CARLSBAD, Calif., Feb. 24, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (Nasdaq: IONS) today reported its financial
results for the fourth quarter of 2020 and recent business
highlights.
"Last year, we laid out a bold new vision for the Company and
took important steps towards our goal of becoming one of the most
successful biotechnology companies. Key to our vision is our
strategy to maximize the value of our pipeline by commercializing
our wholly owned medicines. Our acquisition of Akcea was an
important step in building our commercial capabilities while
enabling us to further strengthen our organization," said
Brett P. Monia, Ph.D., chief
executive officer at Ionis. "Last year, we also advanced our
late-stage pipeline and expanded the utility of our technology.
Looking ahead, we expect data from multiple wholly owned programs
in the first half of this year, followed by Phase 3 tofersen data
in patients with SOD1-ALS in the second half. These key upcoming
catalysts, together with our recent pipeline and technology
achievements, position us well to have 12 or more products on the
market in 2026. Importantly, we continue to have the financial
strength to expand investment in our wholly owned pipeline and
commercial capabilities to drive meaningful and increasing value
for patients and shareholders."
2020 Summary Financial Results
- Achieved 2020 financial guidance
-
- $729 million in total revenues,
with half from marketed products
- $640 million of operating
expenses on a non-GAAP basis(1) and $901 million on a GAAP basis, reflecting
investments in Ionis' wholly owned pipeline
- Net income of $111 million on a
non-GAAP basis(1) and a net loss of $451 million on a GAAP basis
- Strong balance sheet with cash of $1.9
billion at year-end, enabling increasing investment in
advancing the pipeline and technology while also preparing to
commercialize the Company's wholly owned medicines
2020 Marketed Products Highlights
- SPINRAZA: a global foundation-of-care for the treatment of
spinal muscular atrophy (SMA) patients of all ages
-
- $2 billion in worldwide sales in
2020
- More than 11,000 patients worldwide were on therapy at the end
of the fourth quarter across post-marketing, expanded access and
clinical trial settings
- Enrollment began in the RESPOND study evaluating potential
SPINRAZA benefit in SMA patients with a suboptimal clinical
response to gene therapy
- Enrollment began in the pivotal randomized treatment cohort of
the DEVOTE study evaluating higher doses of SPINRAZA
- TEGSEDI and WAYLIVRA: transformational medicines approved for
the treatment of patients with severe rare diseases
-
- Product sales increased more than 65 percent in 2020, compared
to 2019
- Generated growing revenues as major markets launched in
2020
- Restructured European operations through a distribution
agreement with Swedish Orphan Biovitrum AB (Sobi)
Q4 2020 and Recent Pipeline Highlights
- Phase 3 Pipeline: growing pipeline positioned for 12 or
more products on the market in 2026
-
- Advanced IONIS-APOCIII-LRx into the Phase 3 BALANCE
study in patients with FCS
- Completed enrollment in the tofersen Phase 3 VALOR study in
patients with SOD1-ALS
- Mid-stage Pipeline: broad and advancing pipeline of potential
first-in-class and/or best-in-class medicines
-
- Advanced and expanded the IONIS-AGT-LRx development
program
-
- Reported IONIS-AGT-LRx positive topline Phase 2
results in patients with hypertension uncontrolled with two or
three antihypertensive medications
- Advanced ION904, the follow-on medicine targeting AGT, into
Phase 1 development in healthy volunteers
- Advanced vupanorsen into Phase 2b
development with the initiation of the TRANSLATE-TIMI 70
dose-ranging study in statin-treated patients with dyslipidemia,
resulting in a $75 million payment
from Pfizer
- Advanced ION449 (AZD8233), targeting PCSK9, into Phase
2b development in patients with
dyslipidemia and AstraZeneca licensed ION455, a new investigational
