Olezarsen Phase 3 data showed significant
triglyceride lowering, substantial reductions in acute pancreatitis
attacks and favorable safety and tolerability in patients with FCS;
on track for regulatory filings in early 2024
Eplontersen marketing applications accepted
for review in the EU and Canada;
potential U.S. approval in December
2023
On track to achieve 2023 financial
guidance
CARLSBAD, Calif., Nov. 2, 2023
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the
"Company"), today reported financial results for the third quarter
of 2023.
![(PRNewsfoto/Ionis Pharmaceuticals, Inc.) (PRNewsfoto/Ionis Pharmaceuticals, Inc.)](https://mma.prnewswire.com/media/882077/Ionis_Logo.jpg)
"We continue to successfully execute on our strategy to deliver
a steady cadence of potentially transformational medicines to
patients," said Brett P. Monia,
Ph.D., chief executive officer of Ionis. "Eplontersen is on track
for its first potential approval in the U.S. and is under
regulatory review in the EU and Canada. We believe the positive efficacy and
safety data coupled with an attractive self-administration dosing
profile positions eplontersen to be the preferred therapy in the
largely underserved hereditary ATTR polyneuropathy population. We
reported positive data from the olezarsen Phase 3 Balance study in
patients with familial chylomicronemia syndrome, showing
statistically significant triglyceride lowering, substantial
reductions in acute pancreatitis attacks and favorable safety and
tolerability, positioning olezarsen to be our first independent
commercial launch. We also made additional progress across our
wholly owned and partnered pipeline, and further expanded our rich
Phase 3 pipeline with the advance of zilganersen for patients with
Alexander disease into Phase 3 development. Looking ahead, we
expect to continue our positive momentum with the potential
approval and launch of eplontersen and the Phase 3 data readout of
donidalorsen in hereditary angioedema."
Third Quarter 2023 Summary Financial Results:
|
|
Three months
ended
September
30,
|
|
Nine months
ended
September
30,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
(amounts in
millions)
|
Total
revenue
|
|
$144
|
|
$160
|
|
$463
|
|
$435
|
Operating
expenses
|
|
$287
|
|
$219
|
|
$811
|
|
$637
|
Operating expenses on a
non-GAAP basis
|
|
$261
|
|
$195
|
|
$732
|
|
$562
|
Loss from
operations
|
|
($143)
|
|
($59)
|
|
($348)
|
|
($202)
|
Loss from operations on
a non-GAAP basis
|
|
($117)
|
|
($35)
|
|
($269)
|
|
($127)
|
Financial Highlights
- Revenue continued to be substantial and sustained, with
revenues of $144 million and
$463 million in the three and nine
months ended September 30, 2023,
reflecting a 10% decrease and 6% increase compared to the same
periods last year, respectively, driven by the timing of certain
partner payments
- Operating expenses increased in the three and nine months ended
September 30, 2023 compared to the
same periods last year, primarily due to strategic investments to
bring eplontersen, olezarsen and donidalorsen to patients
- Cash and short-term investments of $2.2
billion as of September 30,
2023 enables continued investments to drive increasing
value
- Reaffirmed 2023 financial guidance
Near-Term Commercial Opportunities and Late-Stage Pipeline
Highlights
- Achieved multiple milestones with eplontersen:
- Eplontersen is under regulatory review by the European
Medicines Agency (EMA) and Health Canada for the treatment of
hereditary ATTR polyneuropathy (ATTRv-PN)
- The EMA granted orphan drug designation to eplontersen for the
treatment of ATTR in the EU
- Published positive data from the Phase 3 NEURO-TTRansform study
in patients with ATTRv-PN in the Journal of the American Medical
Association (JAMA) showing eplontersen halted measures of
disease progression and continuously improved quality of life at
35-, 66- and 85-weeks
- Presented positive new data showing continued benefit in
secondary endpoints from the Phase 3 NEURO-TTRansform study in
patients with ATTRv-PN at the European ATTR Amyloidosis (EU-ATTR)
meeting
- Presented positive exploratory data from a pre-defined cardiac
sub-population of patients in NEURO-TTRansform showing improvement
in cardiac function and structure compared to external placebo at
the Heart Failure Society of America (HFSA) Annual Scientific
Meeting
- Completed enrollment of the Phase 3 CARDIO-TTRansform study of
eplontersen in patients with ATTR cardiomyopathy (ATTR-CM), the
largest study ever conducted in ATTR-CM; on track for data readout
as early as H1:2025
- Reported positive data from the Phase 3 Balance study of
olezarsen in patients with familial chylomicronemia syndrome (FCS)
- Olezarsen demonstrated robust, dose-dependent reductions in
APOCIII, statistically significant reductions in triglycerides,
substantial reductions in acute pancreatitis attacks and a
favorable safety and tolerability profile
- On track to file for regulatory approval in the U.S. and EU in
