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): May 3, 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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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 May 3, 2023, Ionis Pharmaceuticals, Inc. (the “Company”) issued a press release announcing the Company’s
financial results for the quarter ended March 31, 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 May 3, 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: May 3, 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 first quarter 2023 financial results
QALSODY approved for SOD1-ALS; MAA under review in EU
Reported positive Phase 3 eplontersen ATTRv-PN data; December 22, 2023 PDUFA date
On track to achieve 2023 financial guidance
CARLSBAD, Calif., May 3, 2023 – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS)
(the “Company”), today reported financial results for the first quarter of 2023. Financial results are summarized below:
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Three months ended
March 31,
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||||||||
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2023
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2022
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|||||||
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(amounts in millions)
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||||||||
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Total revenue
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$
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131
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$
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142
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||||
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Operating expenses
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$
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245
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$
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199
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||||
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Operating expenses on a non-GAAP basis
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$
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218
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$
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173
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||||
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Loss from operations
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$
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(114
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)
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$
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(57
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)
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||
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Loss from operations on a non-GAAP basis
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$
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(87
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)
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$
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(31
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)
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Financial Highlights
| • |
Revenue for the first quarter of 2023 was in line with expectations and included revenue from numerous diverse sources
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| • |
Operating expenses increased in the first quarter of 2023 compared to the prior year as planned, reflecting investments in advancing Ionis’ pipeline, technology and go-to-market
activities for eplontersen, olezarsen and donidalorsen
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| • |
Cash and short-term investments of $2.3 billion at March 31, 2023 enables continued investment in creating future growth opportunities
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| • |
Reaffirmed 2023 financial guidance
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“2023 is off to a strong start. With QALSODY’s approval, it joins SPINRAZA as a new groundbreaking medicine to treat a devastating neurological disease, further
validating our RNA-targeting therapeutic platform. We also achieved another important milestone with our recent positive eplontersen Phase 3 data. We believe the positive efficacy and safety data, and the attractive self-administered dosing
profile, position eplontersen to be an important treatment for ATTRv-PN patients, who today are underserved. We look forward to the first potential approval of eplontersen in the U.S. in December,” said Brett P. Monia, Ph.D., chief executive officer of Ionis. “In addition, we further expanded our industry-leading late-stage pipeline to seven programs across nine indications following the start of GSK’s bepirovirsen
hepatitis B program. In the second half, we plan to report results from our olezarsen Phase 3 FCS study, which if positive, positions us for our first independent commercial launch. These recent achievements, together with our upcoming milestones,
continue to build value for Ionis stakeholders.”
1
Recent Late-Stage Pipeline Highlights
| • |
FDA granted Biogen accelerated approval of QALSODY (tofersen) for patients with SOD1-ALS
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| • |
FDA accepted eplontersen NDA for patients with polyneuropathy caused by hereditary TTR amyloidosis (ATTRv-PN) with a PDUFA date of December 22, 2023
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Presented positive week-35 and week-66 data from the Phase 3 NEURO-TTRansform study of eplontersen in patients with ATTRv-PN
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GSK advanced bepirovirsen into Phase 3 development in patients with chronic hepatitis B
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Recent Additional Pipeline Updates
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Biogen presented data demonstrating IONIS-MAPTRx (BIIB080) substantially reduced tau protein in patients with early-stage Alzheimer’s disease
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Continued to focus R&D efforts by discontinuing two programs that did not meet Ionis’ target product profile, cimdelirsen for acromegaly and sapablursen for beta-thalassemia.
Ionis continues to advance the Phase 2 sapablursen study for polycythemia vera
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2
First Quarter 2023 Financial Results
“Our first quarter results were in line with our expectations. We generated meaningful revenue while continuing to invest in key growth opportunities across our
business. These results keep us on track to achieve our 2023 financial guidance,” said Elizabeth L. Hougen, chief financial officer of Ionis. “We plan to continue investing in areas with the greatest potential to drive growth. As such, we expect
our investments to grow modestly as we advance and expand our late-stage pipeline and move our near-term commercial opportunities toward the market. Additionally, as we keep more programs for ourselves, we expect a greater proportion of commercial
revenues compared to R&D revenues, and our commercial revenues to be the primary driver of future revenue growth.”