medicine for the treatment of nonalcoholic steatohepatitis (NASH),
resulting in $50 million from
AstraZeneca
- Unlocked potential new pulmonary disease franchise with
positive IONIS-ENAC-2.5Rx data
-
- Reported positive healthy volunteer results supporting aerosol
antisense medicine delivery to the lung
- Completed dosing in the Phase 2 study in patients with cystic
fibrosis
- Advanced IONIS-ENAC-2.5Rx into Phase 2 development
in patients with chronic obstructive pulmonary disease (COPD)
- Highlighted IONIS-MAPTRx (BIIB080) Phase 1/2 study
in patients with Alzheimer's disease in which
IONIS-MAPTRx was generally well tolerated and
demonstrated durable, time and dose-dependent reductions in CSF tau
protein
Upcoming 2021 Pipeline Catalysts
Anticipated 2021
Data Readouts(2)
|
Program
|
Phase
|
Anticipated
Indication
|
H1
|
H2
|
IONIS-ENAC-2.5Rx
|
2
|
Cystic
fibrosis
|
•
|
|
IONIS-PKK-LRx
|
2
|
Hereditary angioedema
(top-line)
|
•
|
|
IONIS-GHR-LRx
|
2
|
Acromegaly
(top-line)
|
•
|
|
IONIS-AGT-LRx
|
2
|
Hypertension
|
•
|
|
Tominersen
|
OLE/NHS
|
Huntington's
disease
|
•
|
IONIS-MAPTRx
|
1/2
|
Alzheimer's
disease
|
|
•
|
Vupanorsen
|
2b
|
sHTG/CVD risk
reduction
|
|
•
|
IONIS-C9Rx
|
1/2
|
C9-ALS
|
|
•
|
Tofersen
|
3 (VALOR
study)
|
SOD1-ALS
|
|
•
|
Anticipated 2021
Study Initiations(2)
|
Program
|
Phase
|
Anticipated
Indication
|
H1
|
H2
|
ION363
|
3
|
FUS-ALS
|
•
|
|
ION373
|
2
|
Alexander
disease
|
•
|
|
IONIS-AGT-LRx
|
2b
|
Resistant
hypertension
|
•
|
|
IONIS-AGT-LRx
|
2
|
Heart failure with
reduced ejection fraction
|
•
|
|
ION224
|
2b
|
NASH
|
•
|
|
Tofersen
|
3 (ATLAS
study)
|
Presymptomatic
SOD1-ALS
|
•
|
IONIS-APOCIII-LRx
|
3
|
Second CV
indication
|
|
•
|
IONIS-ENAC-2.5Rx
|
2
|
Cystic fibrosis not
amenable to CFTR modulators
|
|
•
|
|
(2) Timing of
partnered program catalysts based on partners' most recent publicly
available disclosures
|
2020 Financial Results and 2021 Financial Guidance
"We achieved our 2020 financial guidance, even in the
challenging COVID-19 pandemic environment. Moreover, in 2020 we
made significant progress toward our goal of creating a stronger,
more efficient company focused primarily on advancing our wholly
owned medicines to the market. We acquired Akcea enabling us to
retain full value from its rich portfolio. We also restructured our
European operations. Together, these transactions unlocked
substantial cost savings that we plan to reinvest to drive future
revenue growth," said Elizabeth L.
Hougen, chief financial officer of Ionis. "Our 2021 guidance
reflects our new strategy to maximize the value of our wholly owned
pipeline, focused primarily on commercializing our rare
neurological and cardiometabolic disease programs. Our guidance
also reflects the investments we are making in three key areas of
our business - advancing and expanding our wholly owned pipeline,
building commercial capabilities in support of our rich pipeline
and broadening the reach of our technology. We can increase our
investments in these areas while only modestly increasing our
expenses because of the significant cost savings we realized from
acquiring Akcea and restructuring our European operations.
Importantly, with nearly $2 billion
of cash at the end of last year, we remain well capitalized with
the substantial financial resources to achieve our goals."
2021 Financial Guidance
Ionis' full year 2021 financial guidance consists of the
following components (on a non-GAAP basis)(1):
Guidance
|
Revenue
|
>$600
million
|
Operating Expenses
(1)
|
$675 million to $725
million
|
Net Loss
(1)
|
<$75
million
|
|
|
|
|
|
(1)
|
All non-GAAP amounts
referred to in this press release exclude non-cash compensation
expense related to equity awards and expenses related to the Akcea
acquisition and restructured European operations and the related
tax effects. Please refer to the section below titled "Financial
Impacts of Akcea Acquisition and Restructured European Operations"
for a breakdown of the costs specific to these transactions.
