CARLSBAD, Calif., Nov. 3, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (Nasdaq: IONS) today reported financial
results for the three and nine months ended September 30, 2021 and recent business
achievements.
"Among recent business highlights, we further expanded our broad
late-stage pipeline to seven Phase 3 programs now that the
olezarsen Phase 3 CORE study in patients with severe
hypertriglyceridemia is underway. We were encouraged that while
tofersen did not achieve the primary endpoint in the Phase 3 VALOR
study, trends favoring tofersen were seen across multiple secondary
and exploratory measures of disease progression in patients with
SOD1-ALS. We also expanded our LICA platform capabilities by
licensing technology from Bicycle Therapeutics," said Brett P. Monia, Ph.D., chief executive officer
of Ionis. "We are looking forward to multiple near-term catalysts,
beginning with the presentation of donidalorsen Phase 2 data in
patients with hereditary angioedema at the ACAAI Annual Meeting
this weekend. We anticipate initiating the donidalorsen Phase 3
study before year-end. We also expect multiple catalysts in 2022,
including results from the eplontersen Phase 3 study in patients
with TTR polyneuropathy mid-year 2022. Furthermore, at our virtual
investor day on December
9th, we look forward to outlining commercial
preparations for our lead programs ahead of our first potential
launch with eplontersen in patients with TTR polyneuropathy in
2023. Based on our anticipated near- and mid-term catalysts, we
remain on track to have 12 or more products on the market in
2026."
Third Quarter 2021 Financial Results
- Third quarter results reflect Ionis' focus on its strategic
objectives
-
- $133 million in total
revenues
- $185 million of operating
expenses on a non-GAAP basis(1) and $219 million on a GAAP basis
- Net loss of $48 million on a
non-GAAP basis(1) and $82
million on a GAAP basis
- Well capitalized with cash and investments of $2 billion as of September
30, 2021
"Since our last quarterly update, we further advanced our
strategic objectives through investments in our expanding Phase 3
pipeline and technology. We also advanced our commercial readiness
initiatives in anticipation of multiple product launches
potentially beginning as early as 2023," said Elizabeth L. Hougen, chief financial officer of
Ionis. "We remain on track to achieve our 2021 financial guidance
driven by increased R&D revenue in the fourth quarter as
several of our partner programs advance. We also project increased
expenses in the fourth quarter as we continue to invest for growth.
Importantly, with $2 billion in cash,
we remain well-capitalized with the resources we need to achieve
our strategic objectives."
(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
Merger and restructured commercial operations and the related tax
effects. Please refer to the section below titled "Financial
Impacts of Akcea Merger 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 press
release.
|
Third Quarter 2021 Marketed Products Highlights
- SPINRAZA®: the global market leader for the
treatment of spinal muscular atrophy (SMA) patients of all
ages
-
- $444 million in worldwide sales
in the third quarter
- More than 11,000 patients worldwide on therapy at the end of
the third quarter across commercial, expanded access and clinical
trial settings
- Biogen plans to initiate the Phase 3b ASCEND study evaluating the potential benefit
of an investigational higher dose of nusinersen in children, teens
and adults with later-onset SMA previously treated with
Evrysdi® (risdiplam)
- TEGSEDI® and WAYLIVRA®: important
medicines approved for the treatment of patients with severe rare
diseases
-
- TEGSEDI achieved innovative drug pricing in Brazil reflecting the significant unmet
medical need and prevalence of TTR polyneuropathy in Brazil
- WAYLIVRA was approved in Brazil as the first and only treatment for
patients with familial chylomicronemia syndrome
Third Quarter 2021 and Recent Events
- Advancing Ionis' leading cardiovascular and metabolic disease
pipeline
-
- Initiated the Phase 3 CORE study of olezarsen
(IONIS-APOCIII-LRx) in patients with severe
hypertriglyceridemia (sHTG)
- Reached 50 percent enrollment in the Phase 3 Lp(a) HORIZON
