IONS 8-K
Ionis Pharmaceuticals Inc (IONS)
8-K
2023-08-09
For: 2023-08-09
View Original
Added on
April 07, 2026
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): August 9, 2023
(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).
Emerging growth company ☐
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 August 9, 2023, Ionis Pharmaceuticals, Inc. (the “Company”)
issued a press release announcing the Company’s financial results for the quarter ended June 30, 2023. 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 and the related tax effects. The Company is presenting pro forma information excluding non-cash compensation expense and the related tax effects because the Company believes it better enables financial statement users to assess and
compare its historical performance and project its future operating results and cash flows. 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 August 9, 2023.
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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: August 9, 2023
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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 second quarter 2023 financial results
Reported Phase 3 data showing eplontersen continued to show improvement in ATTRv-PN
through 85 weeks; December 22, 2023 PDUFA date
Completed enrollment in donidalorsen OASIS-HAE study and eplontersen CARDIO-TTRansform
ATTR-CM study keeping Phase 3 data readouts on track
On track to achieve 2023 financial guidance
CARLSBAD, Calif., August 9, 2023 – Ionis
Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”), today reported financial results for the second quarter of 2023. Financial results
are summarized below:
|
Three months ended
June 30,
|
Six months ended
June 30,
|
|||||||||||||||
|
2023
|
2022
|
2023
|
2022
|
|||||||||||||
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(amounts in millions)
|
||||||||||||||||
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Total revenue
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$
|
188
|
$
|
134
|
$
|
319
|
$
|
276
|
||||||||
|
Operating expenses
|
$
|
279
|
$
|
220
|
$
|
523
|
$
|
419
|
||||||||
|
Operating expenses on a non-GAAP basis
|
$
|
252
|
$
|
195
|
$
|
469
|
$
|
368
|
||||||||
|
Loss from operations
|
$
|
(91
|
)
|
$
|
(86
|
)
|
$
|
(204
|
)
|
$
|
(143
|
)
|
||||
|
Loss from operations on a non-GAAP basis
|
$
|
(64
|
)
|
$
|
(61
|
)
|
$
|
(150
|
)
|
$
|
(92
|
)
|
||||
Financial Highlights
| • |
Revenue increased for the second quarter and first half of 2023 by 40% and 16% compared to the same periods last year, respectively, driven by significant partner payments
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| • |
Operating expenses increased in the second quarter and first half of 2023 compared to the prior year as planned, reflecting investments in advancing Ionis’ pipeline and go-to-market activities
for eplontersen, olezarsen and donidalorsen
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| • |
Cash and short-term investments of $2.4 billion as of June 30, 2023 enables continued investments to drive increasing value
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| • |
Well-capitalized balance sheet reflects 2024 convertible note refinancing that extended maturity to 2028 while maintaining a low coupon and retaining the flexibility to mitigate potential equity
dilution
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| • |
Reaffirmed 2023 financial guidance
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1
“Ionis is creating significant value in 2023 as we successfully execute on our strategy to bring a steady cadence of transformational medicines to the market. Our
growing late-stage pipeline now includes eight medicines for 10 indications, highlighted by eplontersen. Based on the strong and consistent data generated to date and an attractive self-administration profile, we expect eplontersen to be an
important new medicine for people with ATTRv-polyneuropathy. We also added QALSODY to our commercial portfolio, a breakthrough treatment for people with SOD1-ALS further strengthening Ionis’ leadership in RNA-based therapies for neurological
diseases,” said Brett P. Monia, Ph.D., chief executive officer of Ionis. “In the second half, we expect continued positive momentum including the Phase 3 readout for olezarsen in familial chylomicronemia syndrome and the potential approval of
eplontersen for ATTRv-PN in December.”
