- Advanced Industry-Leading RNAi Therapeutics Pipeline, Including Four Programs in Late-Stage Development -
- On Track to Report APOLLO Phase 3 Top-Line Results for Patisiran in Near Term, and Initiated ATLAS Phase 3 Program for Fitusiran -
- Maintained Strong Balance Sheet with $1.25 Billion in Cash and Plans to End 2017 with Greater than $1.0 Billion in Cash -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the second quarter 2017, and highlighted recent progress in advancing its pipeline.
“2017 is shaping up to be a pivotal year for Alnylam. With patisiran, we expect to report top-line APOLLO Phase 3 study results in the coming weeks. If positive, these study findings will support our first NDA filing, planned by year-end, and our commercial transition in mid-2018 assuming regulatory approval. In parallel, we continue to advance our late-stage pipeline of investigational RNAi therapeutics, with the recently announced initiation of our ATLAS Phase 3 program for fitusiran in hemophilia and expected Phase 3 starts by year-end for givosiran in acute hepatic porphyrias and, with The Medicines Company, inclisiran in hypercholesterolemia,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “We believe that these are all important and defining milestones that position us to fulfill our ‘Alnylam 2020’ vision of becoming a multi-product, commercial-stage company with a deep and sustainable clinical development pipeline by the end of 2020.”
Second Quarter 2017 and Recent Significant Corporate Highlights
- Advanced patisiran, an investigational RNAi therapeutic for the treatment of patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy, 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 APOLLO Phase 3 data expected in mid-2017.
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Advanced fitusiran, an investigational RNAi therapeutic for the
treatment of hemophilia and rare bleeding disorders, with positive new data from the Phase 2 OLE study presented at the International
Society on Thrombosis and Haemostasis 2017 Congress.
- Results from the Phase 1 study were published in The New England Journal of Medicine in a paper titled, “Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy.”
- Alnylam and partner Sanofi Genzyme announced the initiation of the ATLAS Phase 3 program, a global, multicenter clinical program designed to evaluate the safety and efficacy of fitusiran in patients with hemophilia A and B with or without inhibitors.
-
Advanced givosiran, an investigational RNAi therapeutic for the
treatment of acute hepatic porphyrias (AHPs), with positive new data presented at the 2017 International Congress on
Porphyrins and Porphyrias (ICPP) from the ongoing randomized,
double-blind, placebo-controlled Phase 1 study in recurrent attack
porphyria patients, as well as positive initial results from the
ongoing Phase 1 OLE study.
- In addition, received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for givosiran for the prophylaxis of attacks in patients with acute hepatic porphyria.
- Alnylam and partner The Medicines Company announced agreement with the FDA on a Phase 3 clinical program for inclisiran, an investigational RNAi therapeutic for the treatment of hypercholesterolemia, with LDL-C lowering as the primary endpoint for the initial pivotal trial program, which is expected to initiate in late 2017.
- Completed successful public offering of common stock, with concurrent private placement from Sanofi Genzyme, totaling $376.5 million in net proceeds.
Upcoming Events
- Alnylam will continue to host its 4th Annual RNAi Roundtable Series, which kicked off last week. This series consists 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 can be found here.
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Alnylam plans to report top-line results from the patisiran APOLLO
Phase 3 study in mid-2017. Full results are expected to be presented
in late 2017 at the 1st European ATTR Amyloidosis Meeting
for Patients and Doctors, being held November 2-3, 2017 in Paris,
France.
- If the APOLLO Phase 3 data are positive, Alnylam expects to file its first New Drug Application (NDA) in late 2017 and Marketing Authorisation Application (MAA) shortly thereafter.
- Alnylam plans to initiate a Phase 3 study of givosiran in late 2017, pending successful alignment on trial design with global regulatory authorities.
- The Medicines Company has announced its intention to initiate a Phase 3 study of inclisiran in patients with atherosclerotic cardiovascular disease (ASCVD) in late 2017.
Financial results for the quarter ended June 30, 2017
“Alnylam continues to maintain a strong balance sheet,” said Manmeet Soni, Chief Financial Officer of Alnylam. “Our financial strength allows us to continue to invest in a broad pipeline of investigational RNAi therapeutics and prepare to transition towards a commercial-stage company, aligned with our ‘Alnylam 2020’ goals and strategy.”
Cash and Investments
At June 30, 2017, Alnylam had cash,
cash equivalents and fixed income marketable securities, and restricted
investments of $1.25 billion, as compared to $1.09 billion at December
31, 2016.
