Press Releases
Ionis reports third quarter 2020 financial results and recent business achievements
"We took an important step forward in our evolution when we acquired Akcea. This transaction supports our commercial strategy, further enabling us to maximize the value of our Ionis-owned pipeline. As one company, we believe we are stronger and more efficient, with an enhanced ability to achieve even greater future success," said
Third Quarter 2020 Financial Results and Highlights
- On track to achieve financial guidance of being meaningfully profitable this year
- Net income of
$5 million on a non-GAAP basis and a net loss of$31 million on a GAAP basis for the third quarter - Achieved quarter over quarter revenue growth
- Commercial revenue from SPINRAZA® (nusinersen) royalties of
$74 million , in line with the prior quarter - Product sales from TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen) increased more than 15 percent
- R&D revenue increased more than 15 percent from advancing several partnered programs
- Maintained a strong balance sheet with cash of
$2.3 billion - Estimated pro forma cash following the Akcea acquisition of
$1.8 billion
"Our acquisition of Akcea further strengthens our business and financial position in numerous ways. We now retain more value from Akcea's rich pipeline and commercial products. We are also able to use Akcea's current cash and future cash flows to advance Ionis' strategic priorities. And beginning next year, we expect to realize meaningful cost synergies as we continue to integrate the two companies," 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 third quarter- More than 11,000 patients were on SPINRAZA treatment worldwide at the end of the third quarter, including patients across commercial, expanded access and clinical trial settings
- The open-label safety cohort of the DEVOTE study of higher-dose SPINRAZA is fully enrolled and the pivotal randomized treatment cohort will begin enrolling patients next
- The Phase 4 RESPOND study in patients with a suboptimal clinical response to gene therapy is expected to begin early next year
- 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
- Secured pricing and reimbursement in multiple new EU markets and in
Canada in the largest provinces and with multiple private payers - Won 2020
Prix Galien USA Award for the Best Biotechnology Product - WAYLIVRA: the only approved treatment in the EU for adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis
- Commercially available in 4 countries
- Finalized pricing negotiations in additional EU markets, including in the
UK
Third Quarter 2020 and Recent Pipeline Highlights
- Positive Phase 2 vupanorsen and AKCEA-APOCIII-LRx results presented at the
European Society of Cardiology annual meeting - Advanced multiple programs into key mid-stage studies
- Vupanorsen advanced into Phase 2b development with the initiation of the TRANSLATE-TIMI 70 dose-ranging study in statin-treated patients with dyslipidemia, resulting in a
$75 million payment from Pfizer - IONIS-FXI-LRx advanced into Phase 2b development in patients with end-stage renal disease
- IONIS-HBVRx advanced into Phase 2b development in patients with hepatitis B virus infection
- Advanced inhaled delivery
- Positive IONIS-ENAC-2.5Rx healthy volunteer results provided support for inhaled antisense medicine delivery
- Dosing completed in the IONIS-ENAC-2.5Rx Phase 2 study in patients with cystic fibrosis
- Advanced the IONIS-PKK-LRx program
- Proof-of-concept data from the
PKK development program in patients with hereditary angioedema were reported in theNew England Journal of Medicine - Enrollment completed in the IONIS-PKK-LRx Phase 2 study in patients with hereditary angioedema
- IONIS-PKK-LRx advanced into an investigator-initiated study in hospitalized COVID-19 patients in
Brazil - Progressed multiple neurological disease medicines under Ionis' broad collaboration with Biogen, earning more than
$50 million - ION541 advanced into Phase 1/2 development in patients with nearly all forms of ALS
- ION464 advanced into Phase 1/2 development in patients with multiple system atrophy
- IONIS-MAPTRx continued to advance in a long-term extension study in patients with Alzheimer's disease
- The
U.S. FDA granted orphan drug designation to Ionis-owned medicines for people with Alexander disease, β-thalassemia and Lafora diseases
