Full Press Release Details
Ionis reports second quarter 2021 financial results and recent business achievements
Key Phase 3 milestones move tofersen, eplontersen and pelacarsen closer to the market
Exclusive license to Bicycle Therapeutics' technology potentially expands LICA technology capabilities
Webcast today, August 4, 2021, at 11:30 a.m. Eastern Time
CARLSBAD, Calif., August 4, 2021 - Ionis
Pharmaceuticals, Inc. (Nasdaq: IONS) today reported its financial results for the second quarter of 2021 and recent business achievements.
"Since our last quarterly update, we continued to execute on our strategic objectives to prepare for multiple Ionis commercial launches, expand our drug
delivery capabilities and advance new products towards the market. Biogen completed dosing in the tofersen Phase 3 VALOR study and began offering tofersen to SOD1-ALS patients on an individual compassionate use basis. We achieved full enrollment in
the eplontersen Phase 3 NEURO-TTRansform study and 50 percent enrollment in the pelacarsen Phase 3 Lp(a) HORIZON study. Additionally, we licensed Bicycle Therapeutics' technology to expand the capabilities of our LICA technology," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "Looking ahead, we expect data from multiple pipeline programs, including additional data supporting
the potential for our IONIS-PKK-LRx program to change the standard of care for patients with hereditary angioedema. And by this fall, we expect 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 recent achievements and upcoming catalysts keep us on track for a regular cadence of Phase 3 data and new drug applications, leading to 12 or more products
on the market in 2026."
Second Quarter 2021 and Recent Summary Financial Results
"In addition to advancing our pipeline and expanding our drug discovery capabilities, we have taken multiple steps to streamline our operations in
support of our wholly owned medicines. We have completed the integration of Akcea, entered distribution arrangements with Sobi and restructured our commercial
operations. These steps enabled us to unlock significant resources that we are redirecting towards our highest priority programs," said Elizabeth L. Hougen, chief financial officer of Ionis. "We remain on track to achieve our 2021 revenue guidance of more than $600 million. We continue to expect increased R&D revenue in the second half of this year. Already in the third quarter, we earned $25
million from Novartis for the pelacarsen enrollment milestone. We are revising our 2021 operating expense and net loss guidance because of our license of Bicycle's technology. Importantly, we remain well-capitalized with the resources we need to
achieve our strategic objectives."
Revised 2021 Financial Guidance
| Prior 2021 Guidance | Revised 2021 Guidance | |
| Revenue | >$600 million | Unchanged |
| Operating Expenses (1) | $675 million to $725 million | $710 million to $750 million |
| Net Loss (1) | <$75 million | <$110 million |
Second Quarter 2021 Marketed Products Highlights
Second Quarter 2021 and Recent Events
Upcoming 2021 Pipeline Catalysts(2)
| Anticipated Key 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-MAPT Rx | 1/2 | Alzheimer's disease | |||||
| Tofersen | 3 (VALOR) | SOD1-ALS | |||||
| Vupanorsen | 2b | sHTG/CVD risk reduction | |||||
| IONIS-PKK-L Rx | 2 | Hereditary angioedema (full data) | |||||
| IONIS-GHR-L Rx | 2 + OLE | Acromegaly |
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 | 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) | |||||
| ION582 | 2 | Angelman Syndrome |
(2) Timing of partnered program catalysts based on partners' most recent publicly available disclosures
Second Quarter 2021 Financial Results
Ionis' revenue was comprised of the following (amounts in millions):
| Three months ended, June 3 0, | Six months ended June 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| Revenue: | ||||||||||||||||
| Commercial revenue: | ||||||||||||||||
| SPINRAZA royalties | $ | 72 | $ | 72 | $ | 132 | $ | 138 | ||||||||
| TEGSEDI and WAYLIVRA revenue, net | 12 | 16 | 31 | 32 | ||||||||||||
| Licensing and royalty revenue | 2 | 2 | 7 | 4 | ||||||||||||
| Total commercial revenue | 86 | 90 | 170 | 174 | ||||||||||||
| R&D Revenue: | ||||||||||||||||
| Amortization from upfront payments | 20 | 28 | 40 | 49 | ||||||||||||
| Milestone payments | 15 | 7 | 20 | 30 | ||||||||||||
| License fees | - | 15 | - | 15 | ||||||||||||
| Other services | 5 | 6 | 7 | 11 | ||||||||||||
| Total R&D revenue | 40 | 56 | 67 | 105 | ||||||||||||
| Total revenue | $ | 126 | $ | 146 | $ | 237 | $ | 279 |
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.
