Full Press Release Details
Pharmaceuticals Reports First Quarter 2017 Financial Results and
Highlights Recent Period Activity
Advanced Industry-Leading RNAi Therapeutics Pipeline with Eight Clinical
Programs, Including Three Programs in Late-Stage Development -
Maintained Strong Balance Sheet with $962 Million in Cash and Plans to
End 2017 with Greater than $700 Million in Cash -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 5, 2017--Alnylam Pharmaceuticals,
Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today
reported its consolidated financial results for the first quarter 2017,
and highlighted recent progress in advancing its pipeline.
"2017 promises to be a pivotal year for Alnylam. With our patisiran
program, we look forward to the read-out of our APOLLO Phase 3 study
and, if the data are positive, our first NDA filing. In addition, we
expect to advance three additional programs into Phase 3 trials:
fitusiran, givosiran, and - with our partners at The Medicines Company -
inclisiran," said John Maraganore, Ph.D., Chief Executive Officer of
Alnylam. "In the first quarter and recent period, we made great progress
with each of these programs, with positive clinical data reported from
patisiran, fitusiran, and inclisiran, and achievement of PRIME
designation for givosiran. As we look towards the rest of 2017, we
believe our upcoming milestones put us on track to meet our Alnylam
2020' goals of becoming a multi-product, commercial-stage company with a
deep and sustainable clinical development pipeline by the end of 2020."
First Quarter 2017 and Recent Significant Corporate Highlights
Advanced patisiran, an investigational RNAi therapeutic for the
treatment of polyneuropathy due to hereditary ATTR (hATTR)
amyloidosis, with final 24-month data from the Phase 2 open-label
extension (OLE) study presented at the American Academy of Neurology
meeting and top-line results from the APOLLO Phase 3 study expected in
Advanced fitusiran, an investigational RNAi therapeutic for the
treatment of hemophilia and rare bleeding disorders, with positive new
data presented at the 2017 European Association for Haemophilia and
Allied Disorders (EAHAD) meeting.
Advanced givosiran (ALN-AS1), an investigational RNAi therapeutic for
the treatment of the acute hepatic porphyrias, with acceptance into
the European Medicines Agency's PRIME program.
Alnylam's partner, The Medicines Company, announced positive final
results from the ORION-1 Phase 2 study of inclisiran, an
investigational RNAi therapeutic for the treatment of
hypercholesterolemia, at the American College of Cardiology's 66th
Annual Scientific Session.
The Medicines Company initiated the ORION-2 study of inclisiran in
patients with Homozygous Familial Hypercholesterolemia (HoFH) as
well as the ORION-3 study, a Phase 2 open-label cross-over
extension study for patients completing the ORION-1 study.
In addition, The Medicines Company and Alnylam announced agreement
with the FDA on the Phase 3 clinical program for inclisiran.
Upcoming Events in Early and Mid-20171
Alnylam announces today that it plans to host its 4th
Annual RNAi Roundtable Series during the summer. This series will
consist of webinars designed to inform attendees of the latest
progress and upcoming milestones for many of the company's
investigational RNAi therapeutic programs. More details for the series
The Company also announces today that it plans to present additional
clinical data from the Phase 1 study of givosiran at the 2017
International Congress of Porphyrins and Porphyrias (ICPP), being held
June 25 - 28, 2017, in Bordeaux, France, in an oral presentation on
Monday, June 26 at 11:45am Central European Time (5:45 am ET).
In addition, the Company announces that it plans to present additional
data from the Phase 2 OLE study of fitusiran at the International
Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, being held
July 8 - 13, 2017, in Berlin, Germany, in an oral presentation on
Monday, July 10 at 2:45 pm Central European Time (8:45 am ET).
In early 2017, Alnylam plans to initiate the ATLAS Phase 3 program for
In mid-2017, Alnylam plans to report top-line results from the APOLLO
Phase 3 study of patisiran.
Also in mid-2017, The Medicines Company plans to initiate a Phase 3
study of inclisiran in patients with atherosclerotic cardiovascular
"Alnylam continues to maintain a strong balance sheet, ending the first
quarter of 2017 with approximately $962.2 million in cash, including
restricted investments," said Michael Mason, Vice President, Finance and
Treasurer. "Our financial strength allows us to continue to invest in a
broad pipeline of investigational RNAi therapeutics, aligned with
achievement of our Alnylam 2020' goals and strategy. As for 2017
guidance, we remain on track to end 2017 with greater than $700 million
in cash, including $150.0 million in restricted investments."
Cash and Investments
At March 31, 2017, Alnylam had cash, cash
equivalents and fixed income marketable securities, and restricted
investments of $962.2 million, as compared to $1.09 billion at December
The net loss according to accounting principles
generally accepted in the U.S. (GAAP) for the first quarter of 2017 was
$107.3 million, or $1.25 per share on both a basic and diluted basis
(including $15.7 million, or $0.18 per share of non-cash stock-based
compensation expense), as compared to a net loss of $103.0 million, or
$1.21 per share on both a basic and diluted basis (including $23.5
million, or $0.28 per share of non-cash stock-based compensation
expense), for the same period in the previous year.
Revenues were $19.0 million in the first quarter of
2017, as compared to $7.3 million in the first quarter of 2016. Revenues
for the first quarter of 2017 included $12.3 million from the company's
alliance with Sanofi Genzyme, $6.4 million from the company's alliance
with The Medicines Company, and $0.3 million from other sources. The
increase in revenues in the quarter ended March 31, 2017 as compared to
the prior year period was due primarily to higher revenue from the
company's alliance with Sanofi Genzyme.
Research and Development Expenses
Research and development
(R&D) expenses were $87.0 million in the first quarter of 2017, which
included $8.7 million of non-cash stock-based compensation, as compared