Revenue
Ionis’ revenue was comprised of the following:
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Three months ended
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||||||||
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March 31,
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||||||||
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2023
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2022
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|||||||
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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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50
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$
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54
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TEGSEDI and WAYLIVRA revenue, net
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7
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6
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Licensing and royalty revenue
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11
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12
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Total commercial revenue
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68
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72
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Research and development revenue:
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Amortization from upfront payments
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16
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17
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Milestone payments
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23
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27
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License fees
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-
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2
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Other services
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-
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4
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Collaborative agreement revenue
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39
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50
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Eplontersen joint development revenue
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24
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20
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Total research and development revenue
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63
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70
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Total revenue
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$
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131
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$
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142
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Ionis continued to derive its revenue for the first quarter of 2023 from diverse sources, with approximately half coming from commercial products and half from
numerous partnered programs. Commercial revenue for the first quarter of 2023 included $50 million from SPINRAZA royalties. Global SPINRAZA product sales of $443 million decreased six percent in the first quarter of 2023, compared to the same
period last year primarily due to the impact from foreign currency, fewer new patient starts in the U.S. and channel dynamics.
R&D revenue for the first quarter of 2023 included $24 million from AstraZeneca for its share of the global Phase 3 development costs for eplontersen, $20 million
from Biogen for advancing several neurology disease programs and $15 million from GSK for advancing bepirovirsen into Phase 3 development. Already in the second quarter, the Company earned $16 million in a milestone payment from Biogen when QALSODY
was approved in the U.S.
Operating Expenses
Ionis’ operating expenses increased in the first quarter of 2023 compared to the same period 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.
3
Balance Sheet
As of March 31, 2023, Ionis’ cash, cash equivalents and short-term investments increased to $2.3 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.
Additionally, the Company recorded a long-term liability for future royalties due to Royalty Pharma in the first quarter of 2023.
Webcast
Management will host a conference call and webcast to discuss Ionis’ first quarter 2023 results at 11:30 a.m. Eastern time on Wednesday, May 3, 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 first 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, ION363, pelacarsen, bepirovirsen, 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, which is on file with the Securities and Exchange Commission. Copies of this 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. QALSODYTM is a trademark of Biogen. SPINRAZA® is a
registered trademark of Biogen.
Ionis Investor Contact:
D. Wade Walke, Ph.D.
info@ionisph.com 760-603-2331
David Polk, J.D.
ionis_ca@ionisph.com 760-603-4679
4
IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Millions, Except Per Share Data)
|
Three months ended,
March 31,
|
||||||||
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2023
|
2022
|
|||||||
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(unaudited)
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|||||||
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Revenue:
|
||||||||
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Commercial revenue:
|
||||||||
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SPINRAZA royalties
|
$
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50
|
$
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54
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||||
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TEGSEDI and WAYLIVRA revenue, net
|
7
|
6
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||||||
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Licensing and royalty revenue
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11
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12
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||||||
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Total commercial revenue
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68
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72
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||||||
