[August 09, 2017] |
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Alnylam Pharmaceuticals Reports Second Quarter 2017 Financial Results and Highlights Recent Period Activity
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.
-
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.
-
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 (News - Alert) 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.
About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) is
leading the translation of RNA interference (RNAi) into a whole new
class of innovative medicines with the potential to transform the lives
of patients who have limited or inadequate treatment options. Based on
Nobel Prize-winning science, RNAi therapeutics represent a powerful,
clinically validated approach for the treatment of a wide range of
debilitating diseases. Founded in 2002, Alnylam is delivering on a bold
vision to turn scientific possibility into reality, with a robust
discovery platform and deep pipeline of investigational medicines,
including three product candidates that are in late-stage development or
will be in 2017. Looking forward, Alnylam will continue to execute on
its "Alnylam 2020" strategy of building a multi-product,
commercial-stage biopharmaceutical company with a sustainable pipeline
of RNAi-based medicines. For more information about our people, science
and pipeline, please visit www.alnylam.com
and engage with us on Twitter (News - Alert) at @Alnylam.
Use of Non-GAAP Financial Measures This press release
contains non-GAAP financial measures, including net loss adjusted to
exclude certain non-cash expenses. These measures are not in accordance
with, or an alternative to, GAAP, and may be different from non-GAAP
financial measures used by other companies.
The item included in GAAP presentations but excluded for purposes of
determining non-GAAP financial measures for the periods presented in
this press release is stock-based compensation expense. The company has
excluded the impact of stock-based compensation expense, which may
fluctuate from period to period based on factors including the
variability associated with performance-based grants for stock options
and restricted stock units and changes in the company's stock price,
which impacts the fair value of these awards.
The company believes the presentation of non-GAAP net loss provides
useful information to management and investors regarding the company's
financial condition and results of operations. When GAAP financial
measures are viewed in conjunction with non-GAAP financial measures,
investors are provided with a more meaningful understanding of the
company's ongoing operating performance. In addition, non-GAAP net loss
is among those indicators the company uses as a basis for evaluating
performance, allocating resources and planning and forecasting future
periods. Non-GAAP financial measures are not intended to be considered
in isolation or as a substitute for GAAP financial measures. A
reconciliation between GAAP and non-GAAP net loss is provided later in
this press release.
Alnylam Forward Looking Statements Various statements in
this release concerning Alnylam's future expectations, plans and
prospects, including without limitation, Alnylam's views with respect to
the potential for RNAi therapeutics, including patisiran, fitusiran,
givosiran, and inclisiran, its expectations regarding the timing of
clinical studies and the presentation of clinical data, including for
studies for patisiran, fitusiran, givosiran, and inclisiran, its
expectations regarding the potential filing of an NDA and MAA for
patisiran if the APOLLO Phase 3 study is positive and its expected
transition to commercial operations in mid-2018 if regulators approve
patisiran, its expected cash, cash equivalents, fixed marketable
securities and restricted investments position as of December 31, 2017,
and its "Alnylam 2020" guidance for the advancement and
commercialization of RNAi therapeutics, constitute forward-looking
statements for the purposes of the safe harbor provisions under The
Private Securities Litigation Reform Act of 1995. Actual results and
future plans may differ materially from those indicated by these
forward-looking statements as a result of various important risks,
uncertainties and other factors, including, without limitation,
Alnylam's ability to discover and develop novel drug candidates and
delivery approaches, successfully demonstrate the efficacy and safety of
its product candidates, the pre-clinical and clinical results for its
product candidates, which may not be replicated or continue to occur in
other subjects or in additional studies or otherwise support further
development of product candidates for a specified indication or at all,
actions or advice of regulatory agencies, which may affect the design,
initiation, timing, continuation and/or progress of clinical trials or
result in the need for additional pre-clinical and/or clinical testing,
delays, interruptions or failures in the manufacture and supply of our
product candidates, obtaining, maintaining and protecting intellectual
property, Alnylam's ability to enforce its intellectual property rights
against third parties and defend its patent portfolio against challenges
from third parties, obtaining and maintaining regulatory approval,
pricing and reimbursement for products, progress in establishing a
commercial and ex-United States infrastructure, competition from others
using technology similar to Alnylam's and others developing products for
similar uses, Alnylam's ability to manage its growth and operating
expenses, obtain additional funding to support its business activities,
and establish and maintain strategic business alliances and new business
initiatives, Alnylam's dependence on third parties for development,
manufacture and distribution of products, the outcome of litigation, the
risk of government investigations, and unexpected expenditures, as well
as those risks more fully discussed in the "Risk Factors" filed with
Alnylam's most recent Quarterly Report on Form 10-Q filed with
the Securities and Exchange Commission (SEC (News - Alert)) and in other filings that
Alnylam makes with the SEC. In addition, any forward-looking statements
represent Alnylam's views only as of today and should not be relied upon
as representing its views as of any subsequent date. Alnylam explicitly
disclaims any obligation, except to the extent required by law, to
update any forward-looking statements.
