Eplontersen NDA submitted to FDA; tofersen
under regulatory review for marketing approval in U.S. and
EU
Phase 3 data planned for eplontersen and
olezarsen; robust late-stage pipeline expanding with two new Phase
3 programs
Ionis provides full year 2023 financial
guidance
CARLSBAD, Calif., Feb. 22,
2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) (the "Company"), a genetic medicines company, today
reported financial results for the fourth quarter and full year
ended December 31, 2022. Financial
results are summarized below:
|
|
Three months
ended
December 31,
|
|
Year ended
December 31,
|
|
|
2022
|
|
2021
|
|
2022
|
|
2021
|
|
|
(amounts in
millions)
|
|
Total
revenue
|
|
$152
|
|
$440
|
|
$587
|
|
$810
|
|
Operating
expenses
|
|
$360
|
|
$219
|
|
$998
|
|
$840
|
|
Operating expenses on a
non-GAAP basis
|
|
$335
|
|
$196
|
|
$898
|
|
$695
|
|
Net (loss)
income
|
|
($52)
|
|
$225
|
|
($270)
|
|
($29)
|
|
Net (loss) income on a
non-GAAP basis
|
|
($168)
|
|
$248
|
|
($311)
|
|
$116
|
|
Cash, cash equivalents and short-term
investments1
|
|
|
|
|
|
$1,987
|
|
$2,115
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
1
Balance at December 31, 2022 does not include $500 million received
in January 2023 under royalty monetization transaction.
|
Financial Highlights
- 2022 revenue was in line with expectations, reflecting revenue
from numerous diverse sources. The prior year fourth quarter and
full year benefited from the $200
million earned from AstraZeneca to jointly develop and
commercialize eplontersen
- 2022 operating expenses increased as planned compared to the
prior year, reflecting investments in pipeline, technology and
go-to-market activities for eplontersen, olezarsen and
donidalorsen
- Further strengthened financial position with royalty
monetization and sale and leaseback transactions worth up to
$1.5 billion, including more than
$700 million already received
- Initiated construction of a new manufacturing facility to
support Ionis' goal to sustainably deliver genetic medicines to the
market
Recent Late-Stage Pipeline Highlights
- Submitted the eplontersen NDA in the U.S. for patients with
polyneuropathy caused by hereditary TTR amyloidosis
- FDA Advisory Committee meeting planned for March 22, 2023 to review Biogen's NDA for
tofersen for patients with SOD1-ALS (PDUFA date of April 25, 2023); EMA accepted MAA for review
- FDA granted olezarsen Fast Track designation for the treatment
of patients with FCS
- Reported positive Phase 2 data from the open label extension
study of donidalorsen in patients with hereditary angioedema
treated for one year, including new data showing clinically
meaningful improvement in angioedema quality of life score; Phase 3
study expected to complete enrollment in 2023
- GSK advanced bepirovirsen into Phase 3 development in patients
with chronic hepatitis B
Recent Additional Pipeline Highlights
- Biogen initiated a Phase 2 study of IONIS-MAPTRx
(BIIB080) in patients with mild cognitive impairment or mild
dementia due to Alzheimer's disease
- Roche initiated a Phase 2 study of tominersen in patients with
prodromal or early manifest Huntington's disease
- Biogen initiated a Phase 1 study of ION306 (BIIB115) for the
treatment of spinal muscular atrophy with the potential for long
interval dosing
Recent Technology Advancement Highlights
- Partnered with Metagenomi to add gene editing capabilities to
Ionis' technology platform
- Advanced programs incorporating muscle LICA technology and MsPA
backbone chemistry into preclinical development
"We made substantial progress in 2022, marked by important
achievements, including the December submission of the eplontersen
NDA for people with ATTRv-PN. We also delivered multiple positive
data readouts, enabling us to advance and expand our rich late- and
mid-stage pipeline. And we took important steps to expand and
diversify our technology, including our Metagenomi collaboration to
add DNA editing to our platform," said Brett P. Monia, Ph.D., chief executive officer
of Ionis. "As we start 2023, we are positioned to build upon our
achievements by bringing our first near-term commercial
opportunities to the market. We look forward to reporting the
66-week results from the eplontersen NEURO-TTRansform study in the
first half of this year. Importantly, we are prepared to
co-commercialization eplontersen with our partner, AstraZeneca. We
also look forward to Phase 3 data from olezarsen in FCS patients,
positioning us for our first independent launch. With the talent
and resources we have today, we anticipate a highly productive year
that will enable us to drive increasing value for all
stakeholders."
