Reported Phase 3 data showing eplontersen
continued to show improvement in ATTRv-PN through 85 weeks;
December 22, 2023 PDUFA date
Completed enrollment in donidalorsen OASIS-HAE
study and eplontersen CARDIO-TTRansform ATTR-CM study keeping Phase
3 data readouts on track
On track to achieve 2023 financial
guidance
CARLSBAD, Calif., Aug. 9, 2023
/PRNewswire/ -- – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the
"Company"), today reported financial results for the second quarter
of 2023. Financial results are summarized below:
|
|
Three months
ended
June 30,
|
|
Six months
ended
June 30,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
(amounts in
millions)
|
Total
revenue
|
|
$188
|
|
$134
|
|
$319
|
|
$276
|
Operating
expenses
|
|
$279
|
|
$220
|
|
$523
|
|
$419
|
Operating expenses on a
non-GAAP basis
|
|
$252
|
|
$195
|
|
$469
|
|
$368
|
Loss from
operations
|
|
($91)
|
|
($86)
|
|
($204)
|
|
($143)
|
Loss from operations on
a non-GAAP basis
|
|
($64)
|
|
($61)
|
|
($150)
|
|
($92)
|
Financial Highlights
- Revenue increased for the second quarter and first half of 2023
by 40% and 16% compared to the same periods last year,
respectively, driven by significant partner payments
- Operating expenses increased in the second quarter and first
half of 2023 compared to the prior year as planned, reflecting
investments in advancing Ionis' pipeline and go-to-market
activities for eplontersen, olezarsen and donidalorsen
- Cash and short-term investments of $2.4
billion as of June 30, 2023
enables continued investments to drive increasing value
- Well-capitalized balance sheet reflects 2024 convertible note
refinancing that extended maturity to 2028 while maintaining a low
coupon and retaining the flexibility to mitigate potential equity
dilution
- Reaffirmed 2023 financial guidance
"Ionis is creating significant value in 2023 as we successfully
execute on our strategy to bring a steady cadence of
transformational medicines to the market. Our growing late-stage
pipeline now includes eight medicines for 10 indications,
highlighted by eplontersen. Based on the strong and consistent data
generated to date and an attractive self-administration profile, we
expect eplontersen to be an important new medicine for people with
ATTRv-polyneuropathy. We also added QALSODY to our commercial
portfolio, a breakthrough treatment for people with SOD1-ALS
further strengthening Ionis' leadership in RNA-based therapies for
neurological diseases," said Brett P.
Monia, Ph.D., chief executive officer of Ionis. "In the
second half, we expect continued positive momentum including the
Phase 3 readout for olezarsen in familial chylomicronemia syndrome
and the potential approval of eplontersen for ATTRv-PN in
December."
Recent Highlights From Commercial Medicines
- Biogen presented interim data from the Phase 4 RESPOND study of
SMA patients demonstrating improved motor function in most
participants treated with SPINRAZA who had unmet medical needs
after treatment with gene therapy
- FDA granted Biogen accelerated approval of QALSODY (tofersen),
a first-in-class medicine for patients with SOD1-ALS
Recent Highlights From Near-Term Commercial
Opportunities
- Reported positive results from the Phase 3 NEURO-TTRansform
study in patients with ATTRv-PN showing eplontersen continued to
halt neuropathy disease progression and improve quality of life
through 85 weeks
- Completed enrollment of the Phase 3 CARDIO-TTRansform study of
eplontersen in patients with ATTR cardiomyopathy, the largest study
ever conducted in ATTR-CM; on track for data readout as early as
H1:2025
- Licensed eplontersen Latin
America rights to AstraZeneca
- Completed enrollment of the Phase 3 OASIS-HAE study of
donidalorsen in patients with hereditary angioedema; on track for
data readout in H1:2024
- Reported positive topline Phase 2 open label extension data of
donidalorsen in patients with hereditary angioedema treated for two
years
Recent Highlights From Partnered Programs
- Roche advanced IONIS-FB-LRx into Phase 3 development
in patients with immunoglobulin A nephropathy
- GSK presented durable response data from the Phase 2 B-Sure long-term follow-up study of
bepirovirsen in complete responder patients from the Phase
2b B-Clear study of patients with
HBV
- Completed enrollment in the Phase 2 GOLDEN study of
IONIS-FB-LRx in patients with geographic atrophy
- AstraZeneca initiated a Phase 2b
study of ION839 (AZD2693) targeting PNPLA3 to treat patients with
NASH
- Entered collaboration with Novartis to advance a next
generation program targeting Lp(a) for cardiovascular disease
Second Quarter 2023 Financial Results
"Our results for the first half of the year keep us on track to
achieve our 2023 guidance. We continued to generate substantial and
sustained revenue, that together with our well-capitalized balance
sheet, allows us to continue investing in key opportunities across
our business," said Elizabeth L.
