– Advanced Pipeline of Seven Investigational
Clinical Programs and Presented Positive Clinical Results for
Three: Patisiran for Familial Amyloidotic Polyneuropathy (FAP),
ALN-AT3 for Hemophilia, and ALN-CC5 for Complement-Mediated
Diseases –
– In Addition to Upcoming Presentation of
ALN-PCSsc Clinical Results, Announces New Plan to Present Initial
Clinical Data for ALN-AS1 Porphyria Program at Medical Meeting in
September and Provides Update on Revusiran Phase 2 Open-Label
Extension Study –
– Maintained Strong Balance Sheet with $1.40
Billion in Cash and Remains On Track to End 2015 with Greater than
$1.2 Billion in Cash –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, today reported its consolidated financial
results for the second quarter 2015, and highlighted recent
progress in advancing its pipeline.
“The second quarter of 2015 and recent period were marked by
tremendous progress in our efforts to bring innovative medicines to
patients, as we execute on our ‘Alnylam 2020’ strategy. In our
patisiran Phase 2 open-label extension – or ‘OLE’ – study, we
believe to have generated increased evidence for a possible halting
of neuropathy progression after the first 12 months of treatment,
with study drug administration generally well tolerated out to 17
months. In our hemophilia program, we believe that recent results
from our ongoing Phase 1 trial provide confirmatory evidence that
ALN-AT3 has the potential to re-balance hemostasis in people with
severe hemophilia through normalization of thrombin generation. We
also reported initial positive data from our ongoing Phase 1/2
trial with ALN-CC5 – an investigational RNAi therapeutic for
complement-mediated diseases – where we demonstrated what we
believe to be promising preliminary clinical activity for single
doses of study drug with potent and highly durable knockdown of
serum C5 and inhibition of serum complement activity,” said John
Maraganore, Ph.D., Chief Executive Officer of Alnylam. “We also
completed enrollment in our ALN-PCSsc Phase 1 study with results to
be presented later this month, and we started dosing in our
patisiran APOLLO-OLE study and Phase 1 studies with ALN-AS1 and
ALN-AAT. We now enter a very data-rich back half of the year, with
important results being presented from six distinct clinical
programs. We very much look forward to sharing these results in the
weeks and months to come, as they highlight what we believe to be
the significant potential for RNAi therapeutics as a new class of
innovative medicines.”
Second Quarter 2015 and Recent Significant Corporate
Highlights
- Advanced pipeline of investigational
programs in Genetic Medicine Strategic Therapeutic Area (STAr).
- Advanced RNAi therapeutic programs for
the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR
amyloidosis).
- Continued enrollment in APOLLO Phase 3
study of patisiran in ATTR patients with Familial Amyloidotic
Polyneuropathy (FAP).
- Initiated Phase 3 open-label extension
study (“APOLLO-OLE”) with patisiran.
- Reported positive 12-month clinical
data from patisiran Phase 2 OLE study, showing sustained TTR
knockdown of up to a mean 88% and continued evidence for potential
halting of neuropathy progression with a mean 2.5 point decrease in
neuropathy impairment score (mNIS+7) at 12 months, comparing
favorably to a 13-18 point increase estimated from the literature
in untreated FAP patients with similar baseline characteristics;
patisiran was also found to be generally well tolerated out to 17
months of study drug administration.
- Continued enrollment in ENDEAVOUR Phase
3 study of revusiran in ATTR patients with Familial Amyloidotic
Cardiomyopathy (FAC).
- Continued dosing FAC and senile
systemic amyloidosis (SSA) patients in revusiran Phase 2 OLE study,
which was initiated in November 2014 and is designed to evaluate
the tolerability and clinical activity of revusiran with long-term
dosing for up to two years.
- The company reports today that three
patients have discontinued from the revusiran Phase 2 OLE study as
of August 5 due to injection site reactions (ISRs), including some
with associated diffuse rash; the study drug remains otherwise
generally well tolerated in the broader revusiran Phase 2 OLE study
population. The company plans to present initial clinical results
from the revusiran Phase 2 OLE study in late 2015.
