Full Press Release Details
Ionis reports first quarter 2021 financial results and recent business achievements
On track to achieve 2021 guidance
Webcast today, May 5, 2021, at 11:30 a.m. Eastern Time
CARLSBAD, Calif., May 5, 2021 - Ionis Pharmaceuticals, Inc. (Nasdaq: IONS)
today reported its financial results for the first quarter of 2021 and recent business highlights.
"In the first quarter, we took important steps to maximize the value of our wholly owned pipeline. We recently initiated pivotal studies with our wholly owned FUS-ALS
and Alexander disease programs. We delivered positive results from our IONIS-PKK-LRx program, demonstrating its potential to change the standard of care for patients with hereditary angioedema. We also further strengthened the business
and continued executing on our strategic priorities," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "This summer, we expect data from our IONIS-MAPTRx
program in Alzheimer's disease patients. And later this year, we look forward to data from the Phase 3 VALOR study of tofersen in patients with SOD1-ALS. If results from the VALOR study are positive, we expect tofersen to be our next commercial
medicine. These key upcoming catalysts, together with our recent achievements, position us well to have 12 or more products on the market in 2026."
First Quarter 2021 and Recent Summary Financial Results
"So far this year, we have taken important steps in support of developing and commercializing our wholly owned medicines. In addition to
completing the restructuring of our European operations, we expanded our Sobi distribution agreement to include North America. These transactions unlocked significant resources that we are now redirecting towards our highest priority programs,
including IONIS-TTR-LRx and IONIS-APOCIII-LRx," said Elizabeth L. Hougen, chief financial officer of Ionis. "We are on track to meet our 2021 financial
guidance. In the second half of this year, we expect R&D revenue to increase as many of our partnered programs continue to advance. Importantly, we are well-capitalized with the resources we need to expand our manufacturing and R&D capacity
to support the future needs of our wholly owned pipeline. This large capital project, which is now underway, is necessary to successfully execute on our goal to drive growth."
First Quarter 2021 Marketed Products Highlights
First Quarter 2021 and Recent Pipeline Events
Upcoming 2021 Pipeline Catalysts(2)
Anticipated 2021 Data Readouts
| Program | Phase | Anticipated Indication | H1 | H2 | |||
| IONIS-PKK-L Rx | 2 | Hereditary angioedema (top-line data) | |||||
| IONIS-AGT-L Rx | 2 | Hypertension | |||||
| Tominersen | 3 | Huntington's disease | |||||
| IONIS-ENAC-2.5 Rx | 2 | Cystic fibrosis | |||||
| IONIS-GHR-L Rx | 2 + OLE | Acromegaly | |||||
| IONIS-MAPT Rx | 1/2 | Alzheimer's disease | |||||
| IONIS-PKK-L Rx | 2 | Hereditary angioedema (full data) | |||||
| Vupanorsen | 2b | sHTG/CVD risk reduction | |||||
| Tofersen | 3 (VALOR study) | SOD1-ALS |
Anticipated 2021 Study Initiations
| Program | Phase | Anticipated Indication | H1 | H2 | |||
| SPINRAZA | 4 (RESPOND) | SMA, suboptimal gene therapy response | |||||
| Tofersen | 3 (ATLAS study) | Presymptomatic SOD1-ALS | |||||
| ION363 | 3 | FUS-ALS | |||||
| IONIS-AGT-L Rx | 2b | Resistant hypertension | |||||
| IONIS-AGT-L Rx | 2 | Heart failure with reduced ejection fraction | |||||
| ION373 | 2/3 | Alexander disease | |||||
| ION224 | 2b | NASH | |||||
| IONIS-APOCIII-L Rx | 3 | Second TG indication (sHTG) |
(2) Timing of partnered program catalysts based on partners' most recent publicly available disclosures
First Quarter 2021 Financial Results
Ionis' revenue was comprised of the following (amounts in millions):
| Three months ended, | ||||||||
| March 31, | ||||||||
| 2021 | 2020 | |||||||
| Revenue: | ||||||||
| Commercial revenue: | ||||||||
| SPINRAZA royalties | $ | 60 | $ | 66 | ||||
| TEGSEDI and WAYLIVRA revenue, net | 20 | 15 | ||||||
| Licensing and royalty revenue | 5 | 3 | ||||||
| Total commercial revenue | 85 | 84 | ||||||
| R&D Revenue: | ||||||||
| Amortization from upfront payments | 20 | 21 | ||||||
| Milestone payments | 5 | 23 | ||||||
| Other services | 2 | 5 | ||||||
| Total R&D revenue | 27 | 49 | ||||||
| Total revenue | $ | 112 | $ | 133 |
The Company's commercial revenue in the first quarter of 2021 was consistent with the same period last year. As the Company completes its transition of TEGSEDI
operations in North America to Sobi, the Company's commercial revenue from product sales will shift to distribution fees based on net sales generated by Sobi.