Upcoming Catalysts
- Report clinical data, potentially enabling key programs to advance towards the market:
- Subcutaneous and orally delivered ION449 targeting PCSK9 at the American Heart Association Scientific Sessions 2020
- IONIS-AGT-LRx Phase 2 studies in patients with hypertension
- IONIS-ENAC-2.5Rx Phase 2 study in patients with cystic fibrosis
- IONIS-GHR-LRx Phase 2 study in patients with acromegaly
- IONIS-PKK-LRx Phase 2 study in patients with hereditary angioedema
- Advance the pipeline with numerous study initiations:
- AKCEA-APOCIII-LRx Phase 3 study in patients with
FCS - ION363 registrational study in patients with FUS-ALS
- IONIS-ENAC-2.5Rx Phase 2 study in patients with chronic obstructive pulmonary disease
- Phase 1/2 studies of Ionis-owned medicines:
- ION251 for patients with multiple myeloma
- ION373 for patients with Alexander disease
- ION716 for patients with Prion diseases
- Expand TEGSEDI and WAYLIVRA commercial availability in the EU and
Latin America - Refile the WAYLIVRA application for marketing authorization in the
U.S. next year
Revenue
Ionis' revenue was comprised of the following (amounts in millions):
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2020 |
2019 |
2020 |
2019 |
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Revenue: |
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Commercial revenue: |
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SPINRAZA royalties |
|
|
|
|
||||
Product sales, net |
19 |
12 |
51 |
29 |
||||
Licensing and royalty revenue |
2 |
2 |
6 |
11 |
||||
Total commercial revenue |
95 |
96 |
269 |
252 |
||||
R&D Revenue: |
||||||||
Amortization from upfront payments |
19 |
23 |
68 |
100 |
||||
Milestone payments |
44 |
12 |
73 |
64 |
||||
License fees |
- |
26 |
15 |
198 |
||||
Other services |
2 |
11 |
14 |
15 |
||||
Total R&D revenue |
65 |
72 |
170 |
377 |
||||
Total revenue |
|
|
|
|
Operating Expenses
Ionis' operating expenses for the third quarter of 2020 increased compared to the same period in 2019 driven by the Company's investments in 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
Prior to completing its acquisition of Akcea in
Net Income (Loss) Attributable to Ionis Common Stockholders
Ionis recognized a net loss attributable to Ionis' common stockholders for the third quarter of 2020 compared to net income in the same period in 2019 primarily due to higher revenue in 2019, including a
Balance Sheet
Ionis ended
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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
|
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2020 |
2019 |
2020 |
2019 |
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(unaudited) |
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Revenue: |
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Commercial revenue: |
||||||||
SPINRAZA royalties |
|
|
|
|
||||
Product sales, net |
19 |
12 |
51 |
29 |
||||
Licensing and royalty revenue |
2 |
2 |
6 |
11 |
||||
Total commercial revenue |
95 |
96 |
269 |
252 |
||||
Research and development revenue under |
65 |
72 |
170 |
377 |
||||
Total revenue |
160 |
168 |
439 |
629 |
||||
Expenses: |
||||||||
Cost of products sold |
3 |
1 |
9 |
3 |
||||
Research, development and patent |
125 |
104 |
364 |
317 |
||||
Selling, general and administrative |
69 |
60 |
215 |
204 |
||||
Total operating expenses |
197 |
165 |
588 |
524 |
||||
Income (loss) from operations |
(37) |
3 |
(149) |
105 |
||||
Other income (loss), net |
(3) |
1 |
3 |
4 |
||||
Income (loss) before income tax benefit (expense) |
(40) |
4 |
(146) |
109 |
||||
Income tax benefit (expense) |
(3) |
14 |
1 |
(10) |
||||
Net income (loss) |
|
|
|
|
||||
Net loss attributable to noncontrolling interest in Akcea |
12 |
8 |
34 |
11 |
||||
Net income (loss) attributable to |
|
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Basic net income (loss) per share |
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Diluted net income (loss) per share |
|
|
|
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Shares used in computing basic net income (loss) per share |
140 |
141 |
139 |
140 |
||||
Shares used in computing diluted net income (loss) per share |
140 |
143 |
139 |
143 |
Condensed Consolidating Statement of Operations (In Millions) |