outcome study of pelacarsen for patients with established
cardiovascular disease and elevated Lp(a), resulting in a
$25 million payment from
Novartis
- Achieved full enrollment in the Bayer Phase 2b RE-THINc ESRD study of fesomersen
(IONIS-FXI-LRx), with data expected in the first half of
2022
- Achieved proof-of-mechanism, a strong indication of
proof-of-concept and good safety and tolerability in a Phase 2
study and a preliminary assessment from an open-label extension
study of cimdelirsen (IONIS-GHR-LRx) in acromegaly
patients uncontrolled on standard of care therapy, supporting
continued development. Data from the ongoing open-label extension
study and monotherapy study are expected in 2022. The results from
the Phase 2 study of cimdelirsen are posted to Ionis' website and
may be accessed here
- Addressing substantial unmet medical need with Ionis' broad
neurological disease pipeline
-
- The Biogen Phase 3 VALOR study of tofersen in patients
with SOD1-ALS did not meet the primary endpoint of change
from baseline to week 28 in the ALS Functional Rating Scale-Revised
(ALSFRS-R); however, signs of reduced disease progression across
multiple secondary and exploratory endpoints were observed
- Achieved full enrollment in the Phase 3 NEURO-TTRansform study
of eplontersen in patients with TTR polyneuropathy, with data
expected in mid-2022
- Reported data from the Biogen 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
- Investing in expanding the reach of Ionis' technology
-
- Entered a license agreement with Bicycle Therapeutics for
exclusive rights to Bicycle's peptide technology targeting
transferrin receptor 1 to expand the capabilities of Ionis' LICA
technology
- Entered a license agreement with Flamingo Therapeutics for the
development and commercialization of programs from Ionis' oncology
pipeline
2021 Pipeline Milestones(2)
Anticipated Key
2021 Data Readouts
|
Program
|
Phase
|
Anticipated
Indication
|
H1
|
H2
|
Donidalorsen
|
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
|
|
√
|
Cimdelirsen
|
2 + OLE
|
Acromegaly
|
|
√
|
Donidalorsen
|
2
|
Hereditary angioedema
(full data)
|
|
•
|
ION449
(PCSK9)
|
1 (MAD)
|
Dyslipidemia
|
|
•
|
Vupanorsen
|
2b
|
sHTG/CVD risk
reduction
|
|
•
|
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
|
2 & 2b
|
Heart failure &
resistant hypertension
|
√
|
|
ION373
|
2/3
|
Alexander
disease
|
√
|
|
ION224
|
2b
|
NASH
|
√
|
|
Olezarsen
|
3 (CORE)
|
Severe
hypertriglyceridemia (sHTG)
|
|
√
|
Donidalorsen
|
3
|
Hereditary
angioedema
|
|
•
|
SPINRAZA
|
3b
(ASCEND)
|
SMA, previous
risdiplam treatment
|
|
•
|
ION582
|
2
|
Angelman
syndrome
|
|
•
|
|
√ =
achieved • = planned
|
|
(2) Timing of
partnered program catalysts based on partners' most recent publicly
available disclosures
|
Third Quarter 2021 Financial Results
Revenue
Ionis' revenue was comprised of the following (amounts in
millions):
|
|
Three months
ended
|
|
Nine months
ended
|
|
|
September
30,
|
|
September
30,
|
|
|
2021
|
|
2020
|
|
2021
|
|
2020
|
Revenue:
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$67
|
|
$74
|
|
$199
|
|
$212
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
15
|
|
19
|
|
47
|
|
51
|
Licensing and royalty
revenue
|
|
3
|
|
2
|
|
9
|
|
6
|
Total commercial
revenue
|
|
85
|
|
95
|
|
255
|
|
269
|
R&D
revenue:
|
|
|
|
|
|
|
|
|
Amortization from
upfront payments
|
|
17
|
|
19
|
|
57
|
|
68
|
Milestone
payments
|
|
28
|
|
44
|
|
48
|
|
73
|
License
fees
|
|
-
|
|
-
|
|
-
|
|
15
|
Other
services
|
|
3
|
|
2
|
|
10
|
|
14
|
Total R&D
revenue
|
|
48
|
|
65
|
|
115
|
|
170
|
Total
revenue
|
|
$133
|
|
$160
|
|
$370
|
|
$439
|
In the third quarter of 2021, the Company continued to advance
its late-stage pipeline, including reaching 50 percent
enrollment in the Phase 3 Lp(a) HORIZON study of pelacarsen for
which it earned a $25 million
milestone payment from Novartis. As its partnered programs advance,
the Company expects R&D revenue to increase in the fourth
quarter of 2021 compared with the third quarter of 2021.
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. In the
third quarter of 2021, the Company earned a $4 million milestone payment from PTC
Therapeutics when WAYLIVRA was approved in Brazil.
