− Achieved Second Quarter 2019 ONPATTRO® Global
Net Product Revenues of $38.2 Million with More Than 500 Patients
on Commercial Product Worldwide –
− Reported Positive Results from ENVISION Phase
3 Study of Givosiran, Submitted Regulatory Filings in the U.S. and
EU, and Received Priority Review from FDA –
– Completed Enrollment in ILLUMINATE-A Phase 3
Study of Lumasiran and Advanced Multiple Additional Late-Stage
Pipeline Programs –
− Formed Broad Collaboration with Regeneron to
Discover, Develop and Commercialize RNAi Therapeutics Focused on
Ocular and Central Nervous System (CNS) Diseases –
− Strengthened Balance Sheet and Ended Second
Quarter with Approximately $2.0 Billion in Cash; Lowers 2019
Non-GAAP R&D and Non-GAAP SG&A Expense Guidance –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today reported its consolidated financial
results for the second quarter 2019 and reviewed recent business
highlights.
“Over the past quarter, we’re pleased with the continued strong
progress in the global launch of ONPATTRO. We believe that
continued commercial execution with ONPATTRO and expected upcoming
launches of other products puts us on a path toward attaining
self-sustainability in our business, delivering on the promise of
RNAi therapeutics for patients around the world,” said John
Maraganore, Ph.D., Chief Executive Officer of Alnylam. “During this
period we also achieved several key milestones with our late and
earlier stage pipeline, including positive Phase 3 results with
givosiran. We expect this track record of commercial and R&D
execution to continue well into the future. Specifically, as we
turn to the second half of 2019, we look forward to pivotal data
readouts from two programs – inclisiran and lumasiran – and
additional Phase 3 initiations, namely APOLLO-B with patisiran,
HELIOS-B with vutrisiran, and ILLUMINATE-C with lumasiran. Each of
these planned milestones will bring us closer to achieving our
Alnylam 2020 vision of building a multi-product, global
biopharmaceutical company that includes a deep clinical pipeline to
fuel continued growth and a robust product engine for sustainable
and organic innovation for the future, a profile rarely achieved in
the biopharmaceutical industry.”
“As we approach the one year anniversary of the ONPATTRO
approval, we couldn’t be more proud of our commercial execution. We
finished the quarter with over 500 patients on commercial therapy,
and we expect continued and steady growth in the years to come
driven by new patient finding, global expansion, and additional
evidence generation in our ATTR amyloidosis franchise,” said Barry
Greene, President of Alnylam. “We are committed to decreasing the
time to diagnosis and treatment for the benefit of patients with
hATTR amyloidosis with polyneuropathy, and we see increased
utilization of diagnostic programs such as Alnylam Act. With recent
approvals in Japan and Canada, and multiple pricing or
reimbursement approvals enabling commercial sales in over ten
countries across the CEMEA region, we are expanding our global
footprint, bringing ONPATTRO to patients internationally and laying
the groundwork for planned future launches of other RNAi
therapeutics. Finally, we believe that evidence generation for
ONPATTRO will continue to demonstrate the potential benefits and
differentiated profile of ONPATTRO.”
Second Quarter 2019 and Recent Significant Corporate
Highlights
Commercial Performance in Second Quarter 2019
- Achieved global ONPATTRO net product revenues for the second
quarter of 2019 of $38.2 million.
- Attained more than 500 patients worldwide on commercial
ONPATTRO treatment as of June 30, 2019.
- Received marketing authorization approvals for ONPATTRO in
Japan and Canada.
- Continued progress with market access efforts across the CEMEA
region (Canada, Europe, Middle East,
and Africa).
- Achieved recent reimbursement approvals and favorable ratings
from health technology assessment agencies in England, Scotland,
Germany, France, Canada, and Sweden, with significant progress in
several additional markets.
R&D Highlights
- Advanced patisiran (the non-branded name for ONPATTRO), an
intravenously administered investigational RNAi therapeutic in
development for the treatment of ATTR amyloidosis, with plans to
initiate the APOLLO-B Phase 3 study in ATTR amyloidosis with
cardiomyopathy in mid-2019.
