Full Press Release Details
Pharmaceuticals Reports Third Quarter 2018 Financial Results and
Highlights Recent Period Activity
Obtained FDA and EMA Approvals of ONPATTRO (patisiran) - the First-Ever
RNAi Therapeutic - and Launched in U.S. and EU
Received 125 U.S. Patient Start Forms in First Seven Weeks of ONPATTRO
Reported Positive Topline Interim Analysis Results from ENVISION Phase 3
Study of Givosiran in Patients with Acute Hepatic Porphyrias
Advanced Lumasiran into ILLUMINATE Phase 3 Program
Maintained Strong Balance Sheet with $1.27 Billion in Cash and Expects
to End 2018 with Approximately $1.0 Billion in Cash
CAMBRIDGE, Mass.--(BUSINESS WIRE)--November 7, 2018--Alnylam
Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics
company, today reported its consolidated financial results for the third
quarter 2018 and reviewed recent commercial and R&D highlights.
"The third quarter and recent period were truly revolutionary for
Alnylam with the approval of ONPATTRO in both the U.S. and EU, heralding
the arrival of RNAi therapeutics as a whole new class of medicines. With
these approvals and the subsequent launches, we have begun to realize
the promise of RNAi therapeutics on a global scale," said John
Maraganore, Ph.D., Chief Executive Officer of Alnylam. "Our recent
regulatory and commercial launch experiences will be leveraged for our
entire portfolio including late-stage programs such as givosiran, where
we will initiate a rolling NDA submission this year; lumasiran, which we
have advanced into late-stage development with the recent initiation of
the ILLUMINATE-A Phase 3 study; and ALN-TTRsc02, which will enter Phase
3 later this year in the HELIOS-A study. We believe these
accomplishments put us well on our way toward achieving our Alnylam
2020 goal of building a multi-product, global, commercial-stage
company with a deep and sustainable clinical pipeline by the end of
"With the approval and launch of ONPATTRO, Alnylam is now a global
commercial-stage company. With only seven weeks of results for the third
quarter, we're encouraged by the number of U.S. patient start forms, and
emerging prescriber base, highlighting what we believe is strong demand
for ONPATTRO for adults with polyneuropathy caused by hATTR amyloidosis.
Moreover, we believe our regional presence in North America, Europe,
Asia, and, soon, Latin America, along with established medical affairs
and supply chain capabilities, positions us to expand our efforts in
markets around the world," said Barry Greene, President of Alnylam. "We
look forward to continuing our work toward strong commercial execution,
with a focus on raising disease awareness, improving diagnosis, and
bringing ONPATTRO to patients in need."
Third Quarter 2018 and Recent Period Significant Corporate Highlights
Commercial Highlights
Launched ONPATTRO (patisiran) in the U.S. and EU, initially in
Received 125 U.S. patient Start Forms as of September 30, 2018.
Recognized ONPATTRO revenue of $0.5 million for the quarter ended
Announced alignment on value-based agreements with leading health
insurers and launched Alnylam Assist , a comprehensive patient support
services program for ONPATTRO in the U.S.
Achieved the first-ever regulatory approval of an RNAi therapeutic,
ONPATTRO (patisiran), in the U.S. and EU.
Received U.S. Food and Drug Administration (FDA) approval of
ONPATTRO for the treatment of the polyneuropathy of hereditary
transthyretin-mediated (hATTR) amyloidosis in adults.
Received marketing authorization from the European Commission for
ONPATTRO for the treatment of hATTR amyloidosis in adult patients
with stage 1 or stage 2 polyneuropathy.
Continued global efforts to bring ONPATTRO to patients with
submission of a New Drug Application to Japan's Pharmaceuticals
and Medical Devices Agency and receipt of a Priority Review
designation in Canada.
Published results from the APOLLO Phase 3 study of patisiran in
the July 5, 2018 issue of The New England Journal of Medicine
and APOLLO exploratory cardiac endpoint data in the September 14,
2018 issue of Circulation.
Advanced ALN-TTRsc02, a subcutaneously administered investigational
RNAi therapeutic in development for the treatment of ATTR amyloidosis.
Aligned the design of HELIOS-A, a pivotal Phase 3 study of
ALN-TTRsc02 in patients with hATTR amyloidosis polyneuropathy,
with FDA and European Medicines Agency (EMA) feedback.
The Company is on track to start the HELIOS-A study in late 2018
and plans to initiate additional Phase 3 studies of ALN-TTRsc02,
including in hereditary and wild-type ATTR amyloidosis
cardiomyopathy, in 2019.
Advanced givosiran, an investigational RNAi therapeutic in development
for the treatment of acute hepatic porphyrias (AHPs).
Announced positive topline results from the interim analysis of
the ENVISION Phase 3 study of givosiran.
Announced plans to initiate a rolling submission of a New Drug
Application (NDA) and pursue full approval based on complete
results - now expected in early 2019 - from the ENVISION Phase 3
study. The rolling NDA submission is expected to be initiated in
2018, with full clinical sections submitted in mid-2019, assuming
Advanced lumasiran, an investigational RNAi therapeutic in development
for the treatment of primary hyperoxaluria type 1 (PH1).
Announced initiation of ILLUMINATE-A, a global Phase 3 pivotal
trial of lumasiran in children and adults with PH1. Alnylam
expects to report topline results from ILLUMINATE-A in late 2019
and, if positive, submit filings for global regulatory approvals
starting in early 2020.
Presented updated positive results from the Phase 1/2 study in PH1
patients at the 2018 European Society for Paediatric Nephrology
and the American Society of Nephrology annual meetings.
Announced alignment with the FDA on the trial design for
ILLUMINATE-B, a Phase 3 study of lumasiran in PH1 patients less
than six years of age with preserved renal function.
Expanded the Alnylam Act program to include
no-charge, third-party genetic testing and counseling for adults
and children who may carry a mutation in the gene encoding
alanine-glyoxylate aminotransferase (AGXT), which is