Advanced key priorities: initiated
manufacturing infrastructure project to support growth
Increased 2022 cash and investments
guidance
CARLSBAD, Calif., Nov. 9, 2022
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the
"Company") today reported financial results for the third quarter
of 2022. Financial results are summarized below:
|
|
Three months
ended
September
30,
|
|
Nine months
ended
September
30,
|
|
|
2022
|
|
2021
|
|
2022
|
|
2021
|
|
|
(amounts in
millions)
|
Total
revenue
|
|
$160
|
|
$133
|
|
$435
|
|
$370
|
Operating
expenses
|
|
$219
|
|
$219
|
|
$637
|
|
$621
|
Operating expenses on a
non-GAAP basis
|
|
$195
|
|
$185
|
|
$562
|
|
$499
|
Net loss
|
|
($47)
|
|
($82)
|
|
($217)
|
|
($253)
|
Net loss on a non-GAAP
basis
|
|
($23)
|
|
($48)
|
|
($142)
|
|
($131)
|
Financial Highlights
- Revenue increased 20% for the third quarter of 2022 and 18% on
a year-to-date basis compared to the same periods last year driven
by significant partner payments earned across multiple
programs
- Non-GAAP operating expenses increased 5% for the third quarter
of 2022 and 13% on a year-to-date basis compared to the same
periods last year driven by advancing Phase 3 pipeline
- Entered into a long-term lease in October 2022 to construct a new manufacturing
facility supporting continued growth
- Entered into a sale-leaseback transaction in October 2022 for several real estate assets,
generating net proceeds of $240
million plus full funding to expand R&D campus
- Reaffirmed 2022 P&L guidance; increased cash and
investments guidance to approximately $2.0
billion
Late-Stage Pipeline Highlights
- Presented positive data from the Phase 3 NEURO-TTRansform study
of eplontersen in patients with polyneuropathy caused by hereditary
TTR amyloidosis; on track to file U.S. New Drug Application this
year
- Expanded enrollment in the Phase 3 CARDIO-TTRansform study of
eplontersen in patients with ATTR cardiomyopathy; data still
expected first half of 2025
- NDA for tofersen was accepted and granted priority review by
the FDA; Prescription Drug User Fee Act date of April 25, 2023
- Initiated CORE2, a confirmatory Phase 3 study of olezarsen in
patients with severe hypertriglyceridemia (SHTG)
- Initiated ESSENCE, a supporting Phase 3 study of olezarsen in
patients with SHTG or hypertriglyceridemia and cardiovascular
disease
Mid-Stage Pipeline Highlights
- GSK presented positive end of study data from the Phase
2b B-Clear study of bepirovirsen
demonstrating potential for functional cures in patients with
chronic hepatitis B; GSK plans to advance bepirovirsen into Phase 3
development in the first half of 2023
- Presented positive data from the Phase 2 study of
IONIS-FB-LRx in patients with immunoglobulin A
nephropathy; Roche plans to advance IONIS-FB-LRx into
Phase 3 development in the first half of 2023
- Bayer presented positive data from the Phase 2b study of fesomersen in patients with end-stage
renal disease; Ionis regained rights to fesomersen from Bayer and
is assessing next steps
- Roche presented the Phase 2 GENERATION HD2 study design of
tominersen in Huntington's disease patients; Roche plans to begin
enrollment in early 2023
- Reported ION449 (AZD8233) targeting PCSK9 met the primary
endpoint in Phase 2b SOLANO study in patients with
hypercholesterolemia; based on pre-specified efficacy criteria,
AstraZeneca is not advancing ION449
"We have made significant progress on our key priorities this
year, building our commercial pipeline, delivering an abundance of
new medicines to the market and expanding and diversifying our
technology. We delivered positive data from eight key programs,
positioning us to potentially add two new marketed medicines to our
portfolio and expand our rich Phase 3 pipeline to eight medicines
next year. Additionally, we have recently begun work on a
manufacturing facility to support our pipeline growth," said
Brett P. Monia, Ph.D., chief
executive officer of Ionis. "As we advance our near-term
opportunities, including filing the NDA this year for eplontersen,
and expanding our rich late-stage pipeline, we are well positioned
to drive increasing value for all stakeholders."
