− Achieved Third Quarter 2020 ONPATTRO® Global
Net Product Revenue of $82.5 Million, Including Over 20% Quarterly
U.S. Growth, with More Than 1,150 Patients on Commercial Product
Worldwide –
− Achieved Third Quarter 2020 GIVLAARI® Global
Net Product Revenue of $16.7 Million, with More Than 150 Patients
on Commercial Product Worldwide –
– Received Positive Opinions for OXLUMO™
(lumasiran) and LEQVIO® (inclisiran) from the Committee for
Medicinal Products for Human Use (CHMP) of the European Medicines
Agency (EMA), Positioning the Company for Potentially Four Approved
RNAi Therapeutic Medicines by Year-End 2020 –
– Increased 2020 Guidance Range for ONPATTRO
Revenue from $280-$300 Million to $295-$310 Million –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today reported its consolidated financial
results for the third quarter 2020 and reviewed recent business
highlights.
“We are extremely pleased with the performance of ONPATTRO and
GIVLAARI in the third quarter, reflecting strong commercial
execution and improving market conditions following the more
challenging COVID-19 pandemic phase experienced in the second
quarter. We’re also excited about the recent positive CHMP opinions
for OXLUMO and LEQVIO, moving these potentially transformative
investigational RNAi therapeutics closer to approval. We believe
that this positions Alnylam to potentially exit 2020 with four
revenue-generating products bolstering our sustained growth,” said
John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “In
addition, our robust late-stage pipeline of investigational
medicines continued to advance in the quarter. Notably, we achieved
positive results from the ILLUMINATE-B study – demonstrating
efficacy and safety for our investigational RNAi therapeutic
lumasiran in children under the age of six, including infants. We
also achieved continued enrollment in our APOLLO-B and HELIOS-B
Phase 3 studies of patisiran and vutrisiran, respectively, in
development for the treatment of ATTR amyloidosis with
cardiomyopathy. With our recent progress, we now expect to exceed
our Alnylam 2020 vision and goals of building a multi-product,
global biopharma company with a deep clinical pipeline and a
robust, organic product engine to drive future sustainable
innovation and value creation. Finally, we look forward to
highlighting further progress and future plans at our upcoming
virtual R&D Day event in December.”
Third Quarter 2020 and Recent Significant Corporate
Highlights
Commercial Performance
ONPATTRO®
- Achieved global net product revenue for the third quarter of
2020 of $82.5 million, representing 24% quarterly growth globally
and 21% quarterly growth in the U.S. alone.
- Attained over 1,150 patients worldwide on commercial ONPATTRO
treatment as of September 30, 2020.
- Continued progress with market access efforts across the CEMEA
region (Canada, Europe, Middle East,
and Africa), with a recent launch in Portugal, conclusion of
price negotiations in France, and completion of initial access
agreement in Canada.
- Continued global expansion with achievement of regulatory
approval in Israel.
- Received the prestigious 2020 Prix Galien USA Award for Best
Biotechnology Product.
GIVLAARI®
- Achieved global net product revenue for the third quarter of
2020 of $16.7 million.
- Attained over 150 patients worldwide on commercial GIVLAARI
treatment as of September 30, 2020.
- Continued strong market access progress in the U.S., with 10
VBAs finalized to date with commercial payers and confirmed access
for over 90% of covered U.S. lives.
- Continued progress with market access efforts across the CEMEA
region, with ongoing launch in Germany, cohort Temporary
Authorization for Use (ATU) supply in France, and named patient
sales in other countries.
- Received an Improvement of Medical Benefit (ASMR) score of II
in France, concluding that GIVLAARI offers significant additional
therapeutic value. In 2019, only two new commercial medicines
received a similar ASMR score.
- In addition, obtained a “Considerable Benefit” rating in
Germany and secured a strong health technology assessment (HTA)
rating in Italy.
- Continued global expansion with approval in Canada and
submission of a new drug application in Japan.
- Received the NORD 2020 Industry Innovation Award.
