Press Releases
Ionis reports second quarter 2020 financial results and recent business achievements
"The first half of this year was marked by numerous important achievements. Our Phase 3 programs progressed, we advanced our cardio-renal, metabolic and neurological disease franchises and added new medicines to our Ionis-owned pipeline. Together, these catalysts moved us closer to delivering 10 or more marketing applications through 2025, which we expect to result in a number of new commercial medicines. In the second half of this year, we are expanding the reach of our technology in neurological and pulmonary diseases, as studies in sporadic ALS and COPD get underway. We also look forward to proof-of-concept data from additional mid-stage programs," said
Second Quarter 2020 Financial Results and Highlights
- On track to achieve financial guidance of being meaningfully profitable this year
- Net income of
$8 million on a non-GAAP basis and a net loss of$32 million on a GAAP basis - Achieved quarter over quarter growth in commercial and R&D revenues
- Commercial revenue from SPINRAZA® (nusinersen) royalties increased to
$72 million - Product sales from TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen) increased to
$16 million - R&D revenue increased to more than
$55 million , including$26 million from Ionis' neurological disease franchise,$13 million from the oncology franchise and more than$10 million from the cardio-renal franchise - Cash position of more than
$2.3 billion provides substantial financial resources to continue executing on strategic goals
"We ended the second quarter with net income on a non-GAAP basis, an increase compared to our first quarter results. Looking ahead, we are maintaining our 2020 financial guidance and expect revenue and earnings growth in the second half of this year. With our substantial resources, we are well positioned to continue executing on our ambitious agenda and to deliver increasing value near-term and into the future," said
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
$495 million in worldwide sales in the second quarter of this year- More than 11,000 patients were on SPINRAZA treatment worldwide at the end of the second quarter, including patients across commercial, expanded access and clinical trial settings
- The Phase 4 RESPOND study to evaluate SPINRAZA benefit in patients with a suboptimal clinical response to Zolgensma® (onasemnogene abeparvovec) is expected to begin early next year
- The DEVOTE study evaluating a higher dose of SPINRAZA with the potential to deliver even greater efficacy in SMA patients of all ages is progressing
- New clinical data from the NURTURE and SHINE studies, as well as new real-world data, further support SPINRAZA's durable efficacy and established safety profile across SMA patients of all ages
- TEGSEDI: the only approved at-home subcutaneous therapy for the treatment of hereditary transthyretin amyloidosis (hATTR) with polyneuropathy in adult patients
- Commercially available in 15 countries
- Reimbursement approved in
Portugal ,Spain ,Italy andAustria - Expanding commercial availability in additional EU countries and in
Latin America this year - WAYLIVRA: the only approved treatment in the EU for adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis
- Launch progressing in
Germany ,Austria ,Greece and through the ATU inFrance ; launching in additional EU countries this year - Filed for marketing approval in
Brazil ; refiling new drug application forU.S. marketing authorization
Second Quarter 2020 and Recent Pipeline Highlights
- Completed enrollment in the global GENERATION HD1 Phase 3 study of tominersen in patients with Huntington's disease
- Progressed multiple neurological disease medicines under Ionis' broad Biogen collaboration
- Published data from the Phase 1/2 study of tofersen in the
New England Journal of Medicine - Progressed the IONIS-MAPTRx long-term extension study in patients with Alzheimer's disease and achieved a
$12 million milestone payment - Advanced ION464 into a Phase 1/2 study in patients with multiple system atrophy and achieved an
$18 million milestone payment - Advanced medicines for the treatment of cancer and immune-mediated GI disease