Financial Impacts of Akcea Merger and Restructured Commercial
Operations
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. For the three and nine months ended September 30, 2021, the Company incurred
$3 million and $24 million of costs in conjunction with the
Akcea merger and restructuring of the Company's commercial
operations, respectively. The Company excluded these costs
from its non-GAAP amounts for those periods. Please refer to the
detailed reconciliation of non-GAAP and GAAP measures that is
provided later in this press release.
Operating Expenses
Ionis' operating expenses for the three and nine months ended
September 30, 2021 increased compared
with the same periods last year driven by an increase in R&D
expenses, partially offset by a decrease in SG&A expenses.
Higher R&D expenses were primarily driven by the Company's
investments in advancing its late-stage wholly owned pipeline,
including advancing the Phase 3 program for eplontersen and
start-up costs associated with the Phase 3 study for a second
indication for olezarsen. Additionally, the Company recognized
$35 million in R&D expense in the
third quarter of 2021 for licensing Bicycle's technology. Lower
SG&A expenses primarily reflect operating efficiencies achieved
from integrating Akcea and restructuring the Company's commercial
operations. The Company projects its operating expenses to increase
in the fourth quarter as it continues to invest for growth.
Net Loss Attributable to Ionis Common Stockholders
Net loss attributable to Ionis' common stockholders for the
three and nine months ended September 30,
2021 increased compared with the same periods in the prior
year for the reasons discussed above.
Balance Sheet
As of September 30, 2021, Ionis
had cash, cash equivalents and short-term investments of
$2.0 billion, compared with
$1.9 billion as of December 31, 2020. The Company intends to utilize
$62 million of its cash to pay the
remaining principal balance of its 1 percent convertible notes at
maturity 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
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 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" all refer 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,
|
|
Nine months
ended
|
|
|
September
30,
|
|
September
30,
|
|
|
2021
|
|
2020
|
|
2021
|
|
2020
|
|
|
(as
revised*)
|
|
(as
revised*)
|
|
|
(unaudited)
|
Revenue:
|
|
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$67
|
|
$74
|
|
$199
|
|
$212
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
15
|
|
19
|
|
47
|
|
51
|
Licensing and royalty
revenue
|
|
3
|
|
2
|
|
9
|
|
6
|
Total commercial
revenue
|
|
85
|
|
95
|
|
255
|
|
269
|
Research and development revenue under
collaborative agreements
|
|
48
|
|
65
|
|
115
|
|
170
|
Total
revenue
|
|
133
|
|
160
|
|
370
|
|
439
|
Expenses:
|
|
|
|
|
|
|
|
|
Cost of
sales
|
|
3
|
|
3
|
|
9
|
|
9
|
Research, development and patent
|
|
185
|
|
125
|
|
464
|
|
364
|
Selling, general and administrative
|
|
31
|
|
69
|
|
148
|
|
215
|
Total operating
expenses
|
|
219
|
|
197
|
|
621
|
|
588
|
Loss from
operations
|
|
(86)
|
|
(37)
|
|
(251)
|
|
(149)
|
|
|
|
|
|
|
|
|
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
Loss on early
retirement of debt
|
|
-
|
|
-
|
|
(9)
|
|
-
|
Other income,
net
|
|
2
|
|
5
|
|
6
|
|
29
|
Loss before income
tax benefit (expense)
|
|
(84)
|
|
(32)
|
|
(254)
|
|
(120)
|
|
|
|
|
|
|
|
|
|
Income tax benefit
(expense)
|
|
2
|
|
(5)
|
|
1
|
|
(4)
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
($82)
|
|
($37)
|
|
($253)
|
|
($124)
|
Net loss attributable
to noncontrolling interest in Akcea
Therapeutics, Inc.
|
|
$-
|
|
$13
|
|
$-
|
|
$34
|
Net loss attributable
to Ionis Pharmaceuticals, Inc.