R&D Highlights
- Advanced patisiran (the non-proprietary name for
ONPATTRO), in development for the treatment of the cardiomyopathy
of both hereditary and wild-type ATTR amyloidosis.
- Continued enrollment in the APOLLO-B Phase 3 study in ATTR
amyloidosis patients with cardiomyopathy.
- Presented additional clinical data with patisiran – including
24-month results from the global open-label extension (OLE) study
and results from an open-label study in patients with hATTR
amyloidosis post-orthotopic liver transplant – and published
findings from an evaluation of patisiran with concomitant or prior
use of TTR stabilizers.
- Advanced vutrisiran, a subcutaneously administered
investigational RNAi therapeutic in development for the treatment
of ATTR amyloidosis.
- Continued treating patients in the fully enrolled HELIOS-A
Phase 3 study of vutrisiran in hATTR amyloidosis patients with
polyneuropathy, and remain on track to report topline results in
early 2021.
- Continued enrollment in the HELIOS-B Phase 3 study in ATTR
amyloidosis patients with cardiomyopathy.
- Announced potential for a biannual dosing regimen option for
vutrisiran, providing support for further product differentiation
as a potential best-in-class agent.
- Presented new interim data from the Phase 1/2 open-label
extension (OLE) study of givosiran (the non-proprietary name
for GIVLAARI) in acute hepatic porphyria (AHP).
- Advanced lumasiran, an investigational RNAi therapeutic
in development for the treatment of primary hyperoxaluria type 1
(PH1).
- Received a positive CHMP opinion from EMA recommending approval
of lumasiran for the treatment of PH1 in patients of all ages. If
approved, lumasiran will be marketed in Europe under the brand name
OXLUMO™.
- Received a positive scientific opinion from the UK’s Medicines
and Healthcare Products Regulatory Agency (MHRA) through the Early
Access to Medicines Scheme (EAMS).
- Presented positive complete results from ILLUMINATE-B, a global
Phase 3 pediatric study of lumasiran in PH1 patients less than six
years of age, including infants, with preserved renal
function.
- Continued enrollment in the ILLUMINATE-C Phase 3 study of
lumasiran for the treatment of advanced PH1 in patients of all
ages.
- Alnylam’s partner, Novartis, continued advancing
inclisiran, potentially the first and only RNAi therapeutic
cholesterol-lowering treatment. Inclisiran is undergoing regulatory
review in the U.S. and EU.
- Received a positive CHMP opinion from EMA recommending approval
of inclisiran for the treatment of adults with hypercholesterolemia
or mixed dyslipidemia. If approved, inclisiran will be marketed
under the brand name LEQVIO®.
- Alnylam’s partner, Sanofi, continued advancement of the ATLAS
Phase 3 program for fitusiran in patients with hemophilia A
or B with and without inhibitors.
- Advanced early- and mid-stage RNAi therapeutic pipeline
programs.
- Continued dosing in the Phase 1 study of ALN-AGT in
hypertension.
- In collaboration with Regeneron, advanced cemdisiran, an
investigational RNAi therapeutic for the treatment of
complement-mediated diseases.
- Continued enrollment in a Phase 2 clinical trial of cemdisiran
monotherapy in patients with IgA nephropathy, with topline results
expected in 2021.
- Alnylam’s partner Vir Biotechnology presented new data on
VIR-2218 (ALN-HBV02) at the European Association for the
Study of the Liver Digital International Liver Congress.
- In addition, Vir initiated a Phase 2 combination trial of
VIR-2218 with pegylated interferon-alpha (PEG-IFN-α), with initial
clinical data anticipated in 2021.
- The Company is announcing today that it has initiated dosing in
a Phase 1 study of ALN-HSD, an investigational RNAi
therapeutic targeting HSD17B13 in development for the treatment of
nonalcoholic steatohepatitis (NASH). ALN-HSD is being advanced in
collaboration with Regeneron.
- The Company is announcing today that it will delay its planned
filing of an IND for ALN-COV in order to obtain additional
pre-clinical efficacy data in models of COVID-19 infection.
