– Advances Pipeline of Ten Clinical Stage
Programs, with Ten Additional Clinical Data Readouts Anticipated
This Year –
– Expects to Complete Enrollment of Revusiran
Phase 3 ENDEAVOUR Trial in Late 2016 with Data Readout in Mid-2018
–
– On Track to Start Fitusiran Phase 3 Program
in Mid-2016; Plans to Report Updated Hemophilia Patient Data in
July –
– Provides Update on ALN-CC5 Program and
Development Path Forward; Plans to Present Initial Data in
Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients in June –
– Ends Quarter with $1.2 Billion in Cash and
Increases Year-End Cash Guidance to Over $1 Billion, Including $150
Million of Restricted Marketable Securities –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today reported its consolidated financial
results for the first quarter 2016, and highlighted recent progress
in advancing its pipeline.
“At Alnylam, we continue to advance a broad pipeline of
investigational RNAi therapeutics – including 10 programs in
clinical development and 2 programs in Phase 3 – across a broad
range of disease indications. A major milestone in the first
quarter was completion of enrollment in our APOLLO Phase 3 trial
for patisiran, and we’re on track to view results in mid-2017. If
positive, we expect to file our first regulatory applications for
approval later that same year. We’re also making strong progress in
our ENDEAVOUR Phase 3 trial of revusiran, where we now expect
completion of enrollment in late 2016 and data readout in mid-2018.
In our fitusiran program in hemophilia, we look forward to
presenting important new results in July, including initial results
in patients with inhibitors, and are on track to start our two
Phase 3 studies shortly thereafter,” said John Maraganore, Ph.D.,
Chief Executive Officer at Alnylam. “We’re also making progress
with our earlier stage clinical programs. In our ALN-CC5 program,
initial results in PNH patients, which will be presented at EHA
next month, point to an optimal development path forward in PNH for
eculizumab poor responders and for eculizumab sparing, with
parallel efforts in other complement-mediated diseases. Finally, we
also filed Clinical Trial Applications for ALN-HBV and ALN-TTRsc02,
and initiated our Phase 1 study for our ALN-GO1 program in primary
hyperoxaluria. We look forward to sharing our continued progress
throughout the course of a very data rich 2016.”
First Quarter 2016 and Recent Significant Corporate
Highlights
- Advanced investigational pipeline
programs in Genetic Medicine Strategic Therapeutic Area (STAr).
- Advanced investigational RNAi
therapeutics programs for the treatment of transthyrethin
(TTR)-mediated amyloidosis (ATTR amyloidosis).
- Completed enrollment in the APOLLO
Phase 3 trial with patisiran for the treatment of hereditary
TTR-mediated amyloidosis with polyneuropathy (hATTR-PN), also known
as familial amyloidotic polyneuropathy (FAP).
- Based on strong investigator and
patient interest, the study was substantially over enrolled with
225 patients.
- Data from APOLLO are expected in
mid-2017, and assuming positive results, the Company expects to
submit an NDA and MAA for patisiran by the end of 2017 and launch
in 2018.
- Announced complete 18-month data from
ongoing Phase 2 open-label extension (OLE) study with patisiran.
- Data presented at the American Academy
of Neurology (AAN) Meeting provided continued evidence that
patisiran has the potential to halt neuropathy progression in
patients with hATTR-PN. In the first reported exploratory analysis
of its kind, the degree of TTR knockdown observed in patients was
shown to correlate with improvement in neuropathy impairment
scores. Further, patisiran was found to be generally well tolerated
with no drug related serious adverse events up to 25 months of
treatment. The majority of adverse events were mild to
moderate.
- Continued enrollment in ENDEAVOUR Phase
3 study with revusiran for the treatment of hereditary TTR-mediated
amyloidosis with cardiomyopathy (hATTR-CM), also known as familial
amyloidotic cardiomyopathy (FAC).
