Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics
company, today reported its consolidated financial results for the first
quarter 2012, and company highlights.
"We made significant clinical and business progress this quarter,
including important achievements with our 'Alnylam 5x15' product
development and commercialization strategy. First, we reported very
encouraging clinical data from our Phase I ALN-PCS trial showing
statistically significant and durable reductions of PCSK9 plasma levels
of up to 84% and lowering of LDL cholesterol of up to 50%, in addition
to demonstrating continued improved efficacy and tolerability for
Alnylam's second-generation lipid nanoparticle delivery technology.
Further, we initiated dosing in our Phase I clinical trial with
ALN-TTR02, an RNAi therapeutic targeting the transthyretin gene for the
treatment of transthyretin-mediated amyloidosis, which also utilizes our
second-generation lipid nanoparticle delivery technology. We are on
track to report clinical data from this study in the third quarter of
this year," said John Maraganore, Ph.D., Chief Executive Officer of
Alnylam. "These important accomplishments, combined with the recent
strengthening of our balance sheet, gives us great confidence in the
continued execution of our 'Alnylam 5x15' product strategy, focused on
advancement of RNAi therapeutics for the treatment of genetically
defined targets for diseases where there are limited treatment options
for patients and caregivers."
"With clear evidence from our ALN-TTR and ALN-PCS Phase I clinical
trials that the RNAi pathway can be harnessed as a therapeutic strategy,
we continue to focus on our 'Alnylam 5x15' efforts with what we believe
to be our highest value opportunities, driving key programs toward
pivotal trials," said Barry Greene, President and Chief Operating
Officer of Alnylam. "This includes accelerated clinical development
plans for ALN-TTR02, which we believe has the potential to become the
leading innovative medicine for the treatment of transthyretin-mediated
amyloidosis, a devastating and debilitating orphan disease. It also
includes our RNAi therapeutic for the treatment of hemophilia, which we
believe represents an exciting opportunity that could fundamentally
change the management of this inherited orphan bleeding disorder. In
addition, we plan to continue to advance additional programs with
existing partners and new alliances we intend to form."
Cash, Cash Equivalents and Total Marketable Securities
At March 31, 2012, Alnylam had cash, cash equivalents and total
marketable securities of $316.9 million, as compared to $260.8 million
at December 31, 2011. In February 2012, Alnylam sold an aggregate of
8,625,000 shares of its common stock through an underwritten public
offering at a price to the public of $10.75 per share. As a result of
the offering, Alnylam received aggregate net proceeds of approximately
$87 million, after deducting underwriting discounts and commissions and
other estimated offering expenses of approximately $5.9 million.
Net Loss
The net loss according to accounting principles generally accepted in
the U.S. (GAAP) for the first quarter of 2012 was $11.4 million, or
$0.25 per share on both a basic and diluted basis (including $3.2
million, or $0.07 per share of non-cash stock-based compensation
expense), as compared to a net loss of $16.3 million, or $0.38 per share
on both a basic and diluted basis (including $4.1 million, or $0.10 per
share of non-cash stock-based compensation expense), for the same period
in the previous year.
Revenues
Revenues were $20.6 million for the first quarter of 2012, as compared
to $20.9 million for the first quarter of 2011. Revenues for the first
quarter of 2012 included $14.0 million of collaboration revenues related
to the company's alliance with Roche (which assigned its rights and
obligations to Arrowhead Research Corporation during 2011), $5.5 million
of revenues from the company's alliance with Takeda Pharmaceuticals
Company Limited, and $1.1 million of expense reimbursement,
amortization, and/or license fee revenues from Cubist Pharmaceuticals,
Inc., InterfeRx(TM), research reagent and services licensees, and other
sources.
Research and Development Expenses
Research and development (R&D) expenses were $21.1 million in the first
quarter of 2012, which included $2.1 million of non-cash stock-based
compensation, as compared to $26.3 million in the first quarter of 2011,
which included $2.7 million of non-cash stock-based compensation. The
decrease in R&D expenses in the first quarter of 2012 as compared to the
prior year period was due primarily to lower clinical trial and
manufacturing costs related to our ALN-RSV and ALN-VSP programs,
partially offset by additional clinical expenses related to the
advancement of our ALN-TTR program. Lab supplies and materials and
compensation and related expenses also decreased due primarily to the
reduction in workforce in connection with our January 2012 strategic
corporate restructuring. Partially offsetting these decreases was a
one-time charge of $3.9 million related to the restructuring, including
employee severance, benefits and related costs.