- Presented positive 12-month data from the Global Open-Label
Extension (OLE) study, as well as new analyses from the APOLLO
Phase 3 study in patients previously treated with tafamidis and
results from an indirect treatment comparison of patisiran versus
inotersen in hATTR amyloidosis patients with polyneuropathy.
- Advanced vutrisiran (ALN-TTRsc02), a subcutaneously
administered investigational RNAi therapeutic in development for
the treatment of ATTR amyloidosis.
- Continued enrollment in the HELIOS-A Phase 3 study of
vutrisiran in hereditary ATTR amyloidosis patients with
polyneuropathy.
- The Company announces today that it has obtained regulatory
alignment on the design of HELIOS-B – a Phase 3 study of vutrisiran
in patients with both hereditary and wild-type ATTR amyloidosis
cardiomyopathy – which it expects to start in late 2019.
- Advanced givosiran, an investigational RNAi therapeutic in
development for the treatment of acute hepatic porphyria (AHP).
- Presented positive results from the ENVISION Phase 3
study.
- Completed submissions of a New Drug Application (NDA) with the
U.S. Food and Drug Administration (FDA) and a Marketing
Authorisation Application (MAA) with the European Medicines Agency
(EMA); both agencies have accepted the applications for filing. The
FDA also granted the Company’s request for Priority Review and has
set an action date of February 4, 2020, under the Prescription Drug
User Fee Act (PDUFA). At this time, the FDA is not planning to hold
an advisory committee meeting to discuss this application.
- Alnylam announces today that it has inititated an Expanded
Access Program for givosiran to support requests by Health Care
Providers for pre-approval access for AHP patients.
- Advanced lumasiran, an investigational RNAi therapeutic in
development for the treatment of primary hyperoxaluria type 1
(PH1).
- Completed enrollment in the ILLUMINATE-A Phase 3 study of
lumasiran in PH1 patients six years of age or older with
mild-to-moderate renal impairment, and remain on track to report
results by year-end 2019.
- Presented complete positive results from the Phase 1/2 clinical
study and positive results from the ongoing Phase 2 open-label
extension (OLE) study of lumasiran.
- Initiated ILLUMINATE-B, a global Phase 3 pediatric study of
lumasiran in PH1 patients under six years of age.
- Alnylam’s partner, The Medicines Company, reported new results
for inclisiran, an investigational RNAi therapeutic in development
for the treatment of hypercholesterolemia.
- New data included interim results from the ongoing ORION-3 OLE
study in patients with atherosclerotic cardiovascular disease
(ASCVD) or ASCVD-risk equivalents and results from the ORION-2 and
-7 studies in patients with homozygous familial
hypercholesterolemia (HoFH) and in patients with renal impairment,
respectively.
- In addition, The Medicines Company announced that the
Independent Data Monitoring Committee for ongoing inclisiran Phase
3 clinical trials (ORION 9, 10, and 11) completed its seventh
planned review of safety and efficacy data from the ORION trials
and recommended that the trials continue without modification. The
safety database for inclisiran now provides more than 3,500
patient-years of exposure to an RNAi therapeutic, representing the
industry’s most comprehensive body of safety data for an RNA
therapeutic.
- Alnylam’s partner, Sanofi, reported new results from the Phase
2 OLE study of fitusiran, an investigational RNAi therapeutic in
development for the treatment of hemophilia.
- Advanced early- and mid-stage RNAi clinical pipeline.
- Initiated a Phase 1 study of ALN-AGT, an investigational RNAi
therapeutic targeting angiotensinogen (AGT) for the treatment of
hypertension in high unmet need populations, including patients
with resistant or refractory hypertension, chronic kidney disease
or heart failure.