- ALN-COV is part of a multi-target pre-clinical collaboration
with Vir Biotechnology.
- Continued progress with investigational RNAi therapeutics for
CNS and ocular diseases, including advancement of ALN-APP,
in development for the treatment of hereditary cerebral amyloid
angiopathy (hCAA) and autosomal dominant Alzheimer’s Disease
(ADAD), which remains on track for a CTA filing in mid-2021.
- Regeneron exercised its co-development/co-commercialization
option on the ALN-APP program, which Alnylam will lead.
Additional Business Updates
- Barry Greene, former President of Alnylam, transitioned from
Alnylam at the end of the third quarter. Yvonne Greenstreet assumed
an expanded role as President and Chief Operating Officer on
October 1, 2020.
- Closed $150 million R&D funding component of the previously
announced $2 billion strategic financing collaboration with
Blackstone to accelerate the advancement of RNAi therapeutics.
- Expanded global reach of commercialization activities with new
third-party distribution agreement with taiba Middle East.
- Received recognition from Science magazine as a top
employer.
Upcoming Events
- Alnylam announced today that it intends to present interim
results from the Phase 1 trial of ALN-AGT at the American Heart
Association Scientific Sessions 2020 on Friday, November 13.
- The Company also announced today that it plans to present a
review of its R&D and commercial activities at its upcoming
R&D Day event being held virtually December 15 and 16.
In addition, in late 2020, Alnylam intends to:
- Achieve regulatory approval for lumasiran in the EU and an FDA
action for lumasiran in late 2020.
- Alnylam’s partner Novartis expects regulatory approval for
inclisiran in the EU and an FDA action for inclisiran in late
2020.
- Achieve regulatory approval for ONPATTRO in Taiwan.
- Alnylam’s partner Regeneron plans to initiate a Phase 1 study
of cemdisiran in combination with pozelimab.
Financial Results for the Quarter Ended September 30,
2020
“The third quarter was very strong, with a rebound in ONPATTRO
sales growth, particularly in the U.S., and continued impressive
early performance with GIVLAARI. While the pandemic headwinds
haven’t completely receded, we believe our teams have been very
effective in meeting the needs of patients. As a result of this
continued strength and our cautious optimism for the fourth
quarter, we are again revising our full-year revenue guidance for
ONPATTRO, increasing the range from $280-$300 million to $295-$310
million,” said Jeff Poulton, Chief Financial Officer of Alnylam.
“Beyond the commercial business, we also closed the $150 million
R&D funding component of the Blackstone strategic financing
collaboration, finalizing the last piece of a broad relationship
that we believe secures our path to self-sustainability without the
need for future equity offerings. Looking ahead, we believe we are
well positioned to continue executing on our current commercial
portfolio, with potential for two additional RNAi therapeutics
coming to market, and advancing our broad pipeline of
investigational programs to drive future growth.”
Financial Highlights (in thousands, except per share
amounts)
Three Months Ended September
30,
2020
2019
Net product revenues
$
99,206
$
46,066
ONPATTRO net product revenues
$
82,516
$
46,066
GIVLAARI net product revenues
$
16,690
$
—
Net revenue from collaborations
$
26,647
$
23,995
Cost of goods sold
$
21,797
$
5,213
GAAP research and development expenses
$
161,783
$
160,796
Non-GAAP research and development
expenses
$
148,080
$
138,059
GAAP selling, general and administrative
expenses
$
167,472
$
120,351
Non-GAAP selling, general and
administrative expenses
$
114,498
$
97,079
GAAP operating loss
$
(225,199)
$
(216,299)
Non-GAAP operating loss
$
(158,522)
$
(170,290)
GAAP net loss
$
(253,291)
$
(208,535)
Non-GAAP net loss
$
(183,597)
$
(162,526)
GAAP net loss per common share - basic and
diluted
$
(2.18)
$
(1.92)
Non-GAAP net loss per common share - basic
and diluted
$
(1.58)
$
(1.50)
September 30, 2020
December 31, 2019
Cash, cash equivalents and marketable
securities
$
1,833,876
$
1,536,162
Net Product Revenues
- Net product revenues were $99.2 million in the third quarter
2020 representing 115% growth from the third quarter 2019 primarily
as a result of the addition of new patients on therapy and
expansion into new markets for ONPATTRO, as well as the ongoing
U.S. commercial launch and initial European launch of
GIVLAARI.
