01—Abstract
Summary
Summary of the 10-K filed February 13, 2020. Cited figures are marked; the original filing is the authoritative source.
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 milliona 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 millionb 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.
02—Key points
Key Highlights
- Secured FDA approval and launched GIVLAARI (givosiran) for Acute Hepatic Porphyria (AHP) in late 2019, marking its second commercial RNAi therapeutic.
- ONPATTRO (patisiran) generated $166.4 million in net revenue for 2019, with global approvals and market expansions contributing to growth.
- Advanced a robust late-stage pipeline with programs in ATTR amyloidosis (vutrisiran), PH1 (lumasiran), hypercholesterolemia (inclisiran via Novartis), and hemophilia (fitusiran via Sanofi Genzyme).
- Strengthened financial position with $1.55 billion in cash, cash equivalents, and marketable securities as of December 31, 2019.
- Executed 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.
- Continued to build out global commercial infrastructure and patient support programs to support the commercialization of ONPATTRO and GIVLAARI.
- Reported positive topline results for key late-stage programs, including lumasiran (ILLUMINATE-A Phase 3) and initiated rolling submission for its NDA to the FDA.
03—One dollar
04—Financials
Financial Highlights
Figures from 10-KFY2019filed February 13, 2020Financial Statements
FY2019 against FY2018
| Line item | FY2019FY2018 | Change |
|---|---|---|
| Revenue | $219.75M$2.90M | +7477.6% |
| Cost of Revenue | $25.06M$1.80M | +1290.8% |
| Gross Profit | $194.69M$1.10M | +17631.1% |
| R&D Expenses | $655.11M$505.42M | +29.6% |
| SG&A Expenses | $479.00M$382.36M | +25.3% |
| Operating Expenses | $1.16B$889.58M | +30.3% |
| Operating Income | -$939.43M-$814.67M | −15.3% |
| Interest Expense | $0$0 | — |
| Net Income | -$886.12M-$761.50M | −16.4% |
| EPS (Basic) | $-8.11$-7.57 | −7.1% |
| EPS (Diluted) | $-8.11— | — |
| Shares Outstanding (Basic) | 109.26M100.59M | +8.6% |
| Shares Outstanding (Diluted) | 109.26M— | — |
05—Questions
Frequently Asked Questions
What are Alnylam's key commercial products and their performance?
Alnylam has two commercial products: ONPATTRO® (patisiran) for the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) and GIVLAARI® (givosiran) for acute hepatic porphyria (AHP). In 2019, ONPATTRO generated $166.4 million in net revenue, with continued global market expansion. GIVLAARI was approved by the FDA in November 2019 and launched in the U.S. in December 2019, showing strong initial demand.
How is Alnylam funding its operations and pipeline development?
Alnylam ended 2019 with $1.55 billion in cash, cash equivalents, and marketable securities. This strong liquidity, combined with ongoing revenues from its commercial products and strategic collaborations, is expected to fund its operations and extensive pipeline development activities for at least the next 12 months. The company may seek additional funding through various means if needed.
What are Alnylam's key pipeline advancements and collaborations?
Alnylam's pipeline is robust, with several late-stage programs targeting significant unmet medical needs, including lumasiran for PH1, vutrisiran for ATTR amyloidosis, and inclisiran (partnered with Novartis) for hypercholesterolemia. Key advancements include positive Phase 3 results for lumasiran and the initiation of its NDA submission. The company also has significant collaborations, notably with Regeneron for eye and CNS targets, and with Sanofi Genzyme for its genetic medicines portfolio, including fitusiran.
What are the primary risks highlighted in the report for investors?
Key risks for investors include the company's history of operating losses and the need for substantial future funding, the potential for product candidates to fail in development or face regulatory delays, intense competition in the pharmaceutical market, reliance on third-party collaborators and manufacturers, and the inherent uncertainties of developing a novel class of therapeutics like RNAi. The report also mentions potential risks related to intellectual property challenges and market acceptance of its products.
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