− Obtained FDA and EMA Approvals of ONPATTRO™
(patisiran) – the First-Ever RNAi Therapeutic – and Launched in
U.S. and EU −
− Received 125 U.S. Patient Start Forms in
First Seven Weeks of ONPATTRO Launch −
− Reported Positive Topline Interim Analysis
Results from ENVISION Phase 3 Study of Givosiran in Patients with
Acute Hepatic Porphyrias −
− Advanced Lumasiran into ILLUMINATE Phase 3
Program −
− Maintained Strong Balance Sheet with $1.27
Billion in Cash and Expects to End 2018 with Approximately $1.0
Billion in Cash −
Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading
RNAi therapeutics company, today reported its consolidated
financial results for the third quarter 2018 and reviewed recent
commercial and R&D highlights.
“The third quarter and recent period were truly revolutionary
for Alnylam with the approval of ONPATTRO in both the U.S. and EU,
heralding the arrival of RNAi therapeutics as a whole new class of
medicines. With these approvals and the subsequent launches, we
have begun to realize the promise of RNAi therapeutics on a global
scale,” said John Maraganore, Ph.D., Chief Executive Officer of
Alnylam. “Our recent regulatory and commercial launch experiences
will be leveraged for our entire portfolio including late-stage
programs such as givosiran, where we will initiate a rolling NDA
submission this year; lumasiran, which we have advanced into
late-stage development with the recent initiation of the
ILLUMINATE-A Phase 3 study; and ALN-TTRsc02, which will enter Phase
3 later this year in the HELIOS-A study. We believe these
accomplishments put us well on our way toward achieving our Alnylam
2020 goal of building a multi-product, global, commercial-stage
company with a deep and sustainable clinical pipeline by the end of
2020.”
“With the approval and launch of ONPATTRO, Alnylam is now a
global commercial-stage company. With only seven weeks of results
for the third quarter, we’re encouraged by the number of U.S.
patient start forms, and emerging prescriber base, highlighting
what we believe is strong demand for ONPATTRO for adults with
polyneuropathy caused by hATTR amyloidosis. Moreover, we believe
our regional presence in North America, Europe, Asia, and, soon,
Latin America, along with established medical affairs and supply
chain capabilities, positions us to expand our efforts in markets
around the world,” said Barry Greene, President of Alnylam. “We
look forward to continuing our work toward strong commercial
execution, with a focus on raising disease awareness, improving
diagnosis, and bringing ONPATTRO to patients in need.”
Third Quarter 2018 and Recent Period Significant Corporate
Highlights
Commercial Highlights
- Launched ONPATTRO™ (patisiran) in the
U.S. and EU, initially in Germany.
- Received 125 U.S. patient Start Forms
as of September 30, 2018.
- Recognized ONPATTRO revenue of $0.5
million for the quarter ended September 30, 2018.
- Announced alignment on value-based
agreements with leading health insurers and launched Alnylam
Assist™, a comprehensive patient support services program for
ONPATTRO in the U.S.
R&D Highlights
- Achieved the first-ever regulatory
approval of an RNAi therapeutic, ONPATTRO (patisiran), in the U.S.
and EU.
- Received U.S. Food and Drug
Administration (FDA) approval of ONPATTRO for the treatment of the
polyneuropathy of hereditary transthyretin-mediated (hATTR)
amyloidosis in adults.
- Received marketing authorization from
the European Commission for ONPATTRO for the treatment of hATTR
amyloidosis in adult patients with stage 1 or stage 2
polyneuropathy.
- Continued global efforts to bring
ONPATTRO to patients with submission of a New Drug Application to
Japan’s Pharmaceuticals and Medical Devices Agency and receipt of a
Priority Review designation in Canada.
- Published results from the APOLLO Phase
3 study of patisiran in the July 5, 2018 issue of The New England
Journal of Medicine and APOLLO exploratory cardiac endpoint data in
the September 14, 2018 issue of Circulation.
- Advanced ALN-TTRsc02, a subcutaneously
administered investigational RNAi therapeutic in development for
the treatment of ATTR amyloidosis.
- Aligned the design of HELIOS-A, a
pivotal Phase 3 study of ALN-TTRsc02 in patients with hATTR
amyloidosis polyneuropathy, with FDA and European Medicines Agency
(EMA) feedback.
- The Company is on track to start the
HELIOS-A study in late 2018 and plans to initiate additional Phase
3 studies of ALN-TTRsc02, including in hereditary and wild-type
ATTR amyloidosis cardiomyopathy, in 2019.
- Advanced givosiran, an investigational
RNAi therapeutic in development for the treatment of acute hepatic
porphyrias (AHPs).
