WAINUA™ approved with launch
underway; on track for EU and Canada approval decisions this year
Positive Phase 3 olezarsen and donidalorsen
data, preparing regulatory submissions for FCS and HAE,
respectively
Olezarsen granted Breakthough Therapy
designation by the FDA for FCS
Ionis provides full year 2024 financial
guidance
CARLSBAD,
Calif., Feb. 21, 2024 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company"), today
reported financial results for the fourth quarter and full year
ended December 31, 2023.
"This past year included many remarkable
achievements as we continued to advance our vision to bring better
futures to people with serious diseases. Ionis achieved two FDA
approvals, delivered three positive Phase 3 data readouts, expanded
our rich Phase 3 pipeline to nine medicines and advanced our next
wave of wholly owned medicines as well as our technology," said
Brett P. Monia, Ph.D., chief
executive officer of Ionis. "In 2024, we anticipate building on our
success with important catalysts and continued value creation. The
WAINUA U.S. launch is underway for patients with hereditary ATTR
polyneuropathy, and we expect additional approvals in other
countries this year. We plan to present positive Phase 3 data for
olezarsen in familial chylomicronemia syndrome and donidalorsen in
hereditary angioedema, positioning Ionis to independently launch
these two medicines. We also anticipate additional readouts from
multiple mid-stage programs that, if positive, would advance into
Phase 3 development, further strengthening our ability to deliver a
steady cadence of potentially transformational medicines for years
to come."
Fourth Quarter and Full Year 2023 Summary
Financial Results(1):
|
|
Three months
ended
December
31,
|
|
Year ended
December
31,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
(amounts in
millions)
|
Total
revenue
|
|
$325
|
|
$152
|
|
$788
|
|
$587
|
Operating
expenses
|
|
$331
|
|
$360
|
|
$1,141
|
|
$998
|
Operating expenses on a
non-GAAP basis
|
|
$305
|
|
$335
|
|
$1,035
|
|
$898
|
Loss from
operations
|
|
($6)
|
|
($208)
|
|
($353)
|
|
($411)
|
Income (Loss) from
operations on a non-GAAP basis
|
|
$20
|
|
($183)
|
|
($247)
|
|
($311)
|
|
(1) Reconciliation of
GAAP to non-GAAP basis contained later in this release.
|
Financial Highlights
- Revenue more than doubled for the fourth quarter of 2023
compared to the same period in the prior year and increased 34% for
the full year, driven by the successful progression of Ionis'
pipeline and technology platform
- Operating expenses increased compared to the prior year,
primarily due to strategic investments to bring eplontersen,
olezarsen and donidalorsen to patients
- 2023 operating loss significantly improved over prior year due
to substantial revenue earned during the year
- Cash and short-term investments of $2.3
billion as of December 31,
2023 enables continued investments to drive increasing
value, including supporting our potential upcoming launches
Recent Marketed Medicines Highlights
- WAINUA approved in the U.S., resulting in a $50 million milestone payment from AstraZeneca;
launch underway for treatment of adults with polyneuropathy of
hereditary transthyretin-mediated amyloidosis (ATTRv-PN)
- SPINRAZA continued to be the global market leader for the
treatment of spinal muscular atrophy (SMA) with global sales of
$1.7 billion in 2023
Recent Late-Stage Pipeline
Highlights
- Eplontersen granted Fast Track designation by the FDA for the
treatment of patients with ATTR cardiomyopathy (ATTR-CM)
- Olezarsen granted Breakthrough Therapy designation by the FDA
for the treatment of patients with familial chylomicronemia
syndrome (FCS)
- Olezarsen granted orphan drug designation by the FDA for the
treatment of patients with FCS
- Achieved multiple milestones with donidalorsen for the
treatment of patients with hereditary angioedema (HAE):
- Reported positive topline data from the Phase 3 OASIS-HAE study
