10-KPeriod: FY2013

ALNYLAM PHARMACEUTICALS, INC. Annual Report, Year Ended Dec 31, 2013

Filed February 20, 2014For Securities:ALNY

Summary

Alnylam Pharmaceuticals, Inc. (ALNY) reported its annual results for the fiscal year ending December 31, 2013. The company is a biopharmaceutical firm focused on developing RNA interference (RNAi) therapeutics, a novel class of medicines targeting genetic diseases. Key to Alnylam's strategy is its "Alnylam 5x15" program, aiming to have 6-7 genetic medicine candidates in clinical development, including at least two in Phase 3, by the end of 2015. The company's most advanced program, patisiran (ALN-TTR02), targets transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR) and has shown positive results in Phase 2 trials with initiation of a Phase 3 APOLLO trial. Significant recent developments include the planned acquisition of Sirna Therapeutics' RNAi assets from Merck and a major global strategic collaboration with Genzyme, a Sanofi company, for the development and commercialization of its genetic medicine pipeline outside of North America and Western Europe. This collaboration, including a $700 million equity investment from Genzyme, significantly strengthens Alnylam's financial position and provides substantial resources for advancing its pipeline. Financially, Alnylam reported net revenues from collaborators of $47.2 million, a decrease from $66.7 million in 2012, primarily due to the completion of certain collaboration obligations. Operating expenses were $140.1 million, a decrease from $196.2 million in 2012, mainly due to the absence of a significant restructuring charge incurred in the prior year, although R&D expenses increased to support pipeline advancement. The company reported a net loss of $89.2 million for 2013, compared to a net loss of $106.0 million in 2012. Alnylam ended the year with a strong cash position of $350.5 million, bolstered by its January 2013 public offering and the anticipated Genzyme investment, providing ample runway to fund its ongoing research and development activities.

Financial Highlights

38 data points
Beta

Net Income

-$89.22M

EPS (Diluted)

$-1.45

Total Assets

$420.53M

Cash & Equivalents

$53.17M

Free Cash Flow

-$72.66M

Financial Statements
Beta
R&D Expenses$112.96M
Operating Expenses$140.11M
Operating Income-$92.94M
Net Income-$89.22M
EPS (Basic)$-1.45
Shares Outstanding (Basic)61.55M

Key Highlights

  • 1Alnylam is advancing its "Alnylam 5x15" strategy, aiming for 6-7 genetic medicine candidates in clinical development by the end of 2015.
  • 2Patisiran (ALN-TTR02) is Alnylam's most advanced product candidate, targeting TTR-mediated amyloidosis, with positive Phase 2 results and the initiation of a Phase 3 trial.
  • 3The company plans to acquire Sirna Therapeutics' RNAi assets from Merck for $25 million in cash and $150 million in stock, enhancing its RNAi platform.
  • 4A significant global strategic collaboration was formed with Genzyme (Sanofi) in January 2014 to develop and commercialize RNAi therapeutics as genetic medicines, which includes a $700 million investment from Genzyme.
  • 5Alnylam reported net revenues from collaborators of $47.2 million for 2013, with a substantial deferred revenue balance of $126.1 million expected to be recognized in future periods.
  • 6The company ended 2013 with a strong cash and marketable securities balance of $350.5 million, providing significant financial resources for R&D and operations.
  • 7Alnylam continues to invest heavily in research and development, with R&D expenses of $113.0 million in 2013, supporting its extensive pipeline.

Frequently Asked Questions

Alnylam's core business strategy is to develop novel therapeutics based on RNA interference (RNAi) technology, focusing on genetic medicines for diseases with high unmet medical needs. Their "Alnylam 5x15" strategy aims to have 6-7 genetic medicine product candidates in clinical development, with at least two in Phase 3, by the end of 2015. Their most advanced program, patisiran (ALN-TTR02), is targeting transthyretin-mediated amyloidosis (ATTR).

In early 2014, Alnylam announced two major developments: the planned acquisition of Sirna Therapeutics' RNAi assets from Merck for $25 million cash and $150 million in stock, and a significant global strategic collaboration with Genzyme (a Sanofi company). This Genzyme collaboration focuses on developing RNAi therapeutics as genetic medicines and includes a $700 million equity investment from Genzyme, bolstering Alnylam's financial resources and advancing its pipeline globally.

Alnylam reported net revenues from collaborators of $47.2 million for 2013, down from $66.7 million in 2012, mainly due to the completion of certain collaboration obligations. Operating expenses were $140.1 million, with a net loss of $89.2 million. However, the company ended 2013 with a strong cash and marketable securities balance of $350.5 million, providing significant financial flexibility for its extensive research and development activities. The company anticipates continued investment in R&D and expects its cash resources, combined with proceeds from recent and anticipated collaborations, to fund operations through the launch of its first commercial product.

Alnylam faces several risks typical of clinical-stage biopharmaceutical companies, including the inherent uncertainties in developing novel RNAi therapeutics, potential challenges in achieving safe and effective drug delivery, reliance on intellectual property, and the highly competitive pharmaceutical landscape. The company also depends heavily on strategic alliances for funding and development capabilities, and any failure to secure or maintain these partnerships could adversely affect its business. Additionally, obtaining regulatory approval for its product candidates is a significant hurdle.