General and Administrative Expenses
General and administrative (G&A) expenses were $10.4 million in the
first quarter of 2012, which included $1.1 million of non-cash
stock-based compensation, as compared to $10.2 million in the first
quarter of 2011, which included $1.5 million of non-cash stock-based
compensation.
Regulus Therapeutics
Equity in loss of joint venture was $0.9 million and $1.1 million for
the first quarter of 2012 and 2011, respectively, related to our share
of the net losses incurred by Regulus.
Interest Income
Interest income was $0.2 million for the first quarter of 2012, as
compared to $0.4 million for the first quarter of 2011. The decrease in
interest income was due primarily to lower average cash, cash
equivalents and total marketable securities balances as compared to the
prior year.
2012 Financial Guidance
Alnylam expects that its cash, cash equivalents and total marketable
securities balance will be greater than $250 million at December 31,
2012.
"Alnylam further strengthened its balance sheet this quarter with a
public offering of 8.6 million shares of common stock that resulted in
net proceeds of approximately $87 million. This financing, combined with
our recent corporate restructuring that included an approximate 33%
reduction in workforce, results in a balance sheet and operating plan
that we believe will enable us to advance our 'Alnylam 5x15' product
strategy," said Michael Mason, Vice President, Finance and Treasurer of
Alnylam. "We ended this quarter with approximately $317 million in cash,
and expect to end 2012 with greater than $250 million."
First Quarter 2012 and Recent Significant Corporate Highlights
Key "Alnylam 5x15" Program Highlights
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Advanced Clinical Development of ALN-TTR Program for Transthyretin
(TTR)-Mediated Amyloidosis (ATTR). Alnylam completed its ALN-TTR01
Phase I study and expects to report final data at the International
Symposium on Amyloidosis meeting in May 2012. Preliminary data
reported last year showed that ALN-TTR01
was generally safe and well tolerated and resulted in a statistically
significant lowering of TTR serum levels in ATTR patients, thus
demonstrating important human proof of concept for this innovative
medicine.
Alnylam is advancing ALN-TTR02, which uses the
same siRNA as ALN-TTR01, but is formulated in a more potent,
proprietary second-generation lipid nanoparticle (LNP) technology
using the "MC3" lipid. Alnylam has initiated a Phase I trial with
ALN-TTR02 aimed at evaluating safety, tolerability, and clinical
activity of ALN-TTR02 in healthy volunteers. Specifically, the study
is evaluating the clinical activity of ALN-TTR02 toward knockdown of
serum TTR, the disease-causing protein in patients with ATTR. Alnylam
expects to present data from this study in the third quarter of 2012.
In addition, Alnylam plans to start a Phase II multi-dose study of
ALN-TTR02 in ATTR patients in the second half of 2012 and, assuming
positive results, expects to start a pivotal trial for ALN-TTR02 in
2013. Alnylam also plans to advance ALN-TTRsc, which utilizes a
GalNAc-conjugate delivery approach and subcutaneous dose
administration.
Alnylam announced today that it has
received a U.S. patent (U.S. Patent No. 8,168,775) for RNAi
therapeutics targeting the transthyretin gene.
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Continued Development of ALN-APC Program for Hemophilia.
Alnylam is developing an RNAi therapeutic targeting Protein C, an
endogenous anticoagulant pathway, for the treatment of hemophilia.
Alnylam plans to advance its hemophilia program toward the clinic with
a goal of initiating a Phase I clinical trial in the first half of
2013, with data expected in the second half of 2013. The company will
present updated pre-clinical data from this program at the World
Federation of Hemophilia in July 2012.
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Reported Positive Clinical Results in ALN-PCS Severe
Hypercholesterolemia Program. ALN-PCS
is a PCSK9 synthesis inhibitor that reduces intracellular and
extracellular levels of PCSK9 resulting in lowered plasma levels of
low-density lipoprotein cholesterol (LDL-C, or "bad" cholesterol). New
data from a Phase I clinical trial presented at the American Heart
Association's Arteriosclerosis, Thrombosis and Vascular Biology 2012
Scientific Sessions demonstrated that administration of a single dose
of ALN-PCS, in the absence of statin co-administration, resulted in
statistically significant and durable reductions of PCSK9 plasma
levels of up to 84% and lowering of LDL-C of up to 50%. ALN-PCS was
shown to be safe and well tolerated in this study. These data also
highlight continued improved efficacy and tolerability for Alnylam's
second-generation LNP delivery technology. Alnylam plans to partner
this program prior to conducting Phase II clinical studies.