Third Quarter 2022 Financial Results
Revenue
Ionis' revenue was comprised of the following:
|
|
Three months
ended
|
|
Nine months
ended
|
|
|
|
September
30,
|
|
September
30,
|
|
|
|
2022
|
|
2021
|
|
2022
|
|
2021
|
|
Revenue:
|
|
(amounts in
millions)
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$62
|
|
$67
|
|
$175
|
|
$199
|
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
6
|
|
15
|
|
23
|
|
47
|
|
Licensing and royalty
revenue
|
|
5
|
|
3
|
|
25
|
|
9
|
|
Total commercial
revenue
|
|
73
|
|
85
|
|
223
|
|
255
|
|
Research and
development revenue:
|
|
|
|
|
|
|
|
|
|
Amortization from
upfront payments
|
|
18
|
|
17
|
|
54
|
|
57
|
|
Milestone
payments
|
|
15
|
|
28
|
|
60
|
|
48
|
|
License
fees
|
|
35
|
|
-
|
|
37
|
|
-
|
|
Other
services
|
|
1
|
|
3
|
|
6
|
|
10
|
|
Collaborative
agreement revenue
|
|
69
|
|
48
|
|
157
|
|
115
|
|
Eplontersen joint
development revenue
|
|
18
|
|
-
|
|
55
|
|
-
|
|
Total research and
development revenue
|
|
87
|
|
48
|
|
212
|
|
115
|
|
Total
revenue
|
|
$160
|
|
$133
|
|
$435
|
|
$370
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total revenue for the three and nine months ended September 30, 2022 increased 20 percent and 18
percent compared to the same periods last year, respectively. The
increase was driven by significant payments Ionis earned across
multiple partnered programs. R&D revenue for the nine months
ended September 30, 2022 included
$85 million from Biogen for advancing
several neurology disease programs, $63
million from Roche for licensing and advancing
IONIS-FB-LRx and $55
million from AstraZeneca for its share of the global Phase 3
development costs for eplontersen.
Commercial revenue for the three and nine months ended
September 30, 2022 decreased 15
percent and 13 percent compared to the same periods last year,
respectively. SPINRAZA royalties for the three and nine months
ended September 30, 2022 decreased 7
percent and 12 percent compared to the same periods last year,
respectively. In the U.S., SPINRAZA sales were flat in the first
nine months of 2022 compared to the same period last year. Outside
of the U.S. SPINRAZA royalties were lower due to lower SPINRAZA
product sales primarily due to decreased pricing, foreign currency
exchange and competition. TEGSEDI and WAYLIVRA revenue was also
lower due to the shift to distribution fees in 2021.
Operating Expenses
Ionis' non-GAAP operating expenses increased for the three and
nine months ended September 30, 2022
compared to the same periods in 2021, in line with expectations.
For both periods, higher R&D expenses were driven by the
expanded number of Phase 3 studies the Company is conducting, which
doubled from three to six studies in 2021. SG&A expenses
increased for the three months ended September 30, 2022 compared to the same period
last year driven by Ionis' go-to-market activities for eplontersen,
donidalorsen and olezarsen. SG&A expenses were lower for the
nine months ended September 30, 2022
compared to the same period last year largely due to the
substantial savings the Company achieved from integrating Akcea and
restructuring its commercial operations in 2021.
Balance Sheet
As of September 30, 2022, Ionis
had cash, cash equivalents and short-term investments of
$2.0 billion, compared with
$2.1 billion at December 31, 2021. Ionis' debt obligations and
working capital did not change significantly from December 31, 2021 to September 30, 2022.
In October 2022, Ionis entered
into a sale and leaseback transaction for several of its real
estate assets. Under the agreement, Ionis will receive net proceeds
of approximately $240 million plus
full funding to expand the Company's R&D campus. The net
proceeds reflect the Company's extinguishment of its mortgage debt
for the related properties. Ionis' fourth quarter financial results
will reflect the impact this transaction.