Net Revenues from Collaborations
- Net revenues from collaborations were $26.6 million in the
third quarter 2020, an increase from $24.0 million in the third
quarter 2019, primarily due to an increase in revenue recognized
from our Vir collaboration.
Research & Development (R&D) and Selling, General &
Administrative (SG&A) Expenses
- R&D expenses were relatively flat on a GAAP basis and
increased on a non-GAAP basis in the third quarter 2020 compared to
the same period in 2019 primarily due to increased expenses
associated with clinical and preclinical activities, personnel, and
facilities as we continue to support our long-term strategic growth
offset by decreased license fees associated with regulatory
filings.
- SG&A expenses increased in the third quarter 2020 compared
to the same period in 2019 on a GAAP and non-GAAP basis primarily
due to increased investment in commercial and medical affairs
activity to support the ongoing launches of ONPATTRO and GIVLAARI
and initial launch preparation activities for lumasiran. SG&A
expenses on a GAAP basis also increased due to a change in estimate
of contingent liabilities related to our arbitration with
Ionis.
Cash and Investments
- Cash, cash equivalents and marketable securities were $1.83
billion at the end of the third quarter 2020 compared to $1.54
billion at the end of 2019. The increase was primarily due to
$600.0 million in proceeds received in the second quarter of 2020
from the sale of future royalties and issuance of common stock to
Blackstone, partially offset by cash used in our operations to
support overall growth.
A reconciliation of GAAP to non-GAAP results for the current
quarter is included in the tables of this press release.
2020 Updated Financial Guidance
Full year 2020 financial guidance consists of the following:
Item
Provided 8/6/2020 ($
millions)
Updated 11/5/2020 ($
millions)
ONPATTRO net product revenues
$280 - $300
$295 - $310
GIVLAARI net product revenues
No guidance provided
Unchanged
Net revenues from collaborations
$100 - $150
Unchanged
GAAP R&D and SG&A expenses
$1,130 - $1,225
$1,160 - $1,255
Non-GAAP R&D and SG&A
expenses*
$1,000 - $1,075
Unchanged
*Excludes $160-$180 million of expenses
primarily related to stock-based compensation and a change in
estimate of contingent liabilities from projected GAAP R&D and
SG&A expenses.
The strategic financing collaboration with Blackstone under
which Alnylam will receive up to $2 billion is expected to enable
Alnylam’s achievement of a self-sustainable financial profile
without need for future equity financings.
Use of Non-GAAP Financial Measures
This 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
expenses, unrealized gains on marketable equity securities, costs
associated with our strategic financing collaboration, loss on
contractual settlement and change in estimate of contingent
liabilities. 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 unrealized gains on marketable equity securities, costs
associated with our strategic financing collaboration, loss on
contractual settlement and change in estimate of contingent
liabilities because the Company believes these items are
non-recurring transactions 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 Information
Management will provide an update on the Company and discuss
third quarter 2020 results as well as expectations for the future
via conference call on Thursday, November 5, 2020 at 8:30 am ET. To
access the call, please dial 800-239-9838 (domestic) or
+1-323-794-2551 (international) five minutes prior to the start
time and refer to conference ID 6976021. 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
+1-719-457-0820 (international) and refer to conference ID
6976021.
A live audio webcast of the call will be available on the
Investors section of the Company’s website at
www.alnylam.com/events. An archived webcast will be available on
the Alnylam website approximately two hours after the event.
