- Advanced Industry-Leading RNAi Therapeutics
Pipeline, Including Four Programs in Late-Stage Development -
- On Track to Report APOLLO Phase 3 Top-Line
Results for Patisiran in Near Term, and Initiated ATLAS Phase 3
Program for Fitusiran -
- Maintained Strong Balance Sheet with $1.25
Billion in Cash and Plans to End 2017 with Greater than $1.0
Billion in Cash -
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading
RNAi therapeutics company, today reported its consolidated
financial results for the second quarter 2017, and highlighted
recent progress in advancing its pipeline.
“2017 is shaping up to be a pivotal year for Alnylam. With
patisiran, we expect to report top-line APOLLO Phase 3 study
results in the coming weeks. If positive, these study findings will
support our first NDA filing, planned by year-end, and our
commercial transition in mid-2018 assuming regulatory approval. In
parallel, we continue to advance our late-stage pipeline of
investigational RNAi therapeutics, with the recently announced
initiation of our ATLAS Phase 3 program for fitusiran in hemophilia
and expected Phase 3 starts by year-end for givosiran in acute
hepatic porphyrias and, with The Medicines Company, inclisiran in
hypercholesterolemia,” said John Maraganore, Ph.D., Chief Executive
Officer of Alnylam. “We believe that these are all important and
defining milestones that position us to fulfill our ‘Alnylam 2020’
vision of becoming a multi-product, commercial-stage company with a
deep and sustainable clinical development pipeline by the end of
2020.”
Second Quarter 2017 and Recent Significant Corporate
Highlights
- Advanced patisiran, an investigational
RNAi therapeutic for the treatment of patients with hereditary ATTR
(hATTR) amyloidosis with polyneuropathy, with final 24-month data
from the Phase 2 open-label extension (OLE) study presented at the
American Academy of Neurology meeting and top-line APOLLO Phase 3
data expected in mid-2017.
- Advanced fitusiran, an investigational
RNAi therapeutic for the treatment of hemophilia and rare bleeding
disorders, with positive new data from the Phase 2 OLE study
presented at the International Society on Thrombosis and
Haemostasis 2017 Congress.
- Results from the Phase 1 study were
published in The New England Journal of Medicine in a paper titled,
“Targeting of Antithrombin in Hemophilia A or B with RNAi
Therapy.”
- Alnylam and partner Sanofi Genzyme
announced the initiation of the ATLAS Phase 3 program, a global,
multicenter clinical program designed to evaluate the safety and
efficacy of fitusiran in patients with hemophilia A and B with or
without inhibitors.
- Advanced givosiran, an investigational
RNAi therapeutic for the treatment of acute hepatic porphyrias
(AHPs), with positive new data presented at the 2017 International
Congress on Porphyrins and Porphyrias (ICPP) from the ongoing
randomized, double-blind, placebo-controlled Phase 1 study in
recurrent attack porphyria patients, as well as positive initial
results from the ongoing Phase 1 OLE study.
- In addition, received Breakthrough
Therapy designation from the U.S. Food and Drug Administration
(FDA) for givosiran for the prophylaxis of attacks in patients with
acute hepatic porphyria.
- Alnylam and partner The Medicines
Company announced agreement with the FDA on a Phase 3 clinical
program for inclisiran, an investigational RNAi therapeutic for the
treatment of hypercholesterolemia, with LDL-C lowering as the
primary endpoint for the initial pivotal trial program, which is
expected to initiate in late 2017.
- Completed successful public offering of
common stock, with concurrent private placement from Sanofi
Genzyme, totaling $376.5 million in net proceeds.
Upcoming Events
- Alnylam will continue to host its 4th
Annual RNAi Roundtable Series, which kicked off last week. This
series consists of webinars designed to inform attendees of the
latest progress and upcoming milestones for many of the company’s
investigational RNAi therapeutic programs. More details for the
series can be found here.
- Alnylam plans to report top-line
results from the patisiran APOLLO Phase 3 study in mid-2017. Full
results are expected to be presented in late 2017 at the 1st
European ATTR Amyloidosis Meeting for Patients and Doctors, being
held November 2-3, 2017 in Paris, France.
- If the APOLLO Phase 3 data are
positive, Alnylam expects to file its first New Drug Application
(NDA) in late 2017 and Marketing Authorisation Application (MAA)
shortly thereafter.
- Alnylam plans to initiate a Phase 3
study of givosiran in late 2017, pending successful alignment on
trial design with global regulatory authorities.
- The Medicines Company has announced its
intention to initiate a Phase 3 study of inclisiran in patients
with atherosclerotic cardiovascular disease (ASCVD) in late
2017.
