On track to achieve 2022 financial guidance
Webcast today, May 4,
2022, at 11:30 a.m. Eastern
Time
CARLSBAD, Calif., May 4, 2022
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS)
today reported financial results for the first quarter of
2022 and recent business achievements.
"We are off to a strong start this year highlighted by progress
in our rich late- and mid-stage pipeline. We remain on track for
data from the NEURO-TTRansform study of eplontersen in patients
with hereditary ATTR polyneuropathy by mid-year. Assuming positive
data, we plan to file for regulatory approval by the end of this
year. We recently increased the size and duration of our
CARDIO-TTRansform study of eplontersen in patients with ATTR
cardiomyopathy. Our aim is to generate even more robust data and
ensure a highly positive study outcome to successfully compete in
this growing and dynamic market," said Brett P. Monia, Ph.D., chief executive officer
of Ionis. "We also reported positive data from two potential
best-in-class medicines. In the Phase 2b ETESIAN study, ION449, our investigational
medicine targeting PCSK9, demonstrated robust LDL-C and PCSK9
reductions in statin-treated patients with hypercholesterolemia.
ION449 was generally well tolerated in this study. Additional
positive data from the Phase 2 study of
donidalorsen demonstrated significant improvements in quality
of life in people with hereditary angioedema. Looking ahead, we
expect to provide additional key mid-stage data readouts and
updates on our technology advancements. These upcoming catalysts,
together with our recent achievements, position us well to continue
to drive substantial growth and value for all stakeholders."
First Quarter 2022 Summary Financial Results
On track to achieve 2022 financial guidance, based on the
following first quarter results:
- $142 million in total
revenues
- $173 million of operating
expenses on a non-GAAP basis(1) and $199 million on a GAAP basis
- $39 million net loss on a
non-GAAP basis(1) and $65
million on a GAAP basis
- $2.1 billion of cash and
short-term investments
"A key element of Ionis' financial strength is our ability to
generate substantial revenue from multiple diverse sources on a
sustained basis. Our first quarter financial results in which
revenues grew more than 25 percent year over year were an excellent
example of this. We generated revenues from our marketed products,
including SPINRAZA, and from numerous partnered medicines as they
advanced. Our first quarter financial results also reflect our
investments in our rich late-stage pipeline and in activities to
prepare for our launches of eplontersen, olezarsen and
donidalorsen," said Elizabeth L.
Hougen, chief financial officer of Ionis. "We are on track
to meet our 2022 financial guidance. With $2.1 billion of cash, we are well capitalized
with the resources we need to continue investing in our large
agenda to drive substantial future growth."
Recent Marketed Products Highlights
SPINRAZA®: the global market leader for the treatment
of spinal muscular atrophy (SMA) patients of all ages
- $473 million in worldwide
SPINRAZA sales in the first quarter
- Biogen provided updates from the ASCEND, RESPOND and NURTURE
studies of SPINRAZA at the Muscular Dystrophy Association (MDA)
Clinical and Scientific conference and the American Academy of
Neurology (AAN) annual meeting
TEGSEDI® and WAYLIVRA®: important
medicines approved for the treatment of patients with
polyneuropathy caused by hereditary TTR amyloidosis and familial
chylomicronemia syndrome, respectively
- Continued to progress into new and existing markets in
Europe and Latin America in the first quarter through
Swedish Orphan Biovitrum AB (Sobi) and PTC Therapeutics,
respectively
First Quarter 2022 and Recent Events
Advancing Ionis' near-term commercial opportunities toward the
market
- Increased study size and duration in the Phase 3
CARDIO-TTRansform study of eplontersen in patients with ATTR
cardiomyopathy with the aim to generate even more robust data and