in patients treated every four weeks or every eight weeks;
preparing to submit NDA
- Licensed European donidalorsen commercialization rights to
Otsuka; Otsuka preparing to submit MAA
- Donidalorsen granted orphan drug designation by EMA
- Reported positive Phase 2 data from the open label extension
study in patients with HAE treated for two years
- Bepirovirsen granted fast track designation by the FDA for the
treatment of patients with chronic hepatitis B (CHB)
Recent Other Pipeline Highlights
- Achieved multiple milestones with ION582 (BIIB121) for the
treatment of patients with Angelman syndrome:
- Completed enrollment in the Phase 1/2 HALOS study in patients
with Angelman syndrome; on track for data readout in mid-2024
- Presented positive clinical update from ongoing HALOS study at
the FAST (Foundation for Angelman Syndrome Therapeutics)
Summit
- Extended the long-term extension portion of HALOS study
- Sapablursen and ION356 granted fast track designation by the
FDA for the treatment of patients with polycythemia vera (PV) and
Pelizaeus-Merzbacher disease (PMD), respectively
- Initiated the Phase 1/2 PrProfile study of ION717 in patients
with Prion disease
- AstraZeneca licensed ION826 for the treatment of heart failure,
resulting in a $36 million payment
from AstraZeneca
Recent Technology Advancement
Highlights
- Licensed Vect-Horus' blood-brain barrier crossing technology
for the development of RNA-targeted neurological disease
medicines
Fourth Quarter, Full Year 2023 Financial
Results and 2024 Financial Guidance
"In 2023, we earned substantial revenues due to
continued success with our pipeline and technology. As a result, we
exceeded our 2023 revenue guidance, which drove a smaller than
anticipated operating loss," said Elizabeth
L. Hougen, chief financial officer of Ionis. "In 2024, with
WAINUA's launch for ATTRv-PN underway, we are adding a new stream
of royalty revenue to our substantial and sustained revenues. We
will continue to deploy our capital resources toward growth
opportunities that can enable Ionis to unlock next-level value.
This includes continuing to make significant investments in
near-term commercial opportunities, the expansion of our wholly
owned pipeline, and advancing our technology, all of which should
empower future growth."
Revenue
Ionis' revenue was comprised of the
following:
|
|
Three months
ended
|
|
Year ended
|
|
|
December
31,
|
|
December
31,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
Revenue:
|
|
(amounts in
millions)
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$62
|
|
$67
|
|
$240
|
|
$242
|
Other commercial
revenue:
|
|
|
|
|
|
|
|
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
9
|
|
7
|
|
35
|
|
30
|
Licensing and royalty
revenue
|
|
8
|
|
6
|
|
34
|
|
31
|
Total commercial
revenue
|
|
79
|
|
80
|
|
309
|
|
303
|
Research and
development revenue:
|
|
|
|
|
|
|
|
|
Amortization from
upfront payments
|
|
76
|
|
15
|
|
125
|
|
69
|
Milestone
payments
|
|
11
|
|
14
|
|
101
|
|
74
|
License
fees
|
|
92
|
|
-
|
|
117
|
|
37
|
Other
services
|
|
-
|
|
22
|
|
10
|
|
27
|
Collaborative
agreement revenue
|
|
179
|
|
51
|
|
353
|
|
207
|
WAINUA joint
development revenue
|
|
67
|
|
21
|
|
126
|
|
77
|
Total research and
development revenue
|
|
246
|
|
72
|
|
479
|
|
284
|
Total
revenue
|
|
$325
|
|
$152
|
|
$788
|
|
$587
|
Commercial revenues in 2023 were comparable to
2022. Commercial revenue for 2023 included $240 million from SPINRAZA royalties, which was
comparable to 2022. Ionis' commercial revenue in 2023 also included
royalties from QALSODY U.S. product sales.
R&D revenue significantly increased in 2023
compared to 2022 primarily due to continued success with Ionis'
pipeline and technology. As a result, Ionis earned significant
partner payments, including $50
million from AstraZeneca for the FDA approval of WAINUA for
ATTRv-PN in the U.S., $36 million
from AstraZeneca for licensing ION826 and payments from Ionis' new
collaborations with Otsuka, Roche and Novartis.