Financial results for the quarter ended June 30, 2017
“Alnylam continues to maintain a strong balance sheet,” said
Manmeet Soni, Chief Financial Officer of Alnylam. “Our financial
strength allows us to continue to invest in a broad pipeline of
investigational RNAi therapeutics and prepare to transition towards
a commercial-stage company, aligned with our ‘Alnylam 2020’ goals
and strategy.”
Cash and InvestmentsAt June 30, 2017, Alnylam had cash, cash
equivalents and fixed income marketable securities, and restricted
investments of $1.25 billion, as compared to $1.09 billion at
December 31, 2016.
In May 2017, Alnylam sold an aggregate of 5,000,000 shares of
its common stock through an underwritten public offering at a price
to the public of $71.87 per share. As a result of the offering,
Alnylam received aggregate net proceeds of $355.2 million.
In addition, Sanofi Genzyme exercised its right to purchase, in
a concurrent private placement, 297,501 shares of common stock, at
the public offering price of $71.87 per share, resulting in
proceeds to Alnylam of $21.4 million.
GAAP and Non-GAAP Net LossThe net loss according to accounting
principles generally accepted in the U.S. (GAAP) for the second
quarter of 2017 was $118.4 million, or $1.34 per share on both a
basic and diluted basis, as compared to a net loss of $90.1
million, or $1.05 per share on both a basic and diluted basis, for
the same period in the previous year.
The non-GAAP net loss for the second quarter of 2017 was $94.4
million, or $1.07 per share on both a basic and diluted basis, as
compared to a non-GAAP net loss of $74.3 million, or $0.87 per
share on both a basic and diluted basis for the same period in the
previous year.
The non-GAAP net loss excludes stock-based compensation expense.
See “Use of Non-GAAP Financial Measures” below for a description of
non-GAAP financial measures and a reconciliation between GAAP and
non-GAAP net loss appearing later in this press release.
RevenuesRevenues were $15.9 million in the second quarter of
2017, as compared to $8.7 million in the second quarter of 2016.
Revenues for the second quarter of 2017 included $14.4 million from
the company’s alliance with Sanofi Genzyme and $1.5 million from
the company’s alliance with The Medicines Company.
Research and Development ExpensesResearch and development
(R&D) expenses were $90.6 million in the second quarter of
2017, which included $13.3 million of stock-based compensation, as
compared to $83.2 million in the second quarter of 2016, which
included $9.3 million of stock-based compensation.
General and Administrative ExpensesGeneral and administrative
(G&A) expenses were $45.8 million in the second quarter of
2017, which included $10.8 million of stock-based compensation, as
compared to $18.0 million in the second quarter of 2016, which
included $6.5 million of stock-based compensation.
Financial Guidance
Alnylam remains on track to end 2017 with greater than $1.0
billion in cash, cash equivalents and fixed marketable securities
including $150.0 million in restricted investments.
Conference Call InformationManagement will provide an
update on the company and discuss second quarter 2017 results as
well as expectations for the future via conference call on
Wednesday, August 9, 2017 at 4:30 p.m. ET. To access the call,
please dial 877-312-7507 (domestic) or 631-813-4828 (international)
five minutes prior to the start time and refer to conference ID
59171216. A replay of the call will be available beginning at 7:30
p.m. ET on the day of the call. To access the replay, please dial
855-859-2056 (domestic) or 404-537-3406 (international), and refer
to conference ID 59171216.
Alnylam – Sanofi Genzyme AllianceIn January 2014, Alnylam
and Sanofi Genzyme, the specialty care global business unit of
Sanofi, formed an alliance to accelerate the advancement of RNAi
therapeutics as a potential new class of innovative medicines for
patients around the world with rare genetic diseases. The alliance
enables Sanofi Genzyme to expand its rare disease pipeline with
Alnylam’s novel RNAi technology and provides access to Alnylam’s
R&D engine, while Alnylam benefits from Sanofi Genzyme’s proven
global capabilities to advance late-stage development and, upon
commercialization, accelerate market access for these promising
genetic medicine products.
In the case of patisiran, Alnylam will advance the product in
the United States, Canada and Western Europe, while Sanofi Genzyme
will advance the product in the rest of the world. In November
2016, Sanofi Genzyme elected to co-develop (through Sanofi R&D)
and co-commercialize fitusiran in the United States, Canada and
Western Europe, in addition to commercializing fitusiran in its
rest of world territories.
About RNAiRNAi (RNA interference) is a revolution in
biology, representing a breakthrough in understanding protein
synthesis in cells, and a completely new approach to drug discovery
and development. Its discovery has been heralded as "a major
scientific breakthrough that happens once every decade or so," and
represents one of the most promising and rapidly advancing
frontiers in biology and drug discovery today which was awarded the
2006 Nobel Prize for Physiology or Medicine. RNAi is a natural
process of gene silencing that occurs in organisms ranging from
plants to mammals. By harnessing the natural biological process of
RNAi occurring in our cells, the creation of a major new class of
medicines, known as RNAi therapeutics, is on the horizon. Small
interfering RNA (siRNA), the molecules that mediate RNAi and
comprise Alnylam's RNAi therapeutic platform, target the cause of
diseases by potently silencing specific mRNAs, with the goal of
preventing disease-causing proteins from being made.
