Press Releases
Ionis reports first quarter 2020 financial results and recent business achievements
"We achieved numerous value-driving catalysts in the first quarter, setting us up to achieve our strategic objectives for the year. Our commercial medicines continued to perform well, led by SPINRAZA. Our late-stage pipeline continues to advance, and we are particularly pleased that the tominersen Phase 3 study is fully enrolled, bringing us closer to potentially delivering this medicine to patients with Huntington's disease. Additionally, AKCEA-APO(a)-LRx was granted fast track designation in the
Financial Results and Highlights
"We are reaffirming our 2020 financial guidance, including ending 2020 meaningfully profitable. We expect our results to be driven by continued significant commercial revenue and R&D revenue from numerous programs," said
- Growing commercial revenues combined with a substantial base of R&D revenues
- Commercial revenue from SPINRAZA® (nusinersen) royalties increased by more than 10 percent to
$66 million compared to Q1 2019 - Product sales from TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen) more than doubled to
$15 million compared to Q1 2019 - R&D revenue of
$49 million included over$25 million from Ionis' neurology disease franchise and$15 million from its cardiometabolic franchise - First quarter results in line with projections to be meaningfully profitable this year
- Operating loss and net loss of
$61 million and$48 million , respectively, on a GAAP basis - Non-GAAP operating loss and net loss of
$20 million and$15 million , respectively - Cash position of
$2.4 billion provides substantial financial strength to continue executing on strategic goals
All non-GAAP amounts referred to in this press release exclude non-cash compensation expense related to equity awards. Please refer to the reconciliation of non-GAAP and GAAP measures, which is provided later in this release.
Commercial Medicine Highlights
- SPINRAZA: a global foundation-of-care for the treatment of spinal muscular atrophy (SMA) patients of all ages
- Worldwide sales increased to
$565 million in the first quarter, a 9 percent increase compared to the first quarter of 2019 - Worldwide patients on treatment increased to approximately 10,800 at the end of the first quarter, including patients across commercial, expanded access and clinical trial settings
- Patient treatment is underway in the Phase 2/3 DEVOTE study evaluating the safety, tolerability and potential to achieve even greater efficacy with a higher dose of SPINRAZA
- Data from an independent study published in Lancet Neurology demonstrated statistically significant improvement in motor function with SPINRAZA treatment in teens and adults
- TEGSEDI: launched in multiple markets for the treatment of hereditary transthyretin amyloidosis (hATTR) with polyneuropathy in adult patients
- Commercially available in 12 countries
- Launching in additional EU countries this year and expanding in
Latin America through PTC Therapeutics - Results from the NEURO-TTR Phase 3 open-label extension study were published in the
European Journal of Neurology - WAYLIVRA: launched in the EU as the only approved treatment for adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis
- Launch progressing in
Germany ,Austria and through the ATU inFrance - Launching in additional EU countries this year
Pipeline Highlights
- Roche completed enrollment in the global, GENERATION HD1 Phase 3 study in patients with Huntington's disease
- Initiated the CARDIO-TTRansform Phase 3 clinical trial for AKCEA-TTR-LRx in patients with TTR-mediated amyloid cardiomyopathy
- Two medicines granted Fast Track Designation by the
U.S. FDA - AKCEA-APO(a)-LRx for the treatment of cardiovascular disease due to elevated Lp(a) levels
- IONIS-C9Rx for the treatment of C9orf72-ALS
- Ionis generated more than
$20 million as numerous partnered medicines advanced $10 million from AstraZeneca for ION532, targeting APOL1 for the treatment of kidney disease$7.5 million from Biogen for IONIS-MAPTRx for the treatment of Alzheimer's disease$5 million from Dynacure for IONIS-DNM2-2.5Rx for the treatment of centronuclear myopathies- Ionis and Akcea reported positive topline results for AKCEA-APOCIII-LRx and vupanorsen (AKCEA-ANGPTL3-LRx)
- Results from the Phase 2 study of AKCEA-APO(a)-LRx in patients with Lp(a)-driven cardiovascular disease, highlighting the favorable safety and tolerability profile and the potential to address a major area of unmet need, were published in the
New England Journal of Medicine - Initiated a Phase 1 study of ION224, an Ionis-owned medicine in development for the treatment of NASH
Upcoming Catalysts
- Initiate the Phase 3 study of AKCEA-APOCIII-LRx in patients with
FCS - Refile WAYLIVRA new drug application for
U.S. marketing authorization - File for WAYLIVRA marketing approval in
Brazil with PTC Therapeutics - Report clinical proof-of-concept results for four or more programs
- Initiate a first-in-human study of ION541 in patients with sporadic ALS, conducted by Biogen
- Advance five or more new medicines into development, including several Ionis-owned medicines
Revenue
Ionis' revenue was comprised of the following (amounts in millions):
Three months ended, |
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|
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2020 |
2019 |
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Revenue: |
|||||
Commercial revenue: |
|||||
SPINRAZA royalties |
|
|
|||
Product sales, net |
15 |
7 |
|||
Licensing and royalty revenue |
3 |
1 |
|||
Total commercial revenue |
84 |
68 |
|||
R&D Revenue: |
|||||
Amortization from upfront payments |
21 |
36 |
|||
Milestone payments |
23 |
40 |
|||
License fees |
- |
150 |
|||
Other services |
5 |
3 |
|||
Total R&D revenue |
49 |
229 |
|||
Total revenue |
|
|
R&D revenue in the first quarter of 2019 included
Operating Expenses
Operating expenses increased for the first quarter of 2020, compared to the same period in 2019, principally due to Ionis' investments in the global launches of TEGSEDI and WAYLIVRA, the Phase 3 program for AKCEA-TTR-LRx and the Company's Ionis-owned pipeline.