Recent Highlights From Commercial Medicines
| • |
Biogen presented interim data from the Phase 4 RESPOND study of SMA patients demonstrating improved motor function in most participants treated with SPINRAZA who had unmet medical needs after
treatment with gene therapy
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| • |
FDA granted Biogen accelerated approval of QALSODY (tofersen), a first-in-class medicine for patients with SOD1-ALS
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Recent Highlights From Near-Term Commercial Opportunities
| • |
Reported positive results from the Phase 3 NEURO-TTRansform study in patients with ATTRv-PN showing eplontersen continued to halt neuropathy disease progression and improve quality of life
through 85 weeks
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| • |
Completed enrollment of the Phase 3 CARDIO-TTRansform study of eplontersen in patients with ATTR cardiomyopathy, the largest study ever conducted in ATTR-CM; on track for data readout as early as
H1:2025
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| • |
Licensed eplontersen Latin America rights to AstraZeneca
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| • |
Completed enrollment of the Phase 3 OASIS-HAE study of donidalorsen in patients with hereditary angioedema; on track for data readout in H1:2024
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Reported positive topline Phase 2 open label extension data of donidalorsen in patients with hereditary angioedema treated for two years
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Recent Highlights From Partnered Programs
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Roche advanced IONIS-FB-LRx into Phase 3 development in patients with immunoglobulin A nephropathy
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GSK presented durable response data from the Phase 2 B-Sure long-term follow-up study of bepirovirsen in complete responder patients from the Phase 2b B-Clear study of patients with HBV
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Completed enrollment in the Phase 2 GOLDEN study of IONIS-FB-LRx in patients with geographic atrophy
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AstraZeneca initiated a Phase 2b study of ION839 (AZD2693) targeting PNPLA3 to treat patients with NASH
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Entered collaboration with Novartis to advance a next generation program targeting Lp(a) for cardiovascular disease
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2
Second Quarter 2023 Financial Results
“Our results for the first half of the year keep us on track to achieve our 2023 guidance. We continued to generate substantial and sustained revenue, that together
with our well-capitalized balance sheet, allows us to continue investing in key opportunities across our business,” said Elizabeth L. Hougen, chief financial officer of Ionis. “With three near-term commercial opportunities that have a combined
multi-billion-dollar peak sales potential and a steady cadence of medicines poised to follow closely behind, we are positioned to drive substantial revenue growth and long-term value for
shareholders.”
Revenue
Ionis’ revenue was comprised of the following:
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Three months ended
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Six months ended
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|||||||||||||||
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June 30,
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June 30,
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|||||||||||||||
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2023
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2022
|
2023
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2022
|
|||||||||||||
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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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61
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$
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60
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$
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111
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$
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113
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||||||||
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Other commercial revenue:
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||||||||||||||||
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TEGSEDI and WAYLIVRA revenue, net
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11
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10
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17
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17
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||||||||||||
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Licensing and royalty revenue
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6
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8
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18
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20
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||||||||||||
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Total commercial revenue
|
78
|
78
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146
|
150
|
||||||||||||
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Research and development revenue:
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||||||||||||||||
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Amortization from upfront payments
|
15
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18
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29
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36
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||||||||||||
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Milestone payments
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51
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18
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74
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45
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||||||||||||
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License fees
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20
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-
|
20
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2
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||||||||||||
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Other services
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4
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3
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6
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6
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||||||||||||
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Collaborative agreement revenue
|
90
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39
|
129
|
89
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||||||||||||
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Eplontersen joint development revenue
|
20
|
17
|
44
|
37
|
||||||||||||
|
Total research and development revenue
|
110
|
56
|
173
|
126
|
||||||||||||
|
Total revenue
|
$
|
188
|
$
|
134
|
$
|
319
|
$
|
276
|
||||||||
Ionis’ revenue increased in the second quarter and first half of 2023 compared to the same periods in 2022 because of increased payments from partnered programs.
Ionis believes its substantial and sustainable revenue is an important source of funding that supports the Company’s investments to bring potentially transformational medicines to the market.
