10-KPeriod: FY2018

ALNYLAM PHARMACEUTICALS, INC. Annual Report, Year Ended Dec 31, 2018

Filed February 14, 2019For Securities:ALNY

Summary

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 million 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.

Financial Statements
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Key Highlights

  • 1Launched ONPATTRO® (patisiran) in the U.S. and Europe in 2018, generating $12.5 million in net revenue.
  • 2Ended 2018 with $1.13 billion in cash, cash equivalents, and marketable debt securities, supported by a successful $382 million public offering in January 2019.
  • 3Advanced five late-stage investigational programs, including vutrisiran, givosiran (positive Phase 3 interim results), lumasiran, fitusiran (partnered), and inclisiran (partnered).
  • 4Refined 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.
  • 5Continued 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.
  • 6The 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.

Frequently Asked Questions

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.

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.

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.

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.