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Continued Progress with Additional "Alnylam 5x15" Programs.
Alnylam designated ALN-TMP as its fifth "Alnylam 5x15" program.
ALN-TMP comprises a systemically delivered RNAi therapeutic targeting
transmembrane protease, serine 6 (Tmprss6) for the treatment of
hemoglobinopathies, including beta-thalassemia and sickle cell anemia.
Pre-clinical animal model studies with ALN-TMP have demonstrated
corrective effects on iron overload in addition to broader disease
modifying effects including improvements in hemoglobin levels and
spleen histopathology. Alnylam plans to partner this program prior to
conducting a Phase I clinical study. Alnylam also plans to partner its
ALN-HPN program which targets the hepcidin pathway for the treatment
of refractory anemia prior to conducting a Phase I clinical study.
Key Partner-Based Program Highlights
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Completed Enrollment in Phase IIb Clinical Study of ALN-RSV01 for
the Treatment of Respiratory Syncytial Virus (RSV) Infection. The
company has completed enrollment in its Phase IIb trial with ALN-RSV
and expects to report results in mid-2012. The Phase IIb trial was
designed as a multi-center, randomized, double-blind,
placebo-controlled study in RSV-infected lung transplant patients. The
ALN-RSV program is partnered with Kyowa Hakko Kirin Co., Ltd. in Asia
and Cubist Pharmaceuticals, Inc. worldwide except Asia.
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Completed Phase I Clinical Study of ALN-VSP for the Treatment of
Liver Cancers. Alnylam has completed its Phase I study with
ALN-VSP, a systemically delivered RNAi therapeutic targeting both
vascular endothelial growth factor (VEGF) and kinesin spindle protein
(KSP) for the treatment of liver cancers. Data reported to date from
the Phase I study showed that ALN-VSP was generally well tolerated,
demonstrated evidence for anti-tumor activity, and was found to
mediate RNAi activity in both hepatic and extra-hepatic tumors.
Complete data from this Phase I trial will be presented at the
American Society of Clinical Oncology meeting in June 2012. The
company intends to partner ALN-VSP prior to initiating a Phase II
study.
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Provides Update on Huntington's Disease Program. ALN-HTT is the
drug under development as part of a drug-device combination therapy
for the treatment of Huntington's disease being advanced in
collaboration with Medtronic, Inc. and CHDI Foundation, Inc. As part
of its alignment of resources on its ATTR and hemophilia programs,
Alnylam has elected to exercise its option under its agreement with
Medtronic to opt-out of the 50-50 expense/profit share arrangement of
the ALN-HTT program. If Medtronic decides to continue the ALN-HTT
program, Alnylam would continue to supply ALN-HTT drug product for the
program and would be entitled to receive milestones and royalty
payments on future annual net sales.
Business and Organizational Highlights
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Completed Successful Public Offering of Common Stock. Alnylam
completed a public offering of common stock resulting in the issuance
of a total of 8,625,000 shares and receipt of aggregate net proceeds,
after deducting underwriting discounts and commissions and other
estimated offering expenses, of approximately $87 million.
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Formed Delivery Collaboration with Arrowhead Research Corporation.
Alnylam granted Arrowhead a license under its intellectual property
that enables the discovery, development, and commercialization of an
RNAi therapeutic targeting the hepatitis B virus (HBV). In addition,
Alnylam has received a license from Arrowhead to utilize their Dynamic
Polyconjugate (DPC) delivery technology for an RNAi therapeutic
product.
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Implemented Strategic Corporate Restructuring. Alnylam
announced a strategic corporate restructuring, including an
approximate 33% reduction in its workforce. The workforce reduction
was implemented in order to align the company's resources to a more
focused execution on its ATTR and hemophilia programs with accelerated
clinical development plans. Alnylam expects the reduction in personnel
costs, along with other external costs, to result in a savings of
approximately $20 million in 2012 cash operating expenses.
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Expanded Board of Directors and Scientific Advisory Board. Alnylam
has elected Dennis A. Ausiello, M.D. to its Board of Directors and
Scientific Advisory Board. Dr. Ausiello is the Jackson Professor of
Clinical Medicine at Harvard Medical School and Chief of Medicine at
Massachusetts General Hospital (MGH).
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