- Announced positive topline results from
the interim analysis of the ENVISION Phase 3 study of
givosiran.
- Announced plans to initiate a rolling
submission of a New Drug Application (NDA) and pursue full approval
based on complete results – now expected in early 2019 – from the
ENVISION Phase 3 study. The rolling NDA submission is expected to
be initiated in 2018, with full clinical sections submitted in
mid-2019, assuming positive results.
- Advanced lumasiran, an investigational
RNAi therapeutic in development for the treatment of primary
hyperoxaluria type 1 (PH1).
- Announced initiation of ILLUMINATE-A, a
global Phase 3 pivotal trial of lumasiran in children and adults
with PH1. 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 and the American Society of Nephrology
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.
- Expanded the Alnylam Act® program to
include no-charge, third-party genetic testing and counseling for
adults and children who may carry a mutation in the gene encoding
alanine-glyoxylate aminotransferase (AGXT), which is associated
with PH1.
- Alnylam’s partner, The Medicines
Company, announced in October that the Independent Data Monitoring
Committee for the ongoing inclisiran Phase 3 clinical trials (ORION
9, 10, and 11) conducted its fourth 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 1,899 years of patient exposure to an RNAi therapeutic,
representing the industry’s most comprehensive body of safety data
for an RNAi 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.
- 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), which
is based on Alnylam’s Enhanced Stabilization Chemistry-Plus (ESC+)
GalNAc conjugate technology.
- The Company announces today that due to
recruitment challenges, it has discontinued a Phase 2 study of
cemdisiran in atypical hemolytic uremic syndrome (aHUS). Alnylam
will now focus its cemdisiran clinical efforts on a Phase 2 study
in IgA nephropathy.
- Reported new platform innovations at
the Oligonucleotide Therapeutics Society 2018 Annual Meeting,
including pre-clinical results demonstrating CNS and ocular
delivery of RNAi therapeutics in rats and non-human primates.
Additional Business Updates
- Expanded organization with key
appointments and new hires.
- Appointed Dr. Margaret Hamburg, former
FDA Commissioner, to the Board of Directors, effective January 10,
2019. Concurrent with Dr. Hamburg’s appointment, Mr. John Clarke is
resigning from the Board after sixteen years of service.
- Alnylam announces today the promotion
of Andy Orth as Senior Vice President, Head of U.S. In this role,
he is responsible for commercial execution of Alnylam programs in
the U.S. market. He was previously Alnylam’s Vice President of
Commercial Operations, and joined the Company in 2016 from Biogen.
Prior to Biogen, he held commercial and finance leadership roles at
Genzyme and Amgen.
- Alnylam also announces today the
appointment of Norton Oliveira as Senior Vice President, Head of
Latin America. He joins Alnylam from Gilead Sciences where he was
Vice President for Latin America and the Caribbean. Prior to
Gilead, Norton held commercial leadership roles at Merck/MSD and
Shire.
Upcoming Events
In late 2018, Alnylam intends to:
- Initiate a rolling submission of an NDA
with the FDA for givosiran, with full clinical sections to be
submitted in mid-2019, assuming positive results.
- Initiate the HELIOS-A Phase 3 study for
ALN-TTRsc02 in hATTR amyloidosis.
- Initiate the Phase 1/2 study for
ALN-AAT02 in alpha-1 liver disease.
- File a Clinical Trial Authorization
(CTA) application for ALN-HBV02 (also known as VIR-2218), in
partnership with Vir Biotechnology, for the treatment of chronic
hepatitis B virus infection.
- Select its first CNS-targeted
development candidate (DC) program.
- Hold an R&D Day Investor Conference
on December 6 in New York City.
Financial results for the quarter ended September 30,
2018
“We are pleased to have recognized initial revenue for an
Alnylam product for the first time in the Company’s history,
following the August FDA approval of ONPATTRO,” said Manmeet Soni,
Chief Financial Officer of Alnylam. “With cash and investments on
our balance sheet standing at $1.27 billion at September 30, and
expectations to finish the year with approximately $1.0 billion, we
believe Alnylam is in a strong position to continue executing on
global commercial operations while advancing our pipeline programs
through the clinic.”
Cash and InvestmentsAt September 30, 2018, Alnylam had cash,
cash equivalents and marketable debt securities, and restricted
investments, excluding equity securities, of $1.27 billion, as
compared to $1.73 billion at December 31, 2017.
GAAP and Non-GAAP Net LossThe net loss according to accounting
principles generally accepted in the U.S. (GAAP) for the third
quarter of 2018 was $245.3 million, or $2.43 per share on both a
basic and diluted basis, as compared to a net loss of $122.9
million, or $1.34 per share on both a basic and diluted basis, for
the same period in the previous year.
