Full Press Release Details
Ionis reports fourth quarter and full year 2021 financial results and recent business achievements
Exceeded 2021 financial guidance with revenues of more than $800 million
Webcast today, February 24, 2022, at 11:30 a.m. Eastern Time
CARLSBAD, Calif., February 24, 2022 - Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today reported financial results for the fourth quarter and full year ended December 31, 2021, and recent business achievements.
"During 2021, we made significant progress towards achieving our vision of becoming a leading fully-integrated biotechnology company. We advanced our commercial
strategy and go-to-market plans for our near-term commercial opportunities, eplontersen, olezarsen and donidalorsen. Our collaboration with AstraZeneca to jointly develop and commercialize eplontersen enables us to potentially maximize benefit for
patients, bolster our commercial organization and accelerate preparations for our near-term product launches. Most recently, we initiated Phase 3 studies with olezarsen in patients with severely high triglycerides and donidalorsen in patients with
hereditary angioedema. This expands our Phase 3 pipeline to six medicines addressing eight indications. We also advanced our technology, positioning us to build on our leadership in RNA-targeted therapeutics and add value for our future medicines,"
said Brett P. Monia, Ph.D., chief executive officer of Ionis. "We look forward to a steady cadence of catalysts throughout this year, highlighted by eplontersen Phase 3 data in patients with hATTR polyneuropathy planned for mid-year. We expect to
file for regulatory approval for eplontersen before year end, assuming positive data. We also expect to make continued advancements to expand and diversify our technology. Based on our anticipated near- and mid-term catalysts, we believe we are
well positioned to drive increasing value for patients and shareholders."
2021 Summary Financial Results
Recent Marketed Products Highlights
Fourth Quarter 2021 and Recent Events
2022 Pipeline Milestones(2)
| Anticipated 2022 Regulatory Filings | ||||
| Program | Anticipated Indication | H1 | H2 | |
| Eplontersen | hATTR polyneuropathy | |||
| Anticipated Key 2022 Data Readouts | ||||
| Program | Data Readout | Anticipated Indication | H1 | H2 |
| Tominersen | Phase 3 post hoc | Huntington's disease | ||
| Eplontersen | Phase 3 | hATTR polyneuropathy | ||
| ION449 (PCSK9) | Phase 2b | Cardiovascular disease | ||
| Donidalorsen | Phase 2 | HAE | ||
| IONIS-C9 Rx (BIIB078) | Phase 2 | C9-ALS | ||
| IONIS-AGT-L Rx | Phase 2b | Treatment-resistant hypertension | ||
| Fesomersen (FXI) | Phase 2b | Thrombosis | ||
| Bepirovirsen (HBV) | Phase 2b | Hepatitis B virus infection | ||
| Donidalorsen | Phase 2 OLE | HAE | ||
| Cimdelirsen | Phase 2 | Acromegaly (monotherapy) | ||
| Anticipated Key 2022 Study Initiations | ||||
| Program | Phase | Anticipated Indication | H1 | H2 |
| Sapablursen | 2 | Polycythemia vera | ||
| IONIS-MAPT Rx (BIIB080) | 2 | Alzheimer's disease | ||
| ION904 (AGT) | 2 | Uncontrolled hypertension | ||
| 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
2021 Financial Results and 2022 Financial Guidance
"Over the last year, we achieved numerous pipeline and technology milestones, advanced multiple medicines towards the market and accelerated preparations for our
near-term commercial launches. We also exceeded our 2021 financial guidance, driven by revenue from advancing multiple partnered programs and by strengthening and streamlining our business," said Elizabeth L. Hougen, chief financial officer of
Ionis. "We have a long history of financial responsibility that provides us with a strong financial foundation. With more than $2 billion of cash and a substantial and sustainable base of commercial and R&D revenues, we are well positioned to
accelerate our investments in 2022 to drive substantial future growth."
