Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2017 Financial Results and Highlights Recent Period Activity - Reported Final Results from APOLLO Phase 3 Study and Completed Filings of New Drug Application (

Pharmaceuticals Reports Fourth Quarter and Full Year 2017 Financial

Results and Highlights Recent Period Activity

Reported Final Results from APOLLO Phase 3 Study and Completed Filings

of New Drug Application (NDA) and Marketing Authorisation Application

(MAA) for Patisiran, with an Expected Commercial Launch in U.S. and

Europe in Mid- and Late 2018, Respectively -

Advanced Three Additional RNAi Therapeutics in Phase 3 Development,

Including Givosiran in ENVISION, Inclisiran in ORION-9, -10, and -11,

and Restart of Fitusiran in ATLAS -

Strategically Restructured Rare Disease Alliance with Sanofi to Optimize

Advancement of Patisiran, ALN-TTRsc02, and Fitusiran to Patients Around

Maintained Strong Balance Sheet with $1.7 Billion in Cash and Expects to

End 2018 with Approximately $1.0 Billion in Cash -

CAMBRIDGE, Mass.--(BUSINESS WIRE)--February 8, 2018--Alnylam

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

company, today reported its consolidated financial results for the

fourth quarter and full year 2017 and highlighted recent progress in

advancing its pipeline.

"2017 was a defining inflection point in Alnylam's history, with

positive results from the APOLLO Phase 3 study culminating in regulatory

filings for patisiran, a first-ever milestone for RNAi, with the

potential to bring a whole new class of innovative therapeutics to the

forefront of medicine. With newly acquired rest-of-world rights, we

anticipate making patisiran available to hATTR amyloidosis patients

around the world starting with the U.S. in mid-2018, followed by

European countries in late 2018, and then Japan and other countries

shortly thereafter. To this end, we have initiated a staged build of

global medical and commercial capabilities to expand our reach and

ensure that patients and physicians are educated about this rare disease

and the safety and efficacy profile of patisiran, upon market approval,"

said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "Beyond

patisiran, we advanced our late-stage pipeline with three additional

RNAi therapeutics in Phase 3 development, including givosiran for acute

hepatic porphyrias in our ENVISION Phase 3 study, with topline interim

analysis results expected in mid-2018. Also, together with our partners

Sanofi and The Medicines Company, we restarted fitusiran in the ATLAS

Phase 3 study and advanced inclisiran in the ORION-9, -10, and -11 Phase

3 studies, respectively, with results expected for both programs in

2019. In sum, we believe our efforts position the Company to achieve its Alnylam

2020 goals of building a multi-product, commercial-stage company

with a deep clinical-stage pipeline and robust product engine by the end

of 2020, a profile rarely achieved in biotech history."

Fourth Quarter 2017 and Recent Significant Corporate Highlights

Advanced patisiran, an investigational RNAi therapeutic for the

treatment of patients with hereditary ATTR amyloidosis.

Presented positive, final results from the APOLLO Phase 3 pivotal

Completed the rolling submission of a New Drug Application (NDA)

with the U.S. Food and Drug Administration (FDA) and submitted a

Marketing Authorisation Application (MAA) to the European

Medicines Agency (EMA), with the agencies recently accepting both

Received Breakthrough Therapy Designation and Priority Review

status from the FDA, and expansion of patisiran's Orphan Drug

Designation to the "treatment of transthyretin-mediated

amyloidosis (ATTR amyloidosis)." In addition, patisiran received

Accelerated Assessment status from the EMA, and has been

designated as a Promising Innovative Medicine (PIM) by the

Medicines and Healthcare Products Regulatory Agency (MHRA) in the

In response to the urgent need for treatment of patients living

with hATTR amyloidosis, the Company is fulfilling requests from

treating physicians for early access or compassionate use of

patisiran; the Company announces today that, to date, more than

100 eligible patients have begun treatment with patisiran under

these programs in the U.S. and EU.

Advanced ALN-TTRsc02, a once-quarterly, subcutaneously administered

investigational RNAi therapeutic in development for the treatment of

Reaffirmed guidance to initiate a comprehensive Phase 3 program

for ALN-TTRsc02 in late 2018.

Advanced givosiran, an investigational RNAi therapeutic in development

for the treatment of acute hepatic porphyrias (AHPs), with initiation

of the ENVISION Phase 3 study.

The Company reached alignment with global regulatory authorities

on the design of the ENVISION study, including with the FDA on an

interim analysis based on reduction of urinary aminolevulinic acid

(ALA), a biomarker that the FDA considers reasonably likely to

predict clinical benefit.

The Company announces today that the interim analysis is also

designed to conduct a blinded assessment of the porphyria

attack rate for the purpose of a study sample size adjustment

from approximately 75 patients to up to approximately 94

The Company has guided that it expects interim analysis results in

mid-2018 and, pending FDA review of the program at the time of

interim analysis and assuming positive results, it expects to

submit an NDA at or around year-end 2018.

Advanced fitusiran, an investigational RNAi therapeutic in development

for the treatment of hemophilia A and B with or without inhibitors,

and reached alignment with the FDA on safety measures and a risk

mitigation strategy resulting in a lift of the temporary hold on

Alnylam's partner, The Medicines Company, initiated the ORION-9, -10,

and -11 Phase 3 clinical studies for inclisiran in patients with

heterozygous familial hypercholesterolemia (HeFH), atherosclerotic

cardiovascular disease (ASCVD) or ASCVD-risk equivalents, and

completed enrollment in the approximately 1,500 patient ORION-11 study

Advanced lumasiran (formerly known as ALN-GO1), an investigational

RNAi therapeutic in development for the treatment of primary

hyperoxaluria type 1 (PH1), with new positive data from the Phase 1/2

study presented at the American Society of Nephrology Kidney Week 2017

Announced a strategic restructuring of the Company's rare disease

alliance with Sanofi, originally formed in 2014, with Alnylam

obtaining global rights to its ATTR amyloidosis programs - patisiran

and ALN-TTRsc02 - and Sanofi obtaining global rights to fitusiran.