- Received Orphan Drug Designation (ODD)
from the United States Food & Drug Administration (FDA) for
revusiran.
- The company announces today that it is
advancing a Development Candidate (DC) for an ESC-GalNAc-siRNA
targeting TTR that is expected to support a once-monthly and
possibly once-quarterly subcutaneous dose regimen. Further details
and guidance on this program will be presented at the
Oligonucleotide Therapeutics Society meeting to be held October 11
– 14, 2015 in Leiden, The Netherlands.
- Advanced ALN-AT3 for the treatment of
hemophilia and rare bleeding disorders (RBD).
- Reported positive interim results from
the ongoing Phase 1 study, including an up to 86% knockdown of
antithrombin (AT), and evidence for a potential re-balancing of
hemostasis resulting in normalization of thrombin generation up to
a mean increase of 350% and marked improvements in whole blood
clotting. In addition, in a post hoc exploratory analysis, AT
knockdown was found to be associated with reduced bleeding
frequency, with the maximum bleed-free interval of 114 days in a
patient with severe hemophilia A. ALN-AT3 was also shown to be
generally well tolerated, without any clinically significant
increases in D-dimer, a marker of pathologic clot formation.
- Presented new pre-clinical results with
ALN-AT3 and published pre-clinical study results in Nature
Medicine.
- These published pre-clinical data were
the centerpiece of a recent “Clinical Implications” article in the
New England Journal of Medicine.
- Presented initial positive Phase 1/2
study results with ALN-CC5 for the treatment of complement-mediated
diseases.
- Single subcutaneous doses of study drug
achieved potent, statistically significant, and highly durable C5
knockdown of up to 96% and an up to 92% inhibition of serum
complement activity, including an up to 61% inhibition of serum
hemolytic activity; single doses of ALN-CC5 were generally well
tolerated.
- Initiated Phase 1 study with ALN-AS1
for the treatment of acute hepatic porphyrias.
- The trial is being conducted initially
in asymptomatic high excreter (ASHE) patients with acute
intermittent porphyria (AIP), and then in AIP patients with
recurrent attacks.
- As detailed below, the company now
plans to present initial clinical data at a medical meeting in
mid-September 2015.
- Advanced ALN-AAT for the treatment of
alpha-1 antitrypsin (AAT) deficiency-associated liver disease
(alpha-1 liver disease).
- Initiated Phase 1/2 study with ALN-AAT.
The Phase 1/2 trial is being conducted initially in normal healthy
volunteers, and, then in patients with alpha-1 liver disease.
- Presented new pre-clinical data at the
Digestive Disease Week (DDW) meeting held in May 2015.
- Advanced ALN-GO1 for the treatment of
primary hyperoxaluria.
- Continued pre-clinical studies to
support selection of a DC for ALN-GO1 in mid-2015.
- Advanced investigational pipeline
programs in Cardio-Metabolic Disease and Hepatic Infectious Disease
STArs.
- The company announces today that it has
completed enrollment in the ALN-PCSsc Phase 1 study in normal human
volunteers with elevated LDL-C at baseline.
- Continued pre-clinical studies to
support a Clinical Trial Application (CTA) for ALN-HBV in late
2015.
- Signed 295,000 square foot lease with
an affiliate of BioMed Realty for Class A laboratory and office
space in Cambridge for the company’s future corporate
headquarters.
- Appointed David-Alexandre “DA” Gros,
M.D. to the position of Senior Vice President, Chief Business
Officer.
Upcoming 3Q2015 Events
- Alnylam announces today that it now
plans to present initial single ascending dose (SAD) cohort data
from its ALN-AS1 Phase 1 study in ASHE patients at the
International Congress of Porphyrins and Porphyrias (ICPP) 2015,
being held September 14 – 16, 2015 in Düsseldorf, Germany, in an
oral presentation on Tuesday, September 15.
