Full Press Release Details
Ionis reports third quarter 2021 financial results and recent business achievements
Olezarsen (IONIS-APOCIII-LRx) Phase 3 CORE study in patients with sHTG initiated
Donidalorsen (IONIS-PKK-LRx) Phase 2 data to be presented at ACAAI Annual Meeting; Phase 3 initiation on track for year-end
Tofersen Phase 3 VALOR study missed primary endpoint; signs of reduced disease progression observed across multiple secondary and exploratory
Webcast today, November 3, 2021, at 11:30 a.m. Eastern Time
CARLSBAD, Calif., November 3, 2021 - Ionis Pharmaceuticals, Inc. (Nasdaq: IONS)
today reported financial results for the three and nine months ended September 30, 2021 and recent business achievements.
"Among recent business highlights, we further expanded our broad late-stage pipeline to seven Phase 3 programs now that the olezarsen Phase 3 CORE study in patients with
severe hypertriglyceridemia is underway. We were encouraged that while tofersen did not achieve the primary endpoint in the Phase 3 VALOR study, trends favoring tofersen were seen across multiple secondary and exploratory measures of disease
progression in patients with SOD1-ALS. We also expanded our LICA platform capabilities by licensing technology from Bicycle Therapeutics," said Brett P. Monia, Ph.D., chief
executive officer of Ionis. "We are looking forward to multiple near-term catalysts, beginning with the presentation of donidalorsen Phase 2 data in patients with hereditary angioedema at the ACAAI Annual Meeting this weekend. We anticipate
initiating the donidalorsen Phase 3 study before year-end. We also expect multiple catalysts in 2022, including results from the eplontersen Phase 3 study in patients with TTR polyneuropathy mid-year 2022. Furthermore, at our virtual investor day on
December 9th, we look forward to outlining commercial preparations for our lead programs ahead of our first potential launch with eplontersen in patients with TTR polyneuropathy in 2023. Based on our anticipated near- and mid-term catalysts, we
remain on track to have 12 or more products on the market in 2026."
Third Quarter 2021 Financial Results
"Since our last quarterly update, we further advanced our strategic objectives through investments in our expanding Phase 3 pipeline and technology. We also advanced our
commercial readiness initiatives in anticipation of multiple product launches potentially beginning as early as 2023," said Elizabeth L. Hougen, chief financial officer of Ionis.
"We remain on track to achieve our 2021 financial guidance driven by increased R&D revenue in the fourth quarter as several of our partner programs advance. We also project increased expenses in the fourth quarter as we continue to invest for
growth. Importantly, with $2 billion in cash, we remain well-capitalized with the resources we need to achieve our strategic objectives."
Third Quarter 2021 Marketed Products Highlights
Third Quarter 2021 and Recent Events
2021 Pipeline Milestones(2)
Anticipated Key 2021 Data Readouts
| Program | Phase | Anticipated Indication | H1 | H2 | |||
| Donidalorsen | 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-MAPT Rx | 1/2 | Alzheimer's disease | |||||
| Tofersen | 3 (VALOR) | SOD1-ALS | |||||
| Cimdelirsen | 2 + OLE | Acromegaly | |||||
| Donidalorsen | 2 | Hereditary angioedema (full data) | |||||
| ION449 (PCSK9) | 1 (MAD) | Dyslipidemia | |||||
| Vupanorsen | 2b | sHTG/CVD risk reduction |
Anticipated Key 2021 Study Initiations
| Program | Phase | Anticipated Indication | H1 | H2 | |||
| SPINRAZA | 4 (RESPOND) | SMA, suboptimal gene therapy response | |||||
| Tofersen | 3 (ATLAS) | Presymptomatic SOD1-ALS | |||||
| ION363 | 3 | FUS-ALS | |||||
| IONIS-AGT-L Rx | 2 & 2b | Heart failure & resistant hypertension | |||||
| ION373 | 2/3 | Alexander disease | |||||
| ION224 | 2b | NASH | |||||
| Olezarsen | 3 (CORE) | Severe hypertriglyceridemia (sHTG) | |||||
| Donidalorsen | 3 | Hereditary angioedema | |||||
| SPINRAZA | 3b (ASCEND) | SMA, previous risdiplam treatment | |||||
| ION582 | 2 | Angelman syndrome |
= achieved = planned
(2) Timing of partnered program catalysts based on partners' most recent publicly available disclosures
Third Quarter 2021 Financial Results
Ionis' revenue was comprised of the following (amounts in millions):
| Three months ended, September 30, | Nine months ended September 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| Revenue: | ||||||||||||||||
| Commercial revenue: | ||||||||||||||||
| SPINRAZA royalties | $ | 67 | $ | 74 | $ | 199 | $ | 212 | ||||||||
| TEGSEDI and WAYLIVRA revenue, net | 15 | 19 | 47 | 51 | ||||||||||||
| Licensing and royalty revenue | 3 | 2 | 9 | 6 | ||||||||||||
| Total commercial revenue | 85 | 95 | 255 | 269 | ||||||||||||
| R&D revenue: | ||||||||||||||||
| Amortization from upfront payments | 17 | 19 | 57 | 68 | ||||||||||||
| Milestone payments | 28 | 44 | 48 | 73 | ||||||||||||
| License fees | - | - | - | 15 | ||||||||||||
| Other services | 3 | 2 | 10 | 14 | ||||||||||||
| Total R&D revenue | 48 | 65 | 115 | 170 | ||||||||||||
| Total revenue | $ | 133 | $ | 160 | $ | 370 | $ | 439 |
In the third quarter of 2021, the Company continued to advance its late-stage pipeline, including reaching 50 percent enrollment in the Phase 3 Lp(a) HORIZON study of
pelacarsen for which it earned a $25 million milestone payment from Novartis. As its partnered programs advance, the Company expects R&D revenue to increase in the fourth quarter of 2021 compared with the third quarter of 2021.
