WAINUATM launch
on track; EU and Canada approval
decisions expected this year
Olezarsen NDA submitted to FDA for FCS;
preparing EU regulatory submission
Positive Phase 3 donidalorsen data for HAE;
preparing regulatory submissions
On track to achieve 2024 financial
guidance
CARLSBAD, Calif., May 7, 2024
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS)
(the "Company"), today reported financial results for the first
quarter ended March 31, 2024.
"Ionis is off to a great start in 2024, as we continue to
execute on our vision to bring better futures to people with
serious diseases. The WAINUA launch for hereditary ATTR
polyneuropathy is progressing well with AstraZeneca. And we are one
step closer to our first independent launch with our NDA submission
for olezarsen, which is supported by robust data positioning
olezarsen to make a profound difference for people with FCS," said
Brett P. Monia, Ph.D., chief
executive officer of Ionis. "We look forward to presenting positive
Phase 3 donidalorsen data, along with data from our open-label
extension and 'switch' studies in patients with HAE at EAACI later
this month, setting the stage for Ionis' second independent launch.
Additionally, we have multiple upcoming data readouts from our
mid-stage programs that, if positive, could advance into Phase 3
development, further strengthening our ability to deliver a steady
cadence of potentially transformational medicines for years to
come."
First Quarter 2024 Summary Financial
Results(1):
|
|
Three months
ended
March 31,
|
|
|
2024
|
|
2023
|
|
|
(amounts in
millions)
|
Total
revenue
|
|
$119
|
|
$131
|
Operating
expenses
|
|
$269
|
|
$245
|
Operating expenses on a
non-GAAP basis
|
|
$238
|
|
$218
|
Loss from
operations
|
|
($150)
|
|
($114)
|
Loss from operations on
a non-GAAP basis
|
|
($119)
|
|
($87)
|
|
(1)
Reconciliation of GAAP to non-GAAP basis contained later in this
release.
|
Financial Highlights
- Revenue for the first quarter of 2024 earned from numerous
diverse sources, including a new source of royalty revenue with the
launch of WAINUA in the U.S.
- Continued strategic investments to bring WAINUA, olezarsen and
donidalorsen to patients drove increased operating expenses in the
first quarter of 2024 compared to the same period last year
- Cash and short-term investments of $2.2
billion as of March 31, 2024
enable continued investments to drive increasing value, including
supporting our planned upcoming launches
- Reaffirmed 2024 financial guidance
Recent Marketed Medicines Highlights
- WAINUA for the treatment of adults with polyneuropathy of
hereditary transthyretin-mediated amyloidosis (ATTRv-PN) generated
sales of $5 million in the first
partial quarter of launch resulting in royalty revenue of
$1 million for Ionis in the first
quarter of 2024
- SPINRAZA for the treatment of spinal muscular atrophy (SMA)
generated global sales of $341
million resulting in royalty revenue of $38 million in the first quarter of 2024
- Biogen presented new positive neurofilament light chain (NfL)
biomarker data from the Phase 4 RESPOND study of SMA patients
adding further evidence supporting the potential benefit of
SPINRAZA in infants and toddlers who had unmet medical needs after
treatment with gene therapy
Recent Late-Stage Pipeline Highlights
- Eplontersen granted Fast Track designation by the FDA for the
treatment of patients with ATTR cardiomyopathy
- Olezarsen achieved multiple milestones advancing it closer to
potentially addressing two distinct populations of patients with
urgent unmet need, familial chylomicronemia syndrome (FCS) and
severe hypertriglyceridemia (sHTG):
- Submitted NDA to the FDA for FCS
- Presented positive Phase 3 Balance study data in patients with
FCS with a simultaneous publication in the New England Journal
of Medicine
- Presented positive Phase 2b
Bridge study data in patients with HTG and sHTG with a simultaneous
publication in the New England Journal of Medicine
- Opened Expanded Access Program (EAP) for FCS in the U.S.
- Granted Breakthrough Therapy and Orphan Drug designations by
the FDA for the treatment of patients with FCS
- Completed enrollment of the Phase 3 CORE pivotal study and
ESSENCE supportive exposure study for sHTG; CORE2 confirmatory
pivotal study on track to fully enroll mid-year
- Donidalorsen achieved multiple milestones advancing it closer
to potentially becoming a first-in-class RNA-targeted prophylactic
treatment for people 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
- Opened EAP for HAE in the U.S.