About ONPATTRO® (patisiran)
ONPATTRO is an RNAi therapeutic that was approved in the United
States and Canada for the treatment of the polyneuropathy of hATTR
amyloidosis in adults. ONPATTRO is also approved in the European
Union, Switzerland and Brazil for the treatment of hATTR
amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and
in Japan for the treatment of hATTR amyloidosis with
polyneuropathy. ONPATTRO is an intravenously administered RNAi
therapeutic targeting transthyretin (TTR). It is designed to target
and silence TTR messenger RNA, thereby blocking the production of
TTR protein before it is made. ONPATTRO blocks the production of
TTR in the liver, reducing its accumulation in the body’s tissues
in order to halt or slow down the progression of the polyneuropathy
associated with the disease. For more information about ONPATTRO,
visit ONPATTRO.com.
ONPATTRO Important Safety Information
Infusion-Related Reactions
Infusion-related reactions (IRRs) have been observed in patients
treated with ONPATTRO® (patisiran). In a controlled clinical study,
19% of ONPATTRO-treated patients experienced IRRs, compared to 9%
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, acetaminophen, 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
Supplementation
ONPATTRO 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 Reactions
The most common adverse reactions that occurred in patients
treated with ONPATTRO were upper respiratory tract infections (29%)
and infusion-related reactions (19%).
For additional information about ONPATTRO, please see the full
Prescribing Information.
About GIVLAARI® (givosiran)
GIVLAARI is an RNAi therapeutic targeting aminolevulinic acid
synthase 1 (ALAS1) approved in the United States and Brazil for the
treatment of adults with acute hepatic porphyria (AHP). GIVLAARI is
also approved in the European Union for the treatment of AHP in
adults and adolescents aged 12 years and older. In the pivotal
study, givosiran was shown to significantly reduce the rate of
porphyria attacks that required hospitalizations, urgent healthcare
visits or intravenous hemin administration at home compared to
placebo. GIVLAARI is Alnylam’s first commercially available
therapeutic based on its Enhanced Stabilization Chemistry
ESC-GalNAc conjugate technology to increase potency and durability.
GIVLAARI is administered via subcutaneous injection once monthly at
a dose based on actual body weight and should be administered by a
healthcare professional. GIVLAARI works by specifically reducing
elevated levels of aminolevulinic acid synthase 1 (ALAS1) messenger
RNA (mRNA), leading to reduction of toxins associated with attacks
and other disease manifestations of AHP. For more information about
GIVLAARI, visit GIVLAARI.com.
GIVLAARI Important Safety Information
Contraindications
GIVLAARI is contraindicated in patients with known severe
hypersensitivity to givosiran. Reactions have included
anaphylaxis.
Anaphylactic Reaction
Anaphylaxis has occurred with GIVLAARI treatment (<1% of
patients in clinical trials). Ensure that medical support is
available to appropriately manage anaphylactic reactions when
administering GIVLAARI. Monitor for signs and symptoms of
anaphylaxis. If anaphylaxis occurs, immediately discontinue
administration of GIVLAARI and institute appropriate medical
treatment.
Hepatic Toxicity
Transaminase elevations (ALT) of at least 3 times the upper
limit of normal (ULN) were observed in 15% of patients receiving
GIVLAARI in the placebo-controlled trial. Transaminase elevations
primarily occurred between 3 to 5 months following initiation of
treatment.
Measure liver function tests prior to initiating treatment with
GIVLAARI, repeat every month during the first 6 months of
treatment, and as clinically indicated thereafter. Interrupt or
discontinue treatment with GIVLAARI for severe or clinically
significant transaminase elevations. In patients who have dose
interruption and subsequent improvement, reduce the dose to 1.25
mg/kg once monthly. The dose may be increased to the recommended
dose of 2.5 mg/kg once monthly if there is no recurrence of severe
or clinically significant transaminase elevations at the 1.25 mg/kg
dose.