Fourth Quarter and Full Year 2022 Financial Results
Revenue
Ionis' revenue was comprised of the following:
|
|
Three months
ended
|
|
Year ended
|
|
|
|
December 31,
|
|
December 31,
|
|
|
|
2022
|
|
2021
|
|
2022
|
|
2021
|
|
Revenue:
|
|
(amounts in
millions)
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$67
|
|
$69
|
|
$242
|
|
$268
|
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
7
|
|
9
|
|
30
|
|
56
|
|
Licensing and royalty
revenue
|
|
6
|
|
9
|
|
31
|
|
18
|
|
Total commercial
revenue
|
|
80
|
|
87
|
|
303
|
|
342
|
|
Research and
development revenue:
|
|
|
|
|
|
|
|
|
|
Amortization from
upfront payments
|
|
15
|
|
21
|
|
69
|
|
78
|
|
Milestone
payments
|
|
14
|
|
40
|
|
74
|
|
88
|
|
License
fees
|
|
-
|
|
290
|
|
37
|
|
291
|
|
Other
services
|
|
22
|
|
2
|
|
27
|
|
11
|
|
Collaborative
agreement revenue
|
|
51
|
|
353
|
|
207
|
|
468
|
|
Eplontersen joint
development revenue
|
|
21
|
|
-
|
|
77
|
|
-
|
|
Total research and
development revenue
|
|
72
|
|
353
|
|
284
|
|
468
|
|
Total
revenue
|
|
$152
|
|
$440
|
|
$587
|
|
$810
|
|
|
|
|
|
|
|
Ionis' 2022 revenue continued to be derived from diverse
sources, with just over half coming from commercial products and
the balance from numerous partnered programs. SPINRAZA royalties,
the largest contributor to the Company's commercial revenue,
increased each quarter in 2022. Total SPINRAZA product sales
increased six percent in the fourth quarter of 2022 compared to the
prior quarter and also increased four percent compared to the same
quarter in 2021. The increases were driven by stabilization in the
U.S. and growth in Asian markets partially offset by competition in
Europe. Total SPINRAZA product
sales decreased six percent year-over-year due to foreign currency
exchange and competition in Europe. TEGSEDI and WAYLIVRA revenue in 2022
reflected the shift to distribution fees.
R&D revenue for 2022 included $112
million from Biogen for advancing several neurology disease
programs, $77 million from
AstraZeneca for its share of the global Phase 3 development costs
for eplontersen and $64 million from
Roche for licensing and advancing IONIS-FB-LRx, among
other partner payments. R&D revenue was higher in 2021 compared
to 2022 primarily due to the $200
million Ionis earned in the fourth quarter of 2021 from
AstraZeneca to jointly develop and commercialize eplontersen.
Operating Expenses
Ionis' operating expenses increased for the three months and
year ended December 31, 2022 compared
to the same periods in 2021, in line with expectations. For both
periods, higher R&D expenses were driven by the increased
number of Phase 3 studies the Company is conducting, which doubled
from three to six studies in 2021, and the $80 million upfront payment for Ionis'
collaboration with Metagenomi. SG&A expenses increased for the
three months ended December 31, 2022
compared to the same period in 2021 driven by Ionis' go-to-market
activities for eplontersen, olezarsen and donidalorsen. SG&A
expenses were lower for 2022 compared to 2021 largely due to the
substantial savings the Company achieved from integrating Akcea and
restructuring the Company's commercial operations in 2021.
Gain on Sale of Property and Related Tax Impact
In October 2022, Ionis entered
into a sale and leaseback transaction for several of its real
estate assets. Under the agreement, Ionis received net proceeds of
$200 million, with the potential to
receive additional payments of up to $40
million plus funding to expand the Company's R&D campus.
As a result, the Company recognized a $150
million gain on sale of property and $9 million in related income tax expense in the
fourth quarter of 2022. In conjunction with the sale and leaseback
transaction, the Company extinguished its mortgage debt for the
related properties and recorded a new right of lease asset and
liability on its balance sheet.
Balance Sheet
As of December 31, 2022, Ionis had
cash, cash equivalents and short-term investments of $2.0 billion compared to $2.1 billion at December
31, 2021. Ionis' year-end cash balance does not include the
$500 million the Company received
from Royalty Pharma in January 2023.
From December 31, 2021 to
December 31, 2022, Ionis' debt
obligations decreased by $50 million
because the Company repaid its mortgage debt and Ionis' working
capital decreased modestly driven by the Company's slightly lower
cash and short-term investments balance.