common stockholders
|
|
($82)
|
|
($24)
|
|
($253)
|
|
($90)
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share
|
|
($0.58)
|
|
($0.18)
|
|
($1.80)
|
|
($0.64)
|
Shares used in
computing basic and diluted net loss
per share
|
|
141
|
|
140
|
|
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, Income (Loss) From Operations, and
Net Income (Loss)
|
(In
Millions)
|
|
|
|
Three months
ended
September
30,
|
|
Nine months
ended
September
30,
|
|
|
2021
|
|
2020
|
|
2021
|
|
2020
|
|
|
|
(as
revised*)
|
|
(as
revised*)
|
|
|
(unaudited)
|
As reported
research, development and patent
expenses according to GAAP
|
|
$185
|
|
$125
|
|
$464
|
|
$364
|
Excluding compensation expense related to equity
awards
|
|
(23)
|
|
(25)
|
|
(72)
|
|
(77)
|
Excluding Akcea merger and restructured commercial
operation
costs**
|
|
(2)
|
|
-
|
|
(8)
|
|
-
|
Non-GAAP research,
development and patent
expenses
|
|
$160
|
|
$100
|
|
$384
|
|
$287
|
|
As reported
selling, general and administrative
expenses according to GAAP
|
|
$31
|
|
$69
|
|
$148
|
|
$215
|
Excluding compensation expense related to equity
awards
|
|
(7)
|
|
(20)
|
|
(26)
|
|
(57)
|
Excluding Akcea merger and restructured commercial
operation
costs**
|
|
(1)
|
|
-
|
|
(16)
|
|
-
|
Non-GAAP selling,
general and administrative
expenses
|
|
$23
|
|
$49
|
|
$106
|
|
$158
|
|
As reported
operating expenses according to GAAP
|
|
$219
|
|
$197
|
|
$621
|
|
$588
|
Excluding compensation expense related to equity
awards
|
|
(31)
|
|
(46)
|
|
(98)
|
|
(135)
|
Excluding Akcea merger and restructured commercial
operation
costs**
|
|
(3)
|
|
-
|
|
(24)
|
|
-
|
Non-GAAP operating
expenses
|
|
$185
|
|
$151
|
|
$499
|
|
$453
|
|
|
|
|
|
|
|
|
|
As reported loss
from operations according to GAAP
|
|
($86)
|
|
($37)
|
|
($251)
|
|
($149)
|
Excluding compensation expense related to equity
awards
|
|
(31)
|
|
(46)
|
|
(98)
|
|
(135)
|
Excluding Akcea merger and restructured commercial
operation costs**
|
|
(3)
|
|
-
|
|
(24)
|
|
-
|
Non-GAAP income
(loss) from operations
|
|
($52)
|
|
$9
|
|
$(129)
|
|
($14)
|
|
As reported net
loss attributable to Ionis
Pharmaceuticals, Inc. common stockholders
according to GAAP
|
|
($82)
|
|
($24)
|
|
($253)
|
|
($90)
|
Excluding compensation
expense related to equity awards
attributable to Ionis Pharmaceuticals, Inc. common
stockholders
|
|
(31)
|
|
(42)
|
|
(98)
|
|
(126)
|
Excluding Akcea merger
and restructured commercial
operation costs**
|
|
(3)
|
|
-
|
|
(24)
|
|
-
|
Income tax effect
related to compensation expense related
to equity awards attributable to Ionis Pharmaceuticals,
Inc. common stockholders
|
|
-
|
|
6
|
|
-
|
|
18
|
Non-GAAP net
income (loss) attributable to Ionis
Pharmaceuticals, Inc. common stockholders
according to GAAP
|
|
($48)
|
|
$12
|
|
($131)
|
|
$18
|
|
* The Company revised
its 2020 amounts to reflect the simplified convertible instruments
guidance the Company adopted retrospectively on January 1,
2021.
|
|
** 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.
|
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 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 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)
|
|
|
|
|
|
|
|
|
September
30,
|
|
December
31,
|
|
|
|
2021
|
|
2020
|
|
|
|
|
|
(as
revised*)
|
|
|
|
(unaudited)
|
|
Assets:
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$1,987
|
|
$1,892
|
|
Contracts
receivable
|
|
9
|
|
76
|
|
Other current
assets
|
|
160
|
|
162
|
|
Property,
plant and equipment, net
|
|
180
|
|
181
|
|
Other
assets
|
|
79
|
|
79
|
|
Total assets
|
|
$2,415
|
|
$2,390
|
|
|
|
|
|
|
|
Liabilities and
stockholders' equity:
|
|
|
|
|
|
Other current
liabilities
|
|
$120
|
|
$183
|
|
Current
portion of 1% convertible senior notes, net
|
|
62
|
|
309
|
|
Current
portion of deferred contract revenue
|
|
98
|
|
108
|
|
0% convertible
senior notes, net
|
|
618
|
|
-
|
|
0.125%
convertible senior notes, net
|
|
542
|
|
540
|
|
Long-term
obligations, less current portion
|
|
81
|
|
83
|
|
Long-term
deferred contract revenue
|
|
363
|
|
424
|
|
Total
stockholders' equity
|
|
531
|
|
743
|
|
Total liabilities and stockholders' equity
|
|
$2,415
|
|
$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.