2022 Financial Guidance
Ionis' full year 2022 financial guidance consists of the following components (on a non-GAAP basis)(1):
| Guidance | |
| Revenue | >$575 million |
| Operating Expenses (1) | $825 million to $850 million |
| Net Loss (1) | <$275 million |
| Cash and Short-Term Investments | ~$1.7 billion |
Ionis' revenue was comprised of the following (amounts in millions):
| Three months ended December 31, | Year ended December 31, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| Revenue: | ||||||||||||||||
| Commercial revenue: | ||||||||||||||||
| SPINRAZA royalties | $ | 69 | $ | 75 | $ | 268 | $ | 287 | ||||||||
| TEGSEDI and WAYLIVRA revenue, net | 9 | 19 | 56 | 70 | ||||||||||||
| Licensing and royalty revenue | 9 | 2 | 18 | 8 | ||||||||||||
| Total commercial revenue | 87 | 96 | 342 | 365 | ||||||||||||
| R&D revenue: | ||||||||||||||||
| Amortization from upfront payments | 21 | 12 | 78 | 80 | ||||||||||||
| Milestone payments | 40 | 110 | 88 | 183 | ||||||||||||
| License fees | 290 | 71 | 291 | 86 | ||||||||||||
| Other services | 2 | 1 | 11 | 15 | ||||||||||||
| Total R&D revenue | 353 | 194 | 468 | 364 | ||||||||||||
| Total revenue | $ | 440 | $ | 290 | $ | 810 | $ | 729 |
The Company's revenue increased by more than 10 percent compared to 2020 driven in large part by significant partner payments across multiple partnered programs. In 2021,
the Company earned $200 million from its new collaboration with AstraZeneca to jointly develop and commercialize eplontersen. The Company also earned more than $160 million from Biogen for advancing several neurology disease programs.
The Company successfully completed the transition of its TEGSEDI and WAYLIVRA operations in the EU and North America to Sobi in the first and second quarters of 2021,
respectively. The decrease in TEGSEDI and WAYLIVRA revenue in 2021 compared to 2020 was due to the shift from product sales to distribution fees based on net sales generated by Sobi. As part of the transition, Ionis restructured its commercial
operations resulting in substantial cost savings.
Ionis is advancing a large late-stage pipeline and as a result, its non-GAAP operating expenses increased in 2021 compared to 2020. Higher R&D expenses were driven
by the expanded number of Phase 3 studies the Company was conducting, which doubled over the course of 2021 from 3 to 6 studies. Additionally, the Company recognized $35 million in R&D expense in the third quarter of 2021 for licensing Bicycle
Therapeutic's technology. Lower SG&A expenses primarily reflected operating efficiencies achieved from integrating Akcea and restructuring the Company's commercial operations.
Net Loss Attributable to Ionis Common Stockholders
Net loss attributable to Ionis' common stockholders in 2021 decreased compared to 2020 for the reasons discussed above. Also contributing to the decrease in Ionis' net
loss in 2021 compared to 2020 was the non-cash adjustment of the valuation allowance Ionis recorded against its federal net deferred tax assets in 2020.
As of December 31, 2021, Ionis had cash, cash equivalents and short-term investments of $2.1 billion, compared with $1.9 billion as of December 31, 2020.
The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on January 1, 2021.
Ionis will conduct a webcast today at 11:30 a.m. Eastern time to discuss this announcement and related activities. Interested parties may access the webcast here.
A webcast replay will be available for a limited time at the same address.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing the standards of care with its
novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry leading 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.
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, 2020, and the most recent Form 10-Q quarterly filing, which are on file with the SEC. Copies of these and
other documents are available from the Company.
