− Achieved Fourth Quarter 2018 ONPATTRO® Global
Net Product Revenues of $12.1 Million, with Over 200 Patients on
Commercial Product in U.S. and EU as of Year-End 2018 –
− Obtained Regulatory Alignment on APOLLO-B
Phase 3 Study of Patisiran in ATTR Amyloidosis with Cardiomyopathy
and Initiated HELIOS-A Phase 3 Study to Advance Vutrisiran to
Market, Supporting Plan for Sustained and Continuous Growth of ATTR
Franchise –
− Advanced Additional Phase 3 Programs,
Including Givosiran, with Rolling Submission of New Drug
Application (NDA) Initiated and Topline ENVISION Phase 3 Results
Expected in March, and Lumasiran, with ILLUMINATE-A Phase 3 Study
Initiated –
− Maintained Strong Balance Sheet
with $1.13 Billion in Cash as of Year-End 2018 –
− Provides 2019 Non-GAAP R&D and SG&A
Expense Guidance –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading
RNAi therapeutics company, today reported its consolidated
financial results for the fourth quarter and full year ended
December 31, 2018 and reviewed recent commercial and R&D
highlights.
“In 2018, we saw the approval and launch of ONPATTRO, the
world’s first RNAi therapeutic, heralding the arrival of a whole
new class of medicines. With our planned APOLLO-B Phase 3 study to
support potential expansion of ONPATTRO to ATTR amyloidosis
patients with cardiomyopathy and advancement of subcutaneously
administered vutrisiran in the HELIOS-A Phase 3 study, we are
committing further efforts to help ATTR amyloidosis patients and to
support our plans for the sustained and continued growth in our
ATTR amyloidosis franchise for years to come,” said John
Maraganore, Ph.D., Chief Executive Officer of Alnylam. “While
executing on our efforts to bring ONPATTRO to patients around the
world, we also made great strides advancing our pipeline of
investigational RNAi therapeutics. In 2019, we intend to execute on
six Phase 3 programs – of which two are being advanced together
with partners – with Phase 3 data expected from three of these
programs, and expect two NDA submissions, assuming positive
results. In the meanwhile, we’ll continue to benefit from a
sustainable RNAi research engine that we expect will fuel future
innovation and deliver important medicines for patients.”
“We are pleased with our commercial progress to date, as we
strive to make ONPATTRO available to patients around the world.
Specifically, we are encouraged by our fourth quarter 2018 results,
with over 200 patients receiving treatment with commercial ONPATTRO
in the U.S. and EU since launch, reflecting what we believe to be
strong patient and physician demand and excellent performance by
our customer-facing field teams,” said Barry Greene, President of
Alnylam. “In 2019, we’re focused on continued execution of our
ONPATTRO launch in the U.S. and EU, while expanding our global
commercial presence in Asia and Latin America. Longer term, we’re
excited by the opportunity for positive impact for patients,
patient families and caregivers, and meaningful growth potential
for our ATTR amyloidosis franchise.”
Fourth Quarter 2018 and Recent Significant Corporate
Highlights
Commercial Performance at Year-End 2018
- Achieved global net product revenues
for the fourth quarter of 2018 of $12.1 million for ONPATTRO, and
$12.5 million for the full year ended December 31, 2018.
- Attained over 200 patients in the U.S.
and EU on commercial ONPATTRO treatment.
- Including patients on commercial drug
and patients in clinical studies and in the Company’s global
Expanded Access Program (EAP), approximately 550 total patients
worldwide were being treated with patisiran.
- Received a total of 250 Start Forms in
the U.S., with approximately 50 percent from patients not
previously treated in the ONPATTRO EAP.
- Start Forms came from a diverse range
of prescribing physician specialties, including 44 percent from
neurologists, 35 percent from cardiologists, and 21 percent from
other specialties.
- For Start Forms received, 62 percent of
patients were covered by Medicare, 32 percent were covered by
commercial insurers, and 6 percent were covered by other government
insurers.
- Continued significant progress with
value-based agreements (VBAs) with commercial payers in the U.S.
and with market access efforts in the EU.