Operating Expenses
Ionis' operating expenses increased for the year
ended December 31, 2023 compared to
2022 primarily due to certain one-time costs, including a non-cash
charge associated with a lease exit and the license fee Ionis paid
to Vect-Horus. As Ionis advanced its robust pipeline, study costs
increased compared to the same periods in 2022 as many of the
Company's Phase 3 studies are either fully enrolled or approaching
full enrollment, resulting in higher R&D expenses year over
year. R&D expenses for the fourth quarter of 2023 were lower
compared to the fourth quarter of 2022, primarily due to the
$80 million upfront payment Ionis
paid to Metagenomi in 2022. Ionis' SG&A expenses increased year
over year primarily due to launch preparation activities for
WAINUA, olezarsen and donidalorsen.
Balance Sheet
As of December 31,
2023, Ionis' cash, cash equivalents and short-term
investments increased to $2.3 billion
compared to $2.0 billion at
December 31, 2022 primarily due to
the $500 million Ionis received from
Royalty Pharma in January 2023 and
significant partner payments throughout 2023. Ionis' working
capital also increased over the same period primarily due to the
Company's higher cash and short-term investments balance. In 2023,
the Company recorded a long-term liability for future royalties due
to Royalty Pharma. In June 2023,
Ionis issued $575 million of senior
convertible notes due in June 2028
with an interest rate of 1.75%. The Company used the majority of
the proceeds to repurchase $504
million of its 0.125% convertible notes.
2024 Financial Guidance
The Company's 2024 guidance reflects its plan to
deploy its capital resources toward growth opportunities, including
continued investments in its near-term commercial opportunities,
expanding its wholly owned pipeline and advancing its technology
platform. Additionally, the Company expects to continue earning
substantial revenue from its commercial portfolio and partnered
programs.
Full Year 2024
Guidance
|
|
|
|
|
Revenue
|
|
>$575
million
|
|
|
Operating loss on a
non-GAAP basis
|
|
<$475
million
|
|
|
Cash, cash equivalents
and short-term investments
|
|
~$1.7
billion
|
|
|
Webcast
Management will host a conference call and
webcast to discuss Ionis' fourth quarter and full year 2023 results
at 11:30 a.m. Eastern time on
Wednesday, February 21, 2024. 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 2023 earnings slides click here.
For more information about SPINRAZA and QALSODY,
visit https://www.spinraza.com/ and https://www.qalsody.com/,
respectively. QALSODY is approved under accelerated approval
based on reduction in plasma neurofilament light chain (NfL)
observed in patients treated with QALSODY. Continued approval may
be contingent upon verification of clinical benefit in confirmatory
trial(s).
INDICATION for WAINUA™
(eplontersen)
WAINUA injection, for subcutaneous use, 45 mg
is indicated for the treatment of the polyneuropathy of hereditary
transthyretin-mediated amyloidosis in adults.
IMPORTANT SAFETY INFORMATION for
WAINUA™ (eplontersen)
WARNINGS AND PRECAUTIONS
Reduced Serum Vitamin A Levels and Recommended
Supplementation WAINUA leads to a decrease in serum
vitamin A levels. Supplement with recommended daily allowance of
vitamin A. Refer patient to an ophthalmologist if ocular symptoms
suggestive of vitamin A deficiency occur.
ADVERSE REACTIONS
Most common adverse reactions (≥9% in WAINUA-treated patients) were
vitamin A decreased (15%) and vomiting (9%).
Please see link to U.S. Full
Prescribing Information for WAINUA.
About Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines
that bring better futures to people with serious diseases. Ionis
currently has five marketed medicines and a leading pipeline in
neurology, cardiology, and other areas of high patient need. As the
pioneer in RNA-targeted medicines, Ionis continues to drive
innovation in RNA therapies in addition to advancing new approaches
in gene editing. A deep understanding of disease biology and
industry-leading technology propels our work, coupled with a
passion and urgency to deliver life-changing advances for
patients. To learn more about Ionis, visit
Ionispharma.com and follow us on X (Twitter) and
LinkedIn.