In May 2017, Alnylam sold an aggregate of 5,000,000 shares of its common stock through an underwritten public offering at a price to the public of $71.87 per share. As a result of the offering, Alnylam received aggregate net proceeds of $355.2 million.
In addition, Sanofi Genzyme exercised its right to purchase, in a concurrent private placement, 297,501 shares of common stock, at the public offering price of $71.87 per share, resulting in proceeds to Alnylam of $21.4 million.
GAAP and Non-GAAP Net Loss
The net loss according to
accounting principles generally accepted in the U.S. (GAAP) for the
second quarter of 2017 was $118.4 million, or $1.34 per share on both a
basic and diluted basis, as compared to a net loss of $90.1 million, or
$1.05 per share on both a basic and diluted basis, for the same period
in the previous year.
The non-GAAP net loss for the second quarter of 2017 was $94.4 million, or $1.07 per share on both a basic and diluted basis, as compared to a non-GAAP net loss of $74.3 million, or $0.87 per share on both a basic and diluted basis for the same period in the previous year.
The non-GAAP net loss excludes stock-based compensation expense. See “Use of Non-GAAP Financial Measures” below for a description of non-GAAP financial measures and a reconciliation between GAAP and non-GAAP net loss appearing later in this press release.
Revenues
Revenues were $15.9 million in the second quarter
of 2017, as compared to $8.7 million in the second quarter of 2016.
Revenues for the second quarter of 2017 included $14.4 million from the
company’s alliance with Sanofi Genzyme and $1.5 million from the
company’s alliance with The Medicines Company.
Research and Development Expenses
Research and development
(R&D) expenses were $90.6 million in the second quarter of 2017, which
included $13.3 million of stock-based compensation, as compared to $83.2
million in the second quarter of 2016, which included $9.3 million of
stock-based compensation.
General and Administrative Expenses
General and
administrative (G&A) expenses were $45.8 million in the second quarter
of 2017, which included $10.8 million of stock-based compensation, as
compared to $18.0 million in the second quarter of 2016, which included
$6.5 million of stock-based compensation.
Financial Guidance
Alnylam remains on track to end 2017 with greater than $1.0 billion in cash, cash equivalents and fixed marketable securities including $150.0 million in restricted investments.
Conference Call Information
Management will provide an
update on the company and discuss second quarter 2017 results as well as
expectations for the future via conference call on Wednesday, August 9,
2017 at 4:30 p.m. ET. To access the call, please dial 877-312-7507
(domestic) or 631-813-4828 (international) five minutes prior to the
start time and refer to conference ID 59171216. A replay of the call
will be available beginning at 7:30 p.m. ET on the day of the call. To
access the replay, please dial 855-859-2056 (domestic) or 404-537-3406
(international), and refer to conference ID 59171216.
Alnylam – Sanofi Genzyme Alliance
In January 2014, Alnylam
and Sanofi Genzyme, the specialty care global business unit of Sanofi,
formed an alliance to accelerate the advancement of RNAi therapeutics as
a potential new class of innovative medicines for patients around the
world with rare genetic diseases. The alliance enables Sanofi Genzyme to
expand its rare disease pipeline with Alnylam’s novel RNAi technology
and provides access to Alnylam’s R&D engine, while Alnylam benefits from
Sanofi Genzyme’s proven global capabilities to advance late-stage
development and, upon commercialization, accelerate market access for
these promising genetic medicine products.
In the case of patisiran, Alnylam will advance the product in the United States, Canada and Western Europe, while Sanofi Genzyme will advance the product in the rest of the world. In November 2016, Sanofi Genzyme elected to co-develop (through Sanofi R&D) and co-commercialize fitusiran in the United States, Canada and Western Europe, in addition to commercializing fitusiran in its rest of world territories.
About RNAi
RNAi (RNA interference) is a revolution in
biology, representing a breakthrough in understanding protein synthesis
in cells, and a completely new approach to drug discovery and
development. Its discovery has been heralded as "a major scientific
breakthrough that happens once every decade or so," and represents one
of the most promising and rapidly advancing frontiers in biology and
drug discovery today which was awarded the 2006 Nobel Prize for
Physiology or Medicine. RNAi is a natural process of gene silencing that
occurs in organisms ranging from plants to mammals. By harnessing the
natural biological process of RNAi occurring in our cells, the creation
of a major new class of medicines, known as RNAi therapeutics, is on the
horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi
and comprise Alnylam's RNAi therapeutic platform, target the cause of
diseases by potently silencing specific mRNAs, with the goal of
preventing disease-causing proteins from being made.
About LNP Technology
Alnylam has licenses to Arbutus LNP
intellectual property for use in RNAi therapeutic products using LNP
technology.