early 2024
- The FDA granted orphan drug designation to donidalorsen for the
treatment of patients with hereditary angioedema (HAE); on track
for data readout in the Phase 3 OASIS-HAE study in H1:2024
- Advanced zilganersen (GFAP) into Phase 3 development for the
treatment of patients with Alexander disease
- The FDA granted orphan drug designation to ulefnersen (FUS) for
the treatment of patients with FUS-ALS
Partnered Program Highlights
- GSK reported positive data from the Phase 2b B-Together study of bepirovirsen followed by
pegylated interferon in patients with chronic hepatitis B virus
(HBV)
- Reported positive interim data from the ongoing Phase 2 study
of IONIS-FB-LRx in patients with immunoglobulin A
nephropathy (IgAN)
- Biogen reported positive data from the Phase 1/2 study of
IONIS-MAPTRx (BIIB080) in patients with Alzheimer's
disease
- Completed enrollment in the Phase 1/2 HALOS study of ION582
(BIIB121) in patients with Angelman syndrome
- Entered a new agreement with Roche to advance two novel
RNA-targeted programs for Alzheimer's disease and Huntington's
disease
Third Quarter 2023 Financial Results
"Our year-to-date financial results keep us on track to achieve
our 2023 guidance as we execute on a strategy to unlock next-level
value," said Elizabeth L. Hougen,
chief financial officer of Ionis. "Our strong financial foundation
includes more than $2B in cash,
significant royalty revenue with SPINRAZA, and substantial and
sustained R&D revenue from multiple partners. We are well
positioned to continue investing in our key priorities to drive
future positive cash flow, including advancing our go-to-market
activities, growing our wholly owned pipeline and optimizing new
cutting-edge technologies for future medicines. We look forward to
the potential U.S. eplontersen ATTRv-PN approval next month
followed closely by launch. Together with our partner, AstraZeneca,
we believe we are well positioned to identify new patients to
further grow the market and become the treatment of choice for this
population that remains largely underserved by current
therapies."
Revenue
Ionis' revenue was comprised of the following:
|
|
Three months
ended
|
|
Nine months
ended
|
|
|
September
30,
|
|
September
30,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
Revenue:
|
|
(amounts in
millions)
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$67
|
|
$62
|
|
$179
|
|
$175
|
Other commercial
revenue:
|
|
|
|
|
|
|
|
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
8
|
|
6
|
|
25
|
|
23
|
Licensing and royalty
revenue
|
|
9
|
|
5
|
|
26
|
|
25
|
Total commercial
revenue
|
|
84
|
|
73
|
|
230
|
|
223
|
Research and
development revenue:
|
|
|
|
|
|
|
|
|
Amortization from
upfront payments
|
|
18
|
|
18
|
|
47
|
|
54
|
Milestone
payments
|
|
16
|
|
15
|
|
90
|
|
60
|
License
fees
|
|
5
|
|
35
|
|
25
|
|
37
|
Other
services
|
|
5
|
|
1
|
|
11
|
|
6
|
Collaborative
agreement revenue
|
|
44
|
|
69
|
|
173
|
|
157
|
Eplontersen joint
development revenue
|
|
16
|
|
18
|
|
60
|
|
55
|
Total research and
development revenue
|
|
60
|
|
87
|
|
233
|
|
212
|
Total
revenue
|
|
$144
|
|
$160
|
|
$463
|
|
$435
|
Commercial revenue for the three and nine months ended
September 30, 2023 included
$67 million and $179 million from SPINRAZA royalties,
respectively, which were essentially flat compared to the same
periods last year reflecting SPINRAZA's resilience against emerging
competition. Ionis' commercial revenue in the three and nine months
ended September 30, 2023 also
included royalties from the U.S. launch of QALSODY.
R&D revenue decreased for the three months ended
September 30, 2023 and increased for
the nine months ended September 30,
2023 compared to the same periods last year due to the
timing of certain partner payments, including the $35 million license fee for
IONIS-FB-LRx that Ionis earned from Roche in the three
months ended September 30, 2022.
Operating Expenses
Ionis' operating expenses increased in the three and nine months
ended September 30, 2023 compared to
the same periods in 2022, consistent with expectations. As Ionis
advanced its robust pipeline, study costs increased compared to the
same periods in 2022 as most of the Company's Phase 3 studies are
either fully enrolled or approaching full enrollment, resulting in
higher R&D expenses year over year. Ionis' SG&A expenses
also increased year over year primarily due to launch preparation
activities for eplontersen, olezarsen and donidalorsen.
Balance Sheet
As of September 30, 2023, Ionis'
cash, cash equivalents and short-term investments increased to
$2.2 billion compared to $2.0 billion at December
31, 2022 primarily due to the $500
million Ionis received from Royalty Pharma in January 2023. Ionis' working capital also
increased over the same period primarily due to the Company's
higher cash and short-term investments balance. In the first
quarter of 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.