ensure a highly positive study outcome to successfully compete in
this growing and dynamic market. Data from this study are expected
in the first half of 2025
- The U.S. FDA granted orphan drug designation to eplontersen for
the treatment of patients with ATTR
- Published positive data from the Phase 2 study of olezarsen in
patients with hypertriglyceridemia and either at high risk for or
with established cardiovascular disease in the European Heart
Journal
- Initiated a study of olezarsen in patients with
hypertriglyceridemia to support the broad Phase 3 program
- Published positive data from the Phase 2 study of donidalorsen
in patients with hereditary angioedema (HAE) in the New England
Journal of Medicine
- Presented additional positive data from the Phase 2 study of
donidalorsen in patients with HAE at the American Academy of
Allergy, Asthma and Immunology (AAAAI) annual meeting
Advancing Ionis' leading cardiovascular disease franchise
- AstraZeneca presented positive data from the Phase 2b ETESIAN study of ION449 (AZD8233) targeting
PCSK9 in statin treated patients with dyslipidemia at the American
College of Cardiology (ACC) annual scientific session
- Achieved full enrollment in the Phase 2b study of IONIS-AGT-LRx in patients
with treatment-resistant hypertension, with data expected in the
second half of 2022
Advancing Ionis' leading neurological disease franchise
- Roche plans to initiate a new Phase 2 study of tominersen in
patients with Huntington's disease based on findings from a
post-hoc analysis of the GENERATION-HD1 study
- Biogen initiated a Phase 1/2 study of ION260 (BIIB132)
targeting ataxin-3 (ATXN3) in patients with spinocerebellar ataxia
type 3 (SCA3), resulting in an $8
million milestone payment from Biogen
- Biogen advanced the Phase 1/2 study of ION859 (BIIB094)
targeting LRRK2 in patients with Parkinson's disease, resulting in
a $10 million milestone payment from
Biogen
- Announced the discontinuation of IONIS-C9Rx
(BIIB078) due to lack of patient benefit demonstrated in the Phase
1/2 study in patients with C9orf72-ALS
2022 Pipeline Milestones(2)
Anticipated 2022 Regulatory
Updates
|
Program
|
Anticipated Indication
|
Regulatory Action
|
H1
|
H2
|
Eplontersen
|
ATTRv
polyneuropathy
|
NDA filing
|
|
•
|
|
Anticipated Key 2022 Data
Readouts
|
Program
|
Data Readout
|
Anticipated Indication
|
H1
|
H2
|
Tominersen
|
Phase 3 post
hoc
|
Huntington's
disease
|
✓
|
|
ION449
(PCSK9)
|
Phase 2b
(ETESIAN)
|
Cardiovascular
disease
|
✓
|
|
Donidalorsen
|
Phase 2
|
HAE
|
✓
|
|
IONIS-C9Rx
(BIIB078)
|
Phase 1/2
|
C9-ALS
|
✓
|
|
Tofersen
|
Phase 3 OLE
|
SOD1-ALS
|
•
|
|
Eplontersen
|
Phase 3
|
ATTRv
polyneuropathy
|
•
|
IONIS-AGT-LRx
|
Phase 2b
|
Treatment-resistant
hypertension
|
|
•
|
Fesomersen
(FXI)
|
Phase 2b
|
Thrombosis
|
|
•
|
Bepirovirsen
(HBV)
|
Phase 2b
|
Hepatitis B virus
infection
|
|
•
|
Donidalorsen
|
Phase 2 OLE
|
HAE
|
|
•
|
Cimdelirsen
(GHR)
|
Phase 2
(monotherapy)
|
Acromegaly
|
|
•
|
|
Anticipated Key 2022 Study
Initiations
|
Program
|
Phase
|
Anticipated Indication
|
H1
|
H2
|
Sapablursen
|
2
|
Polycythemia
vera
|
✓
|
|
IONIS-MAPTRx
(BIIB080)
|
2
|
Alzheimer's
disease
|
|
•
|
ION904 (AGT)
|
2
|
Uncontrolled
hypertension
|
|
•
|
ION717
(PRNP)
|
1/2
|
Prion
disease
|
|
•
|
|
Anticipated Key 2022 Technology
Advancements
|
Program
|
Anticipated Advancement
|
H1
|
H2
|
SMA
|
Advance follow-on
program
|
✓
|
|
Muscle LICA
|
Advance into
preclinical development (IND-supporting)
|
|
•
|
MsPA
Backbone
|
Advance into
preclinical development (IND-supporting)
|
|
•
|
✓ = achieved • = planned
(1)
|
All non-GAAP amounts
referred to in this press release exclude non-cash compensation
expense related to equity awards. In 2021 all non-GAAP amounts also
excluded expenses
related to the Akcea Merger and restructured commercial operations
and the related tax
effects. Please refer to the detailed reconciliation of non-GAAP
and GAAP measures, which is
provided later in this press release.