Additionally, please refer to the detailed reconciliation of
non-GAAP and GAAP measures, which is provided later in this
release.
|
Revenue
Ionis' revenue was comprised of the following (amounts in
millions):
|
|
Three months
ended,
|
|
Year
ended,
|
|
|
December
31,
|
|
December
31,
|
|
|
2020
|
|
2019
|
|
2020
|
|
2019
|
Revenue:
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$75
|
|
$81
|
|
$287
|
|
$293
|
Product sales,
net
|
|
19
|
|
13
|
|
70
|
|
42
|
Licensing and royalty
revenue
|
|
2
|
|
6
|
|
8
|
|
17
|
Total commercial
revenue
|
|
96
|
|
100
|
|
365
|
|
352
|
R&D
Revenue:
|
|
|
|
|
|
|
|
|
Amortization from
upfront payments
|
|
12
|
|
46
|
|
80
|
|
146
|
Milestone
payments
|
|
110
|
|
51
|
|
183
|
|
115
|
License
fees
|
|
71
|
|
292
|
|
86
|
|
490
|
Other
services
|
|
1
|
|
5
|
|
15
|
|
20
|
Total R&D
revenue
|
|
194
|
|
394
|
|
364
|
|
771
|
Total
revenue
|
|
$290
|
|
$494
|
|
$729
|
|
$1,123
|
Financial Impacts of Akcea Acquisition and Restructured
European Operations
In the fourth quarter of 2020, the Company's non-GAAP amounts
exclude the following expenses related to the Akcea acquisition and
restructured European operations because the costs are not part of
its normal ongoing operating activities. Refer to the detailed
reconciliation of non-GAAP and GAAP measures, which is provided
later in this release. (Amounts in millions):
|
|
Three months
ended
December 31,
2020
|
Severance, retention and
other expenses
|
|
$31
|
Non-cash stock-based
compensation expense for the acceleration of Akcea equity
awards
|
|
59
|
Total expenses
included in GAAP operating expenses
|
|
$90
|
Non-cash adjustment of
the valuation allowance for Ionis' federal net deferred tax assets
and other tax adjustments related to the Akcea
acquisition
|
|
312
|
Total expenses
included in GAAP net loss
|
|
$402
|
As a result of the Akcea acquisition, Ionis and Akcea began
reporting their federal taxes on a consolidated basis in the fourth
quarter of 2020. The Company recorded a valuation allowance against
all Ionis' federal net deferred tax assets in the fourth quarter of
2020, due largely to Akcea's history of losses and the expected
impact of this on Ionis' consolidated federal taxable income. The
Company now maintains a valuation allowance against all its
consolidated federal and state net deferred tax assets.
Operating Expenses
Ionis' operating expenses for the year ended December 31, 2020 increased compared to 2019
driven by the Company's investments in advancing the Phase 3
programs for IONIS-TTR-LRx, IONIS-APOCIII-LRx
and other medicines in its wholly owned pipeline. Additionally, the
Company incurred approximately $90
million in costs related to the Akcea acquisition and
restructured European operations on a GAAP basis. The costs
consisted of $31 million of
severance, retention and other costs and $59
million of non-cash stock-based compensation expense for the
acceleration of Akcea equity awards.
Net Loss Attributable to Noncontrolling Interest in
Akcea
Prior to completing its acquisition of Akcea in October 2020, Ionis owned approximately 76
percent of Akcea. The line titled "Net loss attributable to
noncontrolling interest in Akcea" on Ionis' statement of operations
reflects the portion of Akcea's net income or loss attributable to
the other owners of Akcea's common stock. From mid-October 2020 through December 31, 2020, Ionis did not recognize any
noncontrolling interest in Akcea on its statement of operations
because it owned 100 percent of Akcea. Beginning in 2021, the
Company will no longer have an adjustment for noncontrolling
interest in Akcea.