The scientific information referenced in this news release relating to
Alnylam's investigational therapeutics is preliminary and investigative.
None of Alnylam's investigational therapeutics, including inclisiran
which is partnered with The Medicines Company, have been approved by the
U.S. Food and Drug Administration, European Medicines Agency, or any
other regulatory authority and no conclusions can or should be drawn
regarding the safety or effectiveness of these therapeutics.
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ALNYLAM PHARMACEUTICALS, INC.
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UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
|
(In thousands, except per share amounts)
|
|
|
|
|
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|
Three Months Ended June 30,
|
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Six Months Ended June 30,
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2017
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2016
|
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2017
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2016
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|
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|
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|
Net revenues from collaborators
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|
|
|
$
|
15,932
|
|
|
|
|
$
|
8,709
|
|
|
|
|
|
$
|
34,892
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|
|
$
|
16,054
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|
|
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|
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|
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Operating expenses:
|
|
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|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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Research and development
|
|
|
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|
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90,627
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|
|
|
|
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83,172
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|
|
|
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|
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177,611
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|
|
|
|
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179,445
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General and administrative
|
|
|
|
|
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45,779
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|
|
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|
|
17,987
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|
|
|
|
|
|
84,266
|
|
|
|
|
|
39,087
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Total operating expenses
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|
|
|
|
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136,406
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|
|
|
|
|
101,159
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|
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261,877
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|
|
|
|
218,532
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Loss from operations
|
|
|
|
|
|
(120,474
|
)
|
|
|
|
|
(92,450
|
)
|
|
|
|
|
|
(226,985
|
)
|
|
|
|
|
(202,478
|
)
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Other income (expense):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income
|
|
|
|
|
|
2,577
|
|
|
|
|
|
2,092
|
|
|
|
|
|
|
4,705
|
|
|
|
|
|
3,905
|
|
Other (expense) income
|
|
|
|
|
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(523
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)
|
|
|
|
|
229
|
|
|
|
|
|
|
(3,430
|
)
|
|
|
|
|
5,470
|
|
Total other income
|
|
|
|
|
|
2,054
|
|
|
|
|
|
2,321
|
|
|
|
|
|
|
1,275
|
|
|
|
|
|
9,375
|
|
Net loss
|
|
|
|
|
$
|
(118,420
|
)
|
|
|
|
$
|
(90,129
|
)
|
|
|
|
|
$
|
(225,710
|
)
|
|
|
|
$
|
(193,103
|
)
|
Net loss per common share - basic and diluted
|
|
|
|
|
$
|
(1.34
|
)
|
|
|
|
$
|
(1.05
|
)
|
|
|
|
|
$
|
(2.59
|
)
|
|
|
|
$
|
(2.26
|
)
|
Weighted-average common shares used to compute basic and diluted net
loss per common share
|
|
|
|
|
|
88,098
|
|
|
|
|
|
85,545
|
|
|
|
|
|
|
87,068
|
|
|
|
|
|
85,411
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Comprehensive loss:
|
|
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|
|
|
|
|
|
|
|
|
|
|
|
|
|
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|
Net loss
|
|
|
|
|
$
|
(118,420
|
)
|
|
|
|
$
|
(90,129
|
)
|
|
|
|
|
$
|
(225,710
|
)
|
|
|
|
$
|
(193,103
|
)
|
Unrealized loss on marketable securities, net of tax
|
|
|
|
|
|
(476
|
)
|
|
|
|
|
(18,331
|
)
|
|
|
|
|
|
(2,412
|
)
|
|
|
|
|
(26,555
|
)
|
Reclassification adjustment for realized loss (gain) on marketable
securities included in net loss
|
|
|
|
|
|
345
|
|
|
|
|
|
(954
|
)
|
|
|
|
|
|
1,894
|
|
|
|
|
|
(6,110
|
)
|
Comprehensive loss
|
|
|
|
|
$
|
(118,551
|
)
|
|
|
|
$
|
(109,414
|
)
|
|
|
|
|
$
|
(226,228
|
)
|
|
|
|
$
|
(225,768
|
)
|
|
|
|
|
|
|
|
|
|
|
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|
|
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|
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|
|
|
|
|
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|
|
|
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ALNYLAM PHARMACEUTICALS, INC.