|
|
|
(2)
|
Partnered program
milestones are based on partners' most recent publicly
available
disclosures.
|
First Quarter 2022 Financial Results
Revenue
Ionis' revenue was
comprised of the following (amounts in millions):
|
|
|
|
|
|
Three months ended
March 31,
|
|
|
|
2022
|
2021
|
|
|
Revenue:
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
SPINRAZA royalties
|
|
$54
|
|
$60
|
|
TEGSEDI and WAYLIVRA revenue,
net
|
|
6
|
|
20
|
|
Licensing and royalty
revenue
|
|
12
|
|
5
|
|
Total commercial
revenue
|
|
72
|
|
85
|
|
Research and development revenue:
|
|
|
|
|
|
Amortization from upfront
payments
|
|
17
|
|
20
|
|
Milestone payments
|
|
27
|
|
5
|
|
License fees
|
|
2
|
|
-
|
|
Other services
|
|
4
|
|
2
|
|
Collaborative agreement
revenue
|
|
50
|
|
27
|
|
Eplontersen joint development revenue
|
|
20
|
|
-
|
|
Total research and development
revenue
|
|
70
|
|
27
|
|
Total revenue
|
|
$142
|
|
$112
|
|
The Company's revenue in the first quarter of 2022 increased
more than 25 percent compared to the same period last year. The
increase was driven by significant partner payments across multiple
partnered programs, including $20
million from AstraZeneca for its share of the global Phase 3
program costs for eplontersen. Since Ionis is conducting the
ongoing Phase 3 program for eplontersen, Ionis recognizes revenue
for the reimbursements it receives from AstraZeneca for development
expenses in the same period Ionis recognizes the related
development expenses. Refer to the detailed table of costs and
reimbursements for the eplontersen collaboration provided later in
this release. The Company also earned $40
million from Biogen for advancing several neurology disease
programs.
The Company successfully completed the transition of its TEGSEDI
and WAYLIVRA operations in the EU and North America to Sobi in the first and second
quarters of 2021, respectively. The decrease in TEGSEDI and
WAYLIVRA revenue in the first quarter of 2022 compared to the same
period last year was due to the shift from product sales to
distribution fees based on net sales generated by Sobi. As part of
the transition, Ionis restructured its commercial operations in
2021 resulting in substantial cost savings.
Operating Expenses
Ionis is advancing a large late-stage pipeline and as a result,
its non-GAAP operating expenses increased in the first quarter of
2022 compared to the same period in 2021. Higher R&D expenses
were driven by the expanded number of Phase 3 studies the Company
is conducting, which doubled over the course of 2021 from three to
six studies. Lower SG&A expenses were largely due to the
substantial savings Ionis achieved from integrating Akcea and
restructuring its commercial operations. These savings were offset
in part by the investments Ionis made in advancing its go-to-market
activities for its near-term commercial opportunities.
Net Loss
Ionis' net loss in the first quarter of 2022 decreased compared
to the same period in 2021 primarily related to the changes in
revenue and operating expenses, as discussed above.
Balance Sheet
As of March 31, 2022 and
December 31, 2021, Ionis had cash,
cash equivalents and short-term investments of $2.1 billion. Ionis' debt obligations and working
capital did not change significantly from December 31, 2021 to March
31, 2022.
Webcast
Ionis will conduct a webcast today at 11:30 a.m. Eastern time to discuss this
announcement and related activities. Interested parties may access
the webcast here. A webcast replay will be available for a limited
time at the same address.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been the leader in
RNA-targeted therapy, pioneering new markets and changing the
standards of care with its novel antisense technology. Ionis
currently has three marketed medicines and a premier late-stage
pipeline highlighted by industry leading cardiovascular and
neurological franchises. Our scientific innovation began and
continues with the knowledge that sick people depend on us, which
fuels our vision of becoming a leading, fully integrated
biotechnology company.
