CARLSBAD, Calif., Aug. 4, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (Nasdaq: IONS) today reported its financial
results for the second quarter of 2021 and recent business
achievements.
"Since our last quarterly update, we continued to execute on our
strategic objectives to prepare for multiple Ionis commercial
launches, expand our drug delivery capabilities and advance new
products towards the market. Biogen completed dosing in the
tofersen Phase 3 VALOR study and began offering tofersen to
SOD1-ALS patients on an individual compassionate use basis. We
achieved full enrollment in the eplontersen Phase 3
NEURO-TTRansform study and 50 percent enrollment in the pelacarsen
Phase 3 Lp(a) HORIZON study. Additionally, we licensed Bicycle
Therapeutics' technology to expand the capabilities of our LICA
technology," said Brett P. Monia,
Ph.D., chief executive officer of Ionis. "Looking ahead, we expect
data from multiple pipeline programs, including additional data
supporting the potential for our IONIS-PKK-LRx program
to change the standard of care for patients with hereditary
angioedema. And by this fall, we expect data from the Phase 3 VALOR
study of tofersen in patients with SOD1-ALS. If results from the
VALOR study are positive, we expect tofersen to be our next
commercial medicine. These key recent achievements and upcoming
catalysts keep us on track for a regular cadence of Phase 3 data
and new drug applications, leading to 12 or more products on the
market in 2026."
Second Quarter 2021 and Recent Summary Financial
Results
- Second quarter results reflect focus on Ionis' strategic
objectives
-
- $126 million in total
revenues
- $154 million of operating
expenses on a non-GAAP basis(1) and $199 million on a GAAP basis
- Net loss of $36 million on a
non-GAAP basis(1) and $81
million on a GAAP basis
- Well capitalized with cash and investments of $2.1 billion at the end of the second
quarter
"In addition to advancing our pipeline and expanding our drug
discovery capabilities, we have taken multiple steps to streamline
our operations in support of our wholly owned medicines. We have
completed the integration of Akcea, entered distribution
arrangements with Sobi and restructured our commercial operations.
These steps enabled us to unlock significant resources that we are
redirecting towards our highest priority programs," said
Elizabeth L. Hougen, chief financial
officer of Ionis. "We remain on track to achieve our 2021 revenue
guidance of more than $600 million.
We continue to expect increased R&D revenue in the second half
of this year. Already in the third quarter, we earned $25 million from Novartis for the pelacarsen
enrollment milestone. We are revising our 2021 operating expense
and net loss guidance because of our license of Bicycle's
technology. Importantly, we remain well-capitalized with the
resources we need to achieve our strategic
objectives."
Revised 2021 Financial Guidance
- Updated operating expense and net loss guidance because of
Ionis' license of Bicycle's technology(1)
|
Prior 2021
Guidance
|
Revised 2021
Guidance
|
Revenue
|
>$600
million
|
Unchanged
|
Operating Expenses
(1)
|
$675 million to $725
million
|
$710 million to $750
million
|
Net Loss
(1)
|
<$75
million
|
<$110
million
|
|
|
(1)
|
All non-GAAP amounts
referred to in this press release exclude non-cash compensation
expense related to equity awards and expenses related to the Akcea
acquisition and restructured commercial operations and the related
tax effects. Please refer to the section below titled "Financial
Impacts of Akcea Acquisition and Restructured Commercial
Operations" for a summary of the costs specific to these
transactions. Additionally, please refer to the detailed
reconciliation of non-GAAP and GAAP measures, which is provided
later in this release.