The non-GAAP net loss for the third quarter of 2018 was $157.3
million, or $1.56 per share on both a basic and diluted basis, as
compared to a non-GAAP net loss of $97.0 million, or $1.06 per
share on both a basic and diluted basis, for the same period in the
previous year.
The non-GAAP net loss for the third quarter of 2018 and 2017
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.
ONPATTRO Revenues, NetNet product revenues from sales of
ONPATTRO were $0.5 million in the third quarter of 2018 following
its approval by the FDA in August 2018.
Net Revenues from CollaboratorsNet revenues from collaborators
were $1.6 million in the third quarter of 2018 as compared to $17.1
million in the third quarter of 2017.
GAAP and Non-GAAP Research and Development ExpensesGAAP research
and development (R&D) expenses were $139.9 million in the third
quarter of 2018 as compared to $95.3 million in the third quarter
of 2017.
Non-GAAP R&D expenses were $94.2 million in the third
quarter of 2018 as compared to $80.2 million in the third quarter
of 2017. 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
ExpensesGAAP selling, general and administrative (SG&A)
expenses were $116.5 million in the third quarter of 2018 as
compared to $47.6 million in the third quarter of 2017.
Non-GAAP SG&A expenses were $74.4 million in the third
quarter of 2018 as compared to $36.8 million in the third quarter
of 2017. 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.
2018 Financial GuidanceAlnylam reiterates its
expectations to end 2018 with approximately $1.0 billion of cash,
cash equivalents and marketable debt securities, restricted cash
and restricted investments, excluding equity securities.
The Company reiterates its expectations for 2018 annual non-GAAP
R&D expenses to be in the range of $420 million to $460 million
and non-GAAP SG&A expenses to be in the range of $280 million
to $320 million. Both non-GAAP R&D and non-GAAP SG&A
expenses exclude stock-based compensation expenses.
Use of Non-GAAP Financial MeasuresThis 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.
Conference Call InformationManagement will provide an
update on the Company and discuss third quarter 2018 and recent
period results as well as expectations for the future via
conference call on Wednesday, November 7, 2018 at 8:30 am 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 7650424. A replay of the call will be
available beginning at 11:30 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
7650424.
Alnylam – Sanofi Genzyme AllianceAlnylam 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 RNAiRNAi (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 LNP TechnologyAlnylam has licenses to Arbutus
Biopharma LNP intellectual property for use in RNAi therapeutic
products using LNP technology.
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
specific messenger RNA, potentially blocking the production of TTR
protein before it is made. Patisiran blocks the production of
transthyretin 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 ReactionsInfusion-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
SupplementationONPATTRO 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 ReactionsThe most common adverse reactions that
occurred in patients treated with ONPATTRO were respiratory-tract
infection (29 percent) and infusion-related reactions (19
percent).
About Alnylam PharmaceuticalsAlnylam (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) 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 StatementsVarious 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
givosiran, ALN-TTRsc02, lumasiran, inclisiran, ALN-AAT02 and
cemdisiran, its plans for additional regulatory filings and product
launches for ONPATTRO, including in Japan and Latin America, its
expectations regarding the timing for initiation of a rolling NDA
submission with the FDA for givosiran and the reporting of complete
topline results, its expectations regarding the potential timing of
ILLUMINATE-A Phase 3 study results for lumasiran in late 2019 and a
possible filing of an NDA 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 3 study for ALN-TTRsc02 in
2018 in hATTR amyloidosis and its plans to initiate additional
Phase 3 studies of ALN-TTRsc02, including in hereditary and
wild-type ATTR amyloidosis cardiomyopathy, in 2019, its plan to
initiate the Phase 1/2 study for ALN-AAT02 in alpha-1 liver disease
and to file a CTA application for ALN-HBV02 in partnership with Vir
Biotechnology, its plans to select its first CNS-targeted DC
program, its expected cash, cash equivalents and marketable debt
securities, restricted cash and restricted investments balance,
excluding equity securities, as of December 31, 2018, its expected
range of 2018 annual non-GAAP R&D expenses and non-GAAP
SG&A expenses, 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 Ended
September 30,
Nine Months Ended
September 30,
2018 2017 2018 2017
Revenues: Product revenues, net $ 460 $ — $ 460 $ —
Net revenues from collaborators 1,609 17,096
53,415 51,988
Total revenues $ 2,069 $ 17,096 $
53,875 $ 51,988
Cost and expenses: Cost of goods sold
137 — 137 — Research and development 139,945 95,252 374,384 272,863
Selling, general and administrative 116,545 47,644
273,671 131,910 Total costs and expenses
256,627 142,896 648,192 404,773 Loss from
operations (254,558) (125,800) (594,317)
(352,785)
Other income (expense): Interest income
6,796 3,296 18,691 8,001 Other income (expense) 2,925 (433) 5,468
(3,863) Gain on litigation settlement — —
20,564 — Total other income 9,721 2,863
44,723 4,138 Loss before income taxes (244,837) (122,937)
(549,594) (348,647) Provision for income taxes (445)
— (462) — Net loss $ (245,282) $ (122,937) $
(550,056) $ (348,647) Net loss per common share - basic and diluted
$ (2.43) $ (1.34) $ (5.48) $ (3.93) Weighted-average common shares
used to compute basic and diluted net loss per common share
100,783 91,828 100,430 88,672
Comprehensive loss: Net loss $ (245,282) $ (122,937) $
(550,056) $ (348,647) Unrealized gain (loss) on marketable
securities, net of tax 415 218 1,041 (2,194) Reclassification
adjustment for realized loss on marketable securities included in
net loss — — — 1,894 Comprehensive loss
$ (244,867) $ (122,719) $ (549,015) $ (348,947)
ALNYLAM PHARMACEUTICALS, INC.
