01—Abstract
Summary
Summary of the 10-K filed February 15, 2018. Cited figures are marked; the original filing is the authoritative source.
Alnylam Pharmaceuticals, Inc.'s 2017 10-K highlights a pivotal year marked by significant progress in its RNA interference (RNAi) therapeutic pipeline, particularly with patisiran.
The company reached a major milestone by filing its first New Drug Application (NDA) with the FDA and a Marketing Authorisation Application (MAA) with the EMA for patisiran, targeting hereditary transthyretin-mediated (hATTR) amyloidosis. Regulatory reviews are underway with anticipated action dates in 2018, positioning Alnylam for potential product launch and revenue generation within the year. Financially, Alnylam strengthened its position by completing two public offerings that raised substantial net proceeds, ending the year with a robust cash balance.
The company continues to invest heavily in research and development to advance its Alnylam 2020 strategy, aiming to have three marketed products and ten clinical-stage programs by the end of 2020. Key collaborations, notably with Sanofi Genzyme and The Medicines Company, remain central to its development and commercialization efforts, with recent amendments to the Sanofi Genzyme agreement significantly reshaping global rights for key pipeline assets.
02—Key points
Key Highlights
- Filed first NDA and MAA for patisiran (hATTR amyloidosis) with the FDA and EMA, respectively, with anticipated regulatory action in 2018.
- Successfully raised approximately $1.14 billion in net proceeds through two public equity offerings, significantly bolstering cash reserves.
- Ended 2017 with $1.7 billion in cash, cash equivalents, and marketable securities, providing strong financial footing.
- Advanced multiple pipeline programs into late-stage clinical development, including givosiran (Acute Hepatic Porphyrias) and fitusiran (Hemophilia A and B), with significant clinical trial updates.
- Restructured global rights for patisiran, ALN-TTRsc02, and fitusiran with Sanofi Genzyme, consolidating patisiran rights with Alnylam and global rights for fitusiran with Sanofi Genzyme.
- Continued to invest heavily in R&D, with expenses reaching $390.6 million in 2017, underscoring commitment to pipeline advancement.
- Established commercial readiness by expanding organizational capabilities in anticipation of patisiran's potential launch.
03—One dollar
04—Financials
Financial Highlights
Figures from 10-KFY2017filed February 15, 2018Financial Statements
FY2017 against FY2016
| Line item | FY2017FY2016 | Change |
|---|---|---|
| Revenue | $89.91M$47.16M | +90.7% |
| Cost of Revenue | $0— | — |
| Gross Profit | $89.91M— | — |
| R&D Expenses | $390.63M$382.39M | +2.2% |
| SG&A Expenses | $199.37M$89.35M | +123.1% |
| Operating Expenses | $590.00M$471.75M | +25.1% |
| Operating Income | -$500.09M-$424.59M | −17.8% |
| Net Income | -$490.87M-$410.11M | −19.7% |
| EPS (Basic) | $-5.42$-4.79 | −13.2% |
| Shares Outstanding (Basic) | 90.55M85.60M | +5.8% |
05—Questions
Frequently Asked Questions
What is the most significant regulatory milestone achieved in 2017 for Alnylam Pharmaceuticals?
The most significant regulatory milestone for Alnylam in 2017 was the filing of its first New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) and a Marketing Authorisation Application (MAA) with the European Medicines Agency (EMA) for patisiran, a treatment for hereditary transthyretin-mediated (hATTR) amyloidosis. This marked a critical step towards potentially bringing their first product to market.
How has Alnylam's financial position changed, and what are their funding strategies?
Alnylam significantly strengthened its financial position in 2017 by raising approximately $1.14 billion in net proceeds through two public equity offerings. This increased their cash, cash equivalents, and marketable securities to $1.7 billion by year-end. Their strategy to fund ongoing operations and pipeline advancement relies on these cash reserves, existing and future strategic alliances, and potential milestone and royalty payments.
What are the key updates regarding Alnylam's major collaborations?
Alnylam made significant strategic adjustments to its collaboration with Sanofi Genzyme in early 2018. The key changes include Alnylam gaining exclusive global rights for the development and commercialization of patisiran and ALN-TTRsc02, while Sanofi Genzyme obtained exclusive global rights for fitusiran. This restructuring aims to streamline development and commercialization efforts for these important pipeline assets.
What is Alnylam's overall strategy and outlook for the near future?
Alnylam is focused on executing its 'Alnylam 2020' strategy, which aims to establish the company as a multi-product commercial-stage biopharmaceutical firm with three marketed products and ten clinical-stage RNAi therapeutic programs by the end of 2020. The company is heavily investing in R&D and building its commercial infrastructure in preparation for the potential launch of patisiran in 2018, while continuing to advance its broader pipeline.
06—Continue