Renal Toxicity
Increases in serum creatinine levels and decreases in estimated
glomerular filtration rate (eGFR) have been reported during
treatment with GIVLAARI. In the placebo-controlled study, 15% of
patients receiving GIVLAARI experienced a renally-related adverse
reaction. The median increase in creatinine at Month 3 was 0.07
mg/dL. Monitor renal function during treatment with GIVLAARI as
clinically indicated.
Injection Site Reactions
Injection site reactions were reported in 25% of patients
receiving GIVLAARI in the placebo-controlled trial. Symptoms
included erythema, pain, pruritus, rash, discoloration, or swelling
around the injection site. One (2%) patient experienced a single,
transient, recall reaction of erythema at a prior injection site
with a subsequent dose administration.
Drug Interactions
Concomitant use of GIVLAARI increases the concentration of
CYP1A2 or CYP2D6 substrates, which may increase adverse reactions
of these substrates. Avoid concomitant use of GIVLAARI with CYP1A2
or CYP2D6 substrates for which minimal concentration changes may
lead to serious or life-threatening toxicities. If concomitant use
is unavoidable, decrease the CYP1A2 or CYP2D6 substrate dosage in
accordance with approved product labeling.
Adverse Reactions
The most common adverse reactions that occurred in patients
receiving GIVLAARI were nausea (27%) and injection site reactions
(25%).
For additional information about GIVLAARI, please see full
Prescribing Information.
About LNP Technology
Alnylam has licenses to Arbutus Biopharma LNP intellectual
property for use in RNAi therapeutic products using LNP
technology.
About RNAi
RNAi (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 or disease pathway 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 Alnylam Pharmaceuticals
Alnylam (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 people afflicted with
rare genetic, cardio-metabolic, hepatic infectious, and central
nervous system (CNS)/ocular 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 RNAi therapeutics platform. Alnylam’s commercial RNAi
therapeutic products are ONPATTRO® (patisiran), approved in the
U.S., EU, Canada, Japan, Brazil, and Switzerland, and GIVLAARI®
(givosiran), approved in the U.S, EU, and Brazil. Alnylam has a
deep pipeline of investigational medicines, including six product
candidates that are in late-stage development. Alnylam is executing
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 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 Statements
Various statements in this release concerning Alnylam’s
expectations, plans and prospects, including, without limitation,
expectations regarding the direct or indirect effects on Alnylam’s
business, activities and prospects as a result of the COVID-19
pandemic, or delays or interruptions resulting therefrom and the
success of Alnylam’s mitigation efforts, Alnylam's views and plans
with respect to the potential for RNAi therapeutics, including
ONPATTRO, GIVLAARI, lumasiran, inclisiran, patisiran, vutrisiran,
fitusiran, cemdisiran, ALN-AGT, ALN-HSD, ALN-COV and ALN-APP, its
plans for additional global regulatory filings and the continuing
product launches of ONPATTRO and GIVLAARI, expectations regarding
reimbursement for ONPATTRO and GIVLAARI in various territories and
the status of VBA agreements and confirmed access, the advancement
of lumasiran and inclisiran through regulatory review and toward
the market including the receipt of positive CHMP opinions for both
drug candidates, the potential for a biannual dosing regimen option
for vutrisiran, the achievement of additional pipeline milestones,
including relating to ongoing clinical studies of vutrisiran, the
expected timing for the presentation of interim results from the
Phase 1 trial of ALN-AGT, the expected timing for filing a CTA for
ALN-APP and the revised expectations regarding the filing of an IND
for ALN-COV, the initiation of a Phase 1 clinical study of
cemdisiran in combination with pozelimab by Regeneron, its
expectations relating to continued ONPATTRO and GIVLAARI revenue
growth and the further revised expected range of ONPATTRO net
product revenues for 2020, the expected range for net revenues from
collaborations for 2020, the revised expected range of 2020
aggregate annual non-GAAP and R&D and SG&A expenses and
further revised expected range of 2020 aggregate annual GAAP
R&D and SG&A expenses, Alnylam’s belief that the funding
provided by Blackstone should enable Alnylam to achieve a
self-sustainable profile without the need for future equity
