8-KOther EventsExhibits & Filings

ALNYLAM PHARMACEUTICALS, INC. 8-K Report, Corporate Update (Sep 7, 2017)

Filed September 7, 2017For Securities:ALNY

Summary

Alnylam Pharmaceuticals, Inc. (ALNY) announced a significant update on two of its key investigational RNAi therapeutic programs, fitusiran and givosiran, via an 8-K filing on September 7, 2017. The most critical development concerns fitusiran, a treatment for hemophilia A and B. The company reported a fatal thrombotic event in a patient participating in the Phase 2 open-label extension study of fitusiran. Consequently, Alnylam has proactively suspended dosing in all ongoing fitusiran studies to thoroughly investigate this serious safety event and to develop a strategy for risk mitigation. In parallel, Alnylam reported positive progress with givosiran, its RNAi therapeutic targeting acute hepatic porphyrias. The company has achieved alignment with the U.S. Food and Drug Administration (FDA) on the design for a Phase 3 study for givosiran. While the fitusiran news presents a substantial setback and uncertainty, the advancement of givosiran into Phase 3 development offers a counterpoint of progress in the company's pipeline.

Key Highlights

  • 1Fatal thrombotic event reported in a patient in the fitusiran Phase 2 open-label extension study.
  • 2Alnylam has suspended all ongoing fitusiran studies pending further review and risk mitigation strategy development.
  • 3Fitusiran is an investigational RNAi therapeutic for the treatment of hemophilia A and B.
  • 4Company has reached agreement with the FDA on the Phase 3 study design for givosiran.
  • 5Givosiran is an investigational RNAi therapeutic for acute hepatic porphyrias.
  • 6This filing was made on September 7, 2017.

Frequently Asked Questions

The primary concern is the report of a fatal thrombotic event in a patient receiving fitusiran, an investigational RNAi therapeutic for hemophilia. This has led to the suspension of all ongoing fitusiran studies.

The givosiran program, targeting acute hepatic porphyrias, is progressing. Alnylam has reached agreement with the FDA on the design for its Phase 3 study.

The fatal thrombotic event and subsequent suspension of fitusiran studies introduce significant uncertainty and potential delays for this program, which was a key part of Alnylam's pipeline. Investors should monitor the company's investigation and risk mitigation efforts closely.

This filing primarily serves to report the critical updates on the fitusiran and givosiran programs. The information is presented through a press release dated September 7, 2017.