- Announced new platform advances, including preclinical results
demonstrating oral delivery of GalNAc-conjugated small interfering
RNAs (siRNAs) directed to a liver target. Oral delivery could
broaden the clinical and commercial opportunities for RNAi
therapeutics, which are currently administered with intravenous or
subcutaneous dose administration.
Additional Business Highlights
- Formed a broad collaboration with Regeneron Pharmaceuticals,
Inc. (Regeneron) to discover, develop, and commercialize RNAi
therapeutics focused on ocular and CNS diseases.
- Concluded the research and option phase of the Company’s 2014
collaboration with Sanofi focused on advancing RNAi therapeutics
for rare genetic diseases.
- Entered into a collaboration with 23andMe to support the
addition of a new Genetic Health Risk report for Hereditary
Amyloidosis (TTR-related). Read more about the report here.
- Announced senior leadership changes, including the appointment
of Kelley Boucher as the Company’s Senior Vice President, Chief
Human Resources Officer; and Jeff Poulton as Executive Vice
President, Chief Financial Officer, effective August 13.
Upcoming Events
In the second half of 2019, Alnylam intends to:
- Initiate the APOLLO-B Phase 3 study of patisiran in ATTR
amyloidosis patients with cardiomyopathy in mid-2019.
- Launch ONPATTRO in Japan, England, Switzerland, and multiple
other countries.
- Initiate the HELIOS-B Phase 3 study of vutrisiran in ATTR
amyloidosis patients with cardiomyopathy.
- Initiate the ILLUMINATE-C Phase 3 study of lumasiran in PH1
patients with severe renal impairment.
- Report topline results from the ILLUMINATE-A Phase 3 study of
lumasiran in PH1 patients six years of age or older.
In addition, The Medicines Company intends to report initial
topline results from the ORION-9, 10, and 11 Phase 3 studies of
inclisiran, and assuming positive results, to file an NDA.
Financial Results for the Quarter Ended June 30, 2019
“Alnylam had strong financial performance in the second quarter.
We ended with cash and cash equivalents on our balance sheet of
approximately $2.0 billion, bolstered by robust ONPATTRO sales as
well as $800 million in additional cash received from our recently
announced collaboration with Regeneron,” said Manmeet Soni,
outgoing Chief Financial Officer of Alnylam. “I have thoroughly
enjoyed my time as part of the Alnylam team and am confident that
the foundation created over the past few years will serve the
Company well.”
“I am thrilled to be joining an organization with great near-
and long-term growth prospects driven by advancing innovative
therapies with the potential to transform patients’ lives,” said
Jeff Poulton, recently appointed Executive Vice President, Chief
Financial Officer of Alnylam, effective August 13. “Having
supported the profitable globalization of Shire’s business during
my tenure there, I look forward to partnering with the business
team at Alnylam to develop a roadmap toward financial
self-sustainability.”
Cash and Investments At June 30, 2019, Alnylam had cash, cash
equivalents and marketable debt securities, and restricted
investments, excluding equity securities, of $1.97 billion, as
compared to $1.13 billion at December 31, 2018.
In May 2019, Alnylam received an upfront collaboration payment
from Regeneron of $400 million. In addition, Regeneron purchased
$400 million of Alnylam equity at a price per share of $90.00 (4.44
million common shares).
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 2019 was $219.5 million, or $2.02 per share on both a
basic and diluted basis, as compared to a net loss of $163.6
million, or $1.63 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 2019 was $198.3
million, or $1.83 per share on both a basic and diluted basis, as
compared to a non-GAAP net loss of $161.9 million, or $1.61 per
share on both a basic and diluted basis for the same period in the
previous year.
Reconciling items between GAAP and non-GAAP net loss include
stock-based compensation expense, a gain on the change in fair
value of a liability obligation related to the sale of common stock
to Regeneron, and a gain on a litigation settlement. 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.
ONPATTRO Revenues, Net Net product revenues from sales of
ONPATTRO were $38.2 million in the second quarter of 2019.
