01—Abstract
Summary
Summary of the 10-K filed February 12, 2016. Cited figures are marked; the original filing is the authoritative source.
Alnylam Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing novel therapeutics based on RNA interference (RNAi).
The company's strategy centers on its proprietary GalNAc-conjugate delivery platform for siRNAs, targeting genetically validated, liver-expressed genes. Alnylam has a pipeline across three Strategic Therapeutic Areas (STArs): Genetic Medicines, Cardio-Metabolic Diseases, and Hepatic Infectious Diseases. The company has significant ongoing clinical development, with its most advanced program, patisiran for TTR-mediated amyloidosis (ATTR), in Phase 3 trials and expected to report data in 2017.
Other key programs include revusiran (also for ATTR), fitusiran for hemophilia, and ALN-PCSsc for hypercholesterolemia. The company's financial performance is characterized by substantial R&D expenses and net losses, with revenues primarily driven by collaboration agreements. A substantial cash position and strategic alliances, particularly with Sanofi Genzyme, provide financial runway for continued development towards achieving its 'Alnylam 2020' goals.
02—Key points
Key Highlights
- Alnylam is a leading biopharmaceutical company developing RNAi therapeutics, with a proprietary GalNAc-conjugate delivery platform enabling subcutaneous administration.
- The company has a robust pipeline with multiple programs in clinical development, including two Phase 3 programs: patisiran (ATTR amyloidosis) and revusiran (ATTR amyloidosis).
- Patisiran is expected to report Phase 3 APOLLO trial data in 2017, with potential for regulatory submissions in late 2017.
- Strategic alliances with major pharmaceutical companies, including Sanofi Genzyme and The Medicines Company, provide significant funding, development capabilities, and commercialization reach.
- The company has a strong intellectual property portfolio covering fundamental aspects of siRNA structure, uses, modifications, and delivery technologies.
- Significant investment in R&D is ongoing, reflecting the company's commitment to advancing its pipeline across Genetic Medicines, Cardio-Metabolic Diseases, and Hepatic Infectious Diseases.
- Alnylam maintains a strong balance sheet with substantial cash reserves to fund ongoing operations and development activities.
03—One dollar
04—Financials
Financial Highlights
Figures from 10-KFY2015filed February 12, 2016Financial Statements
FY2015 against FY2014
| Line item | FY2015FY2014 | Change |
|---|---|---|
| R&D Expenses | $276.50M$190.25M | +45.3% |
| Operating Expenses | $337.11M$455.54M | −26.0% |
| Operating Income | -$296.01M-$404.98M | +26.9% |
| Net Income | -$290.07M-$360.39M | +19.5% |
| EPS (Basic) | $-3.45$-4.85 | +28.9% |
| Shares Outstanding (Basic) | 83.99M74.28M | +13.1% |
05—Questions
Frequently Asked Questions
What is Alnylam's core technology and how does it work?
Alnylam's core technology is based on RNA interference (RNAi), a naturally occurring biological process for selectively silencing gene expression. They develop RNAi therapeutics, specifically small interfering RNAs (siRNAs), which can be delivered to silence disease-associated genes, offering a potential new class of medicines. Their proprietary GalNAc-conjugate delivery platform is key to enabling targeted delivery to liver cells and facilitating subcutaneous administration.
What are Alnylam's most advanced product candidates and their indications?
Alnylam's most advanced product candidate is patisiran, an investigational RNAi therapeutic for the treatment of TTR-mediated amyloidosis (ATTR amyloidosis), specifically familial amyloid polyneuropathy (FAP). It is currently in Phase 3 clinical development (APOLLO study). Other advanced candidates include revusiran (also for ATTR amyloidosis, Phase 3 ENDEAVOUR study), fitusiran for hemophilia and rare bleeding disorders (Phase 1 ongoing), and ALN-CC5 for complement-mediated diseases (Phase 1/2 ongoing).
How does Alnylam fund its operations and development?
Alnylam funds its operations primarily through strategic alliances and collaborations with leading pharmaceutical and life sciences companies. These collaborations typically involve upfront payments, research and development funding, milestone payments tied to development and regulatory achievements, and potential royalties on future product sales. The company also maintains a strong cash position from equity financings, providing additional financial runway.
What is the significance of the Sanofi Genzyme collaboration?
The global collaboration with Sanofi Genzyme is crucial for Alnylam's strategy, particularly for its Genetic Medicines pipeline. Under this agreement, Alnylam retains development and commercialization rights in North America and Western Europe, while Sanofi Genzyme has rights for development and commercialization in the rest of the world. Sanofi Genzyme has opted into several of Alnylam's Genetic Medicine programs, including patisiran and fitusiran, providing significant financial and commercial support.
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