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" 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:
Ionis Pharmaceuticals Media Contact:
IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Millions, Except Per Share Data)
| Three months ended, December 31, | Year ended December 31, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| (as revised*) | (as revised*) | |||||||||||||||
| (unaudited) | ||||||||||||||||
| Revenue: | ||||||||||||||||
| Commercial revenue: | ||||||||||||||||
| SPINRAZA royalties | $ | 69 | $ | 75 | $ | 268 | $ | 287 | ||||||||
| TEGSEDI and WAYLIVRA revenue, net | 9 | 19 | 56 | 70 | ||||||||||||
| Licensing and royalty revenue | 9 | 2 | 18 | 8 | ||||||||||||
| Total commercial revenue | 87 | 96 | 342 | 365 | ||||||||||||
| Research and development revenue under collaborative agreements | 353 | 194 | 468 | 364 | ||||||||||||
| Total revenue | 440 | 290 | 810 | 729 | ||||||||||||
| Expenses: | ||||||||||||||||
| Cost of sales | 2 | 3 | 11 | 12 | ||||||||||||
| Research, development and patent | 179 | 171 | 643 | 535 | ||||||||||||
| Selling, general and administrative | 38 | 139 | 186 | 354 | ||||||||||||
| Total operating expenses | 219 | 313 | 840 | 901 | ||||||||||||
| Income (loss) from operations | 221 | (23 | ) | (30 | ) | (172 | ) | |||||||||
| Other income (expense): | ||||||||||||||||
| Loss on early retirement of debt | - | - | (9 | ) | - | |||||||||||
| Other income, net | 4 | 8 | 9 | 37 | ||||||||||||
| Income (loss) before income tax benefit (expense) | 225 | (15 | ) | (30 | ) | (135 | ) | |||||||||
| Income tax benefit (expense) | - | (341 | ) | 1 | (345 | ) | ||||||||||
| Net income (loss) | $ | 225 | $ | (356 | ) | $ | (29 | ) | $ | (480 | ) | |||||
| Net loss attributable to noncontrolling interest in Akcea Therapeutics, Inc. | - | 1 | - | 36 | ||||||||||||
| Net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders | $ | 225 | $ | (355 | ) | $ | (29 | ) | $ | (444 | ) | |||||
| Basic net income (loss) per share | $ | 1.59 | $ | (2.54 | ) | $ | (0.20 | ) | $ | (3.18 | ) | |||||
| Diluted net income (loss) per share | $ | 1.41 | $ | (2.54 | ) | $ | (0.20 | ) | $ | (3.18 | ) | |||||
| Shares used in computing basic net income (loss) per share | 141 | 140 | 141 | 140 | ||||||||||||
| Shares used in computing diluted net income (loss) per share | 160 | 140 | 141 | 140 |
*The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on January 1, 2021.
IONIS PHARMACEUTICALS, INC.
Reconciliation of GAAP to Non-GAAP Basis:
Condensed Consolidated Operating Expenses, Income (Loss) From Operations, and Net Income (Loss)
| Three months ended December 31, | Year ended December 31, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| (as revised*) | (as revised*) | |||||||||||||||
| (unaudited) | ||||||||||||||||
| As reported research, development and patent expenses according to GAAP | $ | 179 | $ | 171 | $ | 643 | $ | 535 | ||||||||
| Excluding compensation expense related to equity awards | (16 | ) | (22 | ) | (88 | ) | (99 | ) | ||||||||
| Excluding Akcea merger and restructured commercial operation costs** | (1 | ) | (26 | ) | (9 | ) | (26 | ) | ||||||||
| Non-GAAP research, development and patent expenses | $ | 162 | $ | 123 | $ | 546 | $ | 410 | ||||||||
| As reported selling, general and administrative expenses according to GAAP | $ | 38 | $ | 139 | $ | 186 | $ | 354 | ||||||||
| Excluding compensation expense related to equity awards | (7 | ) | (15 | ) | (33 | ) | (72 | ) | ||||||||
| Excluding Akcea merger and restructured commercial operation costs** | 1 | (64 | ) | (15 | ) | (64 | ) | |||||||||
| Non-GAAP selling, general and administrative expenses | $ | 32 | $ | 60 | $ | 138 | $ | 218 | ||||||||
| As reported operating expenses according to GAAP | $ | 219 | $ | 313 | $ | 840 | $ | 901 | ||||||||
| Excluding compensation expense related to equity awards | (23 | ) | (36 | ) | (121 | ) | (171 | ) | ||||||||
| Excluding Akcea merger and restructured commercial operation costs** | - | (90 | ) | (24 | ) | (90 | ) | |||||||||
