Alnylam Pharmaceuticals, Inc. — Q1 2024 Form 10-Q
Reporting period: Three months ended March 31, 2024. Financial statements are unaudited. Alnylam is a commercial-stage RNAi therapeutics company with four products it commercializes directly and Leqvio, commercialized by Novartis.
Financial performance
| Metric | Q1 2024 | Q1 2023 | Change |
|---|---|---|---|
| Total revenue | $494.3 million | $319.3 million | +55% |
| Net product revenue | $365.2 million | $276.3 million | +32% |
| Collaboration revenue | $118.5 million | $36.5 million | +225% |
| Royalty revenue | $10.6 million | $6.5 million | +63% |
| Operating expenses | $537.8 million | $469.1 million | +15% |
| Loss from operations | $(43.4) million | $(149.8) million | Loss narrowed |
| Net loss | $(65.9) million | $(174.1) million | Loss narrowed |
| Basic and diluted loss per share | $(0.52) | $(1.40) | |
| Net cash used in operating activities | $(81.5) million | $(166.5) million | Cash use decreased |
Cost of goods sold was 15.0% of net product revenue, unchanged year over year. Revenue growth outpaced expense growth, reducing the operating loss, but the company remained loss-making. Research and development expense rose 13% to $261.0 million and selling, general and administrative expense rose 15% to $210.8 million.
Products and material period-over-period changes
- AMVUTTRA revenue rose 92% to $195.2 million, with growth across the U.S., Europe and rest of world.
- GIVLAARI revenue increased 21% to $58.1 million, and OXLUMO increased 77% to $42.6 million.
- ONPATTRO revenue declined 32% to $69.2 million.
- Collaboration revenue included $74.7 million from Roche. This included $65.0 million recognized for a zilebesiran development milestone; the first patient in KARDIA-3 was dosed in April 2024, after quarter-end.
- Leqvio royalties increased year over year. Alnylam sold part of its future Leqvio royalty stream to Blackstone; the related liability was $1.38 billion at quarter-end.
Liquidity, debt and cash flow
At March 31, 2024, cash and cash equivalents were $681.9 million, marketable debt securities were $1.678 billion, and marketable equity securities were $11.3 million. Together these totaled approximately $2.37 billion. Current assets were $2.99 billion and current liabilities were $941.7 million. Cash, cash equivalents and restricted cash decreased by $130.2 million during the quarter.
Convertible debt carrying value was $1.022 billion, principally 1% convertible senior notes due 2027. The future-royalty liability was $1.383 billion. The company reported an accumulated deficit of $7.08 billion and stockholders’ deficit of $219.3 million. Operating cash use improved from the prior-year quarter, which management attributed primarily to stronger product-sales receipts and lower working-capital cash disbursements.
Outlook, commentary and key risks
- Management believes cash, cash equivalents and marketable securities, together with expected product and collaboration cash generation, will fund working capital and operating needs for at least 12 months from the filing date. The company continues to target financial self-sustainability by the end of 2025, while expecting annual operating losses in the near term.
- Management expects 2024 collaboration and royalty revenue combined to decrease versus 2023, primarily due to lower Roche collaboration revenue; it expects Leqvio royalties to increase. R&D and SG&A expenses combined are expected to increase in 2024.
- Topline HELIOS-B Phase 3 results for vutrisiran in ATTR amyloidosis with cardiomyopathy were expected in late June or early July 2024. The FDA had issued a complete response letter for patisiran in this indication, stating clinical meaningfulness had not been established.
- Roche collaboration revenue and future payments depend on development progress and milestones. The company noted that drug-development outcomes and future milestone, royalty or profit-share receipts are uncertain.
- Principal risks include clinical-trial and regulatory uncertainty, product uptake and reimbursement, competition, reliance on collaborators and contract manufacturers, and continued operating losses. Alnylam’s patent litigation against Pfizer/BioNTech and Moderna was ongoing; the Pfizer/BioNTech trial was expected in the first half of 2025, subject to court scheduling.
Important facts for investors to verify
- HELIOS-B results, regulatory implications, and any resulting AMVUTTRA filing or approval timeline.
- Whether product-sales growth—particularly AMVUTTRA—can offset expected lower Roche-related collaboration revenue and rising R&D and SG&A costs.
- Leqvio sales and royalties, including the effect of the Blackstone royalty-sale terms and the $1.38 billion related liability.
- Cash burn and funding assumptions underlying management’s 12-month liquidity assessment and 2025 self-sustainability goal.
- Developments in the Pfizer/BioNTech and Moderna patent cases and their potential financial or operational impact.