News Press Releases

Alnylam to Report New Clinical Results with Fitusiran at the World Federation of Hemophilia (WFH) 2016 World Congress

ALNY | July 25, 2016

Alnylam to Report New Clinical Results with Fitusiran at the World Federation of Hemophilia (WFH) 2016 World Congress

– New Phase 1 Results to Include Initial Data in Hemophilia A and B Patients with Inhibitors –

– Company to Host Conference Call on Wednesday, July 27, 2016, at 1:00 p.m. ET to Discuss Results –

Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, announced today that Alnylam and collaborators will present interim results from the ongoing Phase 1 clinical trial with fitusiran, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia A and B and rare bleeding disorders (RBD) at the World Federation of Hemophilia (WFH) 2016 World Congress being held July 24 – 28, 2016 in Orlando, Florida.

The data will be presented in an oral presentation titled “A Subcutaneously Administered Investigational RNAi Therapeutic, Fitusiran (ALN- AT3), Targeting Antithrombin for Treatment of Hemophilia: Interim Results in Patients with Hemophilia A or B” by lead author John Pasi, MB, ChB, Ph.D., FRCP, FRCPath, FRCPCH, Professor of Haemostasis and Thrombosis and Consultant Haemotologist at Barts Health NHS Trust. The presentation will take place on Wednesday, July 27, 2016, at 11:30 a.m. ET, in Room 206.

Alnylam will host a webcast conference call on Wednesday, July 27, 2016 at 1:00 p.m. ET to discuss these results. A slide presentation will also be available on the Investors page of the company's website, www.alnylam.com, to accompany the conference call. To access the call, please dial 877-312-7507 (domestic) or 631-813-4828 (international) five minutes prior to the start time and refer to conference ID 51834298. A replay of the call will be available beginning at 4:00 p.m. ET on July 27, 2016. To access the replay, please dial 855-859-2056 (domestic) or 404-537-3406 (international), and refer to conference ID 51834298. In addition, an archived webcast will be available on the Alnylam website approximately two hours after the event.

Finally, Alnylam will also host an investor reception to discuss the new fitusiran clinical results on Wednesday, July 27, 2016 from 5:30 – 6:30 p.m. ET at the Rosen Plaza Hotel in Orlando.

About Alnylam Pharmaceuticals

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines. Alnylam’s pipeline of investigational RNAi therapeutics is focused in 3 Strategic Therapeutic Areas (STArs): Genetic Medicines, with a broad pipeline of RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics toward genetically validated, liver-expressed disease targets for unmet needs in cardiovascular and metabolic diseases; and Hepatic Infectious Disease, with a pipeline of RNAi therapeutics that address the major global health challenges of hepatic infectious diseases. In early 2015, Alnylam launched its “Alnylam 2020” guidance for the advancement and commercialization of RNAi therapeutics as a whole new class of innovative medicines. Specifically, by the end of 2020, Alnylam expects to achieve a company profile with 3 marketed products, 10 RNAi therapeutic clinical programs – including 4 in late stages of development – across its 3 STArs. The company’s demonstrated commitment to RNAi therapeutics has enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen, Roche, Takeda, Kyowa Hakko Kirin, Cubist, GlaxoSmithKline, Ascletis, Monsanto, The Medicines Company, and Sanofi Genzyme. In addition, Alnylam holds an equity position in Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 200 peer-reviewed papers, including many in the world’s top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, Cell, New England Journal of Medicine, and The Lancet. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information about Alnylam’s pipeline of investigational RNAi therapeutics, please visit www.alnylam.com.