Net Revenues from Collaborators Net revenues from collaborators
were $6.5 million in the second quarter of 2019 as compared to
$29.9 million in the second quarter of 2018.
GAAP and Non-GAAP Research and Development Expenses GAAP
research and development (R&D) expenses were $163.9 million in
the second quarter of 2019 as compared to $137.6 million in the
second quarter of 2018.
Non-GAAP R&D expenses were $148.6 million in the second
quarter of 2019 as compared to $126.0 million in the second quarter
of 2018. Non-GAAP R&D expenses exclude stock-based compensation
expense. A reconciliation between GAAP and non-GAAP R&D
expenses appears later in this press release.
GAAP and Non-GAAP Selling, General and Administrative Expenses
GAAP selling, general and administrative (SG&A) expenses were
$112.8 million in the second quarter of 2019 as compared to $84.7
million in the second quarter of 2018.
Non-GAAP SG&A expenses were $97.4 million in the second
quarter of 2019 as compared to $74.1 million in the second quarter
of 2018. Non-GAAP SG&A expenses exclude stock-based
compensation expense. A reconciliation between GAAP and non-GAAP
SG&A expenses appears later in this press release.
2019 Updated Financial Guidance Alnylam is updating its
2019 annual non-GAAP R&D expenses to be in the range of $550 to
$575 million (previously $550 to $590 million) and non-GAAP
SG&A expenses to be in the range of $390 to $400 million
(previously $390 million to $410 million). Both non-GAAP R&D
and non-GAAP SG&A expenses exclude stock-based compensation
expenses.
The Company expects its current cash, cash equivalents, and
marketable debt securities will support company operations for
multiple years based upon its current operating plan.
Use of Non-GAAP Financial Measures This press release
contains non-GAAP financial measures, including expenses adjusted
to exclude certain non-cash expenses and non-recurring gains
outside the ordinary course of the Company’s business. 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 items included in GAAP presentations but excluded for
purposes of determining non-GAAP financial measures for the periods
presented in the press release are stock-based compensation
expense, a gain on the change in fair value of a liability
obligation, and a gain on litigation settlement. 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 has excluded the impact of a gain on the change in fair
value of liability obligation and the gain on litigation settlement
because the Company believes these items are one-time events
occurring outside the ordinary course of the Company’s
business.
The Company believes the presentation of non-GAAP financial
measures 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 and are better able to compare the Company’s
performance between periods. In addition, these non-GAAP financial
measures are 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
measures is provided later in this press release.
The Company does not provide in this press release a
reconciliation of its estimated 2019 non-GAAP R&D and non-GAAP
SG&A expense guidance to the comparable GAAP measures because
it is not able to estimate 2019 stock-based compensation expense
without unreasonable efforts. The Company’s stock-based
compensation expense is subject to significant fluctuations from
period to period due to variability in the probability of
performance-based vesting events for stock options and restricted
stock units and changes in the Company’s stock price which
materially impact the recognition, timing of expense and fair value
of these awards. In addition, the Company believes such
reconciliations for its 2019 financial guidance would imply a
degree of precision that would be confusing or misleading to
investors.
Conference Call Information Management will provide an
update on the Company and discuss second quarter 2019 results as
well as expectations for the future via conference call on Tuesday,
August 6, 2019 at 8:00 am ET. To access the call, please dial
800-263-0877 (domestic) or 646-828-8143 (international) five
minutes prior to the start time and refer to conference ID 9566932.
A replay of the call will be available beginning at 11:00 am ET on
the day of the call. To access the replay, please dial 888-203-1112
(domestic) or 719-457-0820 (international) and refer to conference
ID 9566932.
About ONPATTRO® (patisiran) ONPATTRO is an RNAi
therapeutic that is approved by the U.S. Food and Drug
Administration
(FDA) for the treatment of the polyneuropathy of hATTR
amyloidosis in adults. ONPATTRO is
also approved in the European Union for the treatment of hATTR
amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and
in Japan for the treatment of hATTR amyloidosis with polyneuropathy
by the Japanese Ministry of Health, Labour and Welfare (MHLW).