RECONCILIATION OF SELECTED GAAP
MEASURES TO NON-GAAP MEASURES
(In thousands, except per share
amounts)
Three Months Ended
September 30,
Nine Months Ended
September 30,
2018 2017 2018 2017
Reconciliation of GAAP to Non-GAAP Research and development:
GAAP Research and development $ 139,945 $ 95,252 $ 374,384 $
272,863 Less: Stock-based compensation expenses (45,784)
(15,090) (67,537) (37,035) Non-GAAP Research
and development $ 94,161 $ 80,162 $ 306,847 $ 235,828
Reconciliation of GAAP to Non-GAAP Selling, general and
administrative: GAAP Selling, general and administrative $
116,545 $ 47,644 $ 273,671 $ 131,910 Less: Stock-based compensation
expenses (42,170) (10,865) (62,242)
(28,667) Non-GAAP Selling, general and administrative $ 74,375 $
36,779 $ 211,429 $ 103,243
Reconciliation of GAAP to
Non-GAAP Operating costs and expenses: GAAP Operating costs and
expenses $ 256,627 $ 142,896 $ 648,192 $ 404,773 Less: Stock-based
compensation expenses (87,954) (25,955)
(129,779) (65,702 ) Non-GAAP Operating costs and expenses $
168,673 $ 116,941 $ 518,413 $ 339,071
Reconciliation of
GAAP to Non-GAAP Net loss: GAAP Net loss $ (245,282) $
(122,937) $ (550,056) $ (348,647 ) Add: Stock-based compensation
expenses 87,954 25,955 129,779 65,702 Less: Gain on litigation
settlement — — (20,564) — Non-GAAP Net
loss $ (157,328) $ (96,982) $ (440,841) $ (282,945 )
Reconciliation of GAAP to Non-GAAP Net loss per common
share-basic and diluted: GAAP Net loss per common share - basic
and diluted $ (2.43) $ (1.34) $ (5.48) $ (3.93) Add: Stock-based
compensation expenses 0.87 0.28 1.29 0.74 Less: Gain on litigation
settlement — — (0.20) — Non-GAAP Net
loss per common share - basic and diluted $ (1.56) $ (1.06) $
(4.39) $ (3.19)
ALNYLAM PHARMACEUTICALS, INC.
UNAUDITED CONDENSED CONSOLIDATED
BALANCE SHEETS
(In thousands, except share
amounts)
September
30, December 31, 2018
2017 Cash, cash equivalents and marketable
debt securities $ 1,221,830 $ 1,704,537 Restricted investments
44,825 30,000 Accounts receivable, net 3,362 34,002 Inventory
11,081 — Prepaid expenses and other assets 121,904 44,291 Property,
plant and equipment, net 272,652
181,900
Total assets $ 1,675,654 $
1,994,730 Accounts payable, accrued expenses and other liabilities
$ 119,671 $ 104,905 Total deferred revenue 5,067 84,780 Total
deferred rent 42,797 8,614 Long-term debt 30,000 30,000 Total
stockholders’ equity (101.0 million and 99.7 million common shares
issued and outstanding at September 30, 2018 and December 31, 2017,
respectively) 1,478,119
1,766,431
Total liabilities and stockholders' equity
$ 1,675,654 $ 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/20181107005464/en/
Alnylam Pharmaceuticals, Inc.Christine Regan
Lindenboom(Investors and Media)617-682-4340orJosh
Brodsky(Investors)617-551-8276
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