|
Second Quarter 2021 Marketed Products
Highlights
- SPINRAZA®: The global market leader for the
treatment of spinal muscular atrophy (SMA) patients of all
ages
-
- $500 million in worldwide sales
in the second quarter
- More than 11,000 patients worldwide on therapy at the end of
the second quarter across commercial, expanded access and clinical
trial settings
- New data presented at CureSMA reinforce the potential for
higher-dose SPINRAZA to improve SMA patient outcomes and further
support SPINRAZA's potential long-term benefit for SMA patients of
all ages
- TEGSEDI® and WAYLIVRA®: important
medicines approved for the treatment of patients with severe rare
diseases
-
- Successfully completed the transition of North American TEGSEDI
operations to Swedish Orphan Biovitrum AB (Sobi)
Second Quarter 2021 and Recent Events
- Phase 3 Pipeline: Six Phase 3 studies on track for a regular
cadence of data readouts beginning this year
-
- Completed dosing in the Phase 3 VALOR study of tofersen in
patients with SOD1-ALS, with data expected by this fall
-
- Opened individual compassionate use access for SOD1-ALS
patients with the most rapidly progressive disease
- Achieved full enrollment in the Phase 3 NEURO-TTRansform study
of eplontersen in patients with TTR polyneuropathy, with data
expected by mid-2022
- Achieved 50 percent enrollment in the Phase 3 Lp(a) HORIZON
study of pelacarsen for patients at risk for Lp(a)-driven
cardiovascular disease, resulting in a $25
million payment from Novartis
- Advanced ION363 into a Phase 3 study in patients with
FUS-ALS
- Mid-stage Pipeline: multiple medicines with potential to change
the standard of care for patients with severe diseases
-
- Continued to advance the Phase 2b
RE-THINc ESRD study of IONIS-FXI-LRx, with data expected
in the first half of 2022
- Reported data from the Phase 1/2 study of
IONIS-MAPTRx in patients with Alzheimer's disease,
demonstrating durable, time and dose-dependent reductions in CSF
tau protein; IONIS-MAPTRx was generally well
tolerated
- Advanced the ongoing Phase 2 study of ION541 in patients with
ALS regardless of family history, resulting in a $10 million payment from Biogen
- Advanced ION224 into a Phase 2b
study in patients with non-alcoholic steatohepatitis (NASH)
- Advanced ION373 into the Phase 2 portion of a pivotal study in
patients with Alexander disease
- Strategic and Business Events
-
- Entered a license agreement with Bicycle Therapeutics for
exclusive rights to Bicycle's peptide technology to expand the
capabilities of Ionis' LICA technology
- Announced changes to the Ionis board of directors
-
- Joseph Loscalzo, M.D., Ph.D.,
appointed as chairman and Allene M.
Diaz as a member of the board
- Joseph Wender appointed as lead
independent director
- Ionis founder and executive chairman, Stanley T. Crooke M.D., Ph.D. and Breaux B. Castleman retired from the board
Upcoming 2021 Pipeline Catalysts(2)
Anticipated Key
2021 Data Readouts
|
Program
|
Phase
|
Anticipated
Indication
|
H1
|
H2
|
IONIS-PKK-LRx
|
2
|
Hereditary angioedema
(top-line data)
|
√
|
|
IONIS-AGT-LRx
|
2
|
Hypertension
|
√
|
|
Tominersen
|
3
|
Huntington's
disease
|
√
|
|
IONIS-ENAC-2.5Rx
|
2
|
Cystic
fibrosis
|
√
|
|
IONIS-MAPTRx
|
1/2
|
Alzheimer's
disease
|
|
√
|
Tofersen
|
3 (VALOR)
|
SOD1-ALS
|
|
•
|
Vupanorsen
|
2b
|
sHTG/CVD risk
reduction
|
|
•
|
IONIS-PKK-LRx
|
2
|
Hereditary angioedema
(full data)
|
|
•
|
IONIS-GHR-LRx
|
2 + OLE
|
Acromegaly
|
|
•
|
Anticipated Key
2021 Study Initiations
|
Program
|
Phase
|
Anticipated
Indication
|
H1
|
H2
|
SPINRAZA
|
4
(RESPOND)
|
SMA, suboptimal gene
therapy response
|
√
|
|
Tofersen
|
3 (ATLAS)
|
Presymptomatic
SOD1-ALS
|
√
|
|
ION363
|
3
|
FUS-ALS
|
√
|
|
IONIS-AGT-LRx
|
2b
|
Resistant
hypertension
|
√
|
|
IONIS-AGT-LRx
|
2
|
Heart failure with
reduced ejection fraction
|
√
|
|
ION373
|
2/3
|
Alexander
disease
|
√
|
|
ION224
|
2b
|
NASH
|
√
|
|
IONIS-APOCIII-LRx
|
3
|
Second TG indication
(sHTG)
|
|
•
|
ION582
|
2
|
Angelman
Syndrome
|
|
•
|
|
|
(2)
|
Timing of partnered