- The Company announced today that it
expects to complete ENDEAVOUR enrollment in late 2016 and report
results in mid-2018.
- Filed Clinical Trial Application (CTA)
for ALN-TTRsc02, an ESC-GalNAc-siRNA conjugate targeting TTR for
the treatment of ATTR amyloidosis, which is expected to enable a
once- quarterly subcutaneous dosing regimen.
- Assuming a positive Phase 1 study, the
Company plans to initiate a Phase 3 trial in 2017.
- Advanced fitusiran (ALN-AT3) for the
treatment of hemophilia and rare bleeding disorders (RBD).
- Initiated dosing of hemophilia patients
with inhibitors in Part D of an ongoing Phase 1 clinical trial
evaluating a once-monthly subcutaneous dose regimen of fitusiran.
Both patients with hemophilia A with inhibitors and hemophilia B
with inhibitors have now been dosed with fitusiran.
- Continued dosing patients in an ongoing
Phase 1 OLE, where once-monthly doses of fitusiran are administered
to patients with moderate or severe hemophilia A or B with or
without inhibitors.
- Alnylam is on track to initiate two
Phase 3 trials: the first in mid-2016 in hemophilia A and B
patients with inhibitors; and, the second in late 2016 in moderate
or severe hemophilia A and B patients without inhibitors.
- The Company has initiated discussions
with global regulatory authorities to confirm specific trial
designs.
- Advanced ALN-CC5 for the treatment of
complement-mediated diseases.
- The Company announced today that it has
achieved preliminary evidence for clinical activity in a small
number of paroxysmal nocturnal hemoglobinuria (PNH) patients
enrolled in Part C of its ongoing Phase 1/2 trial, and it believes
that based on LDH data, the optimal development path for ALN-CC5 in
PNH is for eculizumab poor responders and for eculizumab sparing.
- The Company now plans to transition
toward a new Phase 2 study in PNH patients focused on that
development plan, which is expected to start by end of the
year.
- The Company plans to present initial
data from a small cohort of PNH patients in the ongoing Phase 1/2
study at the European Hematology Association (EHA) Meeting in June,
as listed below.
- The Company also expects to initiate
studies of ALN-CC5 as monotherapy and/or in combination with
anti-C5 monoclonal antibodies in additional complement-mediated
disease indications, such as atypical hemolytic uremic syndrome
(aHUS) and myasthenia gravis, starting in early 2017.
- Advanced ALN-AS1 for the treatment of
acute hepatic porphyrias.
- Transitioned to Part C in ongoing Phase
1 study where ALN-AS1 is being evaluated in porphyria patients with
recurrent attacks.
- The Company plans to initiate a Phase 3
trial in 2017, if the Phase 1 study results are positive.
- Advanced ALN-AAT for the treatment of
alpha-1 antitrypsin (AAT) deficiency-associated liver disease.
- Advanced ALN-GO1 for the treatment of
primary hyperoxaluria type 1 (PH1).
- Initiated a Phase 1/2 clinical trial
that is being conducted initially in normal healthy volunteers, and
then will be conducted in patients with PH1.
- Received Orphan Drug Designations for
ALN-GO1 from the European Medicines Agency (EMA) and
the U.S. Food and Drug Administration (FDA).
- Added ALN-F12 as a new program in
development pipeline.
- ALN-F12 is an RNAi therapeutic
targeting factor XII in development for the treatment of hereditary
angioedema and for thromboprophylaxis.
- Advanced investigational pipeline
programs in Cardio-Metabolic Disease STAr.
- The Medicines Company continued
enrollment in the Phase 2 ORION-1 trial for ALN-PCSsc.
- Advanced investigational pipeline
programs in Hepatic Infectious Disease STAr.
- Filed and obtained approval for CTA for
ALN-HBV, an RNAi therapeutic targeting the Hepatitis B Virus (HBV)
genome for the treatment of HBV infection. The Company is on
track to start a Phase 1 study in mid-2016.