Income Tax Expense (Benefit)
Ionis recorded an income tax benefit in the first quarter of 2020, compared to income tax expense in the same period in 2019. Ionis recorded an income tax benefit in the first quarter of 2020 because it generated a pre-tax loss.
Net (Income) Loss Attributable to Noncontrolling Interest in Akcea
At
Net Income (Loss) Attributable to Ionis Common Stockholders
Ionis' net loss attributable to Ionis' common stockholders for the first quarter of 2020 was primarily due to Ionis' investments in advancing its strategic priorities. Ionis' net income attributable to Ionis' common stockholders for the first quarter of 2019 was primarily due to the
Balance Sheet
Ionis ended the first quarter of 2020 with cash, cash equivalents and short-term investments of $2.4 billion, nearly flat compared to
Webcast
Today, at
About
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of diseases, including neurological, cardiovascular, infectious, and pulmonary diseases.
To learn more about Ionis visit www.ionispharma.com or follow us on twitter @ionispharma.
*Spinraza is marketed by Biogen.
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, including the business of
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to
|
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SELECTED FINANCIAL INFORMATION |
|||
Condensed Consolidated Statements of Operations |
|||
(In Millions, Except Per Share Data) |
|||
Three months ended |
|||
|
|||
2020 |
2019 |
||
(unaudited) |
|||
Revenue: |
|||
Commercial revenue: |
|||
SPINRAZA royalties |
|
|
|
Product sales, net |
15 |
7 |
|
Licensing and royalty revenue |
3 |
1 |
|
Total commercial revenue |
84 |
68 |
|
Research and development revenue under collaborative agreements |
49 |
229 |
|
Total revenue |
133 |
297 |
|
Expenses: |
|||
Cost of products sold |
3 |
1 |
|
Research, development and patent |
116 |
107 |
|
Selling, general and administrative |
75 |
68 |
|
Total operating expenses |
194 |
176 |
|
Income (loss) from operations |
(61) |
121 |
|
Other income (expense): |
|||
Investment income |
10 |
12 |
|
Interest expense |
(11) |
(12) |
|
Income (loss) before income tax benefit (expense) |
(62) |
121 |
|
Income tax benefit (expense) |
3 |
(31) |
|
Net income (loss) |
|
|
|
Net (income) loss attributable to noncontrolling interest in |
11 |
(6) |
|
Net income (loss) attributable to |
|
|
|
Basic net income (loss) per share |
|
|
|
Diluted net income (loss) per share |
|
|
|
Shares used in computing basic net income (loss) per share |
139,429 |
138,582 |
|
Shares used in computing diluted net income (loss) per share |
139,429 |
141,537 |
|
|||||||||
SELECTED FINANCIAL INFORMATION |
|||||||||
Condensed Consolidating Statement of Operations |
|||||||||
(In Millions) |
|||||||||
Three months ended, (unaudited) |
|||||||||
Ionis |
Akcea |
Eliminations |
Ionis |
||||||
Revenue: |
|||||||||
Commercial revenue: |
|||||||||
SPINRAZA royalties |
$ |
|
$- |
$ - |
|
||||
Product sales, net |
- |
15 |
- |
15 |
|||||
Licensing and royalty revenue |
3 |
- |
- |
3 |
|||||
Total commercial revenue |
69 |
15 |
- |
84 |
|||||
Research and development revenue under collaborative agreements |
48 |
1 |
- |
49 |
|||||
Intercompany revenue |
1 |
- |
(1) |
- |
|||||
Total revenue |
118 |
16 |
(1) |
133 |
|||||
Expenses: |
|||||||||
Cost of products sold |
- |
5 |
(2) |
3 |
|||||
Research, development and patent expenses |
99 |
17 |
- |
116 |
|||||
Selling, general and administrative |
29 |
46 |
- |
75 |
|||||
Profit/ loss share for TEGSEDI commercialization activities |
7 |
(7) |
- |
- |
|||||
Total operating expenses |
135 |
61 |
(2) |
194 |
|||||
Loss from operations |
(17) |
(45) |
1 |
(61) |
|||||
Other income (expense): |
|||||||||
Investment income |
9 |
1 |
- |
10 |
|||||
Interest expense |
(11) |
- |
- |
(11) |
|||||
Loss on early retirement of debt |
- |
- |
- |
- |
|||||
Loss before income tax benefit |
(19) |
(44) |
1 |
(62) |