Webcast
Management will host a conference call and webcast to discuss
Ionis' third quarter 2023 results at 11:30
a.m. Eastern time on Thursday, November 2, 2023. 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 third quarter 2023 earnings slides click here.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been a leader in RNA-targeted
therapy, pioneering new markets and changing standards of care.
Ionis currently has four marketed medicines and a promising
late-stage pipeline highlighted by cardiovascular and neurological
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision to
become 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 QALSODY (tofersen), SPINRAZA (nusinersen),
TEGSEDI (inotersen), WAYLIVRA (volanesorsen), eplontersen,
olezarsen, donidalorsen, zilganersen, ulefnersen, pelacarsen,
bepirovirsen, IONIS-FB-LRx, 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,
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.
IONIS
PHARMACEUTICALS, INC.
SELECTED FINANCIAL
INFORMATION
Condensed
Consolidated Statements of Operations
(In Millions, Except
Per Share Data)
|
|
|
|
|
|
|
|
Three months
ended,
|
|
Nine months
ended
|
|
|
September
30,
|
|
September
30,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
(unaudited)
|
Revenue:
|
|
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$67
|
|
$62
|
|
$179
|
|
$175
|
Other commercial
revenue
|
|
17
|
|
11
|
|
51
|
|
48
|
Total commercial
revenue
|
|
84
|
|
73
|
|
230
|
|
223
|
Research and
development revenue:
|
|
|
|
|
|
|
|
|
Collaborative
agreement revenue
|
|
44
|
|
69
|
|
173
|
|
157
|
Eplontersen joint
development revenue
|
|
16
|
|
18
|
|
60
|
|
55
|
Total research and
development revenue
|
|
60
|
|
87
|
|
233
|
|
212
|
Total
revenue
|
|
144
|
|
160
|
|
463
|
|
435
|
Expenses:
|
|
|
|
|
|
|
|
|
Cost of
sales
|
|
2
|
|
2
|
|
6
|
|
10
|
Research, development and patent
|
|
215
|
|
183
|
|
643
|
|
525
|
Selling, general and administrative
|
|
70
|
|
34
|
|
162
|
|
102
|
Total operating
expenses
|
|
287
|
|
219
|
|
811
|
|
637
|
Loss from
operations
|
|
(143)
|
|
(59)
|
|
(348)
|
|
(202)
|
|
|
|
|
|
|
|
|
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
Interest expense
related to the sale of future royalties:
|
|
(18)
|
|
-
|
|
(51)
|
|
-
|
Other income
(expense), net
|
|
20
|
|
12
|
|
68
|
|
(12)
|
Loss before income tax
expense
|
|
(141)
|
|
(47)
|
|
(331)
|
|
(214)
|
|
|
|
|
|
|
|
|
|
Income tax
expense
|
|
(6)
|
|
-
|
|
(26)
|
|
(3)
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
($147)
|
|
($47)
|
|
($357)
|
|
($217)
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share
|
|
($1.03)
|
|
($0.33)
|
|
($2.50)
|
|
($1.53)
|
Shares used in
computing basic and diluted net loss per share
|
|
143
|
|
142
|
|
143
|
|
142
|
IONIS
PHARMACEUTICALS, INC.
Reconciliation of
GAAP to Non-GAAP Basis:
Condensed
Consolidated Operating Expenses, Loss From Operations, and Net
Loss
(In
Millions)
|
|
|
|
Three months
ended
September
30,
|
|
Nine months
ended
September
30,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
(unaudited)
|
As reported
research, development and patent expenses according to
GAAP
|
|
$215
|
|
$183
|
|
$643
|
|
$525
|
Excluding compensation expense related to equity
awards
|
|
(19)
|
|
(18)
|
|
(58)
|
|
(55)
|
Non-GAAP research,
development and patent expenses
|
|
$196
|
|
$165
|
|
$585
|
|
$470
|
|
|
|
|
|
|
|
|
|
As reported selling,
general and administrative expenses according to
GAAP
|
|
$70
|
|
$34
|
|
$162
|
|
$102
|
Excluding compensation expense related to equity
awards
|
|
(7)
|
|
(6)
|
|
(22)
|
|
(19)
|
Non-GAAP selling,
general and administrative expenses
