Alnylam Pharmaceuticals Reports First Quarter 2019 Financial Results and Highlights Recent Period Activity Achieved First Quarter 2019 ONPATTRO Global Net Product Revenues of $26.3 Million with Over 400 Patients on Comme

Pharmaceuticals Reports First Quarter 2019 Financial Results and

Highlights Recent Period Activity

Achieved First Quarter 2019 ONPATTRO

Global Net Product Revenues of $26.3 Million with Over 400 Patients on

Commercial Product Worldwide -

Achieved Positive Results in ENVISION Phase 3 Study of Givosiran -

Formed Broad Collaboration with Regeneron to Discover, Develop and

Commercialize RNAi Therapeutics Focused on Ocular and Central Nervous

System (CNS) Diseases -

Strengthened Balance Sheet with New Equity-Based Capital and

Partnership-Based Funding -

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 1, 2019--Alnylam Pharmaceuticals,

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

reported its consolidated financial results for the first quarter 2019

and reviewed recent business highlights.

"The first quarter of 2019 and recent weeks were a remarkable period of

advancement for Alnylam toward our goal of building a leading

biopharmaceutical company. Specifically, we demonstrated good progress

in global commercialization of ONPATTRO, continued success in our R&D

efforts with positive Phase 3 results for givosiran, and a strong

commitment to future pipeline growth through our landmark ocular and CNS

disease alliance with Regeneron. In addition, we significantly

strengthened our balance sheet through both an equity offering in

January and partnership-based equity funding and cash that we'll receive

upon closing of the Regeneron transaction," said John Maraganore, Ph.D.,

Chief Executive Officer of Alnylam. "We believe we are now well

positioned to achieve our Alnylam 2020 goal of building a

multi-product, global biopharma company with a deep clinical pipeline

for future growth and a robust product engine for sustainable

innovation, a profile rarely achieved in our industry. Moreover, as

evidenced by the success of our ONPATTRO launch and the results of our

givosiran Phase 3 study in porphyria, we believe we're delivering on our

most important goal of bringing potentially transformative medicines to

"In the first quarter, we made significant progress in our global

commercialization of ONPATTRO, as we advanced our efforts to help

patients secure access to this important new medicine. With over 400

patients on commercial product, we're very pleased with our team's

launch execution in over 10 countries where ONPATTRO is now available,

and we look forward to continued growth in all existing and many new

countries in the months to come. Of note, we saw strong new patient

demand in the U.S. and EU in the first quarter, beyond the initial

stages of our launch that had been largely driven by conversion of

patients from our expanded access program as well as patients known to

clinical study sites," said Barry Greene, President of Alnylam. "As we

reported last night, we believe an important highlight in our

commercialization efforts is the innovation we've demonstrated with

regard to patient access. Indeed, with 10 value-based agreements now

completed in the U.S. and with favorable HTA ratings and reimbursement

outcomes in key EU countries, we're advancing our goal to ensure that

ONPATTRO reaches all patients in need."

First Quarter 2019 and Recent Significant Corporate Highlights

Commercial Performance in First Quarter 2019

Achieved global net product revenues for the first quarter of 2019 of

$26.3 million for ONPATTRO.

Attained over 400 patients worldwide on commercial ONPATTRO treatment

since launch as of March 31, 2019.

Received 77 Start Forms in the U.S. in the first quarter, with over 90

percent from newly identified patients not previously treated in the

ONPATTRO Expanded Access Program.

Start Forms in the first quarter came from a diverse range of

prescribing physician specialties, including 55 percent from

cardiologists and 35 percent from neurologists, with 65 percent of

patients covered by Medicare.

In addition, the Company reports continued strength in the number

of U.S. patients receiving ONPATTRO from channels outside of its

Alnylam Assist patient hub where the Company does not receive

Reported continued progress with value-based agreements (VBAs) with

commercial payers in the U.S. and with market access efforts globally.

Since launch, Alnylam has achieved access to ONPATTRO, if

prescribed, for greater than 90 percent of U.S. lives across

commercial, Medicare, Medicaid, and other government payer

categories. In addition, Alnylam has completed definitive VBAs

with 10 commercial U.S. payers to date.

In the EU, Alnylam reported favorable Health Technology Assessment

(HTA) ratings from health authorities in several countries,

including in France ("ASMR III"), Germany ("considerable

benefit"), Italy ("innovation status"), and the Netherlands where

a first-of-its-kind VBA was reached with all health insurers to

reimburse ONPATTRO. The Company remains on track with pricing and

reimbursement procedures in nearly all EU markets, and encouraging

discussions with authorities have progressed significantly over

Advanced patisiran (the non-branded name for ONPATTRO), an

intravenously administered investigational RNAi therapeutic in

development for the treatment of ATTR amyloidosis.

Obtained alignment with the U.S. Food and Drug Administration

(FDA) on the design of APOLLO-B, a randomized, double-blind,

placebo-controlled Phase 3 study of patisiran in hereditary and

wild-type ATTR amyloidosis patients with cardiomyopathy, with the

goal of starting the trial in mid-2019.

Advanced vutrisiran (ALN-TTRsc02), a subcutaneously administered

investigational RNAi therapeutic in development for the treatment of

Continued enrollment in the HELIOS-A Phase 3 study of vutrisiran

in hereditary ATTR amyloidosis patients with plans to initiate

additional Phase 3 studies, including in hereditary and wild-type

ATTR amyloidosis cardiomyopathy, in late 2019.

Advanced givosiran, an investigational RNAi therapeutic in development

for the treatment of acute hepatic porphyria (AHP).

Achieved positive results in the ENVISION Phase 3 study and

presented data at the European Association for the Study of the

Liver (EASL) 54th Annual International Liver Congress.