CARLSBAD, Calif., May 5, 2021 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc. (Nasdaq: IONS) today reported its financial
results for the first quarter of 2021 and recent business
highlights.
"In the first quarter, we took important steps to maximize the
value of our wholly owned pipeline. We recently initiated pivotal
studies with our wholly owned FUS-ALS and Alexander disease
programs. We delivered positive results from our
IONIS-PKK-LRx program, demonstrating its potential to
change the standard of care for patients with hereditary
angioedema. We also further strengthened the business and continued
executing on our strategic priorities," said Brett P. Monia, Ph.D., chief executive officer
of Ionis. "This summer, we expect data from our
IONIS-MAPTRx program in Alzheimer's disease patients.
And later this year, we look forward to 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 upcoming catalysts, together with
our recent achievements, position us well to have 12 or more
products on the market in 2026."
First Quarter 2021 and Recent Summary Financial
Results
- On track to achieve 2021 financial guidance reflecting
investments in Ionis' wholly owned pipeline, based on the following
first quarter results
-
- $112 million in total
revenues
- $159 million of operating
expenses on a non-GAAP basis(1) and $204 million on a GAAP basis
- Net loss of $45 million on a
non-GAAP basis(1) and $90
million on a GAAP basis
- Further strengthened the Company's balance sheet with pro forma
cash of $2.1 billion, after
reflecting the convertible notes transaction
-
- Enables expansion of manufacturing and R&D capacity
- $632.5 million principal due in
April 2026 with 0% interest and an
effective conversion price of $76.39
after the purchase of a call spread
-
- Will realize interest expense savings while keeping potential
future dilution nearly flat
- Repurchased approximately 80% of previously outstanding 1%
convertible notes due in November
2021
"So far this year, we have taken important steps in support of
developing and commercializing our wholly owned medicines. In
addition to completing the restructuring of our European
operations, we expanded our Sobi distribution agreement to include
North America. These transactions
unlocked significant resources that we are now redirecting towards
our highest priority programs, including IONIS-TTR-LRx
and IONIS-APOCIII-LRx," said Elizabeth L. Hougen, chief financial officer of
Ionis. "We are on track to meet our 2021 financial guidance. In the
second half of this year, we expect R&D revenue to increase as
many of our partnered programs continue to advance. Importantly, we
are well-capitalized with the resources we need to expand our
manufacturing and R&D capacity to support the future needs of
our wholly owned pipeline. This large capital project, which is now
underway, is necessary to successfully execute on our goal to drive
growth."
(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 European operations and the related
tax effects. Please refer to the section below titled "Financial
Impacts of Akcea Acquisition and Restructured 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.
|
First Quarter 2021 Marketed Products Highlights
- SPINRAZA: a global foundation-of-care for the treatment of
spinal muscular atrophy (SMA) patients of all ages
-
- $521 million in worldwide sales
in the first quarter
- More than 11,000 patients worldwide were on therapy at the end
of the first quarter across post-marketing, expanded access and
clinical trial settings
- Higher-dose SPINRAZA demonstrated safety and tolerability
consistent with the currently approved dose in the open-label
safety cohort of the DEVOTE study, enabling enrollment in the
blinded, pivotal cohort to get underway
- TEGSEDI and WAYLIVRA: important medicines approved for the
treatment of patients with severe rare diseases
-
- Completed the transition of European operations to Swedish
Orphan Biovitrum AB (Sobi) and expanded the distribution agreement
to include North American TEGSEDI operations
First Quarter 2021 and Recent Pipeline Events
- Phase 3 Pipeline: growing and positioned for 12 or more
products on the market in 2026
-
- Advanced ION363 into a Phase 3 study in patients with
FUS-ALS
- Advanced tofersen into the Phase 3 ATLAS study in
presymptomatic SOD1-ALS patients
- Roche reported tominersen data related to the dosing halt in
the Phase 3 program
- Mid-stage Pipeline: advancing multiple medicines with potential
to change the standard of care for patients with severe
diseases
-
- Reported positive topline IONIS-PKK-LRx results in
patients with hereditary angioedema
- Advanced ION373 into the Phase 2 portion of a pivotal study in
patients with Alexander disease
- Advanced the IONIS-AGT-LRx development program:
-
- Reported positive Phase 2 data in JACC: Basic to
Translational Science