Based on Nobel Prize-winning science, ONPATTRO is an intravenously
administered RNAi therapeutic targeting transthyretin (TTR) for the
treatment of hereditary ATTR amyloidosis. It is designed to target
and silence TTR messenger RNA, thereby blocking the production of
TTR protein before it is made. ONPATTRO blocks the production of
TTR in the liver, reducing its accumulation in the body’s tissues
in order to halt or slow down the progression of the disease.
Important Safety Information ONPATTRO is a medicine that
treats the polyneuropathy caused by an illness called hereditary
transthyretin-mediated amyloidosis (hATTR amyloidosis). ONPATTRO is
used in adults only.
Infusion-Related Reactions Infusion-related reactions (IRRs)
have been observed in patients treated with ONPATTRO. In a
controlled clinical study, 19 percent of ONPATTRO-treated patients
experienced IRRs, compared to 9 percent of placebo-treated
patients. The most common symptoms of IRRs with ONPATTRO were
flushing, back pain, nausea, abdominal pain, dyspnea, and
headache.
To reduce the risk of IRRs, patients should receive
premedication with a corticosteroid, paracetamol, and
antihistamines (H1 and H2 blockers) at least 60 minutes prior to
ONPATTRO infusion. Monitor patients during the infusion for signs
and symptoms of IRRs. If an IRR occurs, consider slowing or
interrupting the infusion and instituting medical management as
clinically indicated. If the infusion is interrupted, consider
resuming at a slower infusion rate only if symptoms have resolved.
In the case of a serious or life-threatening IRR, the infusion
should be discontinued and not resumed.
Reduced Serum Vitamin A Levels and Recommended Supplementation
ONPATTRO treatment leads to a decrease in serum vitamin A levels.
Supplementation at the recommended daily allowance (RDA) of vitamin
A is advised for patients taking ONPATTRO. Higher doses than the
RDA should not be given to try to achieve normal serum vitamin A
levels during treatment with ONPATTRO, as serum levels do not
reflect the total vitamin A in the body.
Patients should be referred to an ophthalmologist if they
develop ocular symptoms suggestive of vitamin A deficiency (e.g.
night blindness).
Adverse Reactions The most common adverse reactions that
occurred in patients treated with ONPATTRO were respiratory-tract
infections (29 percent) and infusion-related reactions (19
percent).
About LNP Technology Alnylam has licenses to Arbutus
Biopharma LNP intellectual property for use in RNAi therapeutic
products using LNP technology.
About RNAi RNAi (RNA interference) is a natural cellular
process of gene silencing that represents one of the most promising
and rapidly advancing frontiers in biology and drug development
today. Its discovery has been heralded as “a major scientific
breakthrough that happens once every decade or so,” and was
recognized with the award of the 2006 Nobel Prize for Physiology or
Medicine. By harnessing the natural biological process of RNAi
occurring in our cells, a new class of medicines, known as RNAi
therapeutics, is now a reality. Small interfering RNA (siRNA), the
molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic
platform, function upstream of today’s medicines by potently
silencing messenger RNA (mRNA) – the genetic precursors – that
encode for disease-causing proteins, thus preventing them from
being made. This is a revolutionary approach with the potential to
transform the care of patients with genetic and other diseases.
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 people afflicted with rare genetic, cardio-metabolic,
hepatic infectious, and central nervous system (CNS)/ocular
diseases. Based on Nobel Prize-winning science, RNAi therapeutics
represent a powerful, clinically validated approach for the
treatment of a wide range of severe and debilitating diseases.
Founded in 2002, Alnylam is delivering on a bold vision to turn
scientific possibility into reality, with a robust discovery
platform. Alnylam’s first commercial RNAi therapeutic is ONPATTRO®
(patisiran), approved in the U.S., EU, Canada, and Japan. Alnylam
has a deep pipeline of investigational medicines, including five
product candidates that are in late-stage development. 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 to address the needs of patients who have limited or
inadequate treatment options. Alnylam employs over 1,200 people
worldwide and is headquartered in Cambridge, MA. For more
information about our people, science and pipeline, please visit
www.alnylam.com and engage with us on Twitter at @Alnylam or on
LinkedIn.