- Granted Orphan Drug designation by EMA
- Bepirovirsen granted Fast Track designation by the FDA for the
treatment of patients with chronic hepatitis B (CHB)
Recent Other Pipeline Highlights
- Reported positive Phase 2 data for ION224 (DGAT2) in patients
with metabolic dysfunction-associated steatohepatitis (MASH)
- Initiated the Phase 1/2 Orbit study of ION356 (PLP1) in
patients with Pelizaeus-Merzbacher disease (PMD)
First Quarter 2024 Financial Results
"Our first quarter results keep us on track to achieve our 2024
financial guidance. With the launch of WAINUA in the U.S. underway,
we are excited to add WAINUA royalties to our meaningful revenues
in the first quarter. We believe WAINUA is uniquely positioned in
this growing market to address the needs of ATTRv-PN patients who
remain significantly underserved, especially as it is the only
approved medicine with monthly dosing that can be self-administered
via an auto injector," said Elizabeth L.
Hougen, chief financial officer of Ionis. "We continued to
invest our capital resources in our near-term commercial
opportunities, wholly owned pipeline and technology. We expect our
modest expense growth this year to be driven by our activities to
support the WAINUA launch and planned launches for olezarsen and
donidalorsen with R&D expenses approaching steady state as
several late-stage studies have recently ended. We believe the
investments we are making today and plan to make over the next few
years position Ionis to drive increasing value for patients and
stakeholders."
Revenue
Ionis' revenue was comprised of the following:
|
|
Three months
ended
|
|
|
March 31,
|
|
|
2024
|
|
2023
|
Revenue:
|
|
(amounts in
millions)
|
Commercial
revenue:
|
|
|
|
|
SPINRAZA
royalties
|
|
$38
|
|
$50
|
WAINUA
royalties
|
|
1
|
|
-
|
Other commercial
revenue:
|
|
|
|
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
9
|
|
7
|
Licensing and other
royalty revenue
|
|
11
|
|
11
|
Total commercial
revenue
|
|
59
|
|
68
|
Research and
development revenue:
|
|
|
|
|
Amortization from
upfront payments
|
|
42
|
|
16
|
Milestone
payments
|
|
7
|
|
23
|
Collaborative
agreement revenue
|
|
49
|
|
39
|
WAINUA joint
development revenue
|
|
11
|
|
24
|
Total research and
development revenue
|
|
60
|
|
63
|
Total
revenue
|
|
$119
|
|
$131
|
Commercial revenue in the first quarter of 2024 included a new
source of royalty revenue with the launch of WAINUA in the U.S.
during the first quarter of 2024. While the number of patients on
SPINRAZA treatment remained consistent globally, royalties
decreased year over year primarily due to the timing of shipments
in several markets outside the U.S. Ionis' commercial revenue in
the first quarter of 2024 also included royalties from the net
sales of QALSODY, which Biogen launched in the second quarter of
2023.
R&D revenue in the first quarter of 2024 included increased
revenue from the amortization of upfront payments compared to the
same period last year due to the new collaborations Ionis entered
into last year with Roche and Novartis. This increase was offset by
decreases in milestone payments due to timing and WAINUA joint
development revenue, which decreased as development activities
relating to ATTRv-PN wound down with the launch of WAINUA
underway.
Operating Expenses
Ionis' operating expenses increased in the first quarter of 2024
compared to the same period in 2023, consistent with expectations.
SG&A expenses increased year over year primarily due to the
launch of WAINUA in the U.S. and launch preparation activities for
olezarsen and donidalorsen. R&D expenses increased compared to
the same period last year due to the timing of development
activities and are expected to stabilize in 2024 as several
late-stage studies have ended.
Balance Sheet
As of March 31, 2024, Ionis' cash,
cash equivalents and short-term investments decreased to
$2.2 billion compared to $2.3 billion at December
31, 2023. As the year progresses, the Company plans to
continue deploying its capital resources toward growth
opportunities. Ionis' working capital also decreased over the same
period primarily due to the Company's lower cash and short-term
investments balance.
Webcast
Management will host a conference call and webcast to discuss
Ionis' first quarter 2024 results at 11:30
a.m. Eastern time on Tuesday, May 7, 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 first quarter 2024 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, 2023, 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 Investor Contact:
D. Wade Walke, Ph.D.
info@ionisph.com 760-603-2331
Ionis Media Contact:
Hayley
Soffer
CorporateCommunications@ionisph.com 760-603-4679
IONIS
PHARMACEUTICALS, INC.
