QALSODY approved for SOD1-ALS;
MAA under review in EU
Reported positive Phase 3 eplontersen ATTRv-PN
data; December 22, 2023 PDUFA
date
On track to achieve 2023 financial
guidance
CARLSBAD, Calif., May 3, 2023
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the
"Company"), today reported financial results for the first quarter
of 2023. Financial results are summarized below:
|
|
Three months
ended
March 31,
|
|
|
|
2023
|
|
2022
|
|
|
|
(amounts in
millions)
|
|
Total
revenue
|
|
$131
|
|
$142
|
|
Operating
expenses
|
|
$245
|
|
$199
|
|
Operating expenses on a
non-GAAP basis
|
|
$218
|
|
$173
|
|
Loss from
operations
|
|
($114)
|
|
($57)
|
|
Loss from
operations on a non-GAAP basis
|
|
($87)
|
|
($31)
|
|
Financial Highlights
- Revenue for the first quarter of 2023 was in line with
expectations and included revenue from numerous diverse
sources
- Operating expenses increased in the first quarter of 2023
compared to the prior year as planned, reflecting investments in
advancing Ionis' pipeline, technology and go-to-market activities
for eplontersen, olezarsen and donidalorsen
- Cash and short-term investments of $2.3
billion at March 31, 2023
enables continued investment in creating future growth
opportunities
- Reaffirmed 2023 financial guidance
"2023 is off to a strong start. With QALSODY's approval, it
joins SPINRAZA as a new groundbreaking medicine to treat a
devastating neurological disease, further validating our
RNA-targeting therapeutic platform. We also achieved another
important milestone with our recent positive eplontersen Phase 3
data. We believe the positive efficacy and safety data, and the
attractive self-administered dosing profile, position eplontersen
to be an important treatment for ATTRv-PN patients, who today are
underserved. We look forward to the first potential approval of
eplontersen in the U.S. in December," said Brett P. Monia, Ph.D., chief executive officer
of Ionis. "In addition, we further expanded our industry-leading
late-stage pipeline to seven programs across nine indications
following the start of GSK's bepirovirsen hepatitis B program. In
the second half, we plan to report results from our olezarsen Phase
3 FCS study, which if positive, positions us for our first
independent commercial launch. These recent achievements, together
with our upcoming milestones, continue to build value for Ionis
stakeholders."
Recent Late-Stage Pipeline Highlights
- FDA granted Biogen accelerated approval of QALSODY (tofersen)
for patients with SOD1-ALS
- FDA accepted eplontersen NDA for patients with polyneuropathy
caused by hereditary TTR amyloidosis (ATTRv-PN) with a PDUFA date
of December 22, 2023
- Presented positive week-35 and week-66 data from the Phase 3
NEURO-TTRansform study of eplontersen in patients with
ATTRv-PN
- GSK advanced bepirovirsen into Phase 3 development in patients
with chronic hepatitis B
Recent Additional Pipeline Updates
- Biogen presented data demonstrating IONIS-MAPTRx
(BIIB080) substantially reduced tau protein in patients with
early-stage Alzheimer's disease
- Continued to focus R&D efforts by discontinuing two
programs that did not meet Ionis' target product profile,
cimdelirsen for acromegaly and sapablursen for beta-thalassemia.
Ionis continues to advance the Phase 2 sapablursen study for
polycythemia vera
First Quarter 2023 Financial Results
"Our first quarter results were in line with our expectations.
We generated meaningful revenue while continuing to invest in key
growth opportunities across our business. These results keep us on
track to achieve our 2023 financial guidance," said Elizabeth L. Hougen, chief financial officer of
Ionis. "We plan to continue investing in areas with the greatest
potential to drive growth. As such, we expect our investments to
grow modestly as we advance and expand our late-stage pipeline and
move our near-term commercial opportunities toward the market.
Additionally, as we keep more programs for ourselves, we expect a
greater proportion of commercial revenues compared to R&D
revenues, and our commercial revenues to be the primary driver of
future revenue growth."