Commercial revenue for the second quarter and first half of 2023 included $61 million and $111 million from SPINRAZA royalties, respectively. Global SPINRAZA product
sales of $437 million and $880 million were essentially flat for the second quarter and first half of 2023, respectively, compared to the same periods last year reflecting SPINRAZA’s resilience against emerging competition. Ionis’ commercial revenue in the second quarter and first half of 2023 also included royalties from the U.S. launch of QALSODY.
R&D revenue essentially doubled for the second quarter of 2023 and increased more than 35% for the first half of 2023 compared to the same periods in 2022
reflecting the value Ionis’ technology is creating as numerous partnered programs advanced.
3
Operating Expenses
Ionis’ operating expenses increased in the second quarter and first half of 2023 compared to the same periods in 2022, consistent with expectations. As Ionis
advanced its robust pipeline, study costs increased as most of the Company’s Phase 3 studies were either fully enrolled or approaching full enrollment resulting in higher R&D expenses year over year. Additionally, as Ionis prepares to launch
eplontersen, olezarsen and donidalorsen, the Company’s SG&A expenses also increased year over year.
Balance Sheet
As of June 30, 2023, Ionis’ cash, cash equivalents and short-term investments increased to $2.4 billion compared to $2.0 billion at December 31, 2022 primarily due
to the $500 million Ionis received from Royalty Pharma in January 2023. Ionis’ working capital also increased over the same period primarily due to the Company’s higher cash and short-term
investments balance. In the first quarter of 2023 the Company recorded a long-term liability for future royalties due to Royalty Pharma. In June 2023 Ionis issued $575 million of senior
convertible notes due in June 2028 with an interest rate of 1.75%. Ionis used the majority of the proceeds to repurchase $434 million of its 2024 convertible notes. The Company plans to utilize the residual proceeds to settle the 2024 notes that
remain outstanding.
Webcast
Management will host a conference call and webcast to discuss Ionis’ second quarter 2023 results at 11:30 a.m. Eastern time on Wednesday, August 9, 2023. 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 second quarter 2023 earnings slides
click here.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been a leader in RNA-targeted therapy, pioneering new markets and changing standards of care. Ionis currently has four marketed
medicines and a promising late-stage pipeline highlighted by cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision to become the leader
in genetic medicine, utilizing a multi-platform approach to discover, develop and deliver life-transforming therapies.
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 QALSODY
(tofersen), SPINRAZA (nusinersen), TEGSEDI (inotersen), WAYLIVRA (volanesorsen), eplontersen, olezarsen, donidalorsen, ulefnersen, pelacarsen, bepirovirsen, IONIS-FB-LRx, 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 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, 2022, 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.
4
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. QALSODYTM is a trademark of Biogen. SPINRAZA® is a
registered trademark of Biogen.
Ionis Investor Contact:
D. Wade Walke, Ph.D.
[email protected] 760-603-2331
Ionis Media Contact:
David Polk, J.D.
