Alnylam Pharmaceuticals Reports Third Quarter 2016 Financial Results and Highlights Recent Period Activity - Advanced RNAi Therapeutics Pipeline with Eight Active Clinical Programs and Multiple Clinical Data Presentation

Pharmaceuticals Reports Third Quarter 2016 Financial Results and

Highlights Recent Period Activity

Advanced RNAi Therapeutics Pipeline with Eight Active Clinical Programs

and Multiple Clinical Data Presentations -

Discontinued Revusiran Development, Focusing on Patisiran and ESC-GalNAc

Conjugate Programs -

Company Announces Initiation of Expanded Access Program with Patisiran

for Patients with Hereditary ATTR Amyloidosis with Polyneuropathy -

Maintained Strong Balance Sheet with $1.19 Billion in Cash and Remains

On Track to End 2016 with Greater than $1.0 Billion in Cash -

CAMBRIDGE, Mass.--(BUSINESS WIRE)--November 2, 2016--Alnylam

Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics

company, today reported its consolidated financial results for the third

quarter 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. Despite a

disappointing outcome in our revusiran program, we remain committed to

serving the needs of the ATTR amyloidosis community with patisiran and

to developing RNAi therapeutics with our ESC-GalNAc conjugate platform

as a new class of innovative medicines. Based on a recent recommendation

from the APOLLO Data Monitoring Committee for patisiran and a

comprehensive review of data from over 800 human subjects treated with

RNAi therapeutics - excluding revusiran - for up to nearly three years,

we are encouraged by the overall safety profile for our platform," said

John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "We're

pleased with new positive clinical results we presented in the third

quarter for patisiran for hATTR-PN, fitusiran for hemophilia, ALN-AS1

for porphyria, and ALN-GO1 for primary hyperoxaluria. Now and through

the end of the year, we are anticipating a very data rich period -

including key clinical data presentations for five distinct programs at

the AHA and ASH meetings and at our R&D Day in December - and we look

forward to sharing our progress."

Third Quarter 2016 and Recent Significant Corporate Highlights

Advanced patisiran for the treatment of hereditary ATTR amyloidosis

with polyneuropathy (hATTR-PN), also known as familial amyloidotic

polyneuropathy (FAP).

The Company announced today that it has initiated its patisiran

Expanded Access Program (EAP) for patients with hATTR-PN. The

patisiran EAP provides an opportunity for eligible hATTR-PN

patients, who are unable to participate in an ongoing clinical

trial or whose treatment options are otherwise limited, to receive

patisiran therapy until the drug becomes commercially available,

should it receive approval.

Announced that the patisiran APOLLO Data Monitoring Committee

(DMC) met at the Company's request following its decision to

discontinue development of revusiran, and recommended continuation

of the APOLLO Phase 3 trial without modification.

Reported positive initial 24-month data from ongoing Phase 2

open-label extension (OLE) study with patisiran for the treatment

of hATTR-PN. Results showed that patisiran can potentially halt or

improve neuropathy progression. Patisiran administration was also

found to be generally well tolerated in hATTR-PN patients out to

25 months, with no drug-related serious adverse events (SAEs)

reported through the data transfer date.

Announced decision to discontinue development of revusiran, an

investigational RNAi therapeutic that was being developed for the

treatment of hereditary ATTR amyloidosis with cardiomyopathy

The decision was made following the recommendation by the DMC for

the ENDEAVOUR Phase 3 trial of revusiran to suspend dosing and the

observation of an imbalance in mortality in revusiran-treated

patients as compared to those on placebo.

The Company has initiated a comprehensive evaluation of revusiran

The decision to discontinue development of revusiran does not

affect patisiran or any other Alnylam investigational RNAi

therapeutic program in development.

Advanced fitusiran for the treatment of hemophilia and rare bleeding

Reported positive interim clinical results from Phase 1 study of

fitusiran. Fitusiran achieved a median estimated annualized

bleeding rate (ABR) of zero in hemophilia patients without

inhibitors. In the initial low-dose cohort of patients with

inhibitors, fitusiran achieved antithrombin lowering, increased

thrombin generation, and preliminary evidence for reduced

bleeding. Fitusiran administration was generally well tolerated in

patients with and without inhibitors through the data transfer

Continued dosing hemophilia patients in the ongoing Phase 1/2 OLE

study, with up to 16 months of dosing.

In July, the Company also updated its guidance for Phase 3

initiation, and currently remains on track to start studies in

Reported positive interim clinical results from Phase 1 study of

ALN-AS1 for the treatment of acute hepatic porphyrias.

In asymptomatic high excreter (ASHE) porphyria subjects, single

and multiple doses of ALN-AS1 achieved rapid, dose-dependent, and

durable lowering of aminolevulinic acid (ALA) and porphobilinogen

(PBG) - the toxic heme synthesis intermediates that mediate

porphyria attacks - of up to 95%, with effects sustained for over

ten months after a single dose. ALN-AS1 was generally well

tolerated following single and multiple doses through the data

In addition, both the United States Food and Drug Administration

(FDA) and the European Medicines Agency (EMA) granted orphan drug

designation to ALN-AS1 for the treatment of acute hepatic

Reported positive initial clinical results from Phase 1/2 study of

ALN-GO1 for the treatment of primary hyperoxaluria type 1 (PH1).

ALN-GO1 achieved human proof of concept with statistically

significant increases in glycolate, a biomarker of effective

glycolate oxidase knockdown, in healthy adult volunteers. Single

doses of ALN-GO1 were found to be generally well tolerated through

the data transfer date.

In addition, the Company published pre-clinical data with ALN-GO1

in the Journal of the American Society of Nephrology (Liebow et