Ionis' Forward-looking Statement
This press release includes forward-looking
statements regarding Ionis' business, financial guidance and the
therapeutic and commercial potential of our commercial medicines,
additional medicines in development and technologies. 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. Except as required by law, we undertake
no obligation to update any forward-looking statements for any
reason. 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,
2022, 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. QALSODYTM is a
trademark of Biogen. SPINRAZA® is a registered trademark
of Biogen. WAINUATM is a registered trademark of the
AstraZeneca group of companies.
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,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
(unaudited)
|
Revenue:
|
|
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$62
|
|
$67
|
|
$240
|
|
$242
|
Other commercial
revenue
|
|
17
|
|
13
|
|
69
|
|
61
|
Total commercial
revenue
|
|
79
|
|
80
|
|
309
|
|
303
|
Research and
development revenue:
|
|
|
|
|
|
|
|
|
Collaborative
agreement revenue
|
|
179
|
|
51
|
|
353
|
|
207
|
WAINUA joint
development revenue
|
|
67
|
|
21
|
|
126
|
|
77
|
Total research and
development revenue
|
|
246
|
|
72
|
|
479
|
|
284
|
Total
revenue
|
|
325
|
|
152
|
|
788
|
|
587
|
Expenses:
|
|
|
|
|
|
|
|
|
Cost of
sales
|
|
3
|
|
4
|
|
9
|
|
14
|
Research, development and patent
|
|
257
|
|
308
|
|
900
|
|
833
|
Selling, general and administrative
|
|
71
|
|
48
|
|
232
|
|
151
|
Total operating
expenses
|
|
331
|
|
360
|
|
1,141
|
|
998
|
Loss from
operations
|
|
(6)
|
|
(208)
|
|
(353)
|
|
(411)
|
|
|
|
|
|
|
|
|
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
Interest expense
related to the sale of future royalties
|
|
(18)
|
|
-
|
|
(69)
|
|
-
|
Gain on sale of real
estate assets
|
|
-
|
|
150
|
|
-
|
|
150
|
Other income
(expense), net
|
|
21
|
|
14
|
|
88
|
|
3
|
Loss before income tax
expense
|
|
(3)
|
|
(44)
|
|
(334)
|
|
(258)
|
|
|
|
|
|
|
|
|
|
Income tax
expense
|
|
(6)
|
|
(8)
|
|
(32)
|
|
(12)
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
($9)
|
|
($52)
|
|
($366)
|
|
($270)
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share
|
|
($0.06)
|
|
($0.37)
|
|
($2.56)
|
|
($1.90)
|
Shares used in
computing basic and diluted net loss per share
|
|
144
|
|
142
|
|
143
|
|
142
|
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,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
(unaudited)
|
As reported research, development and patent expenses
according to GAAP
|
|
$257
|
|
$308
|
|
$900
|
|
$833
|
Excluding compensation expense related to equity
awards
|
|
(20)
|
|
(19)
|
|
(78)
|
|
(74)
|
Non-GAAP research, development and patent
expenses
|
|
$237
|
|
$289
|
|
$822
|
|
$759
|
|
|
|
|
|
|
|
|
|
As reported selling, general and administrative
expenses according to GAAP
|
|
$71
|
|
$48
|
|
$232
|
|
$151
|
Excluding compensation expense related to equity
awards
|
|
(6)
|
|
(7)
|
|
(27)
|
|
(26)
|
Non-GAAP selling, general and administrative
expenses
|
|
$65
|
|
$41
|
|
$205
|
|
$125
|
|
|
|
|
|
|
|
|
|
As reported operating expenses according to
GAAP
|
|
$331
|
|
$360
|
|
$1,141
|
|
$998
|
Excluding compensation
expense related to equity
awards
|
|
(26)
|
|
(25)
|
|
(106)
|
|
(100)
|
Non-GAAP operating expenses
|
|
$305
|
|
$335
|
|
$1,035
|
|
$898
|
|
|
|
|
|
|
|
|
|
As reported loss from operations according to
GAAP
|
|
($6)
|
|
($208)
|
|
($353)
|
|
($411)
|
Excluding compensation expense related to equity
awards
|
|
(26)
|
|
(25)
|
|
(106)
|
|
(100)
|
Non-GAAP income (loss) from
operations
|
|
$20
|
|
($183)
|
|
($247)
|
|
($311)
|
|
|
|
|
|
|
|
|
|
As reported net loss according to
GAAP
|
|
($9)
|
|
($52)
|
|
($366)
|
|
($270)
|
Excluding compensation expense related to equity awards and related
tax effects
|
|
(26)
|
|
(25)
|
|
(106)
|
|
(100)
|
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)
|
|
$17
|
|
($168)
|
|
($260)
|
|
($311)
|
*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 real estate assets related
to the sale and leaseback transaction and the related tax effects.