Revenue
Ionis' revenue was comprised of the following:
|
|
Three months
ended
|
|
|
|
|
March 31,
|
|
|
|
|
2023
|
|
2022
|
|
|
Revenue:
|
|
(amounts in
millions)
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$50
|
|
$54
|
|
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
7
|
|
6
|
|
|
Licensing and royalty
revenue
|
|
11
|
|
12
|
|
|
Total commercial
revenue
|
|
68
|
|
72
|
|
|
Research and
development revenue:
|
|
|
|
|
|
|
Amortization from
upfront payments
|
|
16
|
|
17
|
|
|
Milestone
payments
|
|
23
|
|
27
|
|
|
License
fees
|
|
-
|
|
2
|
|
|
Other
services
|
|
-
|
|
4
|
|
|
Collaborative
agreement revenue
|
|
39
|
|
50
|
|
|
Eplontersen joint
development revenue
|
|
24
|
|
20
|
|
|
Total research and
development revenue
|
|
63
|
|
70
|
|
|
Total
revenue
|
|
$131
|
|
$142
|
|
|
Ionis continued to derive its revenue for the first quarter of
2023 from diverse sources, with approximately half coming from
commercial products and half from numerous partnered programs.
Commercial revenue for the first quarter of 2023 included
$50 million from SPINRAZA royalties.
Global SPINRAZA product sales of $443
million decreased six percent in the first quarter of 2023,
compared to the same period last year primarily due to the impact
from foreign currency, fewer new patient starts in the U.S. and
channel dynamics.
R&D revenue for the first quarter of 2023 included
$24 million from AstraZeneca for its
share of the global Phase 3 development costs for eplontersen,
$20 million from Biogen for advancing
several neurology disease programs and $15
million from GSK for advancing bepirovirsen into Phase 3
development. Already in the second quarter, the Company earned
$16 million in a milestone payment
from Biogen when QALSODY was approved in the U.S.
Operating Expenses
Ionis' operating expenses increased in the first quarter of 2023
compared to the same period 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 March 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. Ionis' working capital also
increased over the same period primarily due to the Company's
higher cash and short-term investments balance. Additionally, the
Company recorded a long-term liability for future royalties due to
Royalty Pharma in the first quarter of 2023.
Webcast
Management will host a conference call and webcast to discuss
Ionis' first quarter 2023 results at 11:30
a.m. Eastern time on Wednesday, May 3, 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 first 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, ION363, 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, 2022, which is on file with the
Securities and Exchange Commission. Copies of this 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. QALSODY™ 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,
|
|
|
|
|
March 31,
|
|
|
|
|
2023
|
|
2022
|
|
|
|
|
(unaudited)
|
|
Revenue:
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$50
|
|
$54
|
|
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
7
|
|
6
|
|
|
Licensing and royalty
revenue
|
|
11
|
|
12
|
|
|
Total commercial
revenue
|
|
68
|
|
72
|
|
|
Research and
development revenue:
|
|
|
|
|
|
|
Collaborative
agreement revenue
|
|
39
|
|
50
|
|
|
Eplontersen joint
development revenue
|
|
24
|
|
20
|
|
|
Total research and
development revenue
|
|
63
|
|
70
|
|
|
Total
revenue
|
|
131
|
|
142
|
|
|
Expenses:
|
|
|
|
|
|
|
Cost of
sales
|
|
1
|
|
4
|
|
|
Research, development and patent
|
|
198
|
|
161
|
|
|
Selling, general and administrative