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, vutrisiran, givosiran, lumasiran, inclisiran, fitusiran
and ALN-AGT, its expectations with respect to broadened clinical
and commercial opportunities enabled by oral delivery of RNAi
therapeutics based on preclinical results, its plans for additional
evidence generation, global regulatory filings and product launches
for ONPATTRO, its expectations regarding pricing and reimbursement
procedures outside the U.S., its expectations regarding the timing
for the initiation of its APOLLO-B Phase 3 study of patisiran, its
plans to initiate the HELIOS-B Phase 3 study for vutrisiran in late
2019, its expectations regarding the regulatory review of
givosiran, including the PDUFA date set by the FDA, and
expectations regarding the FDA’s current views on not requiring an
advisory committee meeting to discuss the givosiran application,
its expectations regarding the timing of topline results from its
ILLUMINATE-A Phase 3 study of lumasiran, its plans to initiate the
ILLUMINATE-C Phase 3 study of lumasiran, its expectations regarding
the timing of topline results to be reported by The Medicines
Company from the ORION-11, ORION-9 and ORION-10 studies of
inclisiran and the potential filing of an NDA, its expected range
of 2019 annual non-GAAP R&D expenses and non-GAAP SG&A
expenses, its expectations regarding the length of time its current
cash, cash equivalents and marketable debt securities will support
company operations based on its current operating plan, plans to
develop a roadmap toward financial self-sustainability, and
expectations regarding 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 its 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, successfully launching, marketing and
selling its approved products globally, Alnylam’s ability to
successfully expand the indication for ONPATTRO in the future,
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, including Regeneron, 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) 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.
With the exception of ONPATTRO (patisiran), none of Alnylam’s
investigational therapeutics 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 such investigational
therapeutics.
ALNYLAM PHARMACEUTICALS,
INC.
UNAUDITED CONDENSED
CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
(In thousands, except per
share amounts)
Three Months Ended June
30,
Six Months Ended June
30,
2019
2018
2019
2018
Revenues:
Product revenues, net
$
38,231
$
—
$
64,522
$
—
Net revenues from collaborators
6,483
29,907
13,486
51,806
Total revenues
44,714
29,907
78,008
51,806
Cost and expenses:
Cost of goods sold
$
4,326
$
—
7,673
$
—
Research and development
163,890
137,582
293,017
234,439
Selling, general and administrative
112,769
84,679
202,377
157,126
Total costs and expenses
280,985
222,261
503,067
391,565
Loss from operations
(236,271)
(192,354)
(425,059)
(339,759)
Other income (expense):
Interest income
8,781
6,101
16,306
11,895
Other (expense) income
(453)
2,208
(410)
2,543
Change in fair value of liability
obligation
9,422
—
9,422
—
Gain on litigation settlement
—
20,564
—
20,564
Total other income
17,750
28,873
25,318
35,002
Loss before income taxes
(218,521)
(163,481)
(399,741)
(304,757)
Provision for income taxes
(960)
(79)
(1,655)
(17)
Net loss
$
(219,481)
$
(163,560)
$
(401,396)
$
(304,774)
Net loss per common share - basic and
diluted
$
(2.02)
$
(1.63)
$
(3.75)
$
(3.04)
Weighted-average common shares used to
compute basic and diluted net loss per common share
108,576
100,519
106,997
100,251
Comprehensive loss:
Net loss
$
(219,481)
$
(163,560)
$
(401,396)
$
(304,774)
Unrealized gain on marketable securities,
net of tax
462
1,046
822
626
Foreign currency translation
842
—
842
—
Unrealized loss on pension obligation
(4,282)
—
(4,282)
—
Comprehensive loss
$
(222,459)
$
(162,514)
$
(404,014)
$
(304,148)
ALNYLAM PHARMACEUTICALS,
INC.