- Expanded Management Team
- Expanded Management Team with
appointments of Patrick Berreby, Vice President of Supply Chain;
Jae Kim, Vice President, Clinical Development; and Andy Orth, Vice
President of Commercial Practice.
Upcoming Events in Mid- and Late 2016
- Alnylam announced today that it will:
- Present updated human volunteer data
from the ongoing ALN-CC5 Phase 1/2 study in a poster presentation
on May 22, 2016 at the 53rd Congress of the European Renal
Association – European Dialysis and Transplant Association
(ERA-EDTA) in Vienna, Austria.
- Present initial ALN-CC5 results in PNH
patients during an oral presentation on June 11, 2016 at the 21st
Congress of the European Hematology Association (EHA) Meeting in
Copenhagen, Denmark.
- Present updated fitusiran Phase 1 data
during an oral presentation on July 27, 2016 at the World
Federation of Hemophilia (WFH) 2016 World Congress in Orlando,
Florida.
- Additional upcoming milestones for
Alnylam pipeline programs include:
- In mid-2016, Alnylam plans to:
- Present 24-month Phase 2 OLE data with
patisiran, likely at the International Symposium on Amyloidosis
(ISA) Meeting to be held July 3 – 7, 2016 in Uppsala, Sweden,
pending abstract acceptance;
- Present 12-month Phase 2 OLE data with
revusiran, also likely at the ISA Meeting, pending abstract
acceptance;
- Start first fitusiran Phase 3 study in
hemophilia A and B patients with inhibitors;
- Present initial Phase 1 data for
ALN-AAT;
- Start ALN-HBV Phase 1 study; and
- Start ALN-TTRsc02 Phase 1 study.
- In late 2016, Alnylam plans to:
- Present additional Phase 1 and initial
Phase 1 OLE data with fitusiran;
- Start second fitusiran Phase 3 study in
moderate or severe hemophilia A and B patients without
inhibitors;
- Present initial ALN-AS1 data in
recurrent attack porphyria patients;
- Present initial ALN-GO1 data in PH1
patients;
- Present initial ALN-TTRsc02 Phase 1
data;
- File a CTA for a new Genetic Medicine
program; and
- Consistent with guidance from The
Medicines Company, present initial Phase 2 data for ALN-PCSsc.
Financials
“Alnylam continues to maintain a strong balance sheet, ending
the first quarter of 2016 with approximately $1.2 billion in cash,”
said Michael Mason, Vice President, Finance and Treasurer. “Our
financial strength allows us to continue to invest in a broad
pipeline of investigational RNAi therapeutics across our three
STArs, aligned with achievement of our ‘Alnylam 2020’ goals. As for
financial guidance this year, we are updating cash guidance today
to end 2016 with greater than $1.0 billion in cash, including
$150.0 million of restricted marketable securities that we received
from new credit agreements – related to the build out of our new
manufacturing facility – entered into in April 2016.”
Cash, Cash Equivalents and Total Marketable Securities
At March 31, 2016, Alnylam had cash, cash equivalents and total
marketable securities of $1.21 billion, as compared to $1.28
billion at December 31, 2015. In April 2016, the company entered
into credit agreements described below with proceeds of $150.0
million of restricted marketable securities.
Credit Agreements
In April 2016, Alnylam entered into credit agreements, related
to the build out of the Company’s new manufacturing facility, that
provide for a $150.0 million term loan facility, and mature in
April 2021. Interest on the borrowings will be calculated based on
LIBOR plus 0.45 percent. The obligations under the credit
agreements are secured by cash collateral in an amount equal to, at
any given time, at least 100 percent of the principal amount of all
term loans outstanding under the credit agreements at such
time.