The Company's R&D revenue decreased in the first quarter of 2021 compared to the same period last year primarily because the Company earned more milestone payments
in the first quarter of 2020 than the same period this year. The Company expects its R&D revenue to increase in the second half of 2021 compared to the first half.
Financial Impacts of Akcea Acquisition and Restructured Operations
In conjunction with the Akcea acquisition and restructured European operations, in the first quarter of 2021, the Company incurred $7 million of costs, which it
excluded from its non-GAAP amounts for the period. Refer to the detailed reconciliation of non-GAAP and GAAP measures that is provided later in this release. The Company expects to incur additional expenses in the range of $11 million to $14
million related to the restructuring of its North American TEGSEDI operations from the expanded distribution agreement with Sobi. The company will reflect the North American TEGSEDI restructuring costs primarily in the second quarter of 2021.
Ionis' operating expenses for the first quarter of 2021 increased compared to the same period last year driven primarily by the Company's investments in advancing its
late-stage wholly owned pipeline.
Net Loss Attributable to Ionis Common Stockholders
Ionis' net loss attributable to Ionis' common stockholders for the first quarter of 2021 increased compared to the same period in the prior year for the reasons
Ionis ended March 2021 with cash, cash equivalents and short-term investments of $1.8 billion, compared to $1.9 billion at December 31,
2020. In April 2021, Ionis issued $632.5 million of 0% senior convertible notes due in April 2026 and repurchased $247.9 million of its 1% senior convertible notes. After reflecting these transactions, Ionis' pro forma cash, cash equivalents and
short-term investments was $2.1 billion.
The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on January 1, 2021.
Today, at 11:30 a.m. Eastern Time, Ionis will conduct a live webcast to discuss this earnings release 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 neurological and cardiometabolic franchises. Our scientific innovation began and continues with
the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies.
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) and WAYLIVRA (volanesorsen) and Ionis' technologies and 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" refers to Ionis Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals is a 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:
D. Wade Walke, Ph.D.
Vice President, Investor Relations
Ionis Pharmaceuticals Media Contact:
Vice President, Marketing and Communications
IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Millions, Except Per Share Data)
| Three months ended, March 31, | ||||||||
| 2021 | 2020 | |||||||
| (as revised*) | ||||||||
| (unaudited) | ||||||||
| Revenue: | ||||||||
| Commercial revenue: | ||||||||
| SPINRAZA royalties | $ | 60 | $ | 66 | ||||
| TEGSEDI and WAYLIVRA revenue, net | 20 | 15 | ||||||
| Licensing and royalty revenue | 5 | 3 | ||||||
| Total commercial revenue | 85 | 84 | ||||||
| Research and development revenue under collaborative agreements | 27 | 49 | ||||||
| Total revenue | 112 | 133 | ||||||
| Expenses: | ||||||||
| Cost of sales | 3 | 3 | ||||||
| Research, development and patent | 140 | 116 | ||||||
| Selling, general and administrative | 61 | 75 | ||||||
| Total operating expenses | 204 | 194 | ||||||
| Loss from operations | (92 | ) | (61 | ) | ||||
| Other income, net | 2 | 8 | ||||||
| Loss before income tax benefit | (90 | ) | (53 | ) | ||||
| Income tax benefit | - | 3 | ||||||
| Net loss | $ | (90 | ) | $ | (50 | ) | ||
| Net loss attributable to noncontrolling interest in Akcea Therapeutics, Inc. | - | 10 | ||||||
| Net loss attributable to Ionis Pharmaceuticals, Inc. common stockholders | $ | (90 | ) | $ | (40 | ) | ||
