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Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2016 Financial Results and Highlights Recent Period Activity
Feb. 8, 2017 4:05 p.m. ET
-- Advanced Industry-Leading RNAi Therapeutics Pipeline with Eight Clinical Programs, Including Three Programs in Late-Stage Development; Discontinued Revusiran Development --

-- Maintained Strong Balance Sheet with $1.1 Billion in Cash and Expects to End 2017 with Greater than $700 Million in Cash --

CAMBRIDGE, Mass.--(BUSINESS WIRE)--February 08, 2017--
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the fourth quarter and full year 2016, and highlighted recent progress in advancing its pipeline.

"We continue to advance a broad pipeline of investigational RNAi therapeutics -- including 8 programs in clinical development -- across a wide range of disease indications with high unmet need. In the fourth quarter of 2016 and recent period, we reported positive clinical results with fitusiran for hemophilia, givosiran for porphyria, and ALN-CC5 for complement-mediated diseases. In addition, our partners at The Medicines Company, reported positive interim clinical data with inclisiran for hypercholesterolemia. On the other hand, we were disappointed to announce the discontinuation of our revusiran program due to safety findings, although our investigation to date indicates the findings are unique to this program and the clinical setting," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "2017 promises to be a pivotal year for Alnylam, with our first Phase 3 data read out -- APOLLO for patisiran -- and, if positive, our first NDA filing. In addition, we plan to advance three additional programs into Phase 3 trials. We believe all of these important milestones put us on track to meet our "Alnylam 2020" goals of becoming a multi-product, commercial-stage company with a deep and sustainable clinical development pipeline by the end of 2020."

Fourth Quarter 2016 and Recent Significant Corporate Highlights

-- Advanced patisiran for the treatment of polyneuropathy due to hereditary
ATTR (hATTR) amyloidosis, with APOLLO Phase 3 data expected in mid-2017.

-- Announced decision to discontinue development of revusiran, an
investigational RNAi therapeutic that was being developed for the
treatment of cardiomyopathy due to hATTR amyloidosis.

-- Advanced fitusiran for the treatment of hemophilia and rare bleeding
disorders (RBD), with positive new data presented at the 2016 American
Society of Hematology (ASH) meeting and additional data presented at the
2017 European Association for Haemophilia and Allied Disorders (EAHAD)

-- Alnylam and partner Sanofi Genzyme announced that Sanofi Genzyme
elected to exercise its right to co-develop and co-promote
fitusiran with Alnylam in the United States, Canada and Western
Europe. Sanofi Genzyme retains commercial rights for rest of

-- Advanced givosiran (ALN-AS1) for the treatment of acute hepatic
porphyrias with positive initial clinical activity results reported at
the 2016 ASH meeting from Phase 1 study in acute intermittent porphyria
(AIP) patients with recurring porphyria attacks.

-- Alnylam and partner Sanofi Genzyme announced that Sanofi Genzyme
has elected not to opt in to develop and commercialize givosiran,
providing Alnylam with full global control of the program for
further development and potential commercialization.

-- Alnylam's partner, The Medicines Company, announced positive results from
the Day 90 interim analysis at the 2016 American Heart Association
meeting and top-line Day 180 results for all 501 patients in the ongoing
ORION-1 Phase 2 study of inclisiran (also known as ALN-PCSsc and PCSK9si),
an investigational RNAi therapeutic for the treatment of

-- The Medicines Company initiated the ORION-2 study of inclisiran in
patients with Homozygous Familial Hypercholesterolemia (HoFH) as
well as the ORION-3 study, a Phase 2 open-label cross-over
extension study for patients completing the ORION-1 study.

-- Reported updated clinical results at the 2016 ASH meeting from Phase 1/2
study of ALN-CC5 in patients with paroxysmal nocturnal hemoglobinuria

-- Alnylam and partner Sanofi Genzyme announced that Sanofi Genzyme
has elected not to opt in to develop and commercialize ALN-CC5,
providing Alnylam with full global control of the program for
further development and potential commercialization.

-- Reported positive initial clinical results from Phase 1 study of
ALN-TTRsc02, an ESC-GalNAc conjugate targeting TTR for hATTR amyloidosis.
Upcoming Events in Early and Mid-2017

-- Alnylam announces today that it plans to present complete 24-month data
and additional analyses from the Phase 2 OLE study of patisiran at the
American Academy of Neurology (AAN) 69th Annual Meeting, being held April
22 -- 28, 2017 in Boston, Massachusetts, in an oral presentation on
Wednesday, April 26 at 8:24 am ET.

-- In addition, The Medicines Company plans to present full safety and
efficacy data from the ORION-1 Phase 2 study of inclisiran, with six- to
nine-month follow-up for all patients in the study, at the American
College of Cardiology's 66th Annual Scientific Session, to be held March
17 -- 19, 2017, in Washington, D.C., in a late-breaker oral presentation
on Friday, March 17, at 1:30 pm ET.

-- Also in early 2017, Alnylam plans to initiate the ATLAS Phase 3 program
for fitusiran.

-- In mid-2017, Alnylam plans to:

-- Report top-line results from the APOLLO Phase 3 study of

-- Present additional data from the Phase 2 OLE study of fitusiran,
likely at the International Society on Thrombosis and Haemostasis
(ISTH) 2017 Congress, being held July 8 -- 13, 2017, in Berlin,

-- Present additional data from Part C of the Phase 1 study of
givosiran, likely at the 2017 International Congress of Porphyrins
and Porphyrias (ICPP), being held June 25 -- 28, 2017, in Bordeaux,
France; and,

-- The Medicines Company plans to initiate the Phase 3 program for
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