program catalysts based on partners' most recent publicly available
disclosures
|
Second Quarter 2021 Financial Results
Revenue
Ionis' revenue was comprised of the following (amounts in
millions):
|
|
Three months
ended,
|
|
Six months
ended
|
|
|
June 30,
|
|
June 30,
|
|
|
2021
|
|
2020
|
|
2021
|
|
2020
|
Revenue:
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$72
|
|
$72
|
|
$132
|
|
$138
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
12
|
|
16
|
|
31
|
|
32
|
Licensing and royalty
revenue
|
|
2
|
|
2
|
|
7
|
|
4
|
Total commercial
revenue
|
|
86
|
|
90
|
|
170
|
|
174
|
R&D
Revenue:
|
|
|
|
|
|
|
|
|
Amortization from
upfront payments
|
|
20
|
|
28
|
|
40
|
|
49
|
Milestone
payments
|
|
15
|
|
7
|
|
20
|
|
30
|
License
fees
|
|
-
|
|
15
|
|
-
|
|
15
|
Other
services
|
|
5
|
|
6
|
|
7
|
|
11
|
Total R&D
revenue
|
|
40
|
|
56
|
|
67
|
|
105
|
Total
revenue
|
|
$126
|
|
$146
|
|
$237
|
|
$279
|
In the second quarter of 2021, the Company successfully
completed the transition of its TEGSEDI operations in North America to Sobi. As a result, the
Company's commercial revenue from product sales shifted to
distribution fees based on net sales generated by Sobi.
The Company's R&D revenue decreased in the second quarter of
2021 compared to the same period last year primarily because the
Company earned more milestone payments in the second quarter of
2020 than the same period this year. The Company expects its
R&D revenue to increase in the second half of 2021 compared to
the first half as its partnered programs advance. Already in the
third quarter of 2021, the Company earned a $25 million milestone payment from Novartis when
Novartis achieved 50 percent enrollment in the Phase 3 Lp(a)
HORIZON study of pelacarsen.
Financial Impacts of Akcea Acquisition and Restructured
Commercial Operations
In the second quarter of 2021, the Company incurred $15 million of costs in conjunction with the
Akcea acquisition and restructuring of the Company's commercial
operations. The Company excluded these costs from its non-GAAP
amounts for the period. Refer to the detailed reconciliation of
non-GAAP and GAAP measures that is provided later in this
release.
Operating Expenses
Ionis' operating expenses for the second quarter of 2021
increased slightly compared to the same period last year driven by
an increase in R&D expenses, partially offset by a decrease in
SG&A expenses. Ionis' increased R&D expenses were primarily
driven by the Company's investments in advancing its late-stage
wholly owned pipeline. Ionis' decreased SG&A expenses were
primarily from operating efficiencies achieved from integrating
Akcea and restructuring the Company's commercial operations.
Net Loss Attributable to Ionis Common Stockholders
Ionis' net loss attributable to Ionis' common stockholders for
the second quarter of 2021 increased compared to the same period in
the prior year for the reasons discussed above. Additionally, the
Company recognized an $8.6 million
non-cash loss from the early retirement of a significant portion of
its 1 percent senior convertible notes.
Balance Sheet
Ionis ended June 2021 with cash,
cash equivalents and short-term investments of $2.1 billion, compared to $1.9 billion at December
31, 2020. In April 2021, Ionis
issued $632.5 million of
0 percent senior convertible notes due in April 2026 and repurchased $247.9 million of its 1 percent senior
convertible notes. The Company's remaining $62 million of 1 percent senior convertible
notes mature in November 2021.
The Company revised its 2020 amounts to reflect the simplified
convertible instruments guidance the Company adopted
retrospectively on January 1,
2021.
Webcast
Today, at 11:30 a.m. Eastern Time,
Ionis will conduct a live webcast to discuss this earnings release
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 neurological and
cardiometabolic franchises. Our scientific innovation began and
continues with the knowledge that sick people depend on us, which
fuels our vision of becoming one of the most successful
biotechnology companies.
