Ionis Pharmaceuticals Inc 8-K, filed Nov 9, 2022
Ionis Pharmaceuticals Inc (IONS)
8-K
2022-11-09
For: 2022-11-09
View Original
Added on
April 07, 2026
About this 8-K
Ionis Pharmaceuticals Inc filed this 8-K on November 9, 2022.
Items reported
- Item 2.02: Financial results
- Item 9.01: Financial statements and exhibits
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
PURSUANT TO SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of report (Date of earliest event reported): November 9, 2022
(Exact Name of Registrant as Specified in Charter)
(State or Other Jurisdiction of Incorporation)
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(Commission File No.)
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(IRS Employer Identification No.)
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(Address of Principal Executive Offices and Zip Code)
Registrant’s telephone number, including area code: (760 ) 931-9200
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
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Securities registered pursuant to Section 12(b) of the Act:
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Title of each class
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Trading symbol
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Name of each exchange on which registered
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“
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (Section 230.405 of this chapter) or
Rule 12b-2 of the Securities Exchange Act of 1934 (Section 240.12b-2 of this chapter).
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Emerging growth company
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If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised
financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
| Item 2.02 |
Results of Operations and Financial Condition.
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On November 9, 2022, Ionis Pharmaceuticals, Inc. (the “Company”) issued a press release announcing the
Company’s financial results for the quarter ended September 30, 2022. In addition to disclosing results that are determined in accordance with Generally Accepted Accounting Principles (“GAAP”),
the Company also discloses pro forma or non-GAAP results of operations, which are adjusted from GAAP to exclude non-cash compensation expense related to equity awards, costs related to the Company’s merger transaction with Akcea Therapeutics, Inc. (“Akcea”), and costs related to the Company’s restructured commercial operations and the related tax effects. The Company is presenting pro forma information excluding non-cash compensation
expense related to equity awards, costs related to the Akcea merger, and costs related to the restructured commercial operations and related tax effects because the Company believes it is useful for investors in assessing the Company’s operating
results compared to the prior year. A copy of the release is furnished with this report as an exhibit pursuant to “Item 2.02. Results of Operations and Financial Condition” of Form 8-K in accordance with SEC Release Nos. 33-8216 and 34-47583.
The information in this Current Report on Form 8-K and the Exhibit attached hereto shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act
of 1934 (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933 or the
Exchange Act, regardless of any general incorporation language in such filing.
| Item 9.01. |
Financial Statements and Exhibits.
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(d) Exhibits.
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Exhibit No.
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Description
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Press Release dated November 9, 2022.
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104
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Cover Page Interactive Data File (embedded within the Inline XBRL document).
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SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf
by the undersigned, thereunto duly authorized.
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Ionis Pharmaceuticals, Inc.
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Dated: November 9, 2022
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By:
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/s/ Patrick R. O’Neil
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Patrick R. O’Neil
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Executive Vice President, Chief Legal Officer and General Counsel
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Exhibit 99.1
Ionis reports third quarter financial results
Advanced key priorities: initiated manufacturing infrastructure project to support growth
Increased 2022 cash and investments guidance
CARLSBAD, Calif., November 9, 2022 – 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
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2021
|
|||||||||||||
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(amounts in millions)
|
||||||||||||||||
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Total revenue
|
$
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160
|
$
|
133
|
$
|
435
|
$
|
370
|
||||||||
|
Operating expenses
|
$
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219
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$
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219
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$
|
637
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$
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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
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| • |
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
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| • |
Entered into a long-term lease in October 2022 to construct a new manufacturing facility supporting continued growth
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| • |
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
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| • |
Reaffirmed 2022 P&L guidance; increased cash and investments guidance to approximately $2.0 billion
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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
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| • |
Expanded enrollment in the Phase 3 CARDIO-TTRansform study of eplontersen in patients with ATTR cardiomyopathy; data still expected first half of 2025
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| • |
NDA for tofersen was accepted and granted priority review by the FDA; Prescription Drug User Fee Act date of April 25, 2023
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| • |
Initiated CORE2, a confirmatory Phase 3 study of olezarsen in patients with severe hypertriglyceridemia (SHTG)
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| • |
Initiated ESSENCE, a supporting Phase 3 study of olezarsen in patients with SHTG or hypertriglyceridemia and cardiovascular disease
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1
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
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| • |
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
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| • |
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
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| • |
Roche presented the Phase 2 GENERATION HD2 study design of tominersen in Huntington’s disease patients; Roche plans to begin enrollment in early 2023
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| • |
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
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“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.”
2
Third Quarter 2022 Financial Results
Revenue
Ionis' revenue was comprised of the following:
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Three months ended
September 30,
|
Nine months ended
September 30,
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|||||||||||||||
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2022
|
2021
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2022
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2021
|
|||||||||||||
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Revenue:
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(amounts in millions)
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|||||||||||||||
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Commercial revenue:
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||||||||||||||||
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SPINRAZA royalties
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$
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62
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$
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67
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$
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175
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$
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199
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||||||||
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TEGSEDI and WAYLIVRA revenue, net
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6
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15
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23
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47
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Licensing and royalty revenue
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5
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3
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25
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9
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||||||||||||
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Total commercial revenue
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73
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85
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223
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255
|
||||||||||||
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Research and development revenue:
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||||||||||||||||
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Amortization from upfront payments
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18
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17
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54
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57
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Milestone payments
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15
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28
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60
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48
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License fees
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35
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-
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37
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-
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||||||||||||
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Other services
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1
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3
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6
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10
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||||||||||||
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Collaborative agreement revenue
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69
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48
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157
|
115
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||||||||||||
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Eplontersen joint development revenue
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18
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-
|
55
|
-
|
||||||||||||
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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.
3
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.
4
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 Investor Contact:
760-603-2331
Ionis Pharmaceuticals Media Contact:
760-603-4679
5
IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Millions, Except Per Share Data)
|
Three months ended,
September 30,
|
Nine months ended
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
|
||||||||||||
6
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
|
)
|
||||
7
*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.
8
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.
9
IONIS PHARMACEUTICALS, INC.
Condensed Consolidated Balance Sheets
(In Millions)
|
September 30,
2022
|
December 31,
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
|
||||
10
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.
|
# # #
11