2023 Financial Guidance
The Company's 2023 guidance reflects its ability to earn
substantial revenue from its commercial portfolio and partnered
programs. It also reflects the Company's commitment to investing in
advancing its rich late-stage pipeline and preparing to
commercialize its near-term commercial opportunities, eplontersen,
olezarsen and donidalorsen, while maintaining a healthy balance
sheet to continue investing for future growth.
Full Year 2023
Guidance
|
|
|
|
|
Revenue
|
|
>$575
million
|
|
|
Operating expenses on a
non-GAAP basis
|
|
~$970 - $995
million
|
|
|
Net operating loss on a
non-GAAP basis
|
|
<$425
million
|
|
|
Cash, cash equivalents
and short-term investments
|
|
~$2.0
billion
|
|
|
"Our solid 2022 financial results reflected our ability to earn
substantial revenues while investing in key programs with the
potential to drive substantial future growth, including our
near-term commercial opportunities," said Elizabeth L. Hougen, chief financial officer of
Ionis. "Additionally, we recently bolstered our balance sheet with
more than $700 million from our
royalty monetization and sale and leaseback transactions. With
approximately $2.5 billion in pro
forma cash, we have the resources to continue to advance our
innovative pipeline and achieve commercial readiness for
eplontersen, olezarsen and donidalorsen."
Webcast
Management will host a conference call and webcast to discuss
Ionis' fourth quarter and full year 2022 results at 11:30 a.m. Eastern time on Wednesday, February
22, 2022. Interested parties may access the webcast here. A webcast
replay will be available for a limited time at the same address. To
access the Company's fourth quarter and full year 2022 earnings
slides click here.
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 cardiovascular and
neurological franchises. Our scientific innovation began and
continues with the knowledge that sick people depend on us, which
fuels our vision of becoming the leader in genetic medicine,
utilizing a multi-platform approach to discover, develop and
deliver life-transforming therapies.
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),
WAYLIVRA (volanesorsen), eplontersen, olezarsen, donidalorsen,
ION363, tofersen, pelacarsen, bepirovirsen, Ionis' technologies and
Ionis' other products in development. 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 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,
2021, and most recent Form 10-Q, which are on file with
the Securities and Exchange Commission. 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" all refer to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered 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,
|
|
|
2022
|
|
2021
|
|
2022
|
|
2021
|
|
|
(unaudited)
|
|
|
|
|
Revenue:
|
|
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$67
|
|
$69
|
|
$242
|
|
$268
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
7
|
|
9
|
|
30
|
|
56
|
Licensing and royalty
revenue
|
|
6
|
|
9
|
|
31
|
|
18
|
Total commercial
revenue
|
|
80
|
|
87
|
|
303
|
|
342
|
Research and
development revenue:
|
|
|
|
|
|
|
|
|
Collaborative
agreement revenue
|
|
51
|
|
353
|
|
207
|
|
468
|
Eplontersen joint
development revenue
|
|
21
|
|
-
|
|
77
|
|
-
|
Total research and
development revenue
|
|
72
|
|
353
|
|
284
|
|
468
|
Total
revenue
|
|
152
|
|
440
|
|
587
|
|
810
|
Expenses:
|
|
|
|
|
|
|
|
|
Cost of
sales
|
|
4
|
|
2
|
|
14
|
|
11
|
Research, development and patent
|
|
308
|
|
179
|
|
833
|
|
643
|
Selling, general and administrative
|
|
48
|
|
38
|
|
151
|
|
186
|
Total operating
expenses
|
|
360
|
|
219
|
|
998
|
|
840
|
Income (loss) from
operations
|
|
(208)
|
|
221
|
|
(411)
|
|
(30)
|
|
|
|
|
|
|
|
|
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
Gain on sale of real
estate assets
|
|
150
|
|
-
|
|
150
|
|
-
|
Other income,
net
|
|
14
|
|
4
|
|
3
|
|
-
|
Income (loss) before
income tax benefit (expense)
|
|
(44)
|
|
225
|
|
(258)
|
|
(30)
|
|
|
|
|
|
|
|
|
|
Income tax benefit
(expense)
|
|
(8)
|
|
-
|
|
(12)
|
|
1
|
|
|
|
|
|
|
|
|
|
Net income
(loss)
|
|
($52)
|
|
$225
|
|
($270)
|
|
($29)
|
|
|