Net Income (Loss) Attributable to Ionis Common
Stockholders
Ionis recognized a net loss attributable to Ionis' common
stockholders for 2020 compared to net income in 2019 primarily due
to higher revenue in 2019, including approximately $400 million in license fees Ionis earned from
Pfizer and Novartis. Also contributing to Ionis' net loss in 2020
was the non-cash adjustment of the valuation allowance Ionis
recorded against its federal net deferred tax assets discussed
above. Additionally, Ionis' operating expenses increased in 2020
compared to the same period last year as described above.
Balance Sheet
Ionis ended 2020 with cash, cash equivalents and short-term
investments of $1.9 billion, compared
to $2.5 billion at December 31, 2019. In October 2020, Ionis used approximately
$545 million of its cash for the
Akcea acquisition.
Webcast
Today, at 9:00 a.m. Eastern Time,
Ionis will conduct a live webcast to discuss this earnings release
and related activities. Interested parties may access the webcast
here. A webcast replay will be available for a limited time at the
same address.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been the leader in
RNA-targeted therapy, pioneering new markets and changing the
standards of care with its novel antisense technology. Ionis
currently has three marketed medicines and a premier late-stage
pipeline highlighted by industry-leading neurological and
cardiometabolic franchises. Our scientific innovation began and
continues with the knowledge that sick people depend on us, which
fuels our vision of becoming one of the most successful
biotechnology companies.
To learn more about Ionis visit www.ionispharma.com or
follow us on twitter @ionispharma.
Ionis' Forward-looking Statement
This press release includes forward-looking statements regarding
Ionis' business, financial guidance and the therapeutic and
commercial potential of SPINRAZA (nusinersen),
TEGSEDI (inotersen) and WAYLIVRA (volanesorsen) and
Ionis' technologies and products in development, including the
business of Akcea Therapeutics, Inc., Ionis' wholly owned
subsidiary. Any statement describing Ionis' goals, expectations,
financial or other projections, intentions or beliefs is a
forward-looking statement and should be considered an at-risk
statement. Such statements are subject to certain risks and
uncertainties, including those related to the impact COVID-19 could
have on our business, and including but not limited to those
related to our commercial products and the medicines in our
pipeline, and particularly those inherent in the process of
discovering, developing and commercializing medicines that are safe
and effective for use as human therapeutics, and in the endeavor of
building a business around such medicines. Ionis' forward-looking
statements also involve assumptions that, if they never materialize
or prove correct, could cause its results to differ materially from
those expressed or implied by such forward-looking statements.
Although Ionis' forward-looking statements reflect the good faith
judgment of its management, these statements are based only on
facts and factors currently known by Ionis. As a result, you are
cautioned not to rely on these forward-looking statements. These
and other risks concerning Ionis' programs are described in
additional detail in Ionis' annual report on Form 10-K for the year
ended December 31, 2019, and the most
recent Form 10-Q quarterly filing, which are on file with the
SEC. Copies of these and other documents are available from the
Company.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our," and "us" refers to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals™ is a trademark of Ionis
Pharmaceuticals, Inc. Akcea Therapeutics® is a
registered trademark of Akcea Therapeutics, Inc.
TEGSEDI® is a registered trademark of Akcea
Therapeutics, Inc. WAYLIVRA® is a registered
trademark of Akcea Therapeutics, Inc. SPINRAZA® is
a registered trademark of Biogen.