- Advanced into a Phase 2b study in
patients with hypertension uncontrolled with three or more
antihypertensive medications
- Advanced into a Phase 2 study in patients with chronic heart
failure with reduced injection fraction
- Advanced the ongoing Phase 2 study of ION541 in patients with
ALS regardless of family history, resulting in a $10 million payment from Biogen
Upcoming 2021 Pipeline Catalysts(2)
Anticipated 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-GHR-LRx
|
2+OLE
|
Acromegaly
|
|
•
|
IONIS-MAPTRx
|
1/2
|
Alzheimer's
disease
|
|
•
|
IONIS-PKK-LRx
|
2
|
Hereditary angioedema
(full data)
|
|
•
|
Vupanorsen
|
2b
|
sHTG/CVD risk
reduction
|
|
•
|
Tofersen
|
3 (VALOR
study)
|
SOD1-ALS
|
|
•
|
|
Anticipated 2021
Study Initiations
|
Program
|
Phase
|
Anticipated
Indication
|
H1
|
H2
|
SPINRAZA
|
4
(RESPOND)
|
SMA, suboptimal gene
therapy response
|
√
|
|
Tofersen
|
3 (ATLAS
study)
|
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)
|
|
•
|
|
(2) Timing of
partnered program catalysts based on partners' most recent publicly
available disclosures
|
First Quarter 2021 Financial Results
Revenue
Ionis' revenue was comprised of the following (amounts in
millions):
|
|
Three months
ended,
|
|
|
|
March 31,
|
|
|
|
2021
|
|
2020
|
|
Revenue:
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$60
|
|
$66
|
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
20
|
|
15
|
|
Licensing and royalty
revenue
|
|
5
|
|
3
|
|
Total commercial
revenue
|
|
85
|
|
84
|
|
R&D
Revenue:
|
|
|
|
|
|
Amortization from
upfront payments
|
|
20
|
|
21
|
|
Milestone
payments
|
|
5
|
|
23
|
|
Other
services
|
|
2
|
|
5
|
|
Total R&D
revenue
|
|
27
|
|
49
|
|
Total
revenue
|
|
$112
|
|
$133
|
|
The Company's commercial revenue in the first quarter of 2021
was consistent with the same period last year. As the Company
completes its transition of TEGSEDI operations in North America to Sobi, the Company's
commercial revenue from product sales will shift to distribution
fees based on net sales generated by Sobi.
The Company's R&D revenue decreased in the first quarter of
2021 compared to the same period last year primarily because the
Company earned more milestone payments in the first 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.
Financial Impacts of Akcea Acquisition and Restructured
Operations
In conjunction with the Akcea acquisition and restructured
European operations, in the first quarter of 2021, the Company
incurred $7 million of costs, which
it excluded 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. The Company expects to incur
additional expenses in the range of $11
million to $14 million related
to the restructuring of its North American TEGSEDI operations from
the expanded distribution agreement with Sobi. The company will
reflect the North American TEGSEDI restructuring costs primarily in
the second quarter of 2021.
Operating Expenses
Ionis' operating expenses for the first quarter of 2021
increased compared to the same period last year driven primarily by
the Company's investments in advancing its late-stage wholly owned
pipeline.
Net Loss Attributable to Ionis Common Stockholders
Ionis' net loss attributable to Ionis' common stockholders for
the first quarter of 2021 increased compared to the same period in
the prior year for the reasons discussed above.
Balance Sheet
Ionis ended March 2021 with cash,
cash equivalents and short-term investments of $1.8 billion, compared to $1.9 billion at December
31, 2020. In April 2021, Ionis
issued $632.5 million of 0% senior
convertible notes due in April 2026
and repurchased $247.9 million of its
1% senior convertible notes. After reflecting these transactions,
Ionis' pro forma cash, cash equivalents and short-term investments
was $2.1 billion.
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,
|
|
|
|
|
March 31,
|
|
|
|
|
2021
|
|
2020
|
|
|
|
|
|
(as
revised*)
|
|
|
|
|
(unaudited)
|
|
|
Revenue:
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
|
$60
|
|
$66
|
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
|
20
|
|
15
|
|
Licensing and royalty
revenue
|
|
|
5
|
|
3
|
|
Total commercial
revenue
|
|
|
85
|
|
84
|
|
Research and development revenue under collaborative
agreements
|
|
|
27
|
|
49
|
|
Total
revenue
|
|
|
112
|
|
133
|
|
Expenses:
|
|
|
|
|
|
|
Cost of
sales
|
|
|
3
|
|
3
|
|
Research, development and patent
|
|
|
140
|
|
116
|
|
Selling, general and administrative
|
|
|
61
|
|
75
|
|
Total operating
expenses
|
|
|
204
|
|
194
|
|
|
|
|
|
|
|
|
Loss from
operations
|
|
|
(92)
|
|
(61)
|
|
|
|
|
|
|
|
|
Other income,
net
|
|
|
2
|
|
8
|
|
Loss before income
tax benefit
|
|
|
(90)
|
|
(53)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Income tax
benefit
|
|
|
-
|
|
3
|
|
|
|
|
|
|
|
|
Net loss
|
|
|
($90)
|
|
($50)
|
|
Net loss attributable
to noncontrolling interest in Akcea Therapeutics, Inc.