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RECONCILIATION OF GAAP NET (News - Alert) LOSS TO NON-GAAP NET LOSS
|
(In thousands, except per share amounts)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended June 30,
|
|
|
|
|
Six Months Ended June 30,
|
|
|
|
|
|
2017
|
|
|
|
2016
|
|
|
|
|
2017
|
|
|
|
2016
|
GAAP net loss
|
|
|
|
|
$
|
(118,420
|
)
|
|
|
|
$
|
(90,129
|
)
|
|
|
|
|
$
|
(225,710
|
)
|
|
|
|
$
|
(193,103
|
)
|
Adjustment:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Stock-based compensation expenses
|
|
|
|
|
|
24,030
|
|
|
|
|
|
15,816
|
|
|
|
|
|
|
39,747
|
|
|
|
|
|
39,296
|
|
Non-GAAP net loss
|
|
|
|
|
$
|
(94,390
|
)
|
|
|
|
$
|
(74,313
|
)
|
|
|
|
|
$
|
(185,963
|
)
|
|
|
|
$
|
(153,807
|
)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
GAAP net loss per common share - basic and diluted
|
|
|
|
|
$
|
(1.34
|
)
|
|
|
|
$
|
(1.05
|
)
|
|
|
|
|
$
|
(2.59
|
)
|
|
|
|
$
|
(2.26
|
)
|
Adjustment (as detailed above)
|
|
|
|
|
|
0.27
|
|
|
|
|
|
0.18
|
|
|
|
|
|
|
0.45
|
|
|
|
|
|
0.46
|
|
Non-GAAP net loss per common share - basic and diluted
|
|
|
|
|
$
|
(1.07
|
)
|
|
|
|
$
|
(0.87
|
)
|
|
|
|
|
$
|
(2.14
|
)
|
|
|
|
$
|
(1.80
|
)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
ALNYLAM PHARMACEUTICALS, INC.
|
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
|
(In thousands, except share amounts)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
June 30,
|
|
|
|
December 31,
|
|
|
|
|
|
2017
|
|
|
|
2016
|
Cash, cash equivalents and fixed income marketable securities
|
|
|
|
|
$
|
1,097,841
|
|
|
|
$
|
942,601
|
Restricted investments
|
|
|
|
|
|
150,000
|
|
|
|
|
150,000
|
Billed and unbilled collaboration receivables
|
|
|
|
|
|
15,405
|
|
|
|
|
23,334
|
Prepaid expenses and other assets
|
|
|
|
|
|
23,504
|
|
|
|
|
32,303
|
Property, plant and equipment, net
|
|
|
|
|
|
147,533
|
|
|
|
|
114,572
|
Total assets
|
|
|
|
|
$
|
1,434,283
|
|
|
|
$
|
1,262,810
|
Accounts payable, accrued expenses and other liabilities
|
|
|
|
|
$
|
69,805
|
|
|
|
$
|
99,650
|
Total deferred revenue
|
|
|
|
|
|
81,873
|
|
|
|
|
82,932
|
Total deferred rent
|
|
|
|
|
|
9,639
|
|
|
|
|
10,007
|
Long-term debt
|
|
|
|
|
|
150,000
|
|
|
|
|
150,000
|
Total stockholders' equity (91.7 million and 85.9 million common
shares issued and outstanding at June 30, 2017 and December 31,
2016, respectively)
|
|
|
|
|
|
1,122,966
|
|
|
|
|
920,221
|
Total liabilities and stockholders' equity
|
|
|
|
|
$
|
1,434,283
|
|
|
|
$
|
1,262,810
|
|
|
|
|
|
|
|
|
|
|
|
|
This selected financial information should be read in conjunction with
the consolidated financial statements and notes thereto included in
Alnylam's Annual Report on Form 10-K which includes the audited
financial statements for the year ended December 31, 2016.
View source version on businesswire.com: http://www.businesswire.com/news/home/20170809005821/en/
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