Hougen, chief financial officer of Ionis. "With three
near-term commercial opportunities that have a combined
multi-billion-dollar peak sales potential and a steady cadence of
medicines poised to follow closely behind, we are positioned to
drive substantial revenue growth and long-term value for
shareholders."
Revenue
Ionis' revenue was comprised of the following:
|
|
Three months
ended
|
|
Six months
ended
|
|
|
June 30,
|
|
June 30,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
Revenue:
|
|
(amounts in
millions)
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$61
|
|
$60
|
|
$111
|
|
$113
|
Other commercial
revenue:
|
|
|
|
|
|
|
|
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
11
|
|
10
|
|
17
|
|
17
|
Licensing and royalty
revenue
|
|
6
|
|
8
|
|
18
|
|
20
|
Total commercial
revenue
|
|
78
|
|
78
|
|
146
|
|
150
|
Research and
development revenue:
|
|
|
|
|
|
|
|
|
Amortization from
upfront payments
|
|
15
|
|
18
|
|
29
|
|
36
|
Milestone
payments
|
|
51
|
|
18
|
|
74
|
|
45
|
License
fees
|
|
20
|
|
-
|
|
20
|
|
2
|
Other
services
|
|
4
|
|
3
|
|
6
|
|
6
|
Collaborative
agreement revenue
|
|
90
|
|
39
|
|
129
|
|
89
|
Eplontersen joint
development revenue
|
|
20
|
|
17
|
|
44
|
|
37
|
Total research and
development revenue
|
|
110
|
|
56
|
|
173
|
|
126
|
Total
revenue
|
|
$188
|
|
$134
|
|
$319
|
|
$276
|
Ionis' revenue increased in the second quarter and first half of
2023 compared to the same periods in 2022 because of increased
payments from partnered programs. Ionis believes its substantial
and sustainable revenue is an important source of funding that
supports the Company's investments to bring potentially
transformational medicines to the market.
Commercial revenue for the second quarter and first half of 2023
included $61 million and $111 million from SPINRAZA royalties,
respectively. Global SPINRAZA product sales of $437 million and $880
million were essentially flat for the second quarter and
first half of 2023, respectively, compared to the same periods last
year reflecting SPINRAZA's resilience against emerging competition.
Ionis' commercial revenue in the second quarter and first half of
2023 also included royalties from the U.S. launch of QALSODY.
R&D revenue essentially doubled for the second quarter of
2023 and increased more than 35% for the first half of 2023
compared to the same periods in 2022 reflecting the value Ionis'
technology is creating as numerous partnered programs advanced.
Operating Expenses
Ionis' operating expenses increased in the second quarter and
first half of 2023 compared to the same periods in 2022, consistent
with expectations. As Ionis advanced its robust pipeline, study
costs increased as most of the Company's Phase 3 studies were
either fully enrolled or approaching full enrollment resulting in
higher R&D expenses year over year. Additionally, as Ionis
prepares to launch eplontersen, olezarsen and donidalorsen, the
Company's SG&A expenses also increased year over year.