|
|
|
-
|
|
10
|
|
Net loss attributable
to Ionis Pharmaceuticals, Inc. common stockholders
|
|
|
($90)
|
|
($40)
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share
|
|
|
($0.64)
|
|
($0.28)
|
|
Shares used in
computing basic and diluted net loss per share
|
|
|
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
Loss
|
(In
Millions)
|
|
|
|
Three months
ended,
March 31,
|
|
|
|
2021
|
|
2020
|
|
|
|
|
(as
revised*)
|
|
|
|
(unaudited)
|
|
|
|
|
|
As reported
research, development and patent expenses according to
GAAP
|
|
$140
|
|
$116
|
|
Excluding compensation expense related to equity
awards
|
|
(26)
|
|
(26)
|
|
Excluding Akcea acquisition and restructured European operations
costs
|
|
(3)
|
|
-
|
|
|
|
|
|
|
|
Non-GAAP research,
development and patent expenses
|
|
$111
|
|
$90
|
|
|
|
|
|
|
|
As reported
selling, general and administrative expenses according to
GAAP
|
|
$61
|
|
$75
|
|
Excluding compensation expense related to equity
awards
|
|
(12)
|
|
(15)
|
|
Excluding Akcea acquisition and restructured European operations
costs
|
|
(4)
|
|
-
|
|
|
|
|
|
|
|
Non-GAAP selling,
general and administrative expenses
|
|
$45
|
|
$60
|
|
|
|
|
|
|
|
As reported
operating expenses according to GAAP
|
|
$204
|
|
$194
|
|
Excluding compensation expense related to equity
awards
|
|
(38)
|
|
(41)
|
|
Excluding Akcea acquisition and restructured European operations
costs
|
|
(7)
|
|
-
|
|
|
|
|
|
|
|
Non-GAAP operating
expenses
|
|
$159
|
|
$153
|
|
|
|
|
|
|
|
As reported loss
from operations according to GAAP
|
|
($92)
|
|
($61)
|
|
Excluding compensation expense related to equity awards
|
|
(38)
|
|
(41)
|
|
Excluding Akcea acquisition and restructured European operations
costs
|
|
(7)
|
|
-
|
|
|
|
|
|
|
|
Non-GAAP loss from
operations
|
|
($47)
|
|
($20)
|
|
|
|
|
|
|
|
As reported net
loss attributable to Ionis Pharmaceuticals, Inc. common
stockholders according to GAAP*
|
|
($90)
|
|
($40)
|
|
Excluding compensation
expense related to equity awards attributable to Ionis
Pharmaceuticals, Inc. common stockholders
|
|
(38)
|
|
(39)
|
|
Excluding Akcea
acquisition and restructured European operations costs
|
|
(7)
|
|
-
|
|
Income tax effect
related to compensation expense related to equity awards
attributable to Ionis Pharmaceuticals, Inc. common
stockholders
|
|
-
|
|
8
|
|
Non-GAAP net loss
attributable to Ionis Pharmaceuticals, Inc. common
stockholders*
|
|
($45)
|
|
($9)
|
|
*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 European
operations and the related tax effects. Compensation expense
related to equity awards are non-cash. Costs related to the Akcea
acquisition and restructured European operations include:
severance costs, retention costs and other costs. 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)
|
|
|
|
|
|
|
|
|
March 31,
|
|
December
31,
|
|
|
|
2021
|
|
2020
|
|
|
|
|
|
(as
revised*)
|
|
|
|
(unaudited)
|
|
Assets:
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$1,820
|
|
$1,892
|
|
Contracts
receivable
|
|
23
|
|
76
|
|
Other current
assets
|
|
146
|
|
162
|
|
Property,
plant and equipment, net
|
|
180
|
|
181
|
|
Other
assets
|
|
80
|
|
79
|
|
Total assets
|
|
$2,249
|
|
$2,390
|
|
|
|
|
|
|
|
Liabilities and
stockholders' equity:
|
|
|
|
|
|
Other current
liabilities
|
|
$126
|
|
$183
|
|
Current
portion of 1% convertible senior notes, net
|
|
62
|
|
309
|
|
Current
portion of deferred contract revenue
|
|
107
|
|
108
|
|
1% convertible
senior notes, less current portion
|
|
247
|
|
-
|
|
0.125%
convertible senior notes, net
|
|
541
|
|
540
|
|
Long-term
obligations, less current portion
|
|
83
|
|
83
|
|
Long-term
deferred contract revenue
|
|
402
|
|
424
|
|
Total
stockholders' equity
|
|
681
|
|
743
|
|
Total liabilities and stockholders' equity
|
|
$2,249
|
|
$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.