In the second quarter of 2021, the Company successfully completed the transition of its TEGSEDI operations in North America to Sobi. As a result, the Company's commercial revenue from product sales shifted to distribution fees based on net sales generated by Sobi. In the third quarter of 2021, the Company earned a $4 million milestone payment
from PTC Therapeutics when WAYLIVRA was approved in Brazil.
Financial Impacts of Akcea Merger and Restructured Commercial Operations
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. For the three and nine months ended
September 30, 2021, the Company incurred $3 million and $24 million of costs in conjunction with the Akcea merger and restructuring of the Company's commercial operations, respectively. The Company excluded these costs from its non-GAAP amounts for
those periods. Please refer to the detailed reconciliation of non-GAAP and GAAP measures that is provided later in this press release.
Ionis' operating expenses for the three and nine months ended September 30, 2021 increased compared with the same periods last year driven by an increase in R&D
expenses, partially offset by a decrease in SG&A expenses. Higher R&D expenses were primarily driven by the Company's investments in advancing its late-stage wholly owned pipeline, including advancing the Phase 3 program for eplontersen and
start-up costs associated with the Phase 3 study for a second indication for olezarsen. Additionally, the Company recognized $35 million in R&D expense in the third quarter of 2021 for licensing Bicycle's technology. Lower SG&A expenses
primarily reflect operating efficiencies achieved from integrating Akcea and restructuring the Company's commercial operations. The Company projects its operating expenses to increase in the fourth quarter as it continues to invest for growth.
Net Loss Attributable to Ionis Common Stockholders
Net loss attributable to Ionis' common stockholders for the three and nine months ended September 30, 2021 increased compared with the same periods in the prior year for
the reasons discussed above.
As of September 30, 2021 Ionis had cash, cash equivalents and short-term investments of $2.0 billion, compared with $1.9 billion as of December 31, 2020. The Company
intends to utilize $62 million of its cash to pay the remaining principal balance of its 1 percent convertible notes at maturity in November 2021.
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 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" all refer 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:
Ionis Pharmaceuticals Media Contact:
IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Millions, Except Per Share Data)
| Three months ended, ended, September 30, | Nine months ended, September 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| (as revised*) | (as revised*) | |||||||||||||||
| (unaudited) | ||||||||||||||||
| Revenue: | ||||||||||||||||
| Commercial revenue: | ||||||||||||||||
| SPINRAZA royalties | $ | 67 | $ | 74 | $ | 199 | $ | 212 | ||||||||
| TEGSEDI and WAYLIVRA revenue, net | 15 | 19 | 47 | 51 | ||||||||||||
| Licensing and royalty revenue | 3 | 2 | 9 | 6 | ||||||||||||
| Total commercial revenue | 85 | 95 | 255 | 269 | ||||||||||||
| Research and development revenue under collaborative agreements | 48 | 65 | 115 | 170 | ||||||||||||
| Total revenue | 133 | 160 | 370 | 439 | ||||||||||||
| Expenses: | ||||||||||||||||
| Cost of sales | 3 | 3 | 9 | 9 | ||||||||||||
| Research, development and patent | 185 | 125 | 464 | 364 | ||||||||||||
| Selling, general and administrative | 31 | 69 | 148 | 215 | ||||||||||||
| Total operating expenses | 219 | 197 | 621 | 588 | ||||||||||||
| Loss from operations | (86 | ) | (37 | ) | (251 | ) | (149 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Loss on early retirement of debt | - | - | (9 | ) | - | |||||||||||
| Other income, net | 2 | 5 | 6 | 29 | ||||||||||||
| Loss before income tax benefit (expense) | (84 | ) | (32 | ) | (254 | ) | (120 | ) | ||||||||
| Income tax benefit (expense) | 2 | (5 | ) | 1 | (4 | ) | ||||||||||
| Net loss | $ | (82 | ) | $ | (37 | ) | $ | (253 | ) | $ | (124 | ) | ||||
| Net loss attributable to noncontrolling interest in Akcea Therapeutics, Inc. | $ | - | $ | 13 | $ | - | $ | 34 | ||||||||