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Research and development revenue:
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||||||||
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Collaborative agreement revenue
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39
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50
|
||||||
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Eplontersen joint development revenue
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24
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20
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||||||
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Total research and development revenue
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63
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70
|
||||||
|
Total revenue
|
131
|
142
|
||||||
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Expenses:
|
||||||||
|
Cost of sales
|
1
|
4
|
||||||
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Research, development and patent
|
198
|
161
|
||||||
|
Selling, general and administrative
|
46
|
34
|
||||||
|
Total operating expenses
|
245
|
199
|
||||||
|
Loss from operations
|
(114
|
)
|
(57
|
)
|
||||
|
|
||||||||
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Other income (expense):
|
||||||||
|
Interest expense related to sale of future royalties
|
(16
|
)
|
-
|
|||||
|
Other income (expense), net
|
17
|
(7
|
)
|
|||||
|
Loss before income tax expense
|
(113
|
)
|
(64
|
)
|
||||
|
|
||||||||
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Income tax expense
|
(11
|
)
|
(1
|
)
|
||||
|
|
||||||||
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Net loss
|
$
|
(124
|
)
|
$
|
(65
|
)
|
||
|
Basic and diluted net loss per share
|
$
|
(0.87
|
)
|
$
|
(0.46
|
)
|
||
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Shares used in computing basic and diluted net loss per share
|
143
|
142
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||||||
5
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,
|
||||||||
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2023
|
2022
|
|||||||
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(unaudited)
|
||||||||
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As reported research, development and patent expenses according to GAAP
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$
|
198
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$
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161
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||||
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Excluding compensation expense related to equity awards
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(20
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)
|
(19
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)
|
||||
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Non-GAAP research, development and patent expenses
|
$
|
178
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$
|
142
|
||||
|
|
||||||||
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As reported selling, general and administrative expenses according to GAAP
|
$
|
46
|
$
|
34
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||||
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Excluding compensation expense related to equity awards
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(7
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)
|
(7
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)
|
||||
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Non-GAAP selling, general and administrative expenses
|
$
|
39
|
$
|
27
|
||||
|
|
||||||||
|
As reported operating expenses according to GAAP
|
$
|
245
|
$
|
199
|
||||
|
Excluding compensation expense related to equity awards
|
(27
|
)
|
(26
|
)
|
||||
|
Non-GAAP operating expenses
|
$
|
218
|
$
|
173
|
||||
|
|
||||||||
|
As reported loss from operations according to GAAP
|
$
|
(114
|
)
|
$
|
(57
|
)
|
||
|
Excluding compensation expense related to equity awards
|
(27
|
)
|
(26
|
)
|
||||
|
Non-GAAP loss from operations
|
$
|
(87
|
)
|
$
|
(31
|
)
|
||
|
|
||||||||
|
As reported net loss according to GAAP
|
$
|
(124
|
)
|
$
|
(65
|
)
|
||
|
Excluding compensation expense related to equity awards and related tax effects
|
(27
|
)
|
(26
|
)
|
||||
|
Non-GAAP net loss
|
$
|
(97
|
)
|
$
|
(39
|
)
|
||
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.
6
IONIS PHARMACEUTICALS, INC.
Condensed Consolidated Balance Sheets
(In Millions)
|
March 31,
2023 |
December 31,
2022 |
|||||||
|
|
(unaudited)
|
|||||||
|
Assets:
|
||||||||
|
Cash, cash equivalents and short-term investments
|
$
|
2,347
|
$
|
1,987
|
||||
|
Contracts receivable
|
14
|
26
|
||||||
|
Other current assets
|
182
|
190
|
||||||
|
Property, plant and equipment, net
|
85
|
74
|
||||||
|
Right-of-use assets
|
179
|
182
|
||||||
|
Other assets
|
78
|
75
|
||||||
|
Total assets
|
$
|
2,885
|
$
|
2,534
|
||||
|
|
||||||||
|
Liabilities and stockholders’ equity:
|
||||||||
|
Other current liabilities
|
$
|
167
|
$
|
221
|
||||
|
Current portion of deferred contract revenue
|
92
|
91
|
||||||
|
0% convertible senior notes, net
|
623
|
622
|
||||||
|
0.125% convertible senior notes, net
|
545
|
545
|
||||||
|
Liability related to sale of future royalties, net
|
505
|
-
|
||||||
|
Long-term lease liabilities
|
177
|
178
|
||||||
|
Long-term obligations, less current portion
|
16
|
16
|
||||||
|
Long-term deferred contract revenue
|
273
|
288
|
||||||
|
Total stockholders’ equity
|
487
|
573
|
||||||
|
Total liabilities and stockholders’ equity
|
$
|
2,885
|
$
|
2,534
|
||||
7
2023 Key Value Driving Events(1)
|
Regulatory Actions
|
||
|
Program
|
Indication
|
Regulatory Action
|
|
QALSODY
|
SOD1-ALS
|
NDA approval (achieved)
|
|
EU approval2
|
||
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Eplontersen (TTR)
|
ATTRv polyneuropathy
|
NDA approval
|
|
OUS filings
|
||
|
Key Clinical Achievements
|
||
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Program
|
Indication
|
Event
|
|
Eplontersen (TTR)
|
ATTRv polyneuropathy
|
Phase 3 data (week 35 & 66) (achieved)
|
|
Olezarsen
|
FCS
|
Phase 3 data
|
|
Eplontersen (TTR)
|
ATTR cardiomyopathy
|
Phase 3 full enrollment
|
|
Donidalorsen (PKK)
|
HAE
|
Phase 3 full enrollment
|
|
Phase 3 Initiations
|
||
|
Program
|
Indication
|
Timing
|
|
Bepirovirsen (HBV)
|
Hepatitis B virus infection
|
H1:23 (achieved)
|
|
IONIS-FB-LRx
|
Immunoglobulin A nephropathy
|
H1:23
|
| (1) |
Timing expectations based on current assumptions and subject to change.
|
| (2) |
CHMP opinion anticipated in Q4:2023.
|
# # #
8