- In addition, during the ICPP meeting,
Alnylam plans to present initial data from the EXPLORE trial, a
prospective observational study to characterize the disease burden
in patients with hepatic porphyrias.
- The company also announces today that
it plans to present complete 12-month data from the Phase 2 OLE
study with patisiran at the American Neurological Association (ANA)
2015 Annual Meeting, being held September 27 – 29, 2015 in Chicago,
in a poster presentation on Monday, September 28 at 5:30 pm CT
(6:30 pm ET).
- In addition, Alnylam plans to:
- Present data from the Phase 1 study of
ALN-PCSsc in development for the treatment of hypercholesterolemia
at the European Society of Cardiology (ESC) Congress 2015, being
held August 29 – September 2, 2015 in London, in a poster
presentation on Sunday, August 30 at 8:30 am British Summer Time
(3:30 am ET).
- Present pre-clinical data for ALN-GO1
in development for the treatment of Primary Hyperoxaluria Type 1
(PH1) at the 48th Annual Scientific Meeting of the European Society
of Paediatric Nephrology (ESPN), being held September 3 – 5, 2015
in Brussels, Belgium, in an oral presentation on September 5.
Upcoming RNAi Roundtables
Alnylam plans to continue hosting its series of online “RNAi
Roundtables” in August and September, including events focused on
the following RNAi therapeutics:
ALN-AAT for the treatment of AAT Deficiency-associated liver
diseaseFriday, August 14, 2:00 – 3:00 p.m. ET
- Alfica Sehgal, Ph.D., Principal
Scientist, Research
- Moderator: Akshay Vaishnaw, M.D.,
Ph.D., Executive Vice President of R&D, Chief Medical
Officer
- Guest Speaker: Jeffrey Teckman, M.D.,
Professor, Department of Pediatrics, St. Louis University School of
Medicine
Patisiran and Revusiran for the treatment of Transthyretin
(TTR)-Mediated AmyloidosisThursday, August 20, 9:00 – 10:30
a.m. ET
- Eric Green, Vice President, General
Manager, TTR Program
- Jared Gollob, M.D., Vice President,
Clinical Research
- Moderator: Barry Greene, President and
Chief Operating Officer
- Guest Speaker: Philip Hawkins, Ph.D.,
FRCP, FRCPath, FMedSci, Head, National Amyloidosis Centre, and
Head, Periodic Fever Syndrome Service/Honorary consultant
physician
- Guest Speaker: Isabelle Lousada,
President & CEO, Amyloidosis Research Consortium, and Chairman,
Amyloidosis Foundation
The company announces today that it has scheduled additional
RNAi Roundtables:
ALN-GO1 for the treatment of Primary Hyperoxaluria Type 1
(PH1)Tuesday, September 8, 9:00 – 10:00 a.m. ET
- David Erbe, Ph.D., Director,
Research
- Moderator: Barry Greene, President and
Chief Operating Officer
- Guest Speaker: Sally-Anne Hulton, M.D.,
FRCPCH, MRCP, FCP, MBBCh, Consultant Paediatric Nephrologist and
Clinical Lead, Birmingham Children’s Hospital NHS Trust
ALN-PCSsc for the treatment of
HypercholesterolemiaWednesday, September 16, 9:30 – 10:30 a.m.
ET
- Kevin Fitzgerald, Ph.D., Vice
President, Research
- David Kallend, MBBS, Vice President and
Global Medical Director, The Medicines Company
- Moderator: Barry Greene, President and
Chief Operating Officer
- Guest Speaker: Marc S. Sabatine, M.D.,
M.P.H., Chairman, Thrombolysis in Myocardial Infarction (TIMI)
Study Group at Brigham and Women’s Hospital, Lewis Dexter, MD
Distinguished Chair in Cardiovascular Medicine, and Professor of
Medicine, Harvard Medical School
ALN-AS1 for the treatment of Acute Hepatic
PorphyriasThursday, September 24, 11:00 am – 12:00 p.m. ET
- Bill Querbes, Ph.D., Associate
Director, Research
- Moderator: John Maraganore, Ph.D.,
Chief Executive Officer
- Guest Speaker: Robert J. Desnick, M.D.,
Ph.D., Dean for Genetics and Genomic Medicine, Professor and Chair
Emeritus, Department of Genetics and Genomic Sciences, Icahn School
of Medicine at Mount Sinai Hospital
Financials
“Alnylam continues to maintain a very strong balance sheet, with
approximately $1.4 billion in cash as of the end of the
second quarter of 2015,” said Michael Mason, Vice President,
Finance & Treasurer. “As for financial guidance this year, we
remain on track to end 2015 with greater than $1.2 billion in
cash.”
