Press Releases
Ionis reports first quarter 2021 financial results and recent business achievements
"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
First Quarter 2021 and Recent Summary Financial Results
- On track to achieve 2021 financial guidance reflecting investments in Ionis' wholly owned pipeline, based on the following first quarter results
$112 million in total revenues$159 million of operating expenses on a non-GAAP basis(1) and$204 million on a GAAP basis- Net loss of
$45 million on a non-GAAP basis(1) and$90 million on a GAAP basis - Further strengthened the Company's balance sheet with pro forma cash of
$2.1 billion , after reflecting the convertible notes transaction - Enables expansion of manufacturing and R&D capacity
$632.5 million principal due inApril 2026 with 0% interest and an effective conversion price of$76.39 after the purchase of a call spread- Will realize interest expense savings while keeping potential future dilution nearly flat
- Repurchased approximately 80% of previously outstanding 1% convertible notes due in
November 2021
"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
(1) |
All non-GAAP amounts referred to in this press release exclude non-cash compensation expense related to equity awards and expenses related to the Akcea acquisition and restructured European operations and the related tax effects. Please refer to the section below titled "Financial Impacts of Akcea Acquisition and Restructured Operations" for a summary of the costs specific to these transactions. Additionally, please refer to the detailed reconciliation of non-GAAP and GAAP measures, which is provided later in this release. |
First Quarter 2021 Marketed Products Highlights
- SPINRAZA: a global foundation-of-care for the treatment of spinal muscular atrophy (SMA) patients of all ages
$521 million in worldwide sales in the first quarter- More than 11,000 patients worldwide were on therapy at the end of the first quarter across post-marketing, expanded access and clinical trial settings
- Higher-dose SPINRAZA demonstrated safety and tolerability consistent with the currently approved dose in the open-label safety cohort of the DEVOTE study, enabling enrollment in the blinded, pivotal cohort to get underway
- TEGSEDI and WAYLIVRA: important medicines approved for the treatment of patients with severe rare diseases
- Completed the transition of European operations to Swedish Orphan Biovitrum AB (Sobi) and expanded the distribution agreement to include North American TEGSEDI operations
First Quarter 2021 and Recent Pipeline Events
- Phase 3 Pipeline: growing and positioned for 12 or more products on the market in 2026
- Advanced ION363 into a Phase 3 study in patients with FUS-ALS
- Advanced tofersen into the Phase 3 ATLAS study in presymptomatic SOD1-ALS patients
- Roche reported tominersen data related to the dosing halt in the Phase 3 program
- Mid-stage Pipeline: advancing multiple medicines with potential to change the standard of care for patients with severe diseases
- Reported positive topline IONIS-PKK-LRx results in patients with hereditary angioedema
- Advanced ION373 into the Phase 2 portion of a pivotal study in patients with Alexander disease
- Advanced the IONIS-AGT-LRx development program:
- Reported positive Phase 2 data in JACC: Basic to Translational Science
- Advanced into a Phase 2b study in patients with hypertension uncontrolled with three or more antihypertensive medications
- Advanced into a Phase 2 study in patients with chronic heart failure with reduced injection fraction
- Advanced the ongoing Phase 2 study of ION541 in patients with ALS regardless of family history, resulting in a
$10 million payment from Biogen
Upcoming 2021 Pipeline Catalysts(2)
Anticipated 2021 Data Readouts |
||||
Program |
Phase |
Anticipated Indication |
H1 |
H2 |
IONIS-PKK-LRx |
2 |
Hereditary angioedema (top-line data) |
√ |
|
IONIS-AGT-LRx |
2 |
Hypertension |
√ |
|
Tominersen |
3 |
Huntington's disease |
√ |
|
IONIS-ENAC-2.5Rx |
2 |
Cystic fibrosis |
• |
|
IONIS-GHR-LRx |
2+OLE |
Acromegaly |
• |
|
IONIS-MAPTRx |
1/2 |
Alzheimer's disease |
• |
|
IONIS-PKK-LRx |
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-LRx |
2b |
Resistant hypertension |
√ |
|
IONIS-AGT-LRx |
2 |
Heart failure with reduced ejection fraction |
√ |
|
ION373 |
2/3 |
Alexander disease |
√ |
|
ION224 |
2b |
NASH |
• |
|
IONIS-APOCIII-LRx |
3 |
Second TG indication (sHTG) |
• |
(2) Timing of partnered program catalysts based on partners' most recent publicly available disclosures |
First Quarter 2021 Financial Results
Revenue
Ionis' revenue was comprised of the following (amounts in millions):
Three months ended, |
|||||
|
|||||
2021 |
2020 |
||||
Revenue: |
|||||
Commercial revenue: |
|||||
SPINRAZA royalties |
|
|
|||
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 |
|
|
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
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
Operating Expenses
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 discussed above.