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 SPINRAZA (nusinersen), TEGSEDI (inotersen),
WAYLIVRA (volanesorsen), eplontersen, olezarsen, donidalorsen,
ION363, pelacarsen, tofersen, 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 related to the impact COVID-19 could
have on our business, and 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,
2021, which is on file with the SEC. 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. 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,
|
|
|
|
2022
|
|
2021
|
|
|
|
|
(unaudited)
|
|
|
Revenue:
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
SPINRAZA royalties
|
|
$54
|
|
$60
|
|
|
TEGSEDI and WAYLIVRA revenue, net
|
|
6
|
|
20
|
|
|
Licensing and royalty revenue
|
|
12
|
|
5
|
|
|
Total commercial
revenue
|
|
72
|
|
85
|
|
|
Research and development revenue:
|
|
|
|
|
|
|
Collaborative agreement
revenue
|
|
50
|
|
27
|
|
|
Eplontersen joint development
revenue
|
|
20
|
|
-
|
|
|
Total research and
development revenue
|
|
70
|
|
27
|
|
|
Total revenue
|
|
142
|
|
112
|
|
|
Expenses:
|
|
|
|
|
|
|
Cost of
sales
|
|
4
|
|
3
|
|
|
Research, development and patent
|
|
161
|
|
140
|
|
|
Selling, general and administrative
|
|
34
|
|
61
|
|
|
Total operating
expenses
|
|
199
|
|
204
|
|
|
Loss from
operations
|
|
(57)
|
|
(92)
|
|
|
|
|
|
|
|
|
|
Other income
(expense):
|
|
|
|
|
|
|
Other income (expense), net
|
|
(7)
|
|
2
|
|
|
Loss before income tax
expense
|
|
(64)
|
|
(90)
|
|
|
|
|
|
|
|
|
|
Income tax
expense
|
|
(1)
|
|
-
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
($65)
|
|
($90)
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share
|
|
($0.46)
|
|
($0.64)
|
|
|
Shares used in
computing basic and diluted net loss per share
|
|
142
|
|
141
|
|
|
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
|
|
|
|
2022
|
|
2021
|
|
|
|
(unaudited)
|
As reported
research, development and patent expenses according to
GAAP
|
|
$161
|
|
$140
|
|
Excluding compensation expense related to equity
awards
|
|
(19)
|
|
(26)
|
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
(3)
|
|
Non-GAAP research,
development and patent expenses
|
|
$142
|
|
$111
|
|
|
|
|
|
|
|
As reported selling,
general and administrative expenses according
to GAAP
|
|
$34
|
|
$61
|
|
Excluding compensation expense related to equity
awards
|
|
(7)
|
|
(12)
|
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
(4)
|
|
Non-GAAP selling,
general and administrative expenses
|
|
$27
|
|
$45
|
|
|
|
|
|
|
|
As reported
operating expenses according to GAAP
|
|
$199
|
|
$204
|
|
Excluding compensation expense related to equity
awards
|
|
(26)
|
|
(38)
|
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
(7)
|
|
Non-GAAP operating
expenses
|
|
$173
|
|
$159
|
|
|
|
|
|
|
|
As reported loss
from operations according to GAAP
|
|
($57)
|
|
($92)
|
|
Excluding compensation expense related to equity
awards
|
|
(26)
|
|
(38)
|
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
(7)
|
|
Non-GAAP loss from
operations
|
|
($31)
|
|
($47)
|
|
|
|
|
|
|
|
As reported net loss
according to GAAP
|
|
($65)
|
|
($90)
|
|
Excluding compensation expense related to equity
awards
|
|
(26)
|
|
(38)
|
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
(7)
|
|
Income tax effect related to compensation expense related to equity
awards
|
|
-
|
|
-
|
|
Non-GAAP net loss
according to GAAP
|
|
($39)
|
|
($45)
|
|
|
|
|
|
|
|
|
*In October 2020, Ionis
completed a merger transaction with Akcea such that following the
completion of the merger Akcea became a wholly owned subsidiary of
Ionis. Additionally, in December 2020 and April 2021, Ionis
restructured its European operations and its North American TEGSEDI
operations, respectively, as a result of entering into distribution
agreements with Sobi. The Company excluded these costs 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 loss from
operations, and non-GAAP net loss were adjusted from GAAP to
exclude compensation expense related to equity awards and costs
related to the Akcea merger and restructured commercial operations
and the related tax effects. Compensation expense related to equity
awards are non-cash. Costs related to the Akcea merger and
restructured commercial operations included: severance costs,
retention costs and other costs related to commercial operations.