financing, and expectations regarding Alnylam’s ability to exceed
its “Alnylam 2020” strategic plan announced in 2015 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: the direct or indirect impact of the
COVID-19 global pandemic or any future pandemic, such as the scope
and duration of the outbreak, government actions and restrictive
measures implemented in response, material delays in diagnoses of
rare diseases, initiation or continuation of treatment for diseases
addressed by Alnylam products, or in patient enrollment in clinical
trials, potential supply chain disruptions, and other potential
impacts to Alnylam’s business, the effectiveness or timeliness of
steps taken by Alnylam to mitigate the impact of the pandemic, and
Alnylam’s ability to execute business continuity plans to address
disruptions caused by the COVID-19 or any future pandemic;
Alnylam's ability to discover and develop novel drug candidates and
delivery approaches and successfully demonstrate the efficacy and
safety of its product candidates, including vutrisiran, ALN-AGT,
ALN-HSD, ALN-APP and ALN-COV; 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 or its marketed products, including ONPATTRO,
GIVLAARI, inclisiran, lumasiran and vutrisiran; obtaining,
maintaining and protecting intellectual property; intellectual
property matters including potential patent litigation relating to
its platform, products or product candidates; obtaining regulatory
approval for its product candidates, including lumasiran and
inclisiran, and maintaining regulatory approval and obtaining
pricing and reimbursement for its products, including ONPATTRO and
GIVLAARI; successfully launching, marketing and selling its
approved products globally, including ONPATTRO and GIVLAARI and
achieving net product revenues for ONPATTRO within its further
revised expected range during 2020; 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 within the reduced ranges
of guidance provided by Alnylam through the implementation of
further discipline in operations to moderate spend and its ability
to achieve a self-sustainable financial profile in the future
without the need for future equity financing; Alnylam’s ability to
establish and maintain strategic business alliances and new
business initiatives; Alnylam's dependence on third parties,
including Regeneron, for development, manufacture and distribution
of certain products, including eye and CNS products and ALN-APP,
and Vir for the development of ALN-COV and other potential RNAi
therapeutics targeting SARS-CoV-2 and host factors for SARS-CoV-2;
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.
This release discusses investigational RNAi therapeutics and
uses of previously approved RNAi therapeutics in development and is
not intended to convey conclusions about efficacy or safety as to
those investigational therapeutics or uses. There is no guarantee
that any investigational therapeutics or expanded uses of
commercial products will successfully complete clinical development
or gain health authority approval.
ALNYLAM PHARMACEUTICALS,
INC.
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS
(In thousands, except per
share amounts)
(Unaudited)
Three Months Ended
Nine Months Ended
September 30,
2020
September 30,
2019
September 30,
2020
September 30,
2019
Statements of Operations
Revenues:
Net product revenues
$
99,206
$
46,066
$
248,677
$
110,588
Net revenues from collaborations
26,647
23,995
80,614
37,481
Total revenues
125,853
70,061
329,291
148,069
Operating costs and expenses:
Cost of goods sold
21,797
5,213
55,028
12,886
Research and development
161,783
160,796
486,350
453,813
Selling, general and administrative
167,472
120,351
422,129
322,728
Total operating costs and expenses
351,052
286,360
963,507
789,427
Loss from operations
(225,199)
(216,299)
(634,216)
(641,358)
Other (expense) income:
Interest expense
(28,731)
—
(55,979)
—
Interest income
2,072
9,889
10,717
26,195
Other (expense) income
(594)
(2,519)
67,477
(2,929)
Change in fair value of liability
obligation
—
—
—
9,422
Total other (expense) income
(27,253)
7,370
22,215
32,688
Loss before income taxes
(252,452)
(208,929)
(612,001)
(608,670)
(Provision) benefit for income taxes
(839)
394
(2,740)
(1,261)
Net loss
$
(253,291)
$
(208,535)
$
(614,741)
$
(609,931)
Net loss per common share - basic and
diluted
$
(2.18)
$
(1.92)
$
(5.37)
$
(5.63)
Weighted-average common shares used to
compute basic and diluted net loss per common share
115,986
108,701
114,554
108,427
ALNYLAM PHARMACEUTICALS,
INC.