Cash, Cash Equivalents and Total Marketable Securities
At June 30, 2015, Alnylam had cash, cash equivalents and total
marketable securities of $1.40 billion, as compared to $881.9
million at December 31, 2014.
Non-GAAP Net Loss
The non-GAAP net loss for the second quarter of 2015 was $71.8
million, or $0.85 per share on both a basic and diluted basis as
compared to a non-GAAP net loss of $48.0 million, or $0.63 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 2014
excludes the $3.9 million reduction to in-process research and
development expense for the purchase of the Sirna RNAi assets from
Merck, described below, for which there is no corresponding expense
for the second quarter of 2015.
GAAP Net Loss
The net loss according to accounting principles generally
accepted in the U.S. (GAAP) for the second quarter of 2015 was
$71.8 million, or $0.85 per share on both a basic and diluted basis
(including $10.2 million, or $0.12 per share of non-cash
stock-based compensation expense), as compared to a net loss of
$44.1 million, or $0.58 per share on both a basic and diluted basis
(including $7.7 million, or $0.10 per share of non-cash stock-based
compensation expense), for the same period in the previous
year.
Revenues
Revenues were $8.7 million for the second quarter of 2015, as
compared to $7.3 million for the second quarter of 2014. Revenues
for the second quarter of 2015 included $3.4 million from the
company’s alliance with Takeda Pharmaceuticals Company Limited,
$2.6 million from the company’s alliance with The Medicines
Company, $2.6 million from the company’s alliance with Genzyme and
$0.1 million from research reagent licenses and other sources. The
increase in revenues in the second quarter of 2015 compared to the
second quarter of the previous year was due primarily to services
performed in connection with our performance obligations under
agreements with The Medicines Company and Genzyme, partially offset
by a decrease in revenue recognized under agreements with Monsanto
and Takeda. The company expects net revenues from collaborators to
decrease during the second half of 2015 on a comparative basis due
primarily to the completion of our revenue amortization under the
Takeda agreement in May 2015.
Research and Development Expenses
Research and development (R&D) expenses were $67.0 million
in the second quarter of 2015 which included $6.1 million of
non-cash stock-based compensation, as compared to $44.7 million in
the second quarter of 2014, which included $2.6 million of non-cash
stock-based compensation. The increase in R&D expenses in the
second quarter of 2015 as compared to the second quarter of the
prior year was due primarily to additional expenses related to the
significant advancement of certain of our clinical and pre-clinical
programs. In addition, compensation, non-cash stock-based
compensation and related expenses increased during the second
quarter of 2015 as compared to the second quarter of 2014 due
primarily to a significant increase in headcount during the period
as we continue to expand and advance our development pipeline. The
company expects that research and development expenses will
increase for the second half of 2015 as compared to the first half
of 2015.
In-Process Research and Development Expense
In the second quarter of 2014, the company recorded a reduction
of $3.9 million to in-process research and development expense in
connection with the purchase of the Sirna RNAi assets from Merck.
Upon the completion of certain technology transfer activities in
the second quarter of 2014, the company issued an additional
378,007 shares of common stock to Merck. In the second quarter of
2014, the company re-measured the expense recorded in the first
quarter of 2014 in connection with these shares using the price of
the company’s common stock on the issuance date.