IONIS
PHARMACEUTICALS, INC.
|
SELECTED FINANCIAL
INFORMATION
|
Condensed
Consolidated Statements of Operations
|
(In Millions,
Except Per Share Data)
|
|
|
|
Three months
ended,
|
|
Year
ended,
|
|
|
December
31,
|
|
December
31,
|
|
|
2020
|
|
2019
|
|
2020
|
|
2019
|
|
|
|
|
|
|
|
|
|
(unaudited)
|
Revenue:
|
|
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$75
|
|
$81
|
|
$287
|
|
$293
|
Product sales,
net
|
|
19
|
|
13
|
|
70
|
|
42
|
Licensing and royalty
revenue
|
|
2
|
|
6
|
|
8
|
|
17
|
Total commercial
revenue
|
|
96
|
|
100
|
|
365
|
|
352
|
Research and development revenue under collaborative
agreements
|
|
194
|
|
394
|
|
364
|
|
771
|
Total
revenue
|
|
290
|
|
494
|
|
729
|
|
1,123
|
Expenses:
|
|
|
|
|
|
|
|
|
Cost of products
sold
|
|
3
|
|
1
|
|
12
|
|
4
|
Research, development and patent
|
|
171
|
|
149
|
|
535
|
|
466
|
Selling, general and administrative
|
|
139
|
|
83
|
|
354
|
|
287
|
Total operating
expenses
|
|
313
|
|
233
|
|
901
|
|
757
|
|
|
|
|
|
|
|
|
|
Income (loss) from
operations
|
|
(23)
|
|
261
|
|
(172)
|
|
366
|
|
|
|
|
|
|
|
|
|
Other income (loss),
net
|
|
(1)
|
|
(23)
|
|
2
|
|
(19)
|
Income (loss) before
income tax benefit (expense)
|
|
(24)
|
|
238
|
|
(170)
|
|
347
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Income tax benefit
(expense)
|
|
(318)
|
|
(34)
|
|
(317)
|
|
(44)
|
|
|
|
|
|
|
|
|
|
Net income
(loss)
|
|
($342)
|
|
$204
|
|
($487)
|
|
$303
|
Net loss
(income) attributable to noncontrolling interest in Akcea
Therapeutics, Inc.
|
|
2
|
|
(20)
|
|
36
|
|
(9)
|
Net income (loss)
attributable to Ionis Pharmaceuticals, Inc. common
stockholders
|
|
($340)
|
|
$184
|
|
($451)
|
|
$294
|
|
|
|
|
|
|
|
|
|
Basic net income
(loss) per share
|
|
($2.44)
|
|
$1.31
|
|
$(3.23)
|
|
$2.12
|
Diluted net income
(loss) per share
|
|
($2.44)
|
|
$1.28
|
|
$(3.23)
|
|
$2.08
|
Shares used in
computing basic net income (loss) per share
|
|
140
|
|
141
|
|
140
|
|
140
|
Shares used in
computing diluted net income (loss) per share
|
|
140
|
|
153
|
|
140
|
|
143
|
IONIS
PHARMACEUTICALS, INC.
|
Reconciliation of
GAAP to Non-GAAP Basis:
|
Condensed
Consolidated Operating Expenses, Income (Loss) From Operations, and
Net Income (Loss)
|
(In
Millions)
|
|
|
|
Three months
ended,
December
31,
|
|
Year ended,
December
31,
|
|
|
2020
|
|
2019
|
|
2020
|
|
2019
|
|
|
(unaudited)
|
As reported
operating expenses according to GAAP
|
|
$313
|
|
$233
|
|
$901
|
|
$757
|
Excluding compensation expense related to equity
awards
|
|
(36)
|
|
(35)
|
|
(171)
|
|
(147)
|
Excluding Akcea acquisition and restructured European
operations costs
|
|
(90)
|
|
-
|
|
(90)
|
|
-
|
|
|
|
|
|
|
|
|
|
Non-GAAP operating
expenses
|
|
$187
|
|
$198
|
|
$640
|
|
$610
|
|
|
|
|
|
|
|
|
|
As reported income
(loss) from operations according to GAAP
|
|
($23)
|
|
$261
|
|
($172)
|
|
$366
|
Excluding compensation expense related to equity awards
|
|
(36)
|
|
(35)
|
|
(171)
|
|
(147)
|
Excluding Akcea
acquisition and restructured European
operations costs
|
|
(90)
|
|
-
|
|
(90)
|
|
-
|
Non-GAAP income
(loss) from operations
|
|
$103
|
|
$296
|
|
$89
|
|
$513
|
|
|
|
|
|
|
|
|
|
As reported net
income (loss) attributable to Ionis Pharmaceuticals, Inc. common
stockholders according to GAAP
|
|
($340)
|
|
$184
|
|
($451)
|
|
$294
|
|
|
|
|
|
|
|
|
|
Excluding compensation
