10-QPeriod: Q1 FY2017

ALNYLAM PHARMACEUTICALS, INC. Quarterly Report for Q1 Ended Mar 31, 2017

Filed May 5, 2017For Securities:ALNY

Summary

This Alnylam Pharmaceuticals, Inc. (ALNY) 10-Q filing for the quarter ended March 31, 2017, highlights the company's continued focus on its novel RNA interference (RNAi) therapeutics. Alnylam is advancing a pipeline across three strategic therapeutic areas: Genetic Medicines, Cardio-Metabolic Diseases, and Hepatic Infectious Diseases, with a stated goal of having three marketed products and ten RNAi therapeutic clinical programs by the end of 2020. The company reported a significant increase in net revenues from collaborators, primarily driven by its partnerships with Sanofi Genzyme and The Medicines Company (MDCO). Financially, Alnylam continues to incur substantial operating losses, consistent with its R&D-intensive business model. While R&D expenses saw a slight decrease year-over-year, general and administrative expenses increased significantly due to investments in commercial and medical affairs infrastructure in preparation for potential product launches. The company maintains a robust cash position but faces ongoing risks associated with drug development, regulatory approvals, competition, and the need for future financing. The most advanced candidate, patisiran, is expected to report Phase 3 data in mid-2017, with potential regulatory filings planned by year-end if results are positive.

Financial Statements
Beta

Key Highlights

  • 1Alnylam reported a substantial increase in net revenues from collaborators, up to $18.96 million from $7.34 million in the prior year's quarter, primarily due to increased activity under agreements with Sanofi Genzyme and MDCO.
  • 2Despite increased collaboration revenues, the company continued to experience significant operating losses, with a net loss of $107.29 million for the quarter, compared to $102.97 million in the same period last year.
  • 3Research and development (R&D) expenses decreased by approximately $9.3 million year-over-year, partly due to lower stock-based compensation and reduced external services, though manufacturing expenses increased for late-stage trials.
  • 4General and administrative (G&A) expenses nearly doubled, increasing by approximately $17.4 million, driven by investments in commercial and medical affairs headcount in anticipation of future product launches.
  • 5Patisiran, the most advanced RNAi therapeutic candidate for hereditary TTR-mediated amyloidosis (hATTR amyloidosis), is expected to report top-line data from its APOLLO Phase 3 study in mid-2017, with planned regulatory submissions by year-end if data are positive.
  • 6The company maintained a strong liquidity position with $171.1 million in cash and cash equivalents at the end of the quarter, though this represents a decrease from the prior year-end.
  • 7Alnylam continues to emphasize its proprietary GalNAc-conjugate delivery platform and its broad intellectual property portfolio, which is considered essential to its business strategy.

Frequently Asked Questions

Alnylam Pharmaceuticals is focused on developing novel therapeutics based on RNA interference (RNAi) technology. Their strategy centers on leveraging their proprietary GalNAc-conjugate platform to deliver small interfering RNAs (siRNAs) to genetically validated, liver-expressed target genes. The company aims to advance its pipeline across three Strategic Therapeutic Areas (STArs) and achieve a profile of three marketed products and ten clinical programs by the end of 2020.

The most advanced product candidate, patisiran, for hATTR amyloidosis, is expected to report top-line data from its Phase 3 APOLLO study in mid-2017. If this data is positive, Alnylam plans to submit its first New Drug Application (NDA) and Marketing Authorization Application (MAA) by the end of 2017. The company also anticipates advancing other candidates, like fitusiran and givosiran, into later stages of development.

In the first quarter of 2017, Alnylam saw a significant increase in net revenues from collaborators, driven by its partnerships. However, the company continues to operate at a loss due to substantial investments in research and development. Operating expenses increased overall, primarily due to a rise in general and administrative costs associated with building commercial capabilities. While the company has a substantial cash reserve, it acknowledges the ongoing need for significant funding to support its long-term strategy.

Alnylam faces several risks, including the inherent uncertainties of drug development in a novel field like RNAi, the possibility of clinical trial failures (as seen with the discontinued revusiran program), reliance on strategic alliances for development and commercialization, obtaining regulatory approvals, intense competition, and the continuous need for substantial funding. The company's success is highly dependent on the successful development and commercialization of its RNAi therapeutics, particularly patisiran.