| Basic and diluted net loss per share | $ | (0.64 | ) | $ | (0.28 | ) | ||
| Shares used in computing basic and diluted net loss per share | 141 | 139 |
*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, Loss From Operations, and Net Loss
| Three months ended, March 31 , | ||||||||
| 2021 | 2020 | |||||||
| (as revised*) | ||||||||
| (unaudited) | ||||||||
| As reported research, development and patent expenses according to GAAP | $ | 140 | $ | 116 | ||||
| Excluding compensation expense related to equity awards | (26 | ) | (26 | ) | ||||
| Excluding Akcea acquisition and restructured European operations costs | (3 | ) | - | |||||
| Non-GAAP research, development and patent expenses | $ | 111 | $ | 90 | ||||
| As reported selling, general and administrative expenses according to GAAP | $ | 61 | $ | 75 | ||||
| Excluding compensation expense related to equity awards | (12 | ) | (15 | ) | ||||
| Excluding Akcea acquisition and restructured European operations costs | (4 | ) | - | |||||
| Non-GAAP selling, general and administrative expenses | $ | 45 | $ | 60 | ||||
| As reported operating expenses according to GAAP | $ | 204 | $ | 194 | ||||
| Excluding compensation expense related to equity awards | (38 | ) | (41 | ) | ||||
| Excluding Akcea acquisition and restructured European operations costs | (7 | ) | - | |||||
| Non-GAAP operating expenses | $ | 159 | $ | 153 | ||||
| As reported loss from operations according to GAAP | $ | (92 | ) | $ | (61 | ) | ||
| Excluding compensation expense related to equity awards | (38 | ) | (41 | ) | ||||
| Excluding Akcea acquisition and restructured European operations costs | (7 | ) | - | |||||
| Non-GAAP loss from operations | $ | (47 | ) | $ | (20 | ) | ||
| As reported net loss attributable to Ionis Pharmaceuticals, Inc. common stockholders according to GAAP * | $ | (90 | ) | $ | (40 | ) | ||
| Excluding compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc. common stockholders | (38 | ) | (39 | ) | ||||
| Excluding Akcea acquisition and restructured European operations costs | (7 | ) | - | |||||
| Income tax effect related to compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc. common stockholders | - | 8 | ||||||
| Non-GAAP net loss attributable to Ionis Pharmaceuticals, Inc. common stockholders* | $ | (45 | ) | $ | (9 | ) |
*The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on January 1, 2021.
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 acquisition and restructured European operations and the
related tax effects. Compensation expense related to equity awards are non-cash. Costs related to the Akcea acquisition and restructured European operations include: severance costs, retention costs and other costs. 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
| March 31, 2021 | December 31, 2020 | |||||||
| (as revised*) | ||||||||
| (unaudited) | ||||||||
| Assets: | ||||||||
| Cash, cash equivalents and short-term investments | $ | 1,820 | $ | 1,892 | ||||
| Contracts receivable | 23 | 76 | ||||||
| Other current assets | 146 | 162 | ||||||
| Property, plant and equipment, net | 180 | 181 | ||||||
| Other assets | 80 | 79 | ||||||
| Total assets | $ | 2,249 | $ | 2,390 | ||||
| Liabilities and stockholders' equity: | ||||||||
| Other current liabilities | $ | 126 | $ | 183 | ||||
| Current portion of 1% convertible senior notes, net | 62 | 309 | ||||||
| Current portion of deferred contract revenue | 107 | 108 | ||||||
| 1% convertible senior notes, less current portion | 247 | - | ||||||
| 0.125% convertible senior notes, net | 541 | 540 | ||||||
| Long-term obligations, less current portion | 83 | 83 | ||||||
| Long-term deferred contract revenue | 402 | 424 | ||||||
| Total stockholders' equity | 681 | 743 | ||||||
| Total liabilities and stockholders' equity | $ | 2,249 | $ | 2,390 |
*The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on January 1, 2021.