- Since launch, Alnylam has completed
definitive VBAs with Harvard Pilgrim Healthcare, Humana, and
another top five U.S. payer. Additional VBAs are under negotiation
with over 15 other commercial payers with the potential to cover
over 90 percent of commercial lives in the U.S.
- The Company announces today that it has
advanced pricing & reimbursement procedures with authorities in
15 EU countries – representing the vast majority of the hATTR
amyloidosis opportunity in Europe – with positive feedback from
several EU payers. Recent examples include positive technology
assessment reports from authorities in Germany and Sweden, the
special innovation designation of ONPATTRO by the Italian
authorities, and favorable alignment with authorities in The
Netherlands, among others.
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.
- Continued global efforts to bring
ONPATTRO to patients with filing of a New Drug Submission (NDS) in
Canada and a Marketing Authorisation Application (MAA) in
Switzerland, which has now been accepted.
- Obtained alignment with the U.S. Food
and Drug Administration (FDA) on the design of APOLLO-B, a
randomized, double-blind, placebo-controlled Phase 3 study of
patisiran in hereditary and wild-type ATTR amyloidosis patients
with cardiomyopathy, with the goal of starting the trial in
mid-2019.
- Advanced vutrisiran (ALN-TTRsc02), a
subcutaneously administered investigational RNAi therapeutic in
development for the treatment of ATTR amyloidosis.
- Initiated HELIOS-A Phase 3 study in
hereditary ATTR amyloidosis with polyneuropathy.
- Announced plans to initiate an
additional Phase 3 study, HELIOS-B, of vutrisiran in hereditary and
wild-type ATTR amyloidosis with cardiomyopathy in late 2019.
- Advanced givosiran, an investigational
RNAi therapeutic in development for the treatment of acute hepatic
porphyria (AHP).
- Initiated rolling submission of a New
Drug Application (NDA) with plans to pursue full approval based on
complete results – expected in March 2019 – from the ENVISION Phase
3 study. Assuming positive results, the Company expects to submit
full clinical sections to the FDA in mid-2019.
- Published data from Phase 1 study of
givosiran in The New England Journal of Medicine.
- Presented updated positive Phase 1/2
open-label extension (OLE) results at The Liver Meeting® 2018
of the American Association for the Study of Liver
Diseases (AASLD).
- Advanced lumasiran, an investigational
RNAi therapeutic in development for the treatment of primary
hyperoxaluria type 1 (PH1).
- Initiated ILLUMINATE-A, a global Phase
3 pivotal trial of lumasiran in children and adult PH1 patients
with preserved renal function. 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 (ESPN) and the American Society of
Nephrology (ASN) 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.
- Alnylam’s partner, The Medicines
Company, announced in January 2019 that the Independent Data
Monitoring Committee for the ongoing inclisiran Phase 3 clinical
trials (ORION 9, 10, and 11) conducted its fifth 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 2,450 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, continues
enrollment in the fitusiran Phase 3 ATLAS program in patients with
hemophilia A or B with and without inhibitors.
- Advanced early-stage RNAi pipeline
programs.
- Discontinued a Phase 2 study of
cemdisiran in atypical hemolytic uremic syndrome (aHUS) due to
recruitment challenges. Alnylam announces today that it has
received regulatory approval to initiate a Phase 2 study of
cemdisiran in IgA nephropathy.
- Submitted a Clinical Trial
Authorization (CTA) application for ALN-AAT02, an investigational
RNAi therapeutic for the treatment of alpha-1 antitrypsin
deficiency-associated liver disease (alpha-1 liver disease).
Alnylam announces today that it has initiated a Phase 1 study of
ALN-AAT02, with initial results expected in 2019.
- With Vir Biotechnology, initiated a
Phase 1/2 study of ALN-HBV02 (also known as VIR-2218), with initial
results expected in 2019.
- Reported new platform innovations at
the Oligonucleotide Therapeutics Society 2018 Annual Meeting,
including pre-clinical results demonstrating potent, wide-spread,
and highly durable CNS and ocular delivery of RNAi therapeutics in
rats and non-human primates.