expense related to equity awards attributable to Ionis
Pharmaceuticals, Inc. common stockholders
|
|
(36)
|
|
(33)
|
|
(162)
|
|
(139)
|
|
|
|
|
|
|
|
|
|
Income tax effect
related to compensation expense related to equity awards
attributable to Ionis Pharmaceuticals, Inc. common
stockholders
|
|
(16)
|
|
5
|
|
2
|
|
31
|
Excluding Akcea
acquisition and restructured European
operations costs
|
|
(90)
|
|
-
|
|
(90)
|
|
-
|
Income tax effect
related to the Akcea acquisition and restructured European
operations costs
|
|
(312)
|
|
-
|
|
(312)
|
|
-
|
Non-GAAP net
income (loss) attributable to Ionis Pharmaceuticals, Inc. common
stockholders
|
|
$114
|
|
$212
|
|
$111
|
|
$402
|
Reconciliation of GAAP to Non-GAAP Basis
As illustrated in the Selected Financial Information in this
press release, non-GAAP operating expenses, non-GAAP income (loss)
from operations, and non-GAAP net income (loss) attributable to
Ionis Pharmaceuticals, Inc. common shareholders were adjusted from
GAAP to exclude compensation expense related to equity awards and
costs related to the Akcea acquisition and restructured European
operations and the related tax effects. Compensation expense
related to equity awards are non-cash. Costs related to the Akcea
acquisition and restructured European operations include:
severance costs, retention costs, other costs, non-cash stock-based
compensation expense for the acceleration of Akcea equity
awards and non-cash income tax expense as a result of
establishing a valuation allowance for Ionis' federal net deferred
tax assets. Ionis has regularly reported non-GAAP measures for
operating results as non-GAAP results. These measures are provided
as supplementary information and are not a substitute for financial
measures calculated in accordance with GAAP. Ionis reports these
non-GAAP results to better enable financial statement users to
assess and compare its historical performance and project its
future operating results and cash flows. Further, the presentation
of Ionis' non-GAAP results is consistent with how Ionis' management
internally evaluates the performance of its operations.
IONIS
PHARMACEUTICALS, INC.
Condensed
Consolidated Balance Sheets
(In
Millions)
|
|
|
|
|
|
|
|
|
|
December
31,
|
|
December
31,
|
|
|
|
2020
|
|
2019
|
|
|
|
|
|
|
|
Assets:
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$1,892
|
|
$2,500
|
|
Contracts
receivable
|
|
76
|
|
63
|
|
Other current
assets
|
|
162
|
|
158
|
|
Property,
plant and equipment, net
|
|
181
|
|
154
|
|
Other
assets
|
|
79
|
|
358
|
|
Total assets
|
|
$2,390
|
|
$3,233
|
|
|
|
|
|
|
|
Liabilities and
stockholders' equity:
|
|
|
|
|
|
Other current
liabilities
|
|
$182
|
|
$155
|
|
Current
portion of deferred contract revenue
|
|
108
|
|
118
|
|
1% convertible
senior notes (current in 2020)
|
|
293
|
|
275
|
|
0.125%
convertible senior notes
|
|
456
|
|
435
|
|
Long-term
obligations, less current portion
|
|
84
|
|
75
|
|
Long-term
deferred contract revenue
|
|
424
|
|
490
|
|
Total Ionis
stockholders' equity
|
|
843
|
|
1,471
|
|
Noncontrolling
interest in Akcea Therapeutics, Inc.
|
|
-
|
|
214
|
|
Total
stockholders' equity
|
|
$843
|
|
$1,685
|
|
Total liabilities and stockholders' equity
|
|
$2,390
|
|
$3,233
|
|
|
|
|
|
|
|
|
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SOURCE Ionis Pharmaceuticals, Inc.