Summary
Alnylam Pharmaceuticals, Inc. (ALNY) presented its 2019 Annual Report (10-K) on February 13, 2020, highlighting significant progress in its RNA interference (RNAi) therapeutics platform. The company achieved key milestones, including the U.S. FDA approval and launch of GIVLAARI for acute hepatic porphyria (AHP) and continued commercialization of ONPATTRO for hereditary transthyretin-mediated amyloidosis (hATTR) polyneuropathy, which generated $166.4 million in net revenue in 2019. Alnylam's "Alnylam 2020" strategy is on track, with a robust pipeline focused on four strategic therapeutic areas (STArs): Genetic Medicines, Cardio-Metabolic Diseases, Hepatic Infectious Diseases, and CNS/Ocular Diseases. Financially, the company ended 2019 with $1.55 billion in cash, cash equivalents, and marketable securities, positioning it to fund its operations and continued pipeline development. While the company reported a net loss of $886.1 million for the year, reflecting significant investment in R&D, it anticipates future growth driven by its expanding commercial product portfolio and promising late-stage pipeline candidates like lumasiran for primary hyperoxaluria type 1 (PH1). Strategic collaborations with major players like Regeneron and Sanofi Genzyme continue to be a cornerstone of Alnylam's development and commercialization strategy.
Financial Highlights
50 data points| Revenue | $219.75M |
| Cost of Revenue | $25.06M |
| Gross Profit | $194.69M |
| R&D Expenses | $655.11M |
| SG&A Expenses | $479.00M |
| Operating Expenses | $1.16B |
| Operating Income | -$939.43M |
| Interest Expense | $0 |
| Net Income | -$886.12M |
| EPS (Basic) | $-8.11 |
| EPS (Diluted) | $-8.11 |
| Shares Outstanding (Basic) | 109.26M |
| Shares Outstanding (Diluted) | 109.26M |
Key Highlights
- 1Secured FDA approval and launched GIVLAARI (givosiran) for Acute Hepatic Porphyria (AHP) in late 2019, marking its second commercial RNAi therapeutic.
- 2ONPATTRO (patisiran) generated $166.4 million in net revenue for 2019, with global approvals and market expansions contributing to growth.
- 3Advanced a robust late-stage pipeline with programs in ATTR amyloidosis (vutrisiran), PH1 (lumasiran), hypercholesterolemia (inclisiran via Novartis), and hemophilia (fitusiran via Sanofi Genzyme).
- 4Strengthened financial position with $1.55 billion in cash, cash equivalents, and marketable securities as of December 31, 2019.
- 5Executed a significant strategic collaboration with Regeneron Pharmaceuticals in April 2019 to discover, develop, and commercialize RNAi therapeutics targeting eye and CNS diseases, along with certain liver targets, including an initial $400 million upfront payment.
- 6Continued to build out global commercial infrastructure and patient support programs to support the commercialization of ONPATTRO and GIVLAARI.
- 7Reported positive topline results for key late-stage programs, including lumasiran (ILLUMINATE-A Phase 3) and initiated rolling submission for its NDA to the FDA.