General and Administrative Expenses
General and administrative (G&A) expenses were $14.6 million
in the second quarter of 2015, which included $4.0 million of
non-cash stock-based compensation, as compared to $11.5 million in
the second quarter of 2014, which included $5.1 million of non-cash
stock-based compensation. The increase in G&A expenses in the
second quarter of 2015 as compared to the second quarter of the
prior year was due primarily to an increase in consulting and
professional services related to an increase in general business
activities. For the second half of 2015, the company expects that
general and administrative expenses will increase slightly compared
to the first half of 2015.
Conference Call Information
Management will provide an update on the company, discuss second
quarter 2015 results, and discuss expectations for the future via
conference call on Thursday, August 6, 2015 at 4:30 p.m. ET. To
access the call, please dial 877-312-7507 (domestic) or
631-813-4828 (international) five minutes prior to the start
time and refer to conference ID 95481472. A replay of the call will
be available beginning at 7:30 p.m. ET on August 6, 2015. To access
the replay, please dial 855-859-2056 (domestic) or
404-537-3406 (international), and refer to conference ID
95481472.
About RNAi
RNAi (RNA interference) is a revolution in biology, representing
a breakthrough in understanding how genes are turned on and off in
cells, and a completely new approach to drug discovery and
development. Its discovery has been heralded as “a major scientific
breakthrough that happens once every decade or so,” and represents
one of the most promising and rapidly advancing frontiers in
biology and drug discovery today which was awarded the 2006 Nobel
Prize for Physiology or Medicine. RNAi is a natural process of gene
silencing that occurs in organisms ranging from plants to mammals.
By harnessing the natural biological process of RNAi occurring in
our cells, the creation of a major new class of medicines, known as
RNAi therapeutics, is on the horizon. Small interfering RNA
(siRNA), the molecules that mediate RNAi and comprise Alnylam's
RNAi therapeutic platform, target the cause of diseases by potently
silencing specific mRNAs, thereby preventing disease-causing
proteins from being made. RNAi therapeutics have the potential to
treat disease and help patients in a fundamentally new way.
About GalNAc Conjugates and Enhanced Stabilization Chemistry
(ESC) GalNAc Conjugates
GalNAc-siRNA conjugates are a proprietary Alnylam delivery
platform and are designed to achieve targeted delivery of RNAi
therapeutics to hepatocytes through uptake by the
asialoglycoprotein receptor. Alnylam’s Enhanced Stabilization
Chemistry (ESC) GalNAc-conjugate technology enables subcutaneous
dosing with increased potency, durability, and a wide therapeutic
index, and is being employed in several of Alnylam’s genetic
medicine programs, including programs in clinical development.
About LNP Technology
Alnylam has licenses to Tekmira LNP intellectual property for
use in RNAi therapeutic products using LNP technology.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel
therapeutics based on RNA interference, or RNAi. The company is
leading the translation of RNAi as a new class of innovative
medicines. Alnylam’s pipeline of investigational RNAi therapeutics
is focused in 3 Strategic Therapeutic Areas (STArs): Genetic
Medicines, with a broad pipeline of RNAi therapeutics for the
treatment of rare diseases; Cardio-Metabolic Disease, with a
pipeline of RNAi therapeutics toward genetically validated,
liver-expressed disease targets for unmet needs in cardiovascular
and metabolic diseases; and Hepatic Infectious Disease, with a
pipeline of RNAi therapeutics that address the major global health
challenges of hepatic infectious diseases. In early 2015, Alnylam
launched its “Alnylam 2020” guidance for the advancement and
commercialization of RNAi therapeutics as a whole new class of
innovative medicines. Specifically, by the end of 2020, Alnylam
expects to achieve a company profile with 3 marketed products, 10
RNAi therapeutic clinical programs – including 4 in late stages of
development – across its 3 STArs. The company’s demonstrated
commitment to RNAi therapeutics has enabled it to form major
alliances with leading companies including Merck, Medtronic,
Novartis, Biogen, Roche, Takeda, Kyowa Hakko Kirin, Cubist,