GAAP Net Loss
The net loss according to accounting principles generally
accepted in the U.S. (GAAP) for the first quarter of 2016 was
$103.0 million, or $1.21 per share on both a basic and diluted
basis (including $23.5 million, or $0.28 per share of non-cash
stock-based compensation expense), as compared to a net loss of
$50.8 million, or $0.62 per share on both a basic and diluted basis
(including $8.2 million, or $0.10 per share of non-cash stock-based
compensation expense), for the same period in the previous
year.
Revenues
Revenues were $7.3 million for the first quarter of 2016, as
compared to $18.5 million for the same period last year. Revenues
for the first quarter of 2016 included $4.4 million from the
company’s alliance with Sanofi Genzyme, $2.7 million from the
company’s alliance with The Medicines Company and $0.2 million from
other sources. The decrease in revenues in the quarter ended March
31, 2016 as compared to the prior year period was due primarily to
the completion of the company’s performance obligations under the
Monsanto agreement in February 2015 and the completion of its
revenue amortization under the Takeda agreement in May 2015,
partially offset by higher revenue from its agreement with Sanofi
Genzyme. The company expects net revenues from collaborators to
increase during the remainder of 2016 due to expected increases in
expense reimbursement and an expected milestone payment under its
agreement with Sanofi Genzyme.
Research and Development Expenses
Research and development (R&D) expenses were $96.3 million
in the first quarter of 2016 which included $14.4 million of
non-cash stock-based compensation, as compared to $58.0 million in
the first quarter of 2015, which included $5.3 million of non-cash
stock-based compensation. The increase in R&D expenses for the
quarter ended March 31, 2016 as compared to the prior year period
was due primarily to higher clinical trial and manufacturing and
external services expenses resulting from the significant
advancement of the company’s Genetic Medicine pipeline. In
addition, compensation and related expenses and non-cash
stock-based compensation expenses increased during the quarter
ended March 31, 2016 as compared to the quarter ended March 31,
2015 due primarily to a significant increase in headcount during
the period as the company expands and advances its development
pipeline, as well as the vesting of certain performance-based stock
option awards during the quarter ended March 31, 2016. The company
expects that on a quarterly basis in 2016 R&D expenses will
increase from the first quarter as it continues to develop its
pipeline and advance its product candidates into clinical
trials.
General and Administrative Expenses
General and administrative (G&A) expenses were $21.1 million
in the first quarter of 2016, which included $9.1 million of
non-cash stock-based compensation, as compared to $12.7 million in
the first quarter of 2015, which included $2.9 million of non-cash
stock-based compensation. G&A expenses for the quarter ended
March 31, 2016 as compared to the prior year period increased due
primarily to an increase in non-cash stock-based compensation
expense due to an increase in headcount, as well as the vesting of
certain performance-based stock option awards during the quarter
ended March 31, 2016. The company expects that on a quarterly basis
in 2016 G&A expenses will remain relatively consistent with the
first quarter of 2016.
Conference Call Information
Management will provide an update on the company, discuss first
quarter 2016 results, and discuss expectations for the future via
conference call on Monday, May 2, 2016 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 99665704. A replay of the call will be
available beginning at 7:30 p.m. ET on May 2, 2016. To access
the replay, please dial 855-859-2056 (domestic) or 404-537-3406
(international), and refer to conference 99665704
Sanofi Genzyme Alliance
In January 2014, Alnylam and Sanofi Genzyme, the specialty
care global business unit of Sanofi, formed an alliance to
accelerate and expand the development and commercialization of RNAi
therapeutics across the world. The alliance is structured as a
multi-product geographic alliance in the field of rare diseases.
Alnylam retains product rights in North
America and Western Europe, while Sanofi Genzyme obtained
the right to access certain programs in Alnylam's current and
future Genetic Medicines pipeline in the rest of the world (ROW)
through the end of 2019, together with certain broader
co-development/co-commercialization rights and global rights for
certain products. In the case of patisiran, Alnylam will advance
the product in North America and Western Europe,
while Sanofi Genzyme will advance the product in the ROW. In the
case of revusiran, Alnylam and Sanofi Genzyme will
co-develop/co-commercialize the product in North America and
Western Europe, while Sanofi Genzyme will advance the product in
the ROW. In the case of fitusiran, Sanofi Genzyme has elected to
opt into the program for its ROW rights, while retaining its
further opt-in right to co-develop and co-promote fitusiran with
Alnylam in North America and Western Europe, subject to certain
restrictions.