2022 Financial Guidance
The Company reaffirmed its full year 2022 guidance for total
revenue, operating expenses and net loss, on a non-GAAP basis. The
Company's 2022 operating expense guidance, compared to the prior
year, includes increasing R&D expenses between 25% and 30% and
consistent SG&A expenses. Ionis expects to recognize a
substantial gain on the sale of its real estate assets in the
fourth quarter. The gain will not impact the Company's non-GAAP
results since the sale was non-recurring and not part of the
Company's normal business operations.
The Company increased its full year 2022 cash and investments
guidance to approximately $2.0
billion from the previous guidance of $1.7 billion.
Full Year 2022
Guidance
|
|
Current
As of 3Q22
|
|
Previous
As of 2Q22
|
|
Revenue
|
|
>$575
million
|
|
>$575
million
|
|
Operating expenses on a
non-GAAP basis
|
|
$825-$850
million
|
|
$825-$850
million
|
|
Net loss on a non-GAAP
basis
|
|
<$275
million
|
|
<$275
million
|
|
Cash, cash equivalents
and short-term investments
|
|
~$2.0
billion
|
|
~$1.7
billion
|
|
"Our strong year-to-date results, including year-over-year
revenue growth of nearly 20 percent, keep us on track to meet our
2022 P&L guidance," said Elizabeth L.
Hougen, chief financial officer of Ionis. "Additionally, we
recently bolstered our balance sheet when we unlocked net proceeds
of approximately $240 million plus
full funding to expand our R&D campus by capitalizing on the
favorable life sciences real estate market and monetizing several
of our facilities through a sale and leaseback transaction. As a
result, we are increasing our 2022 cash and short-term investment
guidance to approximately $2
billion."
Webcast
Management will host a conference call and webcast to discuss
Ionis' third quarter 2022 results at 11:30
a.m. Eastern time on Wednesday, November 9, 2022. 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 third quarter 2022 earnings slides click here.
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, 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. 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,
|
|
|
2022
|
|
2021
|
|
2022
|
|
2021
|
|
|
(unaudited)
|
Revenue:
|
|
|
|
|
|
|
|
|
Commercial
revenue:
|
|
|
|
|
|
|
|
|
SPINRAZA
royalties
|
|
$62
|
|
$67
|
|
$175
|
|
$199
|
TEGSEDI and WAYLIVRA
revenue, net
|
|
6
|
|
15
|
|
23
|
|
47
|
Licensing and royalty
revenue
|
|
5
|
|
3
|
|
25
|
|
9
|
Total commercial
revenue
|
|
73
|
|
85
|
|
223
|
|
255
|
Research and
development revenue:
|
|
|
|
|
|
|
|
|
Collaborative
agreement revenue
|
|
69
|
|
48
|
|
157
|
|
115
|
Eplontersen joint
development revenue
|
|
18
|
|
-
|
|
55
|
|
-
|
Total research and
development revenue
|
|
87
|
|
48
|
|
212
|
|
115
|
Total
revenue
|
|
160
|
|
133
|
|
435
|
|
370
|
Expenses:
|
|
|
|
|
|
|
|
|
Cost of
sales
|
|
2
|
|
3
|
|
10
|
|
9
|
Research, development and patent
|
|
183
|
|
185
|
|
525
|
|
464
|
Selling, general and administrative
|
|
34
|
|
31
|
|
102
|
|
148
|
Total operating
expenses
|
|
219
|
|
219
|
|
637
|
|
621
|
Loss from
operations
|
|
(59)
|
|
(86)
|
|
(202)
|
|
(251)
|
|
|
|
|
|
|
|
|
|
Other expense
(income)
|
|
12
|
|
2
|
|
(12)
|
|
(3)
|
Loss before income tax
benefit (expense)
|
|
(47)
|
|
(84)
|
|
(214)
|
|
(254)
|
|
|
|
|
|
|
|
|
|
Income tax benefit
(expense)
|
|
-
|
|
2
|
|
(3)
|
|
1
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
($47)
|
|
($82)
|
|
($217)
|
|
($253)