- Selected its first CNS-targeted
development candidate, ALN-APP, an investigational RNAi therapeutic
targeting amyloid precursor protein (APP) for the treatment of
cerebral amyloid angiopathy (CAA).
Additional Business Highlights
- Entered into an exclusive distribution
agreement with Medison Pharma for the commercialization of certain
RNAi therapeutics in Israel.
- Resolved all litigation worldwide with
Silence Therapeutics.
Upcoming Events in Early 2019
- Alnylam expects to report topline
results from the ENVISION Phase 3 study of givosiran in March 2019,
and announces today that it plans to present full study results in
an oral presentation at the The International Liver Congress™ 2019
of the European Association for the Study of the Liver (EASL) on
Saturday, April 13, 2019 in Vienna, Austria.
Financial Results for the Quarter and Year Ended December 31,
2018
“We ended 2018 with cash and investments on our balance sheet of
$1.13 billion, exceeding our 2018 guidance, and we’re pleased to
have recently strengthened our balance sheet further with our
public offering in January resulting in net proceeds of
approximately $382 million,” said Manmeet Soni, Chief Financial
Officer of Alnylam. “Taken together with our growing product
revenues, we believe Alnylam is in a strong position to continue
executing on global commercialization plans while advancing our
pipeline of late- and early-stage programs.”
Cash and Investments
At December 31, 2018, Alnylam had cash, cash equivalents and
marketable debt securities and restricted investments, excluding
equity securities, of $1.13 billion, as compared to $1.73 billion
at December 31, 2017.
In January 2019, 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 $77.50 per share. As a result
of the offering, Alnylam received aggregate net proceeds
of approximately $382 million.
GAAP and Non-GAAP Net Loss
The net loss according to accounting principles generally
accepted in the U.S. (GAAP) for the fourth quarter of 2018 was
$211.4 million, or $2.09 per share on both a basic and diluted
basis, as compared to a net loss of $142.2 million, or $1.48 per
share on both a basic and diluted basis, for the same period in the
previous year. For the year ended December 31, 2018, the net loss
was $761.5 million, or $7.57 per share on both a basic and diluted
basis, as compared to a net loss of $490.9 million, or $5.42 per
share on both a basic and diluted basis, for the prior year.
The non-GAAP net loss for the fourth quarter of 2018 was $183.5
million, or $1.82 per share on both a basic and diluted basis, as
compared to a non-GAAP net loss of $115.1 million, or $1.20 per
share on both a basic and diluted basis for the same period in the
previous year. The non-GAAP net loss for the year ended December
31, 2018 was $624.3 million, or $6.21 per share on both a basic and
diluted basis, as compared to a non-GAAP net loss of $398.1
million, or $4.40 per share on both a basic and diluted basis, for
the prior year.
The non-GAAP net loss excludes stock-based compensation expense
and gain on 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 $12.1 million
in the fourth quarter of 2018. Net product revenues from sales of
ONPATTRO were $12.5 million in the year ended December 31,
2018.
Net Revenues from Collaborators
Net revenues from collaborators were $9.0 million in the fourth
quarter of 2018, as compared to $37.9 million in the fourth quarter
of 2017. Net revenues from collaborators were $62.4 million in the
year ended December 31, 2018, as compared to $89.9 million in the
year ended December 31, 2017.
GAAP and Non-GAAP Research and Development Expenses
GAAP research and development (R&D) expenses were $131.0
million in the fourth quarter of 2018 as compared to $117.8 million
in the fourth quarter of 2017. GAAP R&D expenses were $505.4
million in the year ended December 31, 2018 as compared to $390.6
million for the prior year.
Non-GAAP R&D expenses were $118.1 million in the fourth
quarter of 2018 as compared to $102.9 million in the fourth quarter
of 2017. Non-GAAP R&D expenses were $424.9 million in the year
ended December 31, 2018 as compared to $338.8 million for the prior
year. 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 $108.7 million in the fourth quarter of 2018 as compared to
$67.5 million in the fourth quarter of 2017. GAAP SG&A expenses
were $382.4 million in the year ended December 31, 2018 as compared
to $199.4 million for the prior year.