GlaxoSmithKline, Ascletis, Monsanto, The Medicines Company, and
Genzyme, a Sanofi company. In addition, Alnylam holds an equity
position in Regulus Therapeutics Inc., a company focused on
discovery, development, and commercialization of microRNA
therapeutics. Alnylam scientists and collaborators have published
their research on RNAi therapeutics in over 200 peer-reviewed
papers, including many in the world’s top scientific journals such
as Nature, Nature Medicine, Nature Biotechnology, Cell, New England
Journal of Medicine, and The Lancet. Founded in 2002, Alnylam
maintains headquarters in Cambridge, Massachusetts. For more
information about Alnylam’s pipeline of investigational RNAi
therapeutics, please visit www.alnylam.com.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam’s future
expectations, plans and prospects, including without limitation,
Alnylam’s expectations regarding its “Alnylam 2020” guidance,
Alnylam’s views with respect to the potential for RNAi
therapeutics, including patisiran, revusiran, ALN-AT3, ALN-CC5,
ALN-PCSsc, ALN-AS1, ALN-AAT, ALN-GO1 and its ESC-GalNAc-siRNA
targeting TTR, its expectations with respect to the timing,
execution, and success of its clinical and pre-clinical trials, the
expected timing of regulatory filings, including its plan to file
IND or IND equivalent applications, its expectations regarding
reporting of data from its clinical and pre-clinical studies,
including its studies for patisiran, revusiran, ALN-PCSsc, ALN-AS1,
ALN-HBV, ALN-GO1 and its ESC-GalNAc-siRNA targeting TTR, its plans
regarding commercialization of RNAi therapeutics, and Alnylam’s
expected cash position as of December 31, 2015, constitute
forward-looking statements for the purposes of the safe harbor
provisions under The Private Securities Litigation Reform Act of
1995. Actual results may differ materially from those indicated by
these forward-looking statements as a result of various important
factors, including, without limitation, Alnylam’s ability to manage
operating expenses, Alnylam’s ability to discover and develop novel
drug candidates and delivery approaches, successfully demonstrate
the efficacy and safety of its drug 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, actions of regulatory agencies, which may affect the
initiation, timing and progress of clinical trials, obtaining,
maintaining and protecting intellectual property, Alnylam’s ability
to enforce its patents against infringers and defend its patent
portfolio against challenges from third parties, obtaining
regulatory approval for products, competition from others using
technology similar to Alnylam’s and others developing products for
similar uses, Alnylam’s ability to 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,
marketing, sales and distribution of products, the outcome of
litigation, 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 to update any forward-looking
statements.
Alnylam Pharmaceuticals, Inc.
Unaudited Condensed Consolidated
Statements of Comprehensive Loss
(In thousands, except per share
amounts)
Three Months EndedJune
30,
Six Months EndedJune 30,
2015 2014 2015
2014 Net revenues from
collaborators $ 8,685 $ 7,295 $ 27,222 $ 15,570
Operating expenses:
Research and development (1)
67,007 44,672 125,042 88,430 In-process research and development —
(3,890) — 220,766 General and administrative (1) 14,622
11,518 27,346 20,443 Total operating expenses
81,629 52,300 152,388 329,639 Loss from
operations (72,944) (45,005) (125,166)
(314,069)
Other income (expense): Interest income 1,619 693
2,633 1,026 Other expense (27) (77) (27)
(159) Total other income 1,592 616
2,606 867 Loss before income taxes (71,352) (44,389)
(122,560) (313,202) (Provision for) Benefit from income taxes
(431) 315 — 18,185 Net loss $ (71,783)
$ (44,074) $ (122,560) $ (295,017) Net loss per common share -
basic and diluted $ (0.85) $ (0.58) $ (1.47) $ (4.11) Weighted
average common shares used to compute basic and diluted net loss
per common share 84,353 75,835 83,219
71,833
Comprehensive loss: Net loss $ (71,783) $
(44,074) $ (122,560) $ (295,017) Unrealized (loss) gain on
marketable securities, net of tax (33,623) (3,047)
(30,001) 2,266 Comprehensive loss $ (105,406) $
(47,121) $ (152,561) $ (292,751) (1) Non-cash
stock-based compensation expenses included in operating expenses
are as follows: Research and development $ 6,149 $ 2,557 $ 11,495 $
6,238 General and administrative 4,030 5,123 6,920 7,033
Alnylam Pharmaceuticals, Inc.