RECONCILIATION OF SELECTED
GAAP MEASURES TO NON-GAAP MEASURES
(In thousands, except per
share amounts)
Three Months Ended June
30,
Six Months Ended June
30,
2019
2018
2019
2018
Reconciliation of GAAP to Non-GAAP
Research and development:
GAAP Research and development
$
163,890
$
137,582
$
293,017
$
234,439
Less: Stock-based compensation
expenses
(15,282)
(11,616)
(31,407)
(21,753)
Non-GAAP Research and development
$
148,608
$
125,966
$
261,610
$
212,686
Reconciliation of GAAP to Non-GAAP
Selling, general and administrative:
GAAP Selling, general and
administrative
$
112,769
$
84,679
$
202,377
$
157,126
Less: Stock-based compensation
expenses
(15,321)
(10,625)
(31,228)
(20,072)
Non-GAAP Selling, general and
administrative
$
97,448
$
74,054
$
171,149
$
137,054
Reconciliation of GAAP to Non-GAAP
Operating expenses:
GAAP Operating expenses
$
280,985
$
222,261
$
503,067
$
391,565
Less: Stock-based compensation
expenses
(30,603)
(22,241)
(62,635)
(41,825)
Non-GAAP Operating expenses
$
250,382
$
200,020
$
440,432
$
349,740
Reconciliation of GAAP to Non-GAAP Net
loss:
GAAP Net loss
$
(219,481)
$
(163,560)
$
(401,396)
$
(304,774)
Add: Stock-based compensation expenses
30,603
22,241
62,635
41,825
Less: Change in fair value of liability
obligation
(9,422)
—
(9,422)
—
Less: Gain on litigation settlement
—
(20,564)
—
(20,564)
Non-GAAP Net loss
$
(198,300)
$
(161,883)
$
(348,183)
$
(283,513)
Reconciliation of GAAP to Non-GAAP Net
loss per common share-basic and diluted:
GAAP Net loss per common share - basic and
diluted
$
(2.02)
$
(1.63)
$
(3.75)
$
(3.04)
Add: Stock-based compensation expenses
0.28
0.22
0.59
0.42
Less: Change in fair value of liability
obligation
(0.09)
—
(0.09)
—
Less: Gain on litigation settlement
—
(0.20)
—
(0.21)
Non-GAAP Net loss per common share - basic
and diluted
$
(1.83)
$
(1.61)
$
(3.25)
$
(2.83)
ALNYLAM PHARMACEUTICALS,
INC.
UNAUDITED CONDENSED
CONSOLIDATED BALANCE SHEETS
(In thousands, except share
amounts)
June 30,
December 31,
2019
2018
Cash, cash equivalents and marketable debt
securities
$
1,926,831
$
1,082,949
Restricted investments
44,825
44,825
Accounts receivable, net
30,739
18,760
Inventory
40,587
24,068
Prepaid expenses and other assets
75,303
83,542
Property, plant and equipment, net
371,769
320,658
Operating lease right-of-use lease
assets
226,357
—
Total assets
$
2,716,411
$
1,574,802
Accounts payable, accrued expenses and
other liabilities
$
219,529
$
177,392
Total deferred revenue
403,129
3,954
Total deferred rent
—
61,491
Operating lease liability
306,558
—
Long-term debt
30,000
30,000
Total stockholders’ equity (111.1 million
shares issued and outstanding at June 30, 2019; 101.2 million
shares issued and outstanding at December 31, 2018)
1,757,195
1,301,965
Total liabilities and stockholders'
equity
$
2,716,411
$
1,574,802
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, 2018.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190806005269/en/
Contacts: Alnylam Pharmaceuticals, Inc. Christine
Regan Lindenboom (Investors and Media) 617-682-4340 Josh Brodsky
(Investors) 617-551-8276
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