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) and WAYLIVRA (volanesorsen) and
Ionis' technologies and 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, 2020, and the most
recent Form 10-Q quarterly filing, which are on file with the
SEC. 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" refers to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals™ is a 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,
|
|
Six months
ended,
|
|
|
June 30,
|
|
June 30,
|
|
|
2021
|
|
2020
|
|
2021
|
|
2020
|
|
|
(as
revised*)
|
|
|
|
(as
revised*)
|
|
|
(unaudited)
|
Revenue:
|
|
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$72
|
|
$72
|
|
$132
|
|
$138
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
12
|
|
16
|
|
31
|
|
32
|
Licensing and royalty
revenue
|
|
2
|
|
2
|
|
7
|
|
4
|
Total commercial
revenue
|
|
86
|
|
90
|
|
170
|
|
174
|
Research and development revenue under
collaborative agreements
|
|
40
|
|
56
|
|
67
|
|
105
|
Total
revenue
|
|
126
|
|
146
|
|
237
|
|
279
|
Expenses:
|
|
|
|
|
|
|
|
|
Cost of
sales
|
|
3
|
|
3
|
|
6
|
|
6
|
Research, development and patent
|
|
139
|
|
122
|
|
279
|
|
239
|
Selling, general and administrative
|
|
57
|
|
73
|
|
117
|
|
147
|
Total operating
expenses
|
|
199
|
|
198
|
|
402
|
|
392
|
Loss from
operations
|
|
(73)
|
|
(52)
|
|
(165)
|
|
(113)
|
|
|
|
|
|
|
|
|
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
Loss on early
retirement of debt
|
|
(8)
|
|
-
|
|
(8)
|
|
-
|
Other income,
net
|
|
-
|
|
17
|
|
3
|
|
25
|
Loss before income
tax (expense) benefit
|
|
(81)
|
|
(35)
|
|
(170)
|
|
(88)
|
|
|
|
|
|
|
|
|
|
Income tax (expense)
benefit
|
|
-
|
|
(3)
|
|
(1)
|
|
1
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
($81)
|
|
($38)
|
|
($171)
|
|
($87)
|
Net loss attributable
to noncontrolling interest in Akcea
Therapeutics, Inc.
|
|
-
|
|
12
|
|
-
|
|
22
|
Net loss attributable
to Ionis Pharmaceuticals, Inc.
common stockholders
|
|
($81)
|
|
($26)
|
|
($171)
|
|
($65)
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share
|
|
($0.57)
|
|
($0.18)
|
|
($1.21)
|
|
($0.47)
|
Shares used in
computing basic and diluted net loss per share
|
|
141
|
|
139
|
|
141
|
|
139
|
|
*The Company revised
its 2020 amounts to reflect the simplified convertible instruments
guidance the Company adopted retrospectively on January 1,
2021.
|
IONIS
PHARMACEUTICALS, INC.
|
Reconciliation of
GAAP to Non-GAAP Basis:
|
Condensed
Consolidated Operating Expenses, Loss From Operations, and Net
Income (Loss)
|
(In
Millions)
|
|
|
|
Three months
ended,
June 30,
|
|
Six months
ended,
June 30,
|
|
|
2021
|
|
2020
|
|
2021
|
|
2020
|
|
|
|
(as
revised*)
|
|
(as
revised*)
|
|
|
(unaudited)
|
As reported
research, development and patent
expenses according to GAAP
|
|
$139
|
|
$122
|
|
$279
|
|
$239
|
Excluding compensation expense related to equity
awards
|
|
(23)
|
|
(26)
|
|
(49)
|
|
(52)
|
Excluding Akcea acquisition and restructured commercial
operation costs
|
|
(4)
|
|
-
|
|
(6)
|
|
-
|
|
|
|
|
|
|
|
|
|
Non-GAAP research,
development and patent
expenses
|
|
$112
|
|
$96
|
|
$224
|
|
$187
|
As reported
selling, general and administrative
expenses according to GAAP
|
|
$57
|
|
$73
|
|
$117
|
|
$147
|
Excluding compensation expense related to equity
awards
|
|
(7)
|
|
(22)
|
|
(19)
|
|
(37)
|
Excluding Akcea acquisition and restructured commercial
operation costs
|
|
(11)
|
|
-
|
|
(16)
|
|
-
|
|
|
|
|
|
|
|
|
|
Non-GAAP selling,
general and administrative
expenses
|
|
$39
|
|
$51
|
|
$82
|
|
$110
|
As reported
operating expenses according to GAAP