- Licensed ION736 to AstraZeneca for the treatment of cancer and achieved a
$13 million license fee - Initiated a Phase 1 study of ION253 for the treatment of immune-mediated GI disease and achieved a
$5 million milestone payment from Janssen - Expanded the Ionis-owned pipeline with the addition of ION363 for the treatment of FUS-ALS
Upcoming Catalysts
- Report clinical proof-of-concept results for four or more programs
- Present positive Phase 2 results from vupanorsen and AKCEA-APOCIII-LRx
- Initiate a Phase 3 study of AKCEA-APOCIII-LRx in patients with
FCS - Initiate a registration study of ION363 in patients with FUS-ALS
- Initiate a Phase 1/2 study of ION541 in patients with sporadic ALS
- Initiate a Phase 2 study of IONIS-ENaC-2.5Rx in patients with chronic obstructive pulmonary disease (COPD)
- Initiate a Phase 2 study of IONIS-FXI-LRx in patients with clotting disorders
- Initiate a Phase 2 study of IONIS-HBVRx in patients with hepatitis B virus infection
- Advance additional new medicines into development
Revenue
Ionis' revenue was comprised of the following (amounts in millions):
Three months ended, |
Six months ended |
|||||||
|
|
|||||||
2020 |
2019 |
2020 |
2019 |
|||||
Revenue: |
||||||||
Commercial revenue: |
||||||||
SPINRAZA royalties |
|
|
|
|
||||
Product sales, net |
16 |
10 |
32 |
17 |
||||
Licensing and royalty revenue |
2 |
7 |
4 |
9 |
||||
Total commercial revenue |
90 |
88 |
174 |
156 |
||||
R&D Revenue: |
||||||||
Amortization from upfront payments |
28 |
41 |
49 |
75 |
||||
Milestone payments |
7 |
12 |
30 |
52 |
||||
License fees |
15 |
22 |
15 |
173 |
||||
Other services |
6 |
1 |
11 |
5 |
||||
Total R&D revenue |
56 |
76 |
105 |
305 |
||||
Total revenue |
|
|
|
|
Operating Expenses
Ionis' operating expenses for the second quarter of 2020 increased compared to the same period in 2019 driven by its investments in advancing its strategic priorities, including advancing the Phase 3 program for AKCEA-TTR-LRx and other medicines in its Ionis-owned pipeline.
Net 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 second quarter of 2020 was larger compared to the same period in 2019 primarily due to higher revenue and lower operating expenses in the same period last year.
Balance Sheet
Ionis ended
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, cardio-renal, metabolic, infectious, and pulmonary diseases.
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, including the business of
In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to
Ionis Pharmaceuticals Investor Contact:
Vice President, Investor Relations
760-603-2741
Ionis Pharmaceuticals Media Contact:
Vice President, Corporate Communications
760-603-2681
SELECTED FINANCIAL INFORMATION Condensed Consolidated Statements of Operations (In Millions, Except Per Share Data) |
||||||||
Three months ended |
Six months ended |
|||||||
|
|
|||||||
2020 |
2019 |
2020 |
2019 |
|||||
(unaudited) |
||||||||
Revenue: |
||||||||
Commercial revenue: |
||||||||
SPINRAZA royalties |
|
|
|
|
||||
Product sales, net |
16 |
10 |
32 |
17 |
||||
Licensing and royalty revenue |
2 |
7 |
4 |
9 |
||||
Total commercial revenue |
90 |
88 |
174 |
156 |
||||
Research and development revenue under collaborative agreements |
56 |
76 |
105 |
305 |
||||
Total revenue |
146 |
164 |
279 |
461 |
||||
Expenses: |
||||||||
Cost of products sold |
3 |
1 |
6 |
2 |
||||
Research, development and patent |
122 |
106 |
239 |
213 |
||||
Selling, general and administrative |
73 |
76 |
147 |
143 |
||||
Total operating expenses |
198 |
183 |
392 |
358 |
||||
Income (loss) from operations |
(52) |
(19) |
(113) |
103 |
||||
Other income, net |
8 |
2 |
7 |
2 |
||||
Income (loss) before income tax benefit (expense) |
(44) |
(17) |
(106) |
105 |
||||
Income tax benefit (expense) |
- |
7 |
4 |
(24) |
||||
Net income (loss) |
|
|
|
|
||||
Net loss attributable to noncontrolling interest in |
12 |
9 |
22 |
3 |
||||
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 |
140 |
139 |
139 |
||||
Shares used in computing diluted net income (loss) per share |
139 |
140 |
139 |
142 |
Condensed Consolidating Statement of Operations (In Millions) |
|||||||||
Six months ended |
|||||||||
Ionis |
Akcea |
Eliminations |
Ionis |
||||||
Revenue: |
|||||||||
Commercial revenue: |
|||||||||
SPINRAZA royalties |
$ |