|
|
|
|
|
|
|
Basic net income (loss)
per share
|
|
($0.37)
|
|
$1.59
|
|
($1.90)
|
|
($0.20)
|
Diluted net income
(loss) per share
|
|
($0.37)
|
|
$1.41
|
|
($1.90)
|
|
($0.20)
|
Shares used in
computing basic net income (loss) per share
|
|
142
|
|
141
|
|
142
|
|
141
|
Shares used in
computing diluted net income (loss) per share
|
|
142
|
|
160
|
|
142
|
|
141
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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,
|
|
|
2022
|
|
2021
|
|
2022
|
|
2021
|
|
|
(unaudited)
|
As reported
research, development and patent expenses according to
GAAP
|
|
$308
|
|
$179
|
|
$833
|
|
$643
|
Excluding compensation expense related to equity
awards
|
|
(19)
|
|
(16)
|
|
(74)
|
|
(88)
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
(1)
|
|
-
|
|
(9)
|
Non-GAAP research,
development and patent expenses
|
|
$289
|
|
$162
|
|
$759
|
|
$546
|
|
|
|
|
|
|
|
|
|
As reported selling,
general and administrative expenses according to
GAAP
|
|
$48
|
|
$38
|
|
$151
|
|
$186
|
Excluding compensation expense related to equity
awards
|
|
(7)
|
|
(7)
|
|
(26)
|
|
(33)
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
1
|
|
-
|
|
(15)
|
Non-GAAP selling,
general and administrative expenses
|
|
$41
|
|
$32
|
|
$125
|
|
$138
|
|
|
|
|
|
|
|
|
|
As reported
operating expenses according to GAAP
|
|
$360
|
|
$219
|
|
$998
|
|
$840
|
Excluding compensation
expense related to equity
awards
|
|
(25)
|
|
(23)
|
|
(100)
|
|
(121)
|
Excluding Akcea merger
and restructured commercial operation
costs*
|
|
-
|
|
-
|
|
-
|
|
(24)
|
Non-GAAP operating
expenses
|
|
$335
|
|
$196
|
|
$898
|
|
$695
|
|
|
|
|
|
|
|
|
|
As reported income
(loss) from operations according to GAAP
|
|
($208)
|
|
$221
|
|
($411)
|
|
($30)
|
Excluding compensation expense related to equity
awards
|
|
(25)
|
|
(23)
|
|
(100)
|
|
(121)
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
-
|
|
-
|
|
(24)
|
Non-GAAP income
(loss) from operations
|
|
($183)
|
|
$244
|
|
($311)
|
|
$115
|
|
|
|
|
|
|
|
|
|
As reported net
income (loss) according to GAAP
|
|
($52)
|
|
$225
|
|
($270)
|
|
($29)
|
Excluding compensation expense related to equity
awards
|
|
(25)
|
|
(23)
|
|
(100)
|
|
(121)
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
-
|
|
-
|
|
(24)
|
Excluding gain on sale of real estate assets**
|
|
150
|
|
-
|
|
150
|
|
-
|
Excluding income tax effect related to gain on sale of real estate
assets
|
|
(9)
|
|
-
|
|
(9)
|
|
-
|
Non-GAAP net income
(loss)
|
|
($168)
|
|
$248
|
|
($311)
|
|
$116
|
|
*In October 2020, Ionis
completed a merger transaction with Akcea such that following the
completion of the merger Akcea became a wholly owned subsidiary of
Ionis. Additionally, in December 2020 and April 2021, Ionis
restructured its European operations and its North American TEGSEDI
operations, respectively, as a result of entering into distribution
agreements with Sobi. The Company excluded the Akcea merger
and restructured commercial operation costs from its non-GAAP
amounts for the applicable periods.
|
|
**In October 2022,
Ionis entered into a sale and leaseback transaction for several of
its real estate assets. As a result, the Company recognized a $150
million gain on sale of real estate assets in the fourth quarter of
2022. The Company excluded the gain on sale of real estate assets
and the related tax effect from its non-GAAP amounts for the
applicable periods.
|
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) were adjusted from
GAAP to exclude compensation expense related to equity awards and
the related tax effects. Compensation expense related to equity
awards are non-cash. In 2022 Ionis' non-GAAP net loss excluded the
gain on property related to the sale and leaseback transaction and
the related tax effect. In 2021 all non-GAAP amounts also excluded
expenses related to the Akcea merger and restructured commercial
operations. Expenses related to the Akcea merger and restructured
commercial operations included: severance costs, retention
costs and other costs related to commercial operations. 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.