|
|
$199
|
|
$198
|
|
$402
|
|
$392
|
Excluding compensation expense related to equity
awards
|
|
(30)
|
|
(48)
|
|
(68)
|
|
(89)
|
Excluding Akcea acquisition and restructured commercial
operation
costs
|
|
(15)
|
|
-
|
|
(22)
|
|
-
|
|
|
|
|
|
|
|
|
|
Non-GAAP operating
expenses
|
|
$154
|
|
$150
|
|
$312
|
|
$303
|
|
|
|
|
|
|
|
|
|
As reported loss
from operations according to GAAP
|
|
$(73)
|
|
$(52)
|
|
$(165)
|
|
($113)
|
Excluding compensation expense related to equity
awards
|
|
(30)
|
|
(48)
|
|
(68)
|
|
(89)
|
Excluding Akcea acquisition and restructured commercial
operation costs
|
|
(15)
|
|
-
|
|
(22)
|
|
-
|
Non-GAAP loss from
operations
|
|
$(28)
|
|
($4)
|
|
$(75)
|
|
($24)
|
As reported net
loss attributable to Ionis
Pharmaceuticals, Inc. common stockholders
according to GAAP
|
|
$(81)
|
|
$(26)
|
|
$(171)
|
|
($65)
|
Excluding compensation
expense related to equity awards
attributable to Ionis Pharmaceuticals,
Inc. common
stockholders
|
|
(30)
|
|
(45)
|
|
(68)
|
|
(84)
|
Excluding Akcea
acquisition and restructured commercial
operation costs
|
|
(15)
|
|
-
|
|
(22)
|
|
-
|
Income tax effect
related to compensation expense related
to equity awards attributable to Ionis
Pharmaceuticals,
Inc. common stockholders
|
|
-
|
|
4
|
|
-
|
|
12
|
Non-GAAP net
income (loss) attributable to Ionis
Pharmaceuticals, Inc. common stockholders
according to GAAP
|
|
$(36)
|
|
$15
|
|
$(81)
|
|
$7
|
|
*The Company revised
its 2020 amounts to reflect the simplified convertible instruments
guidance the Company adopted retrospectively on January 1,
2021.
|
Reconciliation of GAAP to Non-GAAP Basis
As illustrated in the Selected Financial Information in this
press release, non-GAAP operating expenses, non-GAAP income (loss)
from operations, and non-GAAP net income (loss) attributable to
Ionis Pharmaceuticals, Inc. common stockholders were adjusted from
GAAP to exclude compensation expense related to equity awards and
costs related to the Akcea acquisition and restructured commercial
operations and the related tax effects. Compensation expense
related to equity awards are non-cash. Costs related to the Akcea
acquisition and restructured commercial operations include:
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.
|
|
Condensed
Consolidated Balance Sheets
|
|
(In
Millions)
|
|
|
|
|
|
|
|
|
|
June 30,
|
|
December
31,
|
|
|
|
2021
|
|
2020
|
|
|
|
|
|
(as
revised*)
|
|
|
|
(unaudited)
|
|
Assets:
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$2,059
|
|
$1,892
|
|
Contracts
receivable
|
|
24
|
|
76
|
|
Other current
assets
|
|
155
|
|
162
|
|
Property,
plant and equipment, net
|
|
179
|
|
181
|
|
Other
assets
|
|
80
|
|
79
|
|
Total assets
|
|
$2,497
|
|
$2,390
|
|
|
|
|
|
|
|
Liabilities and
stockholders' equity:
|
|
|
|
|
|
Other current
liabilities
|
|
$130
|
|
$183
|
|
Current
portion of 1% convertible senior notes, net
|
|
62
|
|
309
|
|
Current
portion of deferred contract revenue
|
|
102
|
|
108
|
|
0% convertible
senior notes, net
|
|
617
|
|
-
|
|
0.125%
convertible senior notes, net
|
|
541
|
|
540
|
|
Long-term
obligations, less current portion
|
|
82
|
|
83
|
|
Long-term
deferred contract revenue
|
|
380
|
|
424
|
|
Total
stockholders' equity
|
|
583
|
|
743
|
|
Total liabilities and stockholders' equity
|
|
$2,497
|
|
$2,390
|
|
|
|
|
|
|
|
|
|
*The Company revised
its 2020 amounts to reflect the simplified convertible instruments
guidance the Company adopted retrospectively on January 1,
2021.
|
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SOURCE Ionis Pharmaceuticals, Inc.