RECONCILIATION OF SELECTED
GAAP MEASURES TO NON-GAAP MEASURES
(In thousands, except per
share amounts)
Three Months Ended
September 30, 2020
September 30, 2019
Reconciliation of GAAP to Non-GAAP
research and development:
GAAP Research and development
$
161,783
$
160,796
Less: Stock-based compensation
expenses
(13,703)
(22,737)
Non-GAAP Research and development
$
148,080
$
138,059
Reconciliation of GAAP to Non-GAAP
selling, general and administrative:
GAAP Selling, general and
administrative
$
167,472
$
120,351
Less: Stock-based compensation
expenses
(23,561)
(23,272)
Less: Costs associated with the strategic
financing collaboration
(763)
—
Less: Loss on contractual settlement
(650)
—
Less: Change in estimate of contingent
liabilities
(28,000)
—
Non-GAAP Selling, general and
administrative
$
114,498
$
97,079
Reconciliation of GAAP to Non-GAAP
operating loss:
GAAP operating loss
$
(225,199)
$
(216,299)
Add: Stock-based compensation expenses
37,264
46,009
Add: Costs associated with the strategic
financing collaboration
763
—
Add: Loss on contractual settlement
650
—
Add: Change in estimate of contingent
liabilities
28,000
—
Non-GAAP operating loss
$
(158,522)
$
(170,290)
Reconciliation of GAAP to Non-GAAP net
loss:
GAAP net loss
$
(253,291)
$
(208,535)
Add: Stock-based compensation expenses
37,264
46,009
Add: Costs associated with the strategic
financing collaboration
763
—
Add: Loss on contractual settlement
650
—
Add: Change in estimate of contingent
liabilities
28,000
—
Add: Unrealized loss on marketable equity
securities
3,017
—
Non-GAAP net loss
$
(183,597)
$
(162,526)
Reconciliation of GAAP to Non-GAAP net
loss per common share-basic and diluted:
GAAP net loss per common share - basic and
diluted
$
(2.18)
$
(1.92)
Add: Stock-based compensation expenses
0.32
0.42
Add: Costs associated with the strategic
financing collaboration
0.01
—
Add: Loss on contractual settlement
0.01
—
Add: Change in estimate of contingent
liabilities
0.24
—
Add: Unrealized gain on marketable equity
securities
0.02
—
Non-GAAP net loss per common share - basic
and diluted
$
(1.58)
$
(1.50)
Please note that the figures presented above may not sum exactly
due to rounding
ALNYLAM PHARMACEUTICALS,
INC.
CONDENSED CONSOLIDATED BALANCE
SHEETS
(In thousands, except share
amounts)
(Unaudited)
September 30, 2020
December 31, 2019
Cash, cash equivalents and marketable
securities
$
1,833,876
$
1,536,162
Restricted investments
24,725
14,825
Accounts receivable, net
79,118
43,011
Inventory
84,040
56,348
Prepaid expenses and other assets
95,600
98,412
Property, plant and equipment, net
444,690
425,179
Operating lease right-of-use lease
assets
245,234
221,197
Receivable related to the sale of future
royalties
500,000
—
Total assets
$
3,307,283
$
2,395,134
Accounts payable, accrued expenses and
other liabilities
$
339,473
$
256,415
Total deferred revenue
380,309
396,204
Operating lease liability
329,454
303,823
Liability related to the sale of future
royalties
1,043,024
—
Total stockholders’ equity (116.1 million
shares issued and outstanding at September 30, 2020; 112.2 million
shares issued and outstanding at December 31, 2019)
1,215,023
1,438,692
Total liabilities and stockholders'
equity
$
3,307,283
$
2,395,134
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, 2019.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20201105005563/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) 617-682-4340 Josh Brodsky (Investors)
617-551-8276
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