SELECTED FINANCIAL
INFORMATION
Condensed
Consolidated Statements of Operations
(In Millions, Except
Per Share Data)
|
|
|
|
|
|
Three months
ended,
|
March 31,
|
2024
|
|
2023
|
|
|
(unaudited)
|
Revenue:
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
SPINRAZA
royalties
|
|
$38
|
|
$50
|
WAINUA
royalties
|
|
1
|
|
-
|
Other commercial
revenue
|
|
20
|
|
18
|
Total commercial revenue
|
|
59
|
|
68
|
Research and
development revenue:
|
|
|
|
|
Collaborative
agreement revenue
|
|
49
|
|
39
|
WAINUA joint
development revenue
|
|
11
|
|
24
|
Total research and development revenue
|
|
60
|
|
63
|
Total
revenue
|
|
119
|
|
131
|
Expenses:
|
|
|
|
|
Cost of sales
|
|
2
|
|
1
|
Research, development and patent
|
|
214
|
|
198
|
Selling, general and administrative
|
|
53
|
|
46
|
Total operating
expenses
|
|
269
|
|
245
|
Loss from
operations
|
|
(150)
|
|
(114)
|
|
|
|
|
|
Other income
(expense):
|
|
|
|
|
Interest expense
related to the sale of future royalties
|
|
(18)
|
|
(16)
|
Other income,
net
|
|
25
|
|
17
|
Loss before income tax
expense
|
|
(143)
|
|
(113)
|
|
|
|
|
|
Income tax
expense
|
|
-
|
|
(11)
|
|
|
|
|
|
Net loss
|
|
($143)
|
|
($124)
|
|
|
|
|
|
Basic and diluted net
loss per share
|
|
($0.98)
|
|
($0.87)
|
Shares used in
computing basic and diluted net loss per share
|
|
146
|
|
143
|
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,
|
2024
|
|
2023
|
|
|
(unaudited)
|
As reported
research, development and patent expenses according to
GAAP
|
|
$214
|
|
$198
|
Excluding compensation expense related to equity
awards
|
|
(22)
|
|
(20)
|
Non-GAAP research,
development and patent expenses
|
|
$192
|
|
$178
|
|
|
|
|
|
As reported selling,
general and administrative expenses according to
GAAP
|
|
$53
|
|
$46
|
Excluding compensation expense related to equity
awards
|
|
(9)
|
|
(7)
|
Non-GAAP selling,
general and administrative expenses
|
|
$44
|
|
$39
|
|
|
|
|
|
As reported
operating expenses according to GAAP
|
|
$269
|
|
$245
|
Excluding compensation
expense related to equity
awards
|
|
(31)
|
|
(27)
|
Non-GAAP operating
expenses
|
|
$238
|
|
$218
|
|
|
|
|
|
As reported loss
from operations according to GAAP
|
|
($150)
|
|
($114)
|
Excluding compensation expense related to equity
awards
|
|
(31)
|
|
(27)
|
Non-GAAP loss from
operations
|
|
($119)
|
|
($87)
|
|
|
|
|
|
As reported net loss
according to GAAP
|
|
($143)
|
|
($124)
|
Excluding compensation expense related to equity awards and related
tax effects
|
|
(31)
|
|
(27)
|
Non-GAAP net
loss
|
|
($112)
|
|
($97)
|
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,
|
|
|
2024
|
|
2023
|
|
|
(unaudited)
|
|
|
Assets:
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$2,206
|
|
$2,331
|
Contracts
receivable
|
|
5
|
|
98
|
Other current
assets
|
|
204
|
|
213
|
Property, plant
and equipment, net
|
|
73
|
|
71
|
Right-of-use
assets
|
|
169
|
|
172
|
Other
assets
|
|
107
|
|
105
|
Total assets
|
|
$2,764
|
|
$2,990
|
|
|
|
|
|
Liabilities and
stockholders' equity:
|
|
|
|
|
Current portion
of deferred contract revenue
|
|
$130
|
|
$151
|
0.125%
convertible senior notes, net – short-term
|
|
44
|
|
44
|
Other current
liabilities
|
|
153
|
|
253
|
1.75%
convertible senior notes, net
|
|
563
|
|
562
|
0% convertible
senior notes, net
|
|
626
|
|
625
|
Liability
related to sale of future royalties, net
|
|
525
|
|
514
|
Long-term lease
liabilities
|
|
169
|
|
171
|
Long-term
obligations, less current portion
|
|
42
|
|
42
|
Long-term
deferred contract revenue
|
|
215
|
|
241
|
Total
stockholders' equity
|
|
297
|
|
387
|
Total liabilities and stockholders' equity
|
|
$2,764
|
|
$2,990
|
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
|
Olezarsen
|
FCS
|
Balance study full
data
|
•
|
Donidalorsen
|
HAE
|
OASIS-HAE topline
data
|
•
|
Donidalorsen
|
HAE
|
OASIS-HAE full
data
|
|
Donidalorsen
|
HAE
|
OASIS-Plus: OLE +
Switch data
|
|
|
Key Phase 2 Clinical Data
Events
|
Program
|
Indication
|
Event
|
Achieved
|
Donidalorsen
|
HAE
|
3-year Phase 2 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.
|
View original content to download
multimedia:https://www.prnewswire.com/news-releases/ionis-reports-first-quarter-2024-financial-results-302137316.html
SOURCE Ionis Pharmaceuticals, Inc.