmaceuticals reported fourth quarter and full year 2023 results on February 15, 2024, highlighted by full-year global net product revenues of $1,241 milliona, up 39% year over year.
Growth was driven by the TTR franchise, where total net product revenues rose 40% to $912 millionb, led by AMVUTTRA ($558 million, up from $94 million in 2022 after its mid-2022 launch) and partly offset by ONPATTRO, which declined to $355 million from $558 million. The Ultra-Rare franchise (GIVLAARI and OXLUMO) grew 35% to $329 million combined. Full-year net revenues from collaborations surged to $546 million from $135 million, largely reflecting the Roche zilebesiran collaboration executed in July 2023, which included a $310 million upfront payment, and Novartis milestone revenue.
Profitability improved at the operating line but the company remained loss-making. GAAP operating loss narrowed to $282 millionc from $785 million in 2022, and GAAP net loss improved to $440 million ($3.52 per share) from $1,131 million ($9.30 per share). Non-GAAP operating loss was $60 million for the year, compared with $554 million in 2022.
Cost of goods sold rose to 21.6% of net product revenues from 15.7%, reflecting AMVUTTRA royalty tiers and inventory write-downs tied to ONPATTRO's unapproved cardiomyopathy indication. Cash, cash equivalents and marketable securities ended 2023 at $2.44 billiond, up from $2.19 billion, supported by the Roche upfront payment, a $100 million Regeneron milestone, and roughly $150 million from employee option exercises. Looking ahead, management guided to 2024 combined net product revenues of $1.40 billion to $1.50 billion (13% to 21% growth), collaboration and royalty revenue of $325 million to $425 million, and GAAP R&D plus SG&A expenses of $1.90 billion to $2.05 billion.
Key catalysts include topline results from the HELIOS-B Phase 3 study of vutrisiran in late June or early July 2024 (with an updated statistical analysis plan) and the KARDIA-2 Phase 2 zilebesiran data at ACC in April 2024. Investors should note that the company still reported a GAAP net loss and a stockholders' deficit of $221 million at year end, and that much of the 2023 revenue step-up was from one-time collaboration payments.
02—Key points
Key Highlights
- Full-year 2023 net product revenues of $1,241 million, up 39% year over year; Q4 net product revenues of $346 million, up 32%.
- AMVUTTRA became the largest TTR product at $558 million for 2023 versus $94 million in 2022, while ONPATTRO declined to $355 million from $558 million.
- Net revenues from collaborations rose 305% to $546 million for 2023, driven by the Roche zilebesiran deal (including a $310 million upfront payment) and Novartis milestones.
- GAAP operating loss narrowed to $282 million from $785 million, and GAAP net loss per share improved to $(3.52) from $(9.30).
- Cash, cash equivalents and marketable securities of $2.44 billion at year end, up from $2.19 billion, despite the operating loss.
- 2024 guidance: combined net product revenues of $1.40-$1.50 billion, collaboration and royalty revenue of $325-$425 million, and GAAP R&D plus SG&A of $1.90-$2.05 billion.
- Key 2024 catalysts include HELIOS-B Phase 3 topline data for vutrisiran (late June or early July) and KARDIA-2 zilebesiran data at ACC on April 7, 2024.
03—Questions
Frequently Asked Questions
How did Alnylam's revenue perform in 2023?
Total net product revenues reached $1,241 million, up 39% from $894 million in 2022, led by AMVUTTRA's launch ramp. Collaboration revenue rose to $546 million from $135 million, and royalty revenue rose to $41 million from $8 million, bringing total revenues to $1,828 million versus $1,037 million a year earlier.
Is Alnylam closer to profitability?
The company narrowed its losses significantly but remained unprofitable. The GAAP operating loss was $282 million versus $785 million in 2022, and the non-GAAP operating loss was about $60 million versus $554 million. GAAP net loss was $440 million, or $3.52 per share. Management's guidance points to continued operating expense investment in 2024, so the path to sustained profitability depends on revenue growth outpacing spending.
What drove the increase in collaboration revenue, and is it recurring?
The increase was driven mainly by the Roche collaboration executed in July 2023, which included a $310 million upfront payment, and by Novartis milestone payments tied to commercialization and regulatory achievements. Much of this is non-recurring milestone or upfront revenue, so investors should not extrapolate the 2023 level into future periods. The 2024 guidance of $325-$425 million for collaboration and royalty revenue suggests a meaningful normalization.
What are the most important upcoming events for investors?
The HELIOS-B Phase 3 topline readout for vutrisiran in ATTR cardiomyopathy is expected in late June or early July 2024, following an updated statistical analysis plan that changes the primary and secondary endpoint structure. The KARDIA-2 Phase 2 zilebesiran results will be presented as a late-breaker at ACC on April 7, 2024. The company also plans to initiate several Phase 1 and Phase 2 studies, including KARDIA-3, ALN-APP in cerebral amyloid angiopathy, ALN-KHK Part B, and ALN-BCAT.
Are there any balance sheet concerns?
Cash and marketable securities of $2.44 billion provide substantial liquidity. However, total liabilities of $4.05 billion exceed total assets of $3.83 billion, resulting in a stockholders' deficit of $221 million. Significant obligations include $1.02 billion in convertible debt and $1.32 billion in long-term liabilities related to the sale of future royalties. Investors should monitor these obligations alongside the company's ability to fund operations without additional equity financing.
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