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 LNP Technology
Alnylam has licenses to Arbutus 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 Ionis, Novartis, Roche,
Takeda, Merck, Monsanto, The Medicines Company, and Sanofi Genzyme.
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 views with respect to the potential for RNAi
therapeutics, including patisiran, revusiran, fitusiran, ALN-CC5,
ALN-AS1, ALN-AAT, ALN-GO1, ALN-PCSsc, ALN-HBV, ALN-TTRsc02, and
ALN-F12, its expectations regarding its STAr pipeline growth
strategy, its “Alnylam 2020” guidance for the advancement and
commercialization of RNAi therapeutics, its expectations for the
timing of filing of regulatory documents, including but not limited
to submission of an MAA and NDA for patisiran, its expectations
regarding the timing of the start of clinical studies and
presentation of clinical data, including for studies with
patisiran, revusiran, fitusiran, ALN-CC5, ALN-AS1, ALN-AAT,
ALN-GO1, ALN-PCSsc, ALN-HBV, and ALN-TTRsc02, its expected cash
position as of December 31, 2016, and its plans regarding the
pursuit of pre-clinical programs 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 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 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 for a
specified indication or at all, 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
manage operating expenses, 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 Annual Report on Form 10-K 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 Ended March
31,
2016 2015 Net
revenues from collaborators $ 7,345 $ 18,537
Operating expenses: Research and development 96,273
58,035 General and administrative 21,100
12,724 Total operating expenses 117,373
70,759 Loss from operations (110,028 ) (52,222
)
Other income: Interest income 1,813 1,014 Other income
5,241 — Total other income 7,054
1,014 Loss before income taxes (102,974 )
(51,208 ) Benefit from income taxes — 431
Net loss $ (102,974 ) $ (50,777 ) Net loss per common share
- basic and diluted $ (1.21 ) $ (0.62 ) Weighted-average common
shares used to compute basic and diluted net loss per common share
85,277 82,074
Comprehensive
loss: Net loss $ (102,974 ) $ (50,777 ) Unrealized (loss) gain
on marketable securities, net of tax (8,224 ) 3,622
Reclassification adjustment for realized gain on marketable
securities
included in net loss
(5,156 ) — Comprehensive loss $ (116,354 ) $
(47,155 )
ALNYLAM PHARMACEUTICALS, INC.
UNAUDITED CONDENSED CONSOLIDATED
BALANCE SHEETS
(In thousands, except share
amounts)
March 31, December 31,
2016 2015 Cash,
cash equivalents and total marketable securities $ 1,208,290 $
1,280,951 Billed and unbilled collaboration receivables 8,814 8,298
Prepaid expenses and other assets 29,097 18,030 Property and
equipment, net 36,814 27,812 Investment in equity securities of
Regulus Therapeutics Inc. 33,934
51,419
Total assets
$ 1,316,949 $ 1,386,510 Accounts
payable, accrued expenses and other liabilities $ 49,620 $ 46,886
Total deferred revenue 70,896 68,317 Total deferred rent 7,382
6,593 Total stockholders’ equity (85.5 million and 85.1 million
common shares issued and outstanding and at March 31, 2016 and
December 31, 2015, respectively)
1,189,051 1,264,714
Total
liabilities and stockholders' equity $
1,316,949 $ 1,386,510
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, 2015.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20160502005867/en/
Alnylam Pharmaceuticals, Inc.Investors and MediaChristine
Regan Lindenboom, 617-682-4340orInvestorsJosh Brodsky,
617-551-8276
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