[email protected] 760-603-4679
5
IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Millions, Except Per Share Data)
|
Three months ended,
|
Six months ended
|
|||||||||||||||
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June 30,
|
June 30,
|
|||||||||||||||
|
2023
|
2022
|
2023
|
2022
|
|||||||||||||
|
(unaudited)
|
||||||||||||||||
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Revenue:
|
||||||||||||||||
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Commercial revenue:
|
||||||||||||||||
|
SPINRAZA royalties
|
$
|
61
|
$
|
60
|
$
|
111
|
$
|
113
|
||||||||
|
Other commercial revenue
|
17
|
18
|
35
|
37
|
||||||||||||
|
Total commercial revenue
|
78
|
78
|
146
|
150
|
||||||||||||
|
Research and development revenue:
|
||||||||||||||||
|
Collaborative agreement revenue
|
90
|
39
|
129
|
89
|
||||||||||||
|
Eplontersen joint development revenue
|
20
|
17
|
44
|
37
|
||||||||||||
|
Total research and development revenue
|
110
|
56
|
173
|
126
|
||||||||||||
|
Total revenue
|
188
|
134
|
319
|
276
|
||||||||||||
|
Expenses:
|
||||||||||||||||
|
Cost of sales
|
3
|
5
|
4
|
9
|
||||||||||||
|
Research, development and patent
|
230
|
181
|
428
|
342
|
||||||||||||
|
Selling, general and administrative
|
46
|
34
|
91
|
68
|
||||||||||||
|
Total operating expenses
|
279
|
220
|
523
|
419
|
||||||||||||
|
Loss from operations
|
(91
|
)
|
(86
|
)
|
(204
|
)
|
(143
|
)
|
||||||||
|
Other income (expense):
|
||||||||||||||||
|
Interest expense related to the sale of future royalties:
|
(18
|
)
|
-
|
(33
|
)
|
-
|
||||||||||
|
Other income (expense), net
|
32
|
(17
|
)
|
47
|
(24
|
)
|
||||||||||
|
Loss before income tax expense
|
(77
|
)
|
(103
|
)
|
(190
|
)
|
(167
|
)
|
||||||||
|
Income tax expense
|
(8
|
)
|
(2
|
)
|
(20
|
)
|
(3
|
)
|
||||||||
|
Net loss
|
$
|
(85
|
)
|
$
|
(105
|
)
|
$
|
(210
|
)
|
$
|
(170
|
)
|
||||
|
Basic and diluted net loss per share
|
$
|
(0.60
|
)
|
$
|
(0.74
|
)
|
$
|
(1.47
|
)
|
$
|
(1.20
|
)
|
||||
|
Shares used in computing basic and diluted net loss per share
|
143
|
142
|
143
|
142
|
||||||||||||
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
June 30,
|
Six months ended
June 30,
|
|||||||||||||||
|
2023
|
2022
|
2023
|
2022
|
|||||||||||||
|
(unaudited)
|
||||||||||||||||
|
As reported research, development and patent expenses according to GAAP
|
$
|
230
|
$
|
181
|
$
|
428
|
$
|
342
|
||||||||
|
Excluding compensation expense related to equity awards
|
(19
|
)
|
(19
|
)
|
(39
|
)
|
(38
|
)
|
||||||||
|
Non-GAAP research, development and patent expenses
|
$
|
211
|
$
|
162
|
$
|
389
|
$
|
304
|
||||||||
|
As reported selling, general and administrative expenses according to GAAP
|
$
|
46
|
$
|
34
|
$
|
91
|
$
|
68
|
||||||||
|
Excluding compensation expense related to equity awards
|
(7
|
)
|
(6
|
)
|
(14
|
)
|
(13
|
)
|
||||||||
|
Non-GAAP selling, general and administrative expenses
|
$
|
39
|
$
|
28
|
$
|
77
|
$
|
55
|
||||||||
|
As reported operating expenses according to GAAP
|
$
|
279
|
$
|
220
|
$
|
523
|
$
|
419
|
||||||||
|
Excluding compensation expense related to equity awards
|
(27
|
)
|
(25
|
)
|
(54
|
)
|
(51
|
)
|
||||||||
|
Non-GAAP operating expenses
|
$
|
252
|
$
|
195
|
$
|
469
|
$
|
368
|
||||||||
|
As reported loss from operations according to GAAP
|
$
|
(91
|
)
|
$
|
(86
|
)
|
$
|
(204
|
)
|
$
|
(143
|
)
|
||||
|
Excluding compensation expense related to equity awards
|
(27
|
)
|
(25
|
)
|
(54
|
)
|
(51
|
)
|
||||||||
|
Non-GAAP loss from operations
|
$
|
(64
|
)
|
$
|
(61
|
)
|
$
|
(150
|
)
|
$
|
(92
|
)
|
||||
|
As reported net loss according to GAAP
|
$
|
(85
|
)
|
$
|
(105
|
)
|
$
|
(210
|
)
|
$
|
(170
|
)
|
||||
|
Excluding compensation expense related to equity awards and related tax effects
|
(27
|
)
|
(25
|
)
|
(54
|
)
|
(51
|
)
|
||||||||
|
Non-GAAP net loss
|
$
|
(58
|
)
|
$
|
(80
|
)
|
$
|
(156
|
)
|
$
|
(119
|
)
|
||||
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. 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.