| Non-GAAP operating expenses | $ | 196 | $ | 187 | $ | 695 | $ | 640 | ||||||||
| As reported income (loss) from operations according to GAAP | $ | 221 | $ | (23 | ) | $ | (30 | ) | $ | (172 | ) | |||||
| Excluding compensation expense related to equity awards | (23 | ) | (36 | ) | (121 | ) | (171 | ) | ||||||||
| Excluding Akcea merger and restructured commercial operation costs** | - | (90 | ) | (24 | ) | (90 | ) | |||||||||
| Non-GAAP income from operations | $ | 244 | $ | 103 | $ | 115 | $ | 89 | ||||||||
| As reported net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders according to GAAP | $ | 225 | $ | (355 | ) | $ | (29 | ) | $ | (444 | ) | |||||
| Excluding compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc. common stockholders | (23 | ) | (36 | ) | (121 | ) | (162 | ) | ||||||||
| Excluding Akcea merger and restructured commercial operation costs** | - | (90 | ) | (24 | ) | (90 | ) | |||||||||
| Income tax effect related to compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc. common stockholders | - | (16 | ) | - | 2 | |||||||||||
| Income tax effect related to the Akcea merger and restructured commercial operation costs** | - | (340 | ) | - | (340 | ) | ||||||||||
| Non-GAAP net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders according to GAAP | $ | 248 | $ | 127 | $ | 116 | $ | 146 |
*The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on January 1, 2021.
** 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 incurred $24 million and
$90 million of costs in conjunction with the Akcea merger and restructuring of the Company's commercial operations for 2021 and 2020, respectively. The Company excluded these costs from its non-GAAP amounts for those 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 income (loss) from operations, and non-GAAP net
income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders were adjusted from GAAP to exclude compensation expense related to equity awards and costs related to the Akcea merger and restructured commercial operations and the
related tax effects. Compensation expense related to equity awards are non-cash. Costs related to the Akcea merger and restructured commercial operations include: severance costs, retention costs and other costs related to commercial operations.
Ionis has regularly reported non-GAAP measures for operating results as non-GAAP results. These measures are provided as supplementary information and are not a substitute for financial measures calculated in accordance with GAAP. Ionis reports
these non-GAAP results to better enable financial statement users to assess and compare its historical performance and project its future operating results and cash flows. Further, the presentation of Ionis' non-GAAP results is consistent with how
Ionis' management internally evaluates the performance of its operations.
IONIS PHARMACEUTICALS, INC.
Condensed Consolidated Balance Sheets
| December 31, 2021 | December 31, 2020 | |||||||
| (as revised*) | ||||||||
| Assets: | ||||||||
| Cash, cash equivalents and short-term investments | $ | 2,115 | $ | 1,892 | ||||
| Contracts receivable | 62 | 76 | ||||||
| Other current assets | 168 | 162 | ||||||
| Property, plant and equipment, net | 178 | 181 | ||||||
| Other assets | 89 | 79 | ||||||
| Total assets | $ | 2,612 | $ | 2,390 | ||||
| Liabilities and stockholders' equity: | ||||||||
| Other current liabilities | $ | 143 | $ | 183 | ||||
| Current portion of 1% convertible senior notes, net | - | 309 | ||||||
| Current portion of deferred contract revenue | 98 | 108 | ||||||
| 0% convertible senior notes, net | 619 | - | ||||||
| 0.125% convertible senior notes, net | 542 | 540 | ||||||
| Long-term obligations, less current portion | 86 | 83 | ||||||
| Long-term deferred contract revenue | 352 | 424 | ||||||
| Total stockholders' equity | 772 | 743 | ||||||
| Total liabilities and stockholders' equity | $ | 2,612 | $ | 2,390 |
*The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on January 1, 2021.