01—Abstract
Summary
Summary of the 10-K filed February 14, 2019. Cited figures are marked; the original filing is the authoritative source.
Alnylam Pharmaceuticals reported its 2018 fiscal year results, marking a significant transition to a commercial-stage biopharmaceutical company with the launch of its first RNAi therapeutic, ONPATTRO®, in August 2018 for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis.
The company generated $12.5 milliona in net revenue from ONPATTRO sales in 2018, with expansion into Europe occurring in the latter half of the year. Financially, Alnylam ended 2018 with strong liquidity, holding $1.13 billion in cash, cash equivalents, and marketable securities, bolstered by a $382 million public offering in January 2019. The company's pipeline remains robust, with five late-stage investigational programs advancing.
These include vutrisiran for ATTR amyloidosis, givosiran for acute hepatic porphyria (AHP) where top-line Phase 3 data was positive, lumasiran for primary hyperoxaluria type 1, fitusiran for hemophilia (partnered with Sanofi Genzyme), and inclisiran for hypercholesterolemia (partnered with The Medicines Company). Alnylam's strategic restructuring of its alliance with Sanofi Genzyme in January 2018 clarified global rights for ONPATTRO and vutrisiran to Alnylam, and for fitusiran to Sanofi Genzyme, aligning with Alnylam's goal of building a multi-product commercial biopharmaceutical company.
02—Key points
Key Highlights
- Launched ONPATTRO® (patisiran) in the U.S. and Europe in 2018, generating $12.5 million in net revenue.
- Ended 2018 with $1.13 billion in cash, cash equivalents, and marketable debt securities, supported by a successful $382 million public offering in January 2019.
- Advanced five late-stage investigational programs, including vutrisiran, givosiran (positive Phase 3 interim results), lumasiran, fitusiran (partnered), and inclisiran (partnered).
- Refined global rights for ATTR amyloidosis franchise (ONPATTRO and vutrisiran) to Alnylam, and transferred global rights for fitusiran to Sanofi Genzyme through a strategic alliance amendment.
- Continued investment in research and development, with significant progress noted across key therapeutic areas: Genetic Medicines, Cardio-Metabolic Diseases, Hepatic Infectious Diseases, and CNS/Ocular Diseases.
- The company's RNAi platform technology is central to its drug development strategy, with ongoing advancements in delivery technologies like lipid nanoparticles (LNPs) and GalNAc conjugates.
03—One dollar
04—Financials
Financial Highlights
Figures from 10-KFY2018filed February 14, 2019Financial Statements
FY2018 against FY2017
| Line item | FY2018FY2017 | Change |
|---|---|---|
| Revenue | $2.90M$89.91M | −96.8% |
| Cost of Revenue | $1.80M$0 | — |
| Gross Profit | $1.10M$89.91M | −98.8% |
| R&D Expenses | $505.42M$390.63M | +29.4% |
| SG&A Expenses | $382.36M$199.37M | +91.8% |
| Operating Expenses | $889.58M$590.00M | +50.8% |
| Operating Income | -$814.67M-$500.09M | −62.9% |
| Interest Expense | $0— | — |
| Net Income | -$761.50M-$490.87M | −55.1% |
| EPS (Basic) | $-7.57$-5.42 | −39.7% |
| Shares Outstanding (Basic) | 100.59M90.55M | +11.1% |
05—Questions
Frequently Asked Questions
What is Alnylam's primary product and its status?
Alnylam's primary product is ONPATTRO® (patisiran), the first FDA-approved RNAi therapeutic. It was launched in the U.S. in August 2018 for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults. It also received marketing authorization in the European Union.
How is Alnylam's financial position?
As of December 31, 2018, Alnylam had a strong liquidity position with $1.13 billion in cash, cash equivalents, and marketable debt securities. The company also raised approximately $382 million in net proceeds from a public offering in January 2019, indicating a healthy financial footing to support its ongoing operations and pipeline development.
What are Alnylam's key late-stage pipeline programs?
Alnylam has five key late-stage investigational RNAi therapeutics: vutrisiran (for ATTR amyloidosis), givosiran (for acute hepatic porphyria), lumasiran (for primary hyperoxaluria type 1), fitusiran (for hemophilia, partnered with Sanofi Genzyme), and inclisiran (for hypercholesterolemia, partnered with The Medicines Company). Givosiran showed positive top-line interim results from its Phase 3 study.
What significant strategic changes occurred regarding Alnylam's collaborations?
In January 2018, Alnylam amended its collaboration with Sanofi Genzyme. This amendment granted Alnylam exclusive global rights for ONPATTRO and vutrisiran, while Sanofi Genzyme gained exclusive global rights for fitusiran. This restructuring aimed to streamline development and commercialization efforts for these key programs.
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