Alnylam Pharmaceuticals Reports Third Quarter 2014 Financial Results and Highlights Recent Period Progress - Reported Six-Month Clinical Data from Phase 2 Open-Label Extension (OLE) Study with Patisiran, Showing Tolerabi

Pharmaceuticals Reports Third Quarter 2014 Financial Results and

Highlights Recent Period Progress

Reported Six-Month Clinical Data from Phase 2 Open-Label Extension (OLE)

Study with Patisiran, Showing Tolerability, Sustained Transthyretin

(TTR) Knockdown, and Promising Initial Evidence for Stabilization of

Neuropathy Progression -

Completed Phase 2 Enrollment and Initiated Phase 2 OLE Study with

Revusiran (ALN-TTRsc), and Successfully Completed Non-Clinical Chronic

Toxicology Studies to Enable Start of Phase 3 Trial -

Continued Enrollment in Phase 1 Hemophilia Clinical Trial with ALN-AT3 -

Filed Clinical Trial Application (CTA) for ALN-PCSsc, Now Approved -

Advanced Additional Development Programs, Including ALN-CC5 for

Complement-Mediated Diseases -

Maintained Strong Balance Sheet with $915 Million in Cash and Increases

Guidance to End 2014 with Greater than $860 Million in Cash -

CAMBRIDGE, Mass.--(BUSINESS WIRE)--November 5, 2014--Alnylam

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

company, today reported its consolidated financial results for the third

quarter 2014, and highlighted recent progress in advancing its pipeline.

"During the third quarter and recent period we continued advancing RNAi

therapeutics through the clinic and toward the market. Notably, we

reported six-month clinical data from our Phase 2 open-label extension

study with patisiran, showing what we believe to be promising initial

results. Indeed, with the important caveat that this is an open-label

study in a small number of patients, we are encouraged by what we

believe to be evidence for possible stabilization of neuropathy

progression after the first six months of treatment. We believe these

data will be increasingly meaningful as we monitor neuropathy

progression in patisiran-treated patients over time. In the meantime, we

are pleased with the pace of enrollment in our Phase 3 APOLLO trial of

patisiran in patients with polyneuropathy, where we aim to obtain

definitive evidence for patisiran safety and efficacy in a double-blind,

placebo-controlled randomized trial," said John Maraganore, Ph.D., Chief

Executive Officer of Alnylam. "Earlier today, we announced that we have

completed enrollment in our Phase 2 study and initiated our Phase 2 OLE

study with revusiran (ALN-TTRsc) in patients with TTR cardiac

amyloidosis. We remain on track to initiate our Phase 3 trial with

revusiran later this year, and have completed favorable discussions with

U.S. and EU regulatory authorities. Enrollment also continues in our

Phase 1 trial with ALN-AT3 for the treatment of hemophilia and rare

bleeding disorders, and we look forward to sharing our early clinical

data, including preliminary initial results in people with hemophilia,

at ASH in December. In addition, we recently filed our CTA for ALN-PCSsc

for the treatment of hypercholesterolemia, and we expect to initiate

dosing in a Phase 1 trial later this year or early next, with initial

results expected in mid-2015. In aggregate, these latest advancements

from our clinical pipeline highlight what we believe to be the

significant potential for RNAi therapeutics as a new class of genetic

"In addition to these advancements on the clinical front, we have made

strong progress with our pre-clinical programs. First, we presented new

data with ALN-CC5, an investigational RNAi therapeutic in development

for the treatment of complement-mediated diseases, showing potent,

clamped knockdown of serum C5 in non-human primate studies, as well as

promising results in pre-clinical disease models. We remain on track to

file a CTA for ALN-CC5 by the end of this year, with initial clinical

results expected in mid-2015. We also made excellent progress in other

development stage programs, including ALN-AS1 for hepatic porphyrias,

ALN-AAT for liver disease associated with alpha-1 antitrypsin

deficiency, and ALN-HBV for hepatitis B virus infection," said Barry

Greene, President & Chief Operating Officer of Alnylam. "In addition to

these pipeline advancements, we've had a very productive quarter with

regard to our intellectual property estate, specifically with our

Manoharan and Tuschl patent families. We believe these and other

Alnylam-held patents are essential elements of our strategy to bring

important new medicines to patients and build value for our

Cash, Cash Equivalents and Total Marketable Securities

At September 30, 2014, Alnylam had cash, cash equivalents and total

marketable securities of $915.2 million, as compared to $350.5 million

at December 31, 2013.

The net loss according to accounting principles generally accepted in

the U.S. (GAAP) for the third quarter of 2014 was $44.0 million, or

$0.58 per share on both a basic and diluted basis (including $6.4

million, or $0.08 per share of non-cash stock-based compensation

expense), as compared to a net loss of $29.7 million, or $0.48 per share

on both a basic and diluted basis (including $8.8 million, or $0.14 per

share of non-cash stock-based compensation expense), for the same period

in the previous year.

Revenues were $11.0 million for the third quarter of 2014, as compared

to $9.0 million for the same period in the previous year. Revenues for

the third quarter of 2014 included $5.5 million of revenues from the

company's alliance with Takeda Pharmaceuticals Company Limited, $3.4

million of revenues related to the company's collaboration with

Monsanto, and $2.1 million for the company's alliance with The Medicines

Company, research reagent licenses, and other sources.

Research and Development Expenses

Research and development (R&D) expenses were $46.3 million in the third

quarter of 2014, which included $3.8 million of non-cash stock-based

compensation, as compared to $34.5 million in the third quarter of 2013,

which included $6.8 million of non-cash stock-based compensation. The

increase in R&D expenses in the third quarter of 2014 as compared to the

third quarter of 2013 was due primarily to the significant advancement

of the company's clinical and pre-clinical programs. The company expects

that R&D expenses will increase slightly for the fourth quarter of 2014

as compared to the third quarter of 2014 as certain of its programs move

into late clinical stages.

General and Administrative Expenses

General and administrative (G&A) expenses were $9.9 million in the third

quarter of 2014, which included $2.6 million of non-cash stock-based

compensation, as compared to $6.8 million in the third quarter of 2013,

which included $2.0 million of non-cash stock-based compensation. The