Non-GAAP SG&A expenses were $93.7 million in the fourth
quarter of 2018 as compared to $55.2 million in the fourth quarter
of 2017. Non-GAAP SG&A expenses were $305.1 million in the year
ended December 31, 2018 as compared to $158.4 million for the prior
year. 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 Financial Guidance
Alnylam expects its 2019 annual non-GAAP R&D expenses to be
in the range of $520 to $560 million and non-GAAP SG&A expenses
to be in the range of $390 to $420 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
approximately two 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
and the 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 the gain on litigation settlement because
the Company believes this item is a one-time event 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 impacts the recognition, timing of expense and fair
value of these awards. In addition, we believe such reconciliations
for our 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
fourth quarter and year end 2018 results as well as expectations
for the future via conference call on Thursday, February 7, 2019 at
4:30 pm ET. To access the call, please dial 800-667-5617 (domestic)
or 334-323-0509 (international) five minutes prior to the start
time and refer to conference ID 4263166. A replay of the call will
be available beginning at 7:30 pm 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
4263166.
Alnylam – Sanofi Genzyme Alliance
Alnylam and Sanofi Genzyme, the specialty care global business
unit of Sanofi, established an alliance to accelerate the
advancement of RNAi therapeutics as a potential new class of
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 medicines products.
About ONPATTRO® (patisiran)
Patisiran, based on Nobel Prize-winning science, 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. Patisiran 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. In August 2018, patisiran received U.S. Food and Drug
Administration (FDA) approval for the treatment of the
polyneuropathy of hATTR amyloidosis in adults, as well as European
Medicines Agency marketing authorization for the treatment of hATTR
amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy.
Important Safety Information
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 infection (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 new class of innovative medicines with
the potential to improve 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. ONPATTRO® (patisiran) lipid complex
injection, available in the U.S. for the treatment of the
polyneuropathy of hereditary transthyretin-mediated (hATTR)
amyloidosis in adults, is Alnylam’s first U.S. FDA-approved RNAi
therapeutic. In the EU, ONPATTRO is approved for the treatment of
hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy.
Alnylam has a deep pipeline of investigational medicines, including
three 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 1,000 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, ALN-AAT02, cemdisiran, ALN-HBV02
and ALN-APP, its plans for additional global regulatory filings and
product launches for ONPATTRO, its expectations regarding the
completion of additional VBAs with U.S. commercial payers and the
status of 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 2019, its expectations
regarding the timing and reporting of complete topline results from
the ENVISION Phase 3 study of givosiran and, if positive, for the
submission of full clinical sections of an NDA with the FDA, its
expectations regarding the timing of topline results from its
ILLUMINATE-A Phase 3 study of lumasiran and a possible filing of
global regulatory submissions starting in early 2020 if such
results are positive, its plans to initiate the ILLUMINATE-B Phase
3 study of lumasiran, , its plans to initiate a Phase 2 study of
cemdisiran in IgA nephropathy, its expectations regarding the
timing of initial results from its Phase 1/2 study for ALN-AAT02
and Vir’s Phase 1/2 study of ALN-HBV02, 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, 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 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 EndedDecember
31,
Year EndedDecember 31,
2018 2017 2018 2017
Revenues: Product revenues, net $ 12,075 $ — $ 12,535
$ — Net revenues from collaborators 8,958 37,924
62,373 89,912
Total revenues 21,033
37,924 74,908 89,912
Cost and
expenses: Cost of goods sold 1,665 — 1,802 — Research and
development 131,036 117,772 505,420 390,635 Selling, general and
administrative 108,688 67,455 382,359
199,365 Total costs and expenses 241,389 185,227
889,581 590,000 Loss from operations (220,356)
(147,303) (814,673) (500,088)
Other income
(expense): Interest income 10,571 4,235 29,262 12,236 Other
(expense) income (1,295) 841 4,173 (3,022) Gain on litigation
settlement — — 20,564 — Total other
income 9,276 5,076 53,999 9,214 Loss
before income taxes (211,080) (142,227) (760,674) (490,874)
Provision for income taxes (361) — (823)
— Net loss $ (211,441) $ (142,227) $ (761,497) $ (490,874)
Net loss per common share - basic and diluted $ (2.09) $ (1.48) $
(7.57) $ (5.42) Weighted-average common shares used to compute
basic and diluted net loss per common share 101,066
96,139 100,590 90,554
Comprehensive
loss: Net loss $ (211,441) $ (142,227) $ (761,497) $ (490,874)
Unrealized gain (loss) on marketable securities, net of tax 179
(692) 1,220 (2,886) Reclassification adjustment for realized loss
on marketable securities included in net loss — —
— 1,894 Comprehensive loss $ (211,262) $ (142,919) $
(760,277) $ (491,866)
ALNYLAM PHARMACEUTICALS, INC.