Ionis has regularly reported non-GAAP measures for operating
results as non-GAAP results. 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.
Summary of the Financial Impacts of the Eplontersen Collaboration
with AstraZeneca
For the Three Months Ended, March 31, 2022
|
|
|
Collaboration
Activities
|
|
Financial
Statement
Line
|
|
Impact of
Cost-Sharing Provisions
on Ionis' Statement
of Operations
|
|
|
|
|
|
|
|
Phase 3
Development: Ionis
leads and conducts
|
|
Eplontersen
Joint Development
Revenue
(R&D
Revenue)
|
|
$20M
|
|
55% of Ionis' Phase
3
development expenses, including
internal+external costs &
CMC costs
|
|
|
|
|
|
|
|
Development
Expenses
(R&D
Expenses)
|
|
$36M
|
|
100% of Ionis' Phase
3
development expenses
|
Ionis' financial results for the first quarter of 2022 reflected
the cost-sharing provisions related to its eplontersen
collaboration with AstraZeneca to develop and commercialize
eplontersen for the treatment of ATTR. Under the terms of the
collaboration agreement, AstraZeneca is paying 55 percent of the
costs associated with the ongoing global Phase 3 development
program. Because Ionis is leading and conducting the Phase 3
development program, Ionis is recognizing the 55 percent of
cost-share funding AstraZeneca is responsible for as R&D
revenue in the same period Ionis incurs the related development
expenses. Ionis will receive $20
million from AstraZeneca related to development expenses
Ionis incurred in the first quarter of 2022.
As AstraZeneca is responsible for the majority of the medical
affairs and commercial costs in the U.S. and all costs associated
with bringing eplontersen to market outside the U.S., Ionis is
recognizing cost-share funding it receives from AstraZeneca related
to these activities as a reduction of its medical affairs (R&D
expenses) and commercialization expenses (SG&A expenses). In
the first quarter of 2022, Ionis recognized $0.4 million and $0.2
million of medical affairs expenses and commercialization
expenses for eplontersen, respectively, net of cost-share funding
from AstraZeneca. Ionis expects its medical affairs and
commercialization expenses to increase as this collaboration
progresses.
IONIS
PHARMACEUTICALS, INC.
Condensed Consolidated Balance Sheets
(In Millions)
|
|
|
|
|
|
|
|
March 31,
|
|
December 31,
|
|
|
2022
|
|
2021
|
|
|
(unaudited)
|
|
|
Assets:
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$2,052
|
|
$2,115
|
Contracts
receivable
|
|
26
|
|
62
|
Other current
assets
|
|
175
|
|
168
|
Property, plant
and equipment, net
|
|
178
|
|
178
|
Other
assets
|
|
88
|
|
89
|
Total assets
|
|
$2,519
|
|
$2,612
|
|
|
|
|
|
Liabilities and
stockholders' equity:
|
|
|
|
|
Other current
liabilities
|
|
$137
|
|
$143
|
Current portion
of deferred contract revenue
|
|
91
|
|
98
|
0% convertible
senior notes, net
|
|
620
|
|
619
|
0.125%
convertible senior notes, net
|
|
543
|
|
542
|
Long-term
obligations, less current portion
|
|
85
|
|
86
|
Long-term
deferred contract revenue
|
|
333
|
|
352
|
Total
stockholders' equity
|
|
710
|
|
772
|
Total liabilities and stockholders' equity
|
|
$2,519
|
|
$2,612
|
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SOURCE Ionis Pharmaceuticals, Inc.