|
|
46
|
|
34
|
|
|
Total operating
expenses
|
|
245
|
|
199
|
|
|
Loss from
operations
|
|
(114)
|
|
(57)
|
|
|
|
|
|
|
|
|
|
Other income
(expense):
|
Interest expense
related to sale of future royalties
|
|
(16)
|
|
-
|
|
Other income
(expense), net
|
|
17
|
|
(7)
|
|
|
Loss before income tax
expense
|
|
(113)
|
|
(64)
|
|
|
|
|
|
|
|
|
|
Income tax
expense
|
|
(11)
|
|
(1)
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
($124)
|
|
($65)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share
|
|
($0.87)
|
|
($0.46)
|
|
|
Shares used in
computing basic and diluted net loss per share
|
|
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
March
31,
|
|
|
|
2023
|
|
2022
|
|
|
(unaudited)
|
As reported
research, development and patent expenses according to
GAAP
|
|
$198
|
|
$161
|
Excluding compensation expense related to equity
awards
|
|
(20)
|
|
(19)
|
Non-GAAP research,
development and patent expenses
|
|
$178
|
|
$142
|
|
|
|
|
|
As reported selling,
general and administrative expenses according to
GAAP
|
|
$46
|
|
$34
|
Excluding compensation expense related to equity
awards
|
|
(7)
|
|
(7)
|
Non-GAAP selling,
general and administrative expenses
|
|
$39
|
|
$27
|
|
|
|
|
|
As reported
operating expenses according to GAAP
|
|
$245
|
|
$199
|
Excluding compensation
expense related to equity
awards
|
|
(27)
|
|
(26)
|
Non-GAAP operating
expenses
|
|
$218
|
|
$173
|
|
|
|
|
|
As reported loss
from operations according to GAAP
|
|
($114)
|
|
($57)
|
Excluding compensation expense related to equity
awards
|
|
(27)
|
|
(26)
|
Non-GAAP loss from
operations
|
|
($87)
|
|
($31)
|
|
|
|
|
|
As reported net loss
according to GAAP
|
|
($124)
|
|
($65)
|
Excluding compensation expense related to equity awards and related
tax effects
|
|
(27)
|
|
(26)
|
Non-GAAP net
loss
|
|
($97)
|
|
($39)
|
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)
|
|
|
|
|
|
|
|
|
|
March 31,
|
|
December 31,
|
|
|
|
2023
|
|
2022
|
|
|
|
(unaudited)
|
|
|
|
Assets:
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$2,347
|
|
$1,987
|
|
Contracts
receivable
|
|
14
|
|
26
|
|
Other current
assets
|
|
182
|
|
190
|
|
Property, plant
and equipment, net
|
|
85
|
|
74
|
|
Right-of-use
assets
|
|
179
|
|
182
|
|
Other
assets
|
|
78
|
|
75
|
|
Total assets
|
|
$2,885
|
|
$2,534
|
|
|
|
|
|
|
|
Liabilities and
stockholders' equity:
|
|
|
|
|
|
Other current
liabilities
|
|
$167
|
|
$221
|
|
Current portion
of deferred contract revenue
|
|
92
|
|
91
|
|
0% convertible
senior notes, net
|
|
623
|
|
622
|
|
0.125%
convertible senior notes, net
|
|
545
|
|
545
|
|
Liability
related to sale of future royalties, net
|
|
505
|
|
-
|
|
Long-term lease
liabilities
|
|
177
|
|
178
|
|
Long-term
obligations, less current portion
|
|
16
|
|
16
|
|
Long-term
deferred contract revenue
|
|
273
|
|
288
|
|
Total
stockholders' equity
|
|
487
|
|
573
|
|
Total liabilities and stockholders' equity
|
|
$2,885
|
|
$2,534
|
|
2023 Key Value Driving Events(1)
Regulatory
Actions
|
Program
|
Indication
|
Regulatory
Action
|
QALSODY
|
SOD1-ALS
|
NDA approval
(achieved)
|
EU
approval2
|
Eplontersen
(TTR)
|
ATTRv
polyneuropathy
|
NDA approval
|
OUS filings
|
|
Key Clinical
Achievements
|
Program
|
Indication
|
Event
|
Eplontersen
(TTR)
|
ATTRv
polyneuropathy
|
Phase 3 data (week
35
& 66) (achieved)
|
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.
|
(2) CHMP opinion
anticipated in Q4:2023.
|
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SOURCE Ionis Pharmaceuticals, Inc.