The Company's R&D revenue decreased in the second quarter of 2021 compared to the same period last year primarily because the Company earned more
milestone payments in the second 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 as its partnered programs advance. Already in the third quarter of 2021, the Company earned a $25 million milestone payment from Novartis when Novartis achieved 50 percent enrollment in the Phase 3 Lp(a) HORIZON study
Financial Impacts of Akcea Acquisition and Restructured Commercial Operations
In the second quarter of 2021, the Company incurred $15 million of costs in conjunction with the Akcea acquisition and restructuring of the Company's
commercial operations. The Company excluded these costs 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.
Ionis' operating expenses for the second quarter of 2021 increased slightly compared to the same period last year driven by an increase in R&D
expenses, partially offset by a decrease in SG&A expenses. Ionis' increased R&D expenses were primarily driven by the Company's investments in advancing its late-stage wholly owned pipeline. Ionis' decreased
SG&A expenses were primarily from operating efficiencies achieved from integrating Akcea and restructuring the Company's commercial operations.
Net Loss Attributable to Ionis Common Stockholders
Ionis' net loss attributable to Ionis' common stockholders for the second quarter of 2021 increased compared to the same period in the prior year for the
reasons discussed above. Additionally, the Company recognized an $8.6 million non-cash loss from the early retirement of a significant portion of its 1 percent senior convertible notes.
Ionis ended June 2021 with cash, cash equivalents and short-term investments of $2.1 billion, compared to $1.9 billion at
December 31, 2020. In April 2021, Ionis issued $632.5 million of 0 percent senior convertible notes due in April 2026 and repurchased $247.9 million of its 1
percent senior convertible notes. The Company's remaining $62 million of 1 percent senior convertible notes mature in November 2021.
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
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:
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, ended, June 30, | Six months ended, June 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| (as revised*) | (as revised*) | |||||||||||||||
| (unaudited) | ||||||||||||||||
| Revenue: | ||||||||||||||||
| Commercial revenue: | ||||||||||||||||
| SPINRAZA royalties | $ | 72 | $ | 72 | $ | 132 | $ | 138 | ||||||||
| TEGSEDI and WAYLIVRA revenue, net | 12 | 16 | 31 | 32 | ||||||||||||
| Licensing and royalty revenue | 2 | 2 | 7 | 4 | ||||||||||||
| Total commercial revenue | 86 | 90 | 170 | 174 | ||||||||||||
| Research and development revenue under collaborative agreements | 40 | 56 | 67 | 105 | ||||||||||||
| Total revenue | 126 | 146 | 237 | 279 | ||||||||||||
| Expenses: | ||||||||||||||||
| Cost of sales | 3 | 3 | 6 | 6 | ||||||||||||
| Research, development and patent | 139 | 122 | 279 | 239 | ||||||||||||
| Selling, general and administrative | 57 | 73 | 117 | 147 | ||||||||||||
| Total operating expenses | 199 | 198 | 402 | 392 | ||||||||||||
| Loss from operations | (73 | ) | (52 | ) | (165 | ) | (113 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Loss on early retirement of debt | (8 | ) | - | (8 | ) | - | ||||||||||
| Other income, net | - | 17 | 3 | 25 | ||||||||||||
| Loss before income tax (expense) benefit | (81 | ) | (35 | ) | (170 | ) | (88 | ) | ||||||||
| Income tax (expense) benefit | - | (3 | ) | (1 | ) | 1 | ||||||||||
| Net loss | $ | (81 | ) | $ | (38 | ) | $ | (171 | ) | $ | (87 | ) | ||||
| Net loss attributable to noncontrolling interest in Akcea Therapeutics, Inc. | - | 12 | - | 22 | ||||||||||||
| Net loss attributable to Ionis Pharmaceuticals, Inc. common stockholders | $ | (81 | ) | $ | (26 | ) | $ | (171 | ) | $ | (65 | ) | ||||
| Basic and diluted net loss per share | $ | (0.57 | ) | $ | (0.18 | ) | $ | (1.21 | ) | $ | (0.47 | ) | ||||