RECONCILIATION OF SELECTED GAAP
MEASURES TO NON-GAAP MEASURES
(In thousands, except per share
amounts)
Three Months EndedDecember
31,
Year EndedDecember 31,
2018 2017 2018 2017
Reconciliation of GAAP to Non-GAAP Research and development:
GAAP Research and development $ 131,036 $ 117,772 $ 505,420 $
390,635 Less: Stock-based compensation expenses (12,972)
(14,837) (80,509) (51,872) Non-GAAP Research
and development $ 118,064 $ 102,935 $ 424,911 $ 338,763
Reconciliation of GAAP to Non-GAAP Selling, general and
administrative: GAAP Selling, general and administrative $
108,688 $ 67,455 $ 382,359 $ 199,365 Less: Stock-based compensation
expenses (15,001) (12,280) (77,243)
(40,947) Non-GAAP Selling, general and administrative $ 93,687 $
55,175 $ 305,116 $ 158,418
Reconciliation of GAAP to
Non-GAAP Operating costs and expenses: GAAP Operating costs and
expenses $ 241,389 $ 185,227 $ 889,581 $ 590,000 Less: Stock-based
compensation expenses (27,973) (27,117)
(157,752) (92,819) Non-GAAP Operating costs and expenses $
213,416 $ 158,110 $ 731,829 $ 497,181
Reconciliation of
GAAP to Non-GAAP Net loss: GAAP Net loss $ (211,441) $
(142,227) $ (761,497) $ (490,874) Add: Stock-based compensation
expenses 27,973 27,117 157,752 92,819 Less: Gain on litigation
settlement — — (20,564) — Non-GAAP Net
loss $ (183,468) $ (115,110) $ (624,309) $ (398,055)
Reconciliation of GAAP to Non-GAAP Net loss per common
share-basic and diluted: GAAP Net loss per common share - basic
and diluted $ (2.09) $ (1.48) $ (7.57) $ (5.42) Add: Stock-based
compensation expenses 0.27 0.28 1.57 1.02 Less: Gain on litigation
settlement — — (0.21) — Non-GAAP Net
loss per common share - basic and diluted $ (1.82) $ (1.20) $
(6.21) $ (4.40)
ALNYLAM PHARMACEUTICALS, INC.
UNAUDITED CONDENSED CONSOLIDATED
BALANCE SHEETS
(In thousands, except share
amounts)
December 31,
December 31, 2018
2017 Cash, cash equivalents and marketable debt securities $
1,082,949 $ 1,704,537 Restricted investments 44,825 30,000 Accounts
receivable, net 18,760 34,002 Inventory 24,068 — Prepaid expenses
and other assets 83,542 44,291 Property, plant and equipment, net
320,658 181,900
Total assets
$ 1,574,802 $ 1,994,730 Accounts payable, accrued
expenses and other liabilities $ 177,392 $ 104,905 Total deferred
revenue 3,954 84,780 Total deferred rent 61,491 8,614 Long-term
debt 30,000 30,000 Total stockholders’ equity (101.2 million and
99.7 million common shares issued and outstanding at December 31,
2018 and December 31, 2017, respectively) 1,301,965
1,766,431
Total liabilities and stockholders'
equity $ 1,574,802 $ 1,994,730
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, 2017.
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version on businesswire.com: https://www.businesswire.com/news/home/20190207005784/en/
Alnylam Pharmaceuticals, Inc.Christine Regan
Lindenboom(Investors and Media)617-682-4340
Josh Brodsky(Investors)617-551-8276
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