Balance Sheet
Ionis ended
The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on
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About
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.
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 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
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to
|
|||||||
SELECTED FINANCIAL INFORMATION |
|||||||
Condensed Consolidated Statements of Operations |
|||||||
(In Millions, Except Per Share Data) |
|||||||
Three months ended, |
|||||||
|
|||||||
2021 |
2020 |
||||||
(as revised*) |
|||||||
(unaudited) |
|||||||
Revenue: |
|||||||
Commercial revenue: |
|||||||
SPINRAZA royalties |
|
|
|||||
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 |
( |
( |
|||||
Net loss attributable to noncontrolling interest in |
- |
10 |
|||||
Net loss attributable to |
( |
( |
|||||
Basic and diluted net loss per share |
( |
( |
|||||
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
|
|||||
Reconciliation of GAAP to Non-GAAP Basis: |
|||||
Condensed Consolidated Operating Expenses, Loss From Operations, and Net Loss |
|||||
(In Millions) |
|||||
Three months ended, |
|||||
2021 |
2020 |
||||
(as revised*) |
|||||
(unaudited) |
|||||
As reported research, development and patent expenses according to GAAP |
|
|
|||
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 |
|
|
|||
As reported selling, general and administrative expenses according to GAAP |
|
|
|||
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 |
|
|
|||
As reported operating expenses according to GAAP |
|
|
|||
Excluding compensation expense related to equity awards |
(38) |
(41) |
|||
Excluding Akcea acquisition and restructured European operations costs |
(7) |
- |
|||
Non-GAAP operating expenses |
|
|
|||
As reported loss from operations according to GAAP |
( |
( |
|||
Excluding compensation expense related to equity awards |
(38) |
(41) |
|||
Excluding Akcea acquisition and restructured European operations costs |
(7) |
- |
|||
Non-GAAP loss from operations |
( |
( |
|||
As reported net loss attributable to |
( |
( |
|||
Excluding compensation expense related to equity awards attributable to |
(38) |
(39) |
|||
Excluding Akcea acquisition and restructured European operations costs |
(7) |
- |
|||
Income tax effect related to compensation expense related to equity awards attributable to |
- |
8 |
|||
Non-GAAP net loss attributable to |
( |
( |
*The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on
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
|
||||||
Condensed Consolidated Balance Sheets |
||||||
(In Millions) |
||||||
|
|
|||||
2021 |
2020 |
|||||
(as revised*) |
||||||
(unaudited) |
||||||
Assets: |
||||||
Cash, cash equivalents and short-term investments |
|
|
||||
Contracts receivable |
23 |
76 |
||||
Other current assets |
146 |
162 |
||||
Property, plant and equipment, net |
180 |
181 |
||||
Other assets |
80 |
79 |
||||
Total assets |
|
|
||||
Liabilities and stockholders' equity: |
||||||
Other current liabilities |
|
|
||||
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 |
|
|
||||
*The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on
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SOURCE
Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph.D., Vice President, Investor Relations, 760-603-2741; Ionis Pharmaceuticals Media Contact: Roslyn Patterson, Vice President, Marketing and Communications, 760-603-2681