7
IONIS PHARMACEUTICALS, INC.
Condensed Consolidated Balance Sheets
(In Millions)
|
June 30,
|
December 31,
|
|||||||
|
2023
|
2022
|
|||||||
|
(unaudited)
|
||||||||
|
Assets:
|
||||||||
|
Cash, cash equivalents and short-term investments
|
$
|
2,385
|
$
|
1,987
|
||||
|
Contracts receivable
|
28
|
26
|
||||||
|
Other current assets
|
203
|
190
|
||||||
|
Property, plant and equipment, net
|
92
|
74
|
||||||
|
Right-of-use assets
|
177
|
182
|
||||||
|
Other assets
|
86
|
75
|
||||||
|
Total assets
|
$
|
2,971
|
$
|
2,534
|
||||
|
Liabilities and stockholders’ equity:
|
||||||||
|
Other current liabilities
|
$
|
192
|
$
|
221
|
||||
|
Current portion of deferred contract revenue
|
96
|
91
|
||||||
|
1.75% convertible senior notes, net
|
561
|
-
|
||||||
|
0% convertible senior notes, net
|
624
|
622
|
||||||
|
0.125% convertible senior notes, net
|
114
|
545
|
||||||
|
Liability related to sale of future royalties, net
|
510
|
-
|
||||||
|
Long-term lease liabilities
|
175
|
178
|
||||||
|
Long-term obligations, less current portion
|
17
|
16
|
||||||
|
Long-term deferred contract revenue
|
254
|
288
|
||||||
|
Total stockholders’ equity
|
428
|
573
|
||||||
|
Total liabilities and stockholders’ equity
|
$
|
2,971
|
$
|
2,534
|
||||
8
2023 Key Value Driving Events(1)
|
Regulatory Actions
|
|||
|
Program
|
Indication
|
Regulatory Action
|
Achieved
|
|
QALSODY
|
SOD1-ALS
|
NDA approval
|
 |
|
EU approval2
|
|||
|
Eplontersen (TTR)
|
ATTRv polyneuropathy
|
NDA approval
|
|
|
OUS filings
|
|||
|
Key Clinical Data Events
|
|||
|
Program
|
Indication
|
Event
|
Achieved
|
|
Eplontersen (TTR)
|
ATTRv polyneuropathy
|
Phase 3 data (week 35, 66 & 85)
|
|
|
Olezarsen (APOCIII)
|
FCS
|
Phase 3 data
|
|
|
Donidalorsen (PKK)
|
HAE
|
Phase 2, OLE 1-year data
|
|
|
Donidalorsen (PKK)
|
HAE
|
Phase 2, OLE 2-year data
|
|
|
Enrollment Achievements
|
|||
|
Program
|
Indication
|
Event
|
Achieved
|
|
Eplontersen (TTR)
|
ATTR cardiomyopathy
|
Phase 3 full enrollment
|
|
|
Donidalorsen (PKK)
|
HAE
|
Phase 3 full enrollment
|
|
|
IONIS-FB-LRx
|
Geographic Atrophy
|
Phase 2 full enrollment
|
|
|
Phase 3 Initiations
|
||
|
Program
|
Indication
|
Achieved
|
|
Bepirovirsen (HBV)
|
Hepatitis B virus infection
|
|
|
IONIS-FB-LRx
|
Immunoglobulin A nephropathy
|
|
| (1) |
Timing expectations based on current assumptions and subject to change.
|
| (2) |
CHMP opinion anticipated in Q4:2023.
|
# # #
9