Balance Sheet
As of June 30, 2023, Ionis' cash,
cash equivalents and short-term investments increased to
$2.4 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%. Ionis used the majority of the proceeds to repurchase
$434 million of its 2024 convertible
notes. The Company plans to utilize the residual proceeds to settle
the 2024 notes that remain outstanding.
Webcast
Management will host a conference call and webcast to discuss
Ionis' second quarter 2023 results at 11:30
a.m. Eastern time on Wednesday, August 9, 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 second 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, 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,
|
|
Six months
ended
|
|
|
June 30,
|
|
June 30,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
(unaudited)
|
Revenue:
|
|
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$61
|
|
$60
|
|
$111
|
|
$113
|
Other commercial
revenue
|
|
17
|
|
18
|
|
35
|
|
37
|
Total commercial
revenue
|
|
78
|
|
78
|
|
146
|
|
150
|
Research and
development revenue:
|
|
|
|
|
|
|
|
|
Collaborative
agreement revenue
|
|
90
|
|
39
|
|
129
|
|
89
|
Eplontersen joint
development revenue
|
|
20
|
|
17
|
|
44
|
|
37
|
Total research and
development revenue
|
|
110
|
|
56
|
|
173
|
|
126
|
Total
revenue
|
|
188
|
|
134
|
|
319
|
|
276
|
Expenses:
|
|
|
|
|
|
|
|
|
Cost of
sales
|
|
3
|
|
5
|
|
4
|
|
9
|
Research, development and patent
|
|
230
|
|
181
|
|
428
|
|
342
|
Selling, general and administrative
|
|
46
|
|
34
|
|
91
|
|
68
|
Total operating
expenses
|
|
279
|
|
220
|
|
523
|
|
419
|
Loss from
operations
|
|
(91)
|
|
(86)
|
|
(204)
|
|
(143)
|
|
|
|
|
|
|
|
|
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
Interest expense
related to the sale of future royalties:
|
|
(18)
|
|
-
|
|
(33)
|
|
-
|
Other income
(expense), net
|
|
32
|
|
(17)
|
|
47
|
|
(24)
|
Loss before income tax
expense
|
|
(77)
|
|
(103)
|
|
(190)
|
|
(167)
|
|
|
|
|
|
|
|
|
|
Income tax
expense
|
|
(8)
|
|
(2)
|
|
(20)
|
|
(3)
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
($85)
|
|
($105)
|
|
($210)
|
|
($170)
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share
|
|
($0.60)
|
|
($0.74)
|
|
($1.47)
|
|
($1.20)
|
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
June
30,
|
|
Six months
ended
June 30,
|
|
|
2023
|
|
2022
|
|
2023
|
|
2022
|
|
|
(unaudited)
|
As reported
research, development and patent expenses according to
GAAP
|
|
$230
|
|
$181
|
|
$428
|
|
$342
|
Excluding compensation expense related to equity
awards
|
|
(19)
|
|
(19)
|
|
(39)
|
|
(38)
|
Non-GAAP research,
development and patent expenses
|
|
$211
|
|
$162
|
|
$389
|
|
$304
|
|
|
|
|
|
|
|
|
|
As reported selling,
general and administrative expenses according to
GAAP
|
|
$46
|
|
$34
|
|
$91
|
|
$68
|
Excluding compensation expense related to equity
awards
|
|
(7)
|
|
(6)
|
|
(14)
|
|
(13)
|
Non-GAAP selling,
general and administrative expenses
|
|
$39
|
|
$28
|
|
$77
|
|
$55
|
|
|
|
|
|
|
|
|
|
As reported
operating expenses according to GAAP
|
|
$279
|
|
$220
|
|
$523
|
|
$419
|