|
|
$63
|
|
$28
|
|
$140
|
|
$83
|
|
|
|
|
|
|
|
|
|
As reported
operating expenses according to GAAP
|
|
$287
|
|
$219
|
|
$811
|
|
$637
|
Excluding compensation
expense related to equity
awards
|
|
(26)
|
|
(24)
|
|
(79)
|
|
(75)
|
Non-GAAP operating
expenses
|
|
$261
|
|
$195
|
|
$732
|
|
$562
|
|
|
|
|
|
|
|
|
|
As reported loss
from operations according to GAAP
|
|
($143)
|
|
($59)
|
|
($348)
|
|
($202)
|
Excluding compensation expense related to equity
awards
|
|
(26)
|
|
(24)
|
|
(79)
|
|
(75)
|
Non-GAAP loss from
operations
|
|
($117)
|
|
($35)
|
|
($269)
|
|
($127)
|
|
|
|
|
|
|
|
|
|
As reported net loss
according to GAAP
|
|
($147)
|
|
($47)
|
|
($357)
|
|
($217)
|
Excluding compensation expense related to equity awards and related
tax effects
|
|
(26)
|
|
(24)
|
|
(79)
|
|
(75)
|
Non-GAAP net
loss
|
|
($121)
|
|
($23)
|
|
($278)
|
|
($142)
|
Reconciliation of GAAP to Non-GAAP Basis
As illustrated in the Selected Financial Information in this
press release, non-GAAP operating expenses, non-GAAP loss from
operations, and non-GAAP net 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. 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)
|
|
|
|
|
|
|
|
|
|
September
30,
|
|
December 31,
|
|
|
|
2023
|
|
2022
|
|
|
|
(unaudited)
|
|
|
|
Assets:
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$2,236
|
|
$1,987
|
|
Contracts
receivable
|
|
142
|
|
26
|
|
Other current
assets
|
|
207
|
|
190
|
|
Property, plant
and equipment, net
|
|
71
|
|
74
|
|
Right-of-use
assets
|
|
174
|
|
182
|
|
Other
assets
|
|
104
|
|
75
|
|
Total assets
|
|
2,934
|
|
$2,534
|
|
|
|
|
|
|
|
Liabilities and
stockholders' equity:
|
|
|
|
|
|
Other current
liabilities
|
|
$199
|
|
$221
|
|
Current portion
of deferred contract revenue
|
|
205
|
|
91
|
|
1.75%
convertible senior notes, net
|
|
562
|
|
-
|
|
0% convertible
senior notes, net
|
|
625
|
|
622
|
|
0.125%
convertible senior notes, net
|
|
44
|
|
545
|
|
Liability
related to sale of future royalties, net
|
|
513
|
|
-
|
|
Long-term lease
liabilities
|
|
173
|
|
178
|
|
Long-term
obligations, less current portion
|
|
49
|
|
16
|
|
Long-term
deferred contract revenue
|
|
249
|
|
288
|
|
Total
stockholders' equity
|
|
315
|
|
573
|
|
Total liabilities and stockholders' equity
|
|
$2,934
|
|
$2,534
|
|
|
|
|
|
|
|
|
|
Key 2023 Value Driving Events(1)
Regulatory
Actions
|
Program
|
Indication
|
Regulatory
Action
|
Achieved
|
QALSODY
|
SOD1-ALS
|
NDA approval
|
•
|
EU
approval2
|
|
Eplontersen
(TTR)
|
ATTRv-PN
|
NDA approval
|
|
OUS filings
|
•
|
|
Key Clinical Data
Events
|
Program
|
Indication
|
Event
|
Achieved
|
Eplontersen
(TTR)
|
ATTRv-PN
|
Phase 3 data (week 35,
66 & 85)
|
•
|
Olezarsen
(APOCIII)
|
FCS
|
Phase 3 data
|
•
|
Donidalorsen
(PKK)
|
HAE
|
Phase 2, OLE 1-year
data
|
•
|
Donidalorsen
(PKK)
|
HAE
|
Phase 2, OLE 2-year
data
|
•
|
Bepirovirsen
|
HBV
|
Phase 2b B-Together
data
|
•
|
IONIS-FB-LRx
|
IgAN
|
Phase 2 interim
data
|
•
|
|
Enrollment
Achievements
|
Program
|
Indication
|
Event
|
Achieved
|
Eplontersen
(TTR)
|
ATTR-CM
|
Phase 3 full
enrollment
|
•
|
Donidalorsen
(PKK)
|
HAE
|
Phase 3 full
enrollment
|
•
|
IONIS-FB-LRx
|
GA
|
Phase 2 full
enrollment
|
•
|
ION582
(UBE3A)
|
Angelman
syndrome
|
Phase 1/2 full
enrollment
|
•
|
|
Phase 3
Initiations
|
Program
|
Indication
|
Achieved
|
Zilganersen
(GFAP)
|
Alexander
disease
|
•
|
Bepirovirsen
|
HBV
|
•
|
IONIS-FB-LRx
|
IgAN
|
•
|
|
(1)
Timing expectations based on current assumptions and subject to
change.
|
(2)
CHMP opinion anticipated in Q4:2023.
|
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SOURCE Ionis Pharmaceuticals, Inc.