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share
|
|
($0.33)
|
|
($0.58)
|
|
($1.53)
|
|
($1.80)
|
Shares used in
computing basic and diluted net loss per share
|
|
142
|
|
141
|
|
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
September
30,
|
|
Nine months
ended
September
30,
|
|
|
2022
|
|
2021
|
|
2022
|
|
2021
|
|
|
(unaudited)
|
As reported
research, development and patent expenses according to
GAAP
|
|
$183
|
|
$185
|
|
$525
|
|
$464
|
Excluding compensation expense related to equity
awards
|
|
(18)
|
|
(23)
|
|
(55)
|
|
(72)
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
(2)
|
|
-
|
|
(8)
|
Non-GAAP research,
development and patent expenses
|
|
$165
|
|
$160
|
|
$470
|
|
$384
|
|
|
|
|
|
|
|
|
|
As reported selling,
general and administrative expenses according to
GAAP
|
|
$34
|
|
$31
|
|
$102
|
|
$148
|
Excluding compensation expense related to equity
awards
|
|
(6)
|
|
(7)
|
|
(19)
|
|
(26)
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
(1)
|
|
-
|
|
(16)
|
Non-GAAP selling,
general and administrative expenses
|
|
$28
|
|
$23
|
|
$83
|
|
$106
|
|
|
|
|
|
|
|
|
|
As reported
operating expenses according to GAAP
|
|
$219
|
|
$219
|
|
$637
|
|
$621
|
Excluding compensation
expense related to equity
awards
|
|
(24)
|
|
(31)
|
|
(75)
|
|
(98)
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
(3)
|
|
-
|
|
(24)
|
Non-GAAP operating
expenses
|
|
$195
|
|
$185
|
|
$562
|
|
$499
|
|
|
|
|
|
|
|
|
|
As reported loss
from operations according to GAAP
|
|
($59)
|
|
($86)
|
|
($202)
|
|
($251)
|
Excluding compensation expense related to equity
awards
|
|
(24)
|
|
(31)
|
|
(75)
|
|
(98)
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
(3)
|
|
-
|
|
(24)
|
Non-GAAP loss from
operations
|
|
($35)
|
|
($52)
|
|
($127)
|
|
($129)
|
|
|
|
|
|
|
|
|
|
As reported net loss
according to GAAP
|
|
($47)
|
|
($82)
|
|
($217)
|
|
($253)
|
Excluding compensation expense related to equity
awards
|
|
(24)
|
|
(31)
|
|
(75)
|
|
(98)
|
Excluding Akcea merger and restructured commercial operation
costs*
|
|
-
|
|
(3)
|
|
-
|
|
(24)
|
Non-GAAP net
loss
|
|
($23)
|
|
($48)
|
|
($142)
|
|
($131)
|
|
*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 the Akcea merger
and restructured commercial operation 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 the
related tax effects. Compensation expense related to equity awards
are non-cash. In 2021 all non-GAAP amounts also excluded expenses
related to the Akcea merger and restructured commercial operations.
Expenses 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 Nine Months
Ended, September 30, 2022
|
(Unaudited)
|
|
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)
|
|
$55M
|
|
55% of Total Phase
3
development expenses,
including
internal+external costs &
CMC costs, net of Ionis'
share of AstraZeneca's
Phase 3 development
expenses
|
|
|
|
|
|
|
|
Development
Expenses
(R&D
Expenses)
|
|
$107M
|
|
100% of Ionis' Phase
3
development expenses
|
Ionis' financial results for the nine months ended September 30, 2022 reflected the cost-sharing
provisions related to its 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, net of Ionis'
share of AstraZeneca's development expenses, as R&D revenue in
the same period Ionis incurs the related development expenses. For
the nine months ended September 30,
2022 Ionis earned $55 million
in joint development revenue under this collaboration.