Summary of the
Financial Impacts of the Eplontersen Collaboration with
AstraZeneca
For the Year Ended,
December 31, 2022
(Unaudited)
|
|
Collaboration
Activities
|
|
Financial
Statement Line
|
|
Impact of
Cost-Sharing Provisions
on Ionis' Statement
of Operations
|
|
|
|
|
|
|
|
Phase 3
Development:
Ionis leads and conducts
|
|
Eplontersen Joint
Development
Revenue
(R&D
Revenue)
|
|
$77M
|
|
55% of Total Phase
3
development expenses,
including
internal+external costs &
CMC costs, net of Ionis'
share of AstraZeneca's
Phase 3 development
expenses
|
|
|
|
|
|
|
|
Development
Expenses
(R&D
Expenses)
|
|
$147M
|
|
100% of Ionis' Phase
3
development expenses
|
Ionis' financial results for the year ended December 31, 2022 reflected the cost-sharing
provisions related to its collaboration with AstraZeneca to develop
and commercialize eplontersen for the treatment of ATTR. Under the
terms of the collaboration agreement, AstraZeneca is currently
paying 55 percent of the costs associated with the ongoing global
Phase 3 development program. Because Ionis is leading and
conducting the Phase 3 development program, Ionis is recognizing
the 55 percent of cost-share funding AstraZeneca is responsible
for, net of Ionis' share of AstraZeneca's development expenses, as
R&D revenue in the same period Ionis incurs the related
development expenses. For the year ended December 31, 2022 Ionis earned $77 million in joint development revenue under
this collaboration.
Because AstraZeneca is responsible for the majority of the
medical affairs and commercial costs in the U.S. and all costs
associated with commercializing eplontersen outside the U.S., Ionis
is recognizing cost-share funding it receives from AstraZeneca
related to these activities as a reduction of its medical affairs
(R&D expenses) and commercialization expenses (SG&A
expenses). For the year ended December 31,
2022 Ionis recognized $2.0
million and $2.6 million of
medical affairs expenses and commercialization expenses for
eplontersen, respectively, net of cost-share funding from
AstraZeneca. Ionis expects its medical affairs and
commercialization expenses to increase as this collaboration
progresses.
IONIS
PHARMACEUTICALS, INC.
Condensed Consolidated Balance Sheets
(In Millions)
|
|
|
|
|
|
|
|
|
December 31,
|
|
December 31,
|
|
|
|
2022
|
|
2021
|
|
|
|
(unaudited)
|
|
|
|
Assets:
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$1,987
|
|
$2,115
|
|
Contracts
receivable
|
|
26
|
|
62
|
|
Other current
assets
|
|
190
|
|
168
|
|
Property, plant
and equipment, net
|
|
74
|
|
178
|
|
Right-of-use
assets
|
|
182
|
|
18
|
|
Other
assets
|
|
75
|
|
71
|
|
Total assets
|
|
$2,534
|
|
$2,612
|
|
|
|
|
|
|
|
Liabilities and
stockholders' equity:
|
|
|
|
|
|
Other current
liabilities
|
|
$221
|
|
$143
|
|
Current portion
of deferred contract revenue
|
|
91
|
|
98
|
|
0% convertible
senior notes, net
|
|
622
|
|
619
|
|
0.125%
convertible senior notes, net
|
|
545
|
|
542
|
|
Long-term lease
liabilities
|
|
178
|
|
19
|
|
Long-term
obligations, less current portion
|
|
16
|
|
67
|
|
Long-term
deferred contract revenue
|
|
288
|
|
352
|
|
Total
stockholders' equity
|
|
573
|
|
772
|
|
Total liabilities and stockholders' equity
|
|
$2,534
|
|
$2,612
|
|
|
|
|
|
|
|
|
|
2023 Key Value Driving Events(1)
Regulatory
Actions
|
Program
|
Indication
|
Regulatory
Action
|
Tofersen
|
SOD1-ALS
|
NDA approval
|
EU approval
|
Eplontersen
(TTR)
|
ATTRv
polyneuropathy
|
NDA approval
|
OUS filings
|
|
Key Clinical
Achievements
|
Program
|
Indication
|
Event
|
Eplontersen
(TTR)
|
ATTRv
polyneuropathy
|
Phase 3 data (week 35
& 66)
|
Olezarsen
|
FCS
|
Phase 3 data
|
Eplontersen
(TTR)
|
ATTR
cardiomyopathy
|
Phase 3 full
enrollment
|
Donidalorsen
(PKK)
|
HAE
|
Phase 3 full
enrollment
|
|
Phase 3
Initiations
|
Program
|
Indication
|
Timing
|
Bepirovirsen
(HBV)
|
Hepatitis B virus
infection
|
H1:23
(achieved)
|
IONIS-FB-LRx
|
Immunoglobulin A
nephropathy
|
H1:23
|
|
(1) Timing
expectations based on current assumptions and subject to
change.
|
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SOURCE Ionis Pharmaceuticals, Inc.