| Net loss attributable to Ionis Pharmaceuticals, Inc. common stockholders | $ | (82 | ) | $ | (24 | ) | $ | (253 | ) | $ | (90 | ) | ||||
| Basic and diluted net loss per share | $ | (0.58 | ) | $ | (0.18 | ) | $ | (1.80 | ) | $ | (0.64 | ) | ||||
| Shares used in computing basic and diluted net loss per share | 141 | 140 | 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, Income (Loss) From Operations, and Net Income (Loss)
| Three months ended, September 30 , | Nine months ended, September 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| (as revised*) | (as revised*) | |||||||||||||||
| (unaudited) | ||||||||||||||||
| As reported research, development and patent expenses according to GAAP | $ | 185 | $ | 125 | $ | 464 | $ | 364 | ||||||||
| Excluding compensation expense related to equity awards | (23 | ) | (25 | ) | (72 | ) | (77 | ) | ||||||||
| Excluding Akcea merger and restructured commercial operation costs** | (2 | ) | - | (8 | ) | - | ||||||||||
| Non-GAAP research, development and patent expenses | $ | 160 | $ | 100 | $ | 384 | $ | 287 | ||||||||
| As reported selling, general and administrative expenses according to GAAP | $ | 31 | $ | 69 | $ | 148 | $ | 215 | ||||||||
| Excluding compensation expense related to equity awards | (7 | ) | (20 | ) | (26 | ) | (57 | ) | ||||||||
| Excluding Akcea merger and restructured commercial operation costs** | (1 | ) | - | (16 | ) | - | ||||||||||
| Non-GAAP selling, general and administrative expenses | $ | 23 | $ | 49 | $ | 106 | $ | 158 | ||||||||
| As reported operating expenses according to GAAP | $ | 219 | $ | 197 | $ | 621 | $ | 588 | ||||||||
| Excluding compensation expense related to equity awards | (31 | ) | (46 | ) | (98 | ) | (135 | ) | ||||||||
| Excluding Akcea merger and restructured commercial operation costs** | (3 | ) | - | (24 | ) | - | ||||||||||
| Non-GAAP operating expenses | $ | 185 | $ | 151 | $ | 499 | $ | 453 | ||||||||
| As reported loss from operations according to GAAP | $ | (86 | ) | $ | (37 | ) | $ | (251 | ) | $ | (149 | ) | ||||
| Excluding compensation expense related to equity awards | (31 | ) | (46 | ) | (98 | ) | (135 | ) | ||||||||
| Excluding Akcea merger and restructured commercial operation costs** | (3 | ) | - | (24 | ) | - | ||||||||||
| Non-GAAP income (loss) from operations | $ | (52 | ) | $ | 9 | $ | (129 | ) | $ | (14 | ) | |||||
| As reported net loss attributable to Ionis Pharmaceuticals, Inc. common stockholders according to GAAP | $ | (82 | ) | $ | (24 | ) | $ | (253 | ) | $ | (90 | ) | ||||
| Excluding compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc. common stockholders | (31 | ) | (42 | ) | (98 | ) | (126 | ) | ||||||||
| Excluding Akcea merger and restructured commercial operation costs** | (3 | ) | - | (24 | ) | - | ||||||||||
| Income tax effect related to compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc. common stockholders | - | 6 | - | 18 | ||||||||||||
| Non-GAAP net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders according to GAAP | $ | (48 | ) | $ | 12 | $ | (131 | ) | $ | 18 |
*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.
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
| September 30, | December 31, | |||||||
| 2021 | 2020 | |||||||
| (as revised*) | ||||||||
| (unaudited) | ||||||||
| Assets: | ||||||||
| Cash, cash equivalents and short-term investments | $ | 1,987 | $ | 1,892 | ||||
| Contracts receivable | 9 | 76 | ||||||
| Other current assets | 160 | 162 | ||||||
| Property, plant and equipment, net | 180 | 181 | ||||||
| Other assets | 79 | 79 | ||||||
| Total assets | $ | 2,415 | $ | 2,390 | ||||
| Liabilities and stockholders' equity: | ||||||||
| Other current liabilities | $ | 120 | $ | 183 | ||||
| Current portion of 1% convertible senior notes, net | 62 | 309 | ||||||
| Current portion of deferred contract revenue | 98 | 108 | ||||||
| 0 % convertible senior notes, net | 618 | - | ||||||
| 0.125% convertible senior notes, net | 542 | 540 | ||||||
| Long-term obligations, less current portion | 81 | 83 | ||||||
| Long-term deferred contract revenue | 363 | 424 | ||||||
| Total stockholders' equity | 531 | 743 | ||||||
| Total liabilities and stockholders' equity | $ | 2,415 | $ | 2,390 |
*The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on January 1, 2021.