10-QPeriod: Q3 FY2019

ALNYLAM PHARMACEUTICALS, INC. Quarterly Report for Q3 Ended Sep 30, 2019

Filed October 31, 2019For Securities:ALNY

Summary

Alnylam Pharmaceuticals, Inc. reported strong growth in product revenues for the third quarter and first nine months of 2019, primarily driven by its first commercial product, ONPATTRO®. Total revenues saw a substantial increase year-over-year. Despite this revenue growth, the company continues to incur significant operating losses, consistent with its business model focused on research and development of novel RNAi therapeutics. The company has a robust pipeline with several late-stage investigational programs, notably givosiran, for which an NDA was accepted by the FDA with Priority Review and an MAA filed with the EMA. Financially, Alnylam bolstered its cash position through a significant public offering and a strategic collaboration with Regeneron, which included a substantial upfront payment and stock purchase. The company's cash reserves remain strong, providing ample runway to fund its ongoing development and commercialization efforts under its Alnylam 2020 strategy. Management believes current resources are sufficient for multiple years, though continued investment in R&D and commercial capabilities is expected.

Financial Statements
Beta

Key Highlights

  • 1ONPATTRO® (patisiran) net product revenues significantly increased to $46.1 million in Q3 2019 and $110.6 million for the first nine months of 2019, a substantial jump from the prior year.
  • 2Total revenues grew by 3,286% in Q3 2019 and 175% year-to-date, reflecting the commercialization of ONPATTRO® and collaboration revenues.
  • 3Givosiran received FDA Priority Review with a PDUFA date of February 4, 2020, and its Marketing Authorisation Application (MAA) was validated by the EMA.
  • 4The company secured significant funding through a $400 million upfront payment and $400 million stock purchase from Regeneron as part of a broad strategic collaboration.
  • 5Alnylam's cash position strengthened, ending Q3 2019 with $925.75 million, boosted by a $381.9 million net proceeds from a public offering and the Regeneron collaboration.
  • 6Research and development expenses increased by 15% for the quarter and 21% year-to-date, reflecting continued investment in pipeline advancement, including clinical trials and license fees.
  • 7Despite revenue growth, the company reported net losses of $208.5 million for Q3 2019 and $609.9 million for the first nine months of 2019, with an accumulated deficit of $3.45 billion, which is typical for a biopharmaceutical company at this stage.

Frequently Asked Questions

ONPATTRO® (patisiran) is Alnylam's first commercial product, approved for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). The company reported $46.1 million in net product revenues for ONPATTRO in the third quarter of 2019, indicating successful global expansion with launches in the U.S., Europe, Japan, and Canada.

The most significant pipeline development is the progress of givosiran for acute hepatic porphyria (AHP). The U.S. FDA accepted its New Drug Application (NDA) for Priority Review with a PDUFA date set for February 4, 2020, and the European Medicines Agency (EMA) has validated its Marketing Authorisation Application (MAA). Other late-stage programs like vutrisiran and lumasiran are also advancing.

Alnylam significantly strengthened its financial position. Key financing activities include a public offering that yielded approximately $381.9 million in net proceeds and a strategic collaboration with Regeneron, providing a $400 million upfront payment and a $400 million stock purchase. This has resulted in a robust cash balance of $925.75 million as of September 30, 2019, which management believes is sufficient to fund operations for multiple years.

Alnylam continues to invest heavily in research and development, which leads to significant operating losses and an accumulated deficit. This is typical for a biopharmaceutical company focused on developing novel therapeutics. While net losses were reported for the quarter and year-to-date, the strong revenue growth from ONPATTRO® and substantial cash reserves provide a strong foundation to fund its 'Alnylam 2020' strategy and future development activities.