About LNP TechnologyAlnylam has licenses to Arbutus LNP
intellectual property for use in RNAi therapeutic products using
LNP technology.
About Alnylam PharmaceuticalsAlnylam (Nasdaq: ALNY) is
leading the translation of RNA interference (RNAi) into a whole new
class of innovative medicines with the potential to transform the
lives of patients who have limited or inadequate treatment options.
Based on Nobel Prize-winning science, RNAi therapeutics represent a
powerful, clinically validated approach for the treatment of a wide
range of debilitating diseases. Founded in 2002, Alnylam is
delivering on a bold vision to turn scientific possibility into
reality, with a robust discovery platform and deep pipeline of
investigational medicines, including three product candidates that
are in late-stage development or will be in 2017. Looking forward,
Alnylam will continue to execute on its “Alnylam 2020” strategy of
building a multi-product, commercial-stage biopharmaceutical
company with a sustainable pipeline of RNAi-based medicines. For
more information about our people, science and pipeline, please
visit www.alnylam.com and engage with us on Twitter at
@Alnylam.
Use of Non-GAAP Financial MeasuresThis press release
contains non-GAAP financial measures, including net loss adjusted
to exclude certain non-cash expenses. These measures are not in
accordance with, or an alternative to, GAAP, and may be different
from non-GAAP financial measures used by other companies.
The item included in GAAP presentations but excluded for
purposes of determining non-GAAP financial measures for the periods
presented in this press release is stock-based compensation
expense. The company has excluded the impact of stock-based
compensation expense, which may fluctuate from period to period
based on factors including the variability associated with
performance-based grants for stock options and restricted stock
units and changes in the company’s stock price, which impacts the
fair value of these awards.
The company believes the presentation of non-GAAP net loss
provides useful information to management and investors regarding
the company’s financial condition and results of operations. When
GAAP financial measures are viewed in conjunction with non-GAAP
financial measures, investors are provided with a more meaningful
understanding of the company’s ongoing operating performance. In
addition, non-GAAP net loss is among those indicators the company
uses as a basis for evaluating performance, allocating resources
and planning and forecasting future periods. Non-GAAP financial
measures are not intended to be considered in isolation or as a
substitute for GAAP financial measures. A reconciliation between
GAAP and non-GAAP net loss is provided later in this press
release.
Alnylam Forward Looking 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
patisiran, fitusiran, givosiran, and inclisiran, its expectations
regarding the timing of clinical studies and the presentation of
clinical data, including for studies for patisiran, fitusiran,
givosiran, and inclisiran, its expectations regarding the potential
filing of an NDA and MAA for patisiran if the APOLLO Phase 3 study
is positive and its expected transition to commercial operations in
mid-2018 if regulators approve patisiran, its expected cash, cash
equivalents, fixed marketable securities and restricted investments
position as of December 31, 2017, and its “Alnylam 2020” guidance
for the advancement and commercialization of RNAi therapeutics,
constitute forward-looking statements for the purposes of the safe
harbor provisions under The Private Securities Litigation Reform
Act of 1995. Actual results and future plans may differ materially
from those indicated by these forward-looking statements as a
result of various important risks, uncertainties and other factors,
including, without limitation, Alnylam's ability to discover and
develop novel drug candidates and delivery approaches, successfully
demonstrate the efficacy and safety of its product candidates, the
pre-clinical and clinical results for its product candidates, which
may not be replicated or continue to occur in other subjects or in
additional studies or otherwise support further development of
product candidates for a specified indication or at all, actions or
advice of regulatory agencies, which may affect the design,
initiation, timing, continuation and/or progress of clinical trials
or result in the need for additional pre-clinical and/or clinical
testing, delays, interruptions or failures in the manufacture and
supply of our product candidates, obtaining, maintaining and
protecting intellectual property, Alnylam's ability to enforce its
intellectual property rights against third parties and defend its
patent portfolio against challenges from third parties, obtaining
and maintaining regulatory approval, pricing and reimbursement for
products, progress in establishing a commercial and ex-United
States infrastructure, competition from others using technology
similar to Alnylam's and others developing products for similar
uses, Alnylam's ability to manage its growth and operating
expenses, obtain additional funding to support its business
activities, and establish and maintain strategic business alliances
and new business initiatives, Alnylam's dependence on third parties
for development, manufacture and distribution of products, the
outcome of litigation, the risk of government investigations, and
unexpected expenditures, as well as those risks more fully
discussed in the "Risk Factors" filed with Alnylam's most recent
Quarterly Report on Form 10-Q filed with the Securities and
Exchange Commission (SEC) and in other filings that Alnylam
makes with the SEC. In addition, any forward-looking
statements represent Alnylam's views only as of today and should
not be relied upon as representing its views as of any subsequent
date. Alnylam explicitly disclaims any obligation, except to the
extent required by law, to update any forward-looking
statements.