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,
|
|
|
2023
|
|
2022
|
|
|
(unaudited)
|
|
|
Assets:
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$2,331
|
|
$1,987
|
Contracts
receivable
|
|
98
|
|
26
|
Other current
assets
|
|
213
|
|
190
|
Property, plant
and equipment, net
|
|
71
|
|
74
|
Right-of-use
assets
|
|
172
|
|
182
|
Other
assets
|
|
105
|
|
75
|
Total assets
|
|
$2,990
|
|
$2,534
|
|
|
|
|
|
Liabilities and
stockholders' equity:
|
|
|
|
|
Current portion
of deferred contract revenue
|
|
$151
|
|
$91
|
0.125%
convertible senior notes, net – short-term
|
|
44
|
|
-
|
Other current
liabilities
|
|
253
|
|
221
|
1.75%
convertible senior notes, net
|
|
562
|
|
-
|
0% convertible
senior notes, net
|
|
625
|
|
622
|
0.125%
convertible senior notes, net – long-term
|
|
-
|
|
545
|
Liability
related to sale of future royalties, net
|
|
514
|
|
-
|
Long-term lease
liabilities
|
|
171
|
|
178
|
Long-term
obligations, less current portion
|
|
42
|
|
16
|
Long-term
deferred contract revenue
|
|
241
|
|
288
|
Total
stockholders' equity
|
|
387
|
|
573
|
Total liabilities and stockholders' equity
|
|
$2,990
|
|
$2,534
|
Key 2024 Value Driving Events(1)
New Product Launches
|
Program
|
Indication
|
|
Achieved
|
WAINUA
|
ATTRv-PN
|
|
•
|
Olezarsen
|
FCS
|
|
|
QALSODY (EU)
|
SOD1-ALS
|
|
|
|
Regulatory Actions
|
Program
|
Indication
|
Regulatory Action
|
Achieved
|
Eplontersen
|
ATTRv-PN
|
Additional OUS
filings
|
•
|
EMA approval
decision
|
|
Additional OUS approval
decision(s)
|
|
Olezarsen
|
FCS
|
NDA filing
|
|
FDA approval
decision
|
|
EU filing
|
|
Canada
filing
|
|
Donidalorsen
|
HAE
|
NDA filing
|
|
QALSODY
|
SOD1-ALS
|
EMA approval
decision
|
|
|
Key Phase 3 Clinical Data
Events
|
Program
|
Indication
|
Event
|
Achieved
|
Donidalorsen
|
HAE
|
OASIS-HAE topline
data
|
•
|
Donidalorsen
|
HAE
|
OASIS-HAE full
data
|
|
Donidalorsen
|
HAE
|
OASIS-Plus: OLE +
Switch data
|
|
Olezarsen
|
FCS
|
Balance study full
data
|
|
|
Key Phase 2 Clinical Data
Events
|
Program
|
Indication
|
Event
|
Achieved
|
Donidalorsen
|
HAE
|
3-year OLE
data
|
|
IONIS-FB-LRx
|
IgAN
|
Phase 2 data
|
|
IONIS-FB-LRx
|
GA
|
GOLDEN study
data
|
|
ION224
(DGAT2)
|
NASH
|
Phase 2 data
|
|
ION582
(UBE3A)
|
Angelman
syndrome
|
HALOS study
data
|
|
ION541
(ATXN2)
|
ALS
|
ALSpire study
data
|
|
|
(1) Timing expectations
based on current assumptions and subject to
change.
|
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SOURCE Ionis Pharmaceuticals, Inc.