Because 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). For the nine months ended September 30, 2022 Ionis recognized $1.4 million and $1.5
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)
|
|
|
|
|
|
|
|
|
|
September
30,
|
|
December 31,
|
|
|
|
2022
|
|
2021
|
|
|
|
(unaudited)
|
|
|
|
Assets:
|
|
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$1,982
|
|
$2,115
|
|
Contracts
receivable
|
|
7
|
|
62
|
|
Other current
assets
|
|
164
|
|
168
|
|
Property, plant
and equipment, net
|
|
181
|
|
178
|
|
Other
assets
|
|
88
|
|
89
|
|
Total assets
|
|
$2,422
|
|
$2,612
|
|
|
|
|
|
|
|
Liabilities and
stockholders' equity:
|
|
|
|
|
|
Other current
liabilities
|
|
$184
|
|
$143
|
|
Current portion
of deferred contract revenue
|
|
100
|
|
98
|
|
0% convertible
senior notes, net
|
|
621
|
|
619
|
0.125%
convertible senior notes, net
|
|
544
|
|
542
|
|
Long-term
obligations, less current portion
|
|
84
|
|
86
|
|
Long-term
deferred contract revenue
|
|
295
|
|
352
|
|
Total
stockholders' equity
|
|
594
|
|
772
|
|
Total liabilities and stockholders' equity
|
|
$2,422
|
|
$2,612
|
|
|
|
|
|
|
|
|
2022 Pipeline Milestones(1)
Anticipated 2022
Regulatory Updates
|
Program
|
Regulatory
Action
|
Anticipated
Indication
|
H1
|
H2
|
Tofersen
|
NDA
acceptance
|
SOD1-ALS
|
|
✓
|
Eplontersen
(TTR)
|
NDA filing
|
ATTRv
polyneuropathy
|
|
•
|
|
Anticipated Key 2022
Data Readouts
|
Program
|
Data
Readout
|
Anticipated
Indication
|
H1
|
H2
|
Eplontersen
(TTR)
|
Phase 3
|
ATTRv
polyneuropathy
|
✓
|
|
Tofersen
|
Phase 3 OLE
|
SOD1-ALS
|
✓
|
|
Tominersen
(HTT)
|
Phase 3 post
hoc
|
Huntington's
disease
|
✓
|
|
ION449
(PCSK9)
|
Phase 2b
(ETESIAN)
|
Cardiovascular
disease
|
✓
|
|
Bepirovirsen
(HBV)
|
Phase 2b
|
Hepatitis B virus
infection
|
✓
|
|
Donidalorsen
(PKK)
|
Phase 2
|
HAE
|
✓
|
|
IONIS-C9Rx
(BIIB078)
|
Phase 1/2
|
C9-ALS
|
✓
|
|
Fesomersen
(FXI)
|
Phase 2b
|
Thrombosis
|
|
✓
|
IONIS-FB-LRx
|
Phase 2
|
Immunoglobulin A
nephropathy
|
|
✓
|
ION449
(PCSK9)
|
Phase 2b
(SOLANO)
|
Cardiovascular
disease
|
|
✓
|
Donidalorsen
(PKK)
|
Phase 2 OLE
|
HAE
|
|
•
|
IONIS-AGT-LRx
|
Phase 2b
|
Treatment-resistant
hypertension
|
|
•
|
Cimdelirsen
(GHR)
|
Phase 2
(monotherapy)
|
Acromegaly
|
|
•
|
|
Anticipated Key 2022
Study Initiations
|
Program
|
Phase
|
Anticipated
Indication
|
H1
|
H2
|
Sapablursen
(TMPRSS6)
|
2
|
Polycythemia
vera
|
✓
|
|
ION904 (AGT)
|
2
|
Uncontrolled
hypertension
|
✓
|
|
IONIS-MAPTRx
(BIIB080)
|
2
|
Alzheimer's
disease
|
|
•
|
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)
Timing expectations based on current assumptions and subject to
change.
|
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SOURCE Ionis Pharmaceuticals, Inc.