Excluding compensation
expense related to equity
awards
|
|
(27)
|
|
(25)
|
|
(54)
|
|
(51)
|
Non-GAAP operating
expenses
|
|
$252
|
|
$195
|
|
$469
|
|
$368
|
|
|
|
|
|
|
|
|
|
As reported loss
from operations according to GAAP
|
|
($91)
|
|
($86)
|
|
($204)
|
|
($143)
|
Excluding compensation expense related to equity
awards
|
|
(27)
|
|
(25)
|
|
(54)
|
|
(51)
|
Non-GAAP loss from
operations
|
|
($64)
|
|
($61)
|
|
($150)
|
|
($92)
|
|
|
|
|
|
|
|
|
|
As reported net loss
according to GAAP
|
|
($85)
|
|
($105)
|
|
($210)
|
|
($170)
|
Excluding compensation expense related to equity awards and related
tax effects
|
|
(27)
|
|
(25)
|
|
(54)
|
|
(51)
|
Non-GAAP net
loss
|
|
($58)
|
|
($80)
|
|
($156)
|
|
($119)
|
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) |
|
|
|
|
|
|
|
|
|
June 30,
|
|
December 31,
|
|
|
|
2023
|
|
2022
|
|
|
|
(unaudited)
|
|
|
|
Assets:
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$2,385
|
|
$1,987
|
|
Contracts
receivable
|
|
28
|
|
26
|
|
Other current
assets
|
|
203
|
|
190
|
|
Property, plant
and equipment, net
|
|
92
|
|
74
|
|
Right-of-use
assets
|
|
177
|
|
182
|
|
Other
assets
|
|
86
|
|
75
|
|
Total assets
|
|
$2,971
|
|
$2,534
|
|
|
|
|
|
|
|
Liabilities and
stockholders' equity:
|
|
|
|
|
|
Other current
liabilities
|
|
$192
|
|
$221
|
|
Current portion
of deferred contract revenue
|
|
96
|
|
91
|
|
1.75%
convertible senior notes, net
|
|
561
|
|
-
|
0% convertible
senior notes, net
|
|
624
|
|
622
|
|
0.125%
convertible senior notes, net
|
|
114
|
|
545
|
|
Liability
related to sale of future royalties, net
|
|
510
|
|
-
|
Long-term lease
liabilities
|
|
175
|
|
178
|
|
Long-term
obligations, less current portion
|
|
17
|
|
16
|
|
Long-term
deferred contract revenue
|
|
254
|
|
288
|
|
Total
stockholders' equity
|
|
428
|
|
573
|
|
Total liabilities and stockholders' equity
|
|
$2,971
|
|
$2,534
|
|
|
|
|
|
|
|
|
|
2023 Key Value Driving Events(1)
Regulatory
Actions
|
Program
|
Indication
|
Regulatory
Action
|
Achieved
|
QALSODY
|
SOD1-ALS
|
NDA approval
|
•
|
EU
approval2
|
|
Eplontersen
(TTR)
|
ATTRv
polyneuropathy
|
NDA approval
|
|
OUS filings
|
|
|
Key Clinical Data
Events
|
Program
|
Indication
|
Event
|
Achieved
|
Eplontersen
(TTR)
|
ATTRv
polyneuropathy
|
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
|
•
|
|
Enrollment
Achievements
|
Program
|
Indication
|
Event
|
Achieved
|
Eplontersen
(TTR)
|
ATTR
cardiomyopathy
|
Phase 3 full
enrollment
|
•
|
Donidalorsen
(PKK)
|
HAE
|
Phase 3 full
enrollment
|
•
|
IONIS-FB-LRx
|
Geographic
Atrophy
|
Phase 2 full
enrollment
|
•
|
|
Phase 3
Initiations
|
Program
|
Indication
|
Achieved
|
Bepirovirsen
(HBV)
|
Hepatitis B virus
infection
|
•
|
IONIS-FB-LRx
|
Immunoglobulin A
nephropathy
|
•
|
|
(1) Timing
expectations based on current assumptions and subject to
change.
(2) CHMP opinion anticipated in Q4:2023.
|
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multimedia:https://www.prnewswire.com/news-releases/ionis-reports-second-quarter-2023-financial-results-301896301.html
SOURCE Ionis Pharmaceuticals, Inc.