Unaudited GAAP to Non-GAAP
Reconciliation: Net Loss and Net Loss Per Share
(In thousands, except per share
amounts)
Three Months EndedJune
30,
Six Months EndedJune 30,
2015 2014 2015
2014 GAAP net loss $ (71,783) $ (44,074) $
(122,560) $ (295,017) Adjustment: In-process research and
development — (3,890) — 220,766
Non-GAAP net loss $ (71,783) $ (47,964) $ (122,560) $ (74,251)
GAAP net loss per common share - basic and diluted $
(0.85) $ (0.58) $ (1.47) $ (4.11) Adjustment (as detailed above)
— (0.05) — 3.08 Non-GAAP net loss per
common share - basic and diluted $ (0.85) $ (0.63) $ (1.47) $
(1.03)
Use of Non-GAAP Financial Measures
The company supplements its condensed consolidated financial
statements presented on a GAAP basis by providing additional
measures that are considered “non-GAAP” financial measures under
applicable SEC rules. These non-GAAP financial measures are not
prepared in accordance with generally accepted accounting
principles in the United States (GAAP) and should not be viewed in
isolation or as a substitute for GAAP net loss and basic and
diluted net loss per common share.
The company evaluates items on an individual basis, and
considers both the quantitative and qualitative aspects of the
item, including (i) its size and nature, (ii) whether or not it
relates to the company’s ongoing business operations, and (iii)
whether or not the company expects it to occur as part of its
normal business on a regular basis. In the second quarter of 2014
and first half of 2014, the company’s Non-GAAP net loss and
Non-GAAP loss per common share – basic and diluted financial
measures excludes the in-process research and development reduction
to expense of $3.9 million and expense of $220.8 million,
respectively, related to the purchase of the Sirna RNAi assets from
Merck. The company believes that the exclusion of this item
provides management and investors with supplemental measures of
performance that better reflect the underlying economics of the
company’s business. In addition, the company believes the exclusion
of this item is important in comparing current results with prior
period results and understanding projected operating performance.
Management uses these non-GAAP financial measures to establish
budgets and operational goals and to manage the company’s
business.
ALNYLAM PHARMACEUTICALS, INC.
UNAUDITED CONDENSED CONSOLIDATED
BALANCE SHEETS
(In thousands, except share
amounts)
June 30,
December 31, 2015
2014 Cash, cash equivalents and total marketable
securities $ 1,396,800 $ 881,929 Billed and unbilled collaboration
receivables 9,403 39,937 Prepaid expenses and other current assets
15,117 9,739 Deferred tax assets 19,654 31,667 Property and
equipment, net 25,169 21,740 Investment in equity securities of
Regulus Therapeutics Inc. 64,628 94,583
Total assets $ 1,530,771 $ 1,079,595 Accounts
payable and accrued expenses $ 33,473 $ 38,791 Deferred tax
liabilities 19,654 31,667 Total deferred revenue 62,716 66,854
Total deferred rent 6,324 6,016 Total stockholders’ equity (84.5
million and 77.2 million common shares issued and outstanding and
at June 30, 2015 and December 31, 2014, respectively)
1,408,604 936,267
Total liabilities and
stockholders' equity $ 1,530,771 $ 1,079,595
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, 2014.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20150806006291/en/
Alnylam Pharmaceuticals, Inc.Michael Mason,
617-551-8327Vice President, Finance and TreasurerorJoshua Brodsky,
617-551-8276Senior Manager, Investor Relations andCorporate
Communications
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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From Mar 2024 to Apr 2024
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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From Apr 2023 to Apr 2024