| Shares used in computing basic and diluted net loss per share | 141 | 139 | 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 Income (Loss)
| Three months ended, June 30 , | Six months ended, June 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| (as revised*) | (as revised*) | |||||||||||||||
| (unaudited) | ||||||||||||||||
| As reported research, development and patent expenses according to GAAP | $ | 139 | $ | 122 | $ | 279 | $ | 239 | ||||||||
| Excluding compensation expense related to equity awards | (23 | ) | (26 | ) | (49 | ) | (52 | ) | ||||||||
| Excluding Akcea acquisition and restructured commercial operation costs | (4 | ) | - | (6 | ) | - | ||||||||||
| Non-GAAP research, development and patent expenses | $ | 112 | $ | 96 | $ | 224 | $ | 187 | ||||||||
| As reported selling, general and administrative expenses according to GAAP | $ | 57 | $ | 73 | $ | 117 | $ | 147 | ||||||||
| Excluding compensation expense related to equity awards | (7 | ) | (22 | ) | (19 | ) | (37 | ) | ||||||||
| Excluding Akcea acquisition and restructured commercial operation costs | (11 | ) | - | (16 | ) | - | ||||||||||
| Non-GAAP selling, general and administrative expenses | $ | 39 | $ | 51 | $ | 82 | $ | 110 | ||||||||
| As reported operating expenses according to GAAP | $ | 199 | $ | 198 | $ | 402 | $ | 392 | ||||||||
| Excluding compensation expense related to equity awards | (30 | ) | (48 | ) | (68 | ) | (89 | ) | ||||||||
| Excluding Akcea acquisition and restructured commercial operation costs | (15 | ) | - | (22 | ) | - | ||||||||||
| Non-GAAP operating expenses | $ | 154 | $ | 150 | $ | 312 | $ | 303 | ||||||||
| As reported loss from operations according to GAAP | $ | (73 | ) | $ | (52 | ) | $ | (165 | ) | $ | (113 | ) | ||||
| Excluding compensation expense related to equity awards | (30 | ) | (48 | ) | (68 | ) | (89 | ) | ||||||||
| Excluding Akcea acquisition and restructured commercial operation costs | (15 | ) | - | (22 | ) | - | ||||||||||
| Non-GAAP loss from operations | $ | (28 | ) | $ | (4 | ) | $ | (75 | ) | $ | (24 | ) | ||||
| As reported net loss attributable to Ionis Pharmaceuticals, Inc. common stockholders according to GAAP | $ | (81 | ) | $ | (26 | ) | $ | (171 | ) | $ | (65 | ) | ||||
| Excluding compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc. common stockholders | (30 | ) | (45 | ) | (68 | ) | (84 | ) | ||||||||
| Excluding Akcea acquisition and restructured commercial operation costs | (15 | ) | - | (22 | ) | - | ||||||||||
| Income tax effect related to compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc. common stockholders | - | 4 | - | 12 | ||||||||||||
| Non-GAAP net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders according to GAAP | $ | (36 | ) | $ | 15 | $ | (81 | ) | $ | 7 |
*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 commercial
operations and the related tax effects. Compensation expense related to equity awards are non-cash. Costs related to the Akcea acquisition 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
| June 30, 2021 | December 31, 2020 | |||||||
| (as revised*) | ||||||||
| (unaudited) | ||||||||
| Assets: | ||||||||
| Cash, cash equivalents and short-term investments | $ | 2,059 | $ | 1,892 | ||||
| Contracts receivable | 24 | 76 | ||||||
| Other current assets | 155 | 162 | ||||||
| Property, plant and equipment, net | 179 | 181 | ||||||
| Other assets | 80 | 79 | ||||||
| Total assets | $ | 2,497 | $ | 2,390 | ||||
| Liabilities and stockholders' equity: | ||||||||
| Other current liabilities | $ | 130 | $ | 183 | ||||
| Current portion of 1% convertible senior notes, net | 62 | 309 | ||||||
| Current portion of deferred contract revenue | 102 | 108 | ||||||
| 0 % convertible senior notes, net | 617 | - | ||||||
| 0.125% convertible senior notes, net | 541 | 540 | ||||||
| Long-term obligations, less current portion | 82 | 83 | ||||||
| Long-term deferred contract revenue | 380 | 424 | ||||||
| Total stockholders' equity | 583 | 743 | ||||||
| Total liabilities and stockholders' equity | $ | 2,497 | $ | 2,390 |
*The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on January 1, 2021.