|
$- |
$- |
|
||||
Product sales, net |
- |
32 |
- |
32 |
|||||
Licensing and royalty revenue |
4 |
- |
- |
4 |
|||||
Total commercial revenue |
142 |
32 |
- |
174 |
|||||
Research and development revenue under collaborative agreements |
98 |
7 |
- |
105 |
|||||
Intercompany revenue |
7 |
- |
(7) |
- |
|||||
Total revenue |
247 |
39 |
(7) |
279 |
|||||
Expenses: |
|||||||||
Cost of products sold |
- |
10 |
(4) |
6 |
|||||
Research, development and patent expenses |
197 |
47 |
(5) |
239 |
|||||
Selling, general and administrative |
59 |
88 |
- |
147 |
|||||
Profit/ loss share for TEGSEDI commercialization activities |
10 |
(10) |
- |
- |
|||||
Total operating expenses |
266 |
135 |
(9) |
392 |
|||||
Loss from operations |
(19) |
(96) |
2 |
(113) |
|||||
Other income, net |
4 |
3 |
- |
7 |
|||||
Loss before income tax benefit |
(15) |
(93) |
2 |
(106) |
|||||
Income tax benefit |
3 |
1 |
- |
4 |
|||||
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 |
Six months ended |
|||||||
2020 |
2019 |
2020 |
2019 |
|||||
(unaudited) |
||||||||
As reported research, development and patent expenses according to GAAP |
|
|
|
|
||||
Excluding compensation expense related to equity awards |
(26) |
(24) |
(52) |
(49) |
||||
Non-GAAP research, development and patent expenses |
|
|
|
|
||||
As reported selling, general and administrative expenses according to GAAP |
|
|
|
|
||||
Excluding compensation expense related to equity awards |
(22) |
(18) |
(37) |
(39) |
||||
Non-GAAP selling, general and administrative expenses |
|
|
|
|
||||
As reported operating expenses according to GAAP |
|
|
|
|
||||
Excluding compensation expense related to equity awards |
(48) |
(42) |
(89) |
(87) |
||||
Non-GAAP operating expenses |
|
|
|
|
||||
As reported income (loss) from operations according to GAAP |
|
|
|
|
||||
Excluding compensation expense related to equity awards |
(48) |
(42) |
(89) |
(87) |
||||
Non-GAAP income (loss) from operations |
|
|
|
|
||||
As reported net income (loss) attributable to |
|
|
|
|
||||
Excluding compensation expense related to equity awards attributable to |
(45) |
(39) |
(84) |
(78) |
||||
Income tax effect related to compensation expense related to equity awards attributable to |
5 |
14 |
11 |
13 |
||||
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
Condensed Consolidated Balance Sheets (In Millions) |
|||||
|
|
||||
2020 |
2019 |
||||
(unaudited) |
|||||
Assets: |
|||||
Cash, cash equivalents and short-term investments |
|
|
|||
Contracts receivable |
28 |
63 |
|||
Other current assets |
155 |
158 |
|||
Property, plant and equipment, net |
173 |
154 |
|||
Other assets |
374 |
358 |
|||
Total assets |
|
|
|||
Liabilities and stockholders' equity: |
|||||
Other current liabilities |
|
|
|||
Current portion of deferred contract revenue |
100 |
118 |
|||
0.125% convertible senior notes |
445 |
435 |
|||
1% convertible senior notes |
284 |
275 |
|||
Long-term obligations, less current portion |
75 |
75 |
|||
Long-term deferred contract revenue |
448 |
490 |
|||
Total Ionis stockholders' equity |
1,377 |
1,471 |
|||
Noncontrolling interest in |
218 |
214 |
|||
Total stockholders' equity |
|
|
|||
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 |
14 |
14 |
- |
28 |
||||
Other current assets |
136 |
24 |
(5) |
155 |
||||
Property, plant and equipment, net |
167 |
6 |
- |
173 |
||||
Other assets |
1,122 |
94 |
(842) |
374 |
||||
Total assets |
|
|
|
|
||||
Liabilities and stockholders' equity: |
||||||||
Other current liabilities |
|
|
|
|
||||
Current portion of deferred contract revenue |
100 |
- |
- |
100 |
||||
0.125% convertible senior notes |
445 |
- |
- |
445 |
||||
1% convertible senior notes |
284 |
- |
- |
284 |
||||
Long-term obligations, less current portion |
61 |
14 |
- |
75 |
||||
Long-term deferred contract revenue |
451 |
- |
(3) |
448 |
||||
Total stockholders' equity before noncontrolling interest |
1,955 |
479 |
(1,057) |
1,377 |
||||
Noncontrolling interest in |
- |
- |
218 |
218 |
||||
Total stockholders' equity |
|
|
|
|
||||
Total liabilities and stockholders' equity |
|
|
|
|
View original content to download multimedia:http://www.prnewswire.com/news-releases/ionis-reports-second-quarter-2020-financial-results-and-recent-business-achievements-301106244.html
SOURCE