|||||
Income tax benefit |
2 |
1 |
- |
3 |
|||||
Net loss |
|
|
|
|
|||||
Net loss attributable to noncontrolling interest in |
$- |
$- |
|
|
|||||
Net loss attributable to |
|
|
|
|
|
|||
Reconciliation of GAAP to Non-GAAP Basis: |
|||
Condensed Consolidated Operating Expenses, Income (Loss) From Operations, and Net Income (Loss) |
|||
(In Millions) |
|||
Three months ended, |
|||
2020 |
2019 |
||
(unaudited) |
|||
As reported research, development and patent expenses according to GAAP |
|
|
|
Excluding compensation expense related to equity awards |
(26) |
(25) |
|
Non-GAAP research, development and patent expenses |
|
|
|
As reported selling, general and administrative expenses according to GAAP |
|
|
|
Excluding compensation expense related to equity awards |
(15) |
(21) |
|
Non-GAAP selling, general and administrative expenses |
|
|
|
As reported operating expenses according to GAAP |
|
|
|
Excluding compensation expense related to equity awards |
(41) |
(46) |
|
Non-GAAP operating expenses |
|
|
|
As reported income (loss) from operations according to GAAP |
|
|
|
Excluding compensation expense related to equity awards |
(41) |
(46) |
|
Non-GAAP income (loss) from operations |
|
|
|
As reported net income (loss) attributable to |
|
|
|
Excluding compensation expense related to equity awards attributable to |
(39) |
(42) |
|
Income tax effect related to compensation expense related to equity awards attributable to |
6 |
(1) |
|
Non-GAAP net income (loss) attributable to |
|
|
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
|
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Condensed Consolidated Balance Sheets |
|||
(In Millions) |
|||
|
|
||
2020 |
2019 |
||
(unaudited) |
|||
Assets: |
|||
Cash, cash equivalents and short-term investments |
|
|
|
Contracts receivable |
29 |
63 |
|
Other current assets |
145 |
158 |
|
Property, plant and equipment, net |
164 |
154 |
|
Other assets |
369 |
358 |
|
Total assets
|
|
|
|
Liabilities and stockholders' equity: |
|||
Other current liabilities |
|
|
|
Current portion of deferred contract revenue |
121 |
118 |
|
0.125% convertible senior notes |
440 |
435 |
|
1% convertible senior notes |
280 |
275 |
|
Long-term obligations, less current portion |
75 |
75 |
|
Long-term deferred contract revenue |
468 |
490 |
|
Total Ionis stockholders' equity |
1,360 |
1,471 |
|
Noncontrolling interest in |
210 |
214 |
|
Total stockholders' equity |
1,570 |
1,685 |
|
Total liabilities and stockholders' equity |
|
|
|
|||||||
Condensed Consolidating Balance Sheet |
|||||||
(In Millions) |
|||||||
(unaudited) |
|||||||
Ionis |
|||||||
Ionis |
Akcea |
Eliminations |
Consolidated |
||||
Assets: |
|||||||
Cash, cash equivalents and short-term investments |
|
|
$- |
|
|||
Contracts receivable |
17 |
12 |
- |
29 |
|||
Other current assets |
121 |
26 |
(2) |
145 |
|||
Property, plant and equipment, net |
159 |
5 |
- |
164 |
|||
Other assets |
1,116 |
96 |
(843) |
369 |
|||
Total assets |
|
|
|
|
|||
Liabilities and stockholders' equity: |
|||||||
Other current liabilities |
104 |
35 |
(2) |
137 |
|||
Current portion of deferred contract revenue |
120 |
1 |
- |
121 |
|||
0.125% convertible senior notes |
440 |
- |
- |
440 |
|||
1% convertible senior notes |
280 |
- |
- |
280 |
|||
Long-term obligations, less current portion |
61 |
14 |
- |
75 |
|||
Long-term deferred contract revenue |
470 |
- |
(2) |
468 |
|||
Total stockholders' equity before noncontrolling interest |
1,901 |
510 |
(1,051) |
1,360 |
|||
Noncontrolling interest in |
- |
- |
210 |
210 |
|||
Total stockholders' equity |
|
|
|
|
|||
Total liabilities and stockholders' equity |
|
|
|
|
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SOURCE
Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph.D., Vice President, Investor Relations, 760-603-2741, or Ionis Pharmaceuticals Media Contact: Roslyn Patterson, Vice President, Corporate Communications, 760-603-2681