The scientific information referenced in this news release
relating to Alnylam’s investigational therapeutics is preliminary
and investigative. None of Alnylam’s investigational therapeutics,
including inclisiran which is partnered with The Medicines Company,
have been approved by the U.S. Food and Drug Administration,
European Medicines Agency, or any other regulatory authority and no
conclusions can or should be drawn regarding the safety or
effectiveness of these therapeutics.
ALNYLAM
PHARMACEUTICALS, INC. UNAUDITED CONDENSED CONSOLIDATED
STATEMENTS OF COMPREHENSIVE LOSS (In thousands, except per
share amounts)
Three Months Ended June
30,
Six Months Ended June
30,
2017 2016 2017
2016 Net revenues from
collaborators $ 15,932 $ 8,709 $ 34,892 $
16,054
Operating expenses: Research and
development 90,627 83,172 177,611 179,445 General and
administrative 45,779 17,987
84,266 39,087 Total operating expenses
136,406 101,159 261,877
218,532 Loss from operations (120,474 )
(92,450 ) (226,985 ) (202,478 )
Other income
(expense): Interest income 2,577 2,092 4,705 3,905 Other
(expense) income (523 ) 229 (3,430 )
5,470 Total other income 2,054
2,321 1,275 9,375 Net loss $
(118,420 ) $ (90,129 ) $ (225,710 ) $ (193,103 ) Net loss per
common share - basic and diluted $ (1.34 ) $ (1.05 ) $ (2.59 ) $
(2.26 ) Weighted-average common shares used to compute basic and
diluted net loss per common share 88,098
85,545 87,068 85,411
Comprehensive loss: Net loss $ (118,420 ) $ (90,129 ) $
(225,710 ) $ (193,103 ) Unrealized loss on marketable securities,
net of tax (476 ) (18,331 ) (2,412 ) (26,555 ) Reclassification
adjustment for realized loss (gain) on marketable securities
included in net loss 345 (954 ) 1,894
(6,110 ) Comprehensive loss $ (118,551 ) $ (109,414 )
$ (226,228 ) $ (225,768 )
ALNYLAM PHARMACEUTICALS, INC.
RECONCILIATION OF GAAP NET LOSS TO NON-GAAP NET LOSS (In
thousands, except per share amounts)
Three Months Ended June
30,
Six Months Ended June
30,
2017 2016 2017
2016 GAAP net loss $ (118,420 ) $ (90,129 ) $
(225,710 ) $ (193,103 ) Adjustment: Stock-based compensation
expenses 24,030 15,816 39,747
39,296 Non-GAAP net loss $ (94,390 ) $ (74,313
) $ (185,963 ) $ (153,807 ) GAAP net loss per common
share - basic and diluted $ (1.34 ) $ (1.05 ) $ (2.59 ) $ (2.26 )
Adjustment (as detailed above) 0.27 0.18
0.45 0.46 Non-GAAP net loss per
common share - basic and diluted $ (1.07 ) $ (0.87 ) $ (2.14 ) $
(1.80 )
ALNYLAM PHARMACEUTICALS, INC. UNAUDITED CONDENSED
CONSOLIDATED BALANCE SHEETS (In thousands, except share
amounts)
June 30, December 31,
2017
2016 Cash, cash equivalents and fixed income marketable
securities $ 1,097,841 $ 942,601 Restricted investments 150,000
150,000 Billed and unbilled collaboration receivables 15,405 23,334
Prepaid expenses and other assets 23,504 32,303 Property, plant and
equipment, net 147,533
114,572
Total assets
$ 1,434,283 $ 1,262,810 Accounts
payable, accrued expenses and other liabilities $ 69,805 $ 99,650
Total deferred revenue 81,873 82,932 Total deferred rent 9,639
10,007 Long-term debt 150,000 150,000 Total stockholders’ equity
(91.7 million and 85.9 million common shares issued and outstanding
at June 30, 2017 and December 31, 2016, respectively)
1,122,966 920,221
Total liabilities and stockholders' equity
$ 1,434,283 $ 1,262,810
This selected financial information should be read in
conjunction with the consolidated financial statements and notes
thereto included in Alnylam’s Annual Report on Form 10-K which
includes the audited financial statements for the year ended
December 31, 2016.
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version on businesswire.com: http://www.businesswire.com/news/home/20170809005821/en/
Alnylam Pharmaceuticals, Inc.Investors and MediaChristine
Regan Lindenboom, 617-682-4340orInvestorsJosh Brodsky,
617-551-8276
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