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Nine months ended, (unaudited) |
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Ionis |
Akcea |
Eliminations |
Ionis |
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Revenue: |
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Commercial revenue: |
||||||||||
SPINRAZA royalties |
|
$- |
$- |
|
||||||
Product sales, net |
- |
51 |
- |
51 |
||||||
Licensing and royalty revenue |
6 |
- |
- |
6 |
||||||
Total commercial revenue |
218 |
51 |
- |
269 |
||||||
Research and development revenue under |
163 |
7 |
- |
170 |
||||||
Intercompany revenue |
8 |
- |
(8) |
- |
||||||
Total revenue |
389 |
58 |
(8) |
439 |
||||||
Expenses: |
||||||||||
Cost of products sold |
- |
16 |
(7) |
9 |
||||||
Research, development and patent expenses |
297 |
72 |
(5) |
364 |
||||||
Selling, general and administrative |
88 |
127 |
- |
215 |
||||||
Profit/ loss share for TEGSEDI |
12 |
(12) |
- |
- |
||||||
Total operating expenses |
397 |
203 |
(12) |
588 |
||||||
Loss from operations |
(8) |
(145) |
4 |
(149) |
||||||
Other income, net |
- |
3 |
- |
3 |
||||||
Loss before income tax benefit |
(8) |
(142) |
4 |
(146) |
||||||
Income tax benefit |
1 |
- |
- |
1 |
||||||
Net loss |
|
|
|
|
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Net loss attributable to noncontrolling interest in |
$- |
$- |
|
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Net loss attributable to |
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2020 |
2019 |
2020 |
2019 |
|||||
(unaudited) |
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As reported research, development and patent |
|
|
|
|
||||
Excluding compensation expense related to equity awards |
(25) |
(24) |
(77) |
(72) |
||||
Non-GAAP research, development and patent |
|
|
|
|
||||
As reported selling, general and administrative |
|
|
|
|
||||
Excluding compensation expense related to equity awards |
(20) |
- |
(57) |
(39) |
||||
Non-GAAP selling, general and administrative |
|
|
|
|
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As reported operating expenses according to GAAP |
|
|
|
|
||||
Excluding compensation expense related to equity awards |
(46) |
(24) |
(135) |
(112) |
||||
Non-GAAP operating expenses |
|
|
|
|
||||
As reported income (loss) from operations according |
|
|
|
|
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Excluding compensation expense related to equity awards |
(46) |
(24) |
(135) |
(112) |
||||
Non-GAAP income (loss) from operations |
|
|
|
|
||||
As reported net income (loss) attributable to Ionis |
|
|
|
|
||||
Excluding compensation expense related to equity awards |
(42) |
(25) |
(126) |
(104) |
||||
Income tax effect related to compensation expense related |
6 |
12 |
18 |
25 |
||||
Non-GAAP net income (loss) attributable to Ionis |
|
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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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2020 |
2019 |
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(unaudited) |
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Assets: |
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Cash, cash equivalents and short-term investments |
|
|
||||
Contracts receivable |
39 |
63 |
||||
Other current assets |
169 |
158 |
||||
Property, plant and equipment, net |
182 |
154 |
||||
Other assets |
377 |
358 |
||||
Total assets |
|
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Liabilities and stockholders' equity: |
||||||
Other current liabilities |
|
|
||||
Current portion of deferred contract revenue |
105 |
118 |
||||
0.125% convertible senior notes |
450 |
435 |
||||
1% convertible senior notes |
289 |
275 |
||||
Long-term obligations, less current portion |
76 |
75 |
||||
Long-term deferred contract revenue |
430 |
490 |
||||
Total Ionis stockholders' equity |
1,383 |
1,471 |
||||
Noncontrolling interest in |
223 |
214 |
||||
Total stockholders' equity |
|
|
||||
Total liabilities and stockholders' equity |
|
|
||||
|
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(unaudited) |
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Ionis |
||||||||
Ionis |
Akcea |
Eliminations |
Consolidated |
|||||
Assets: |
||||||||
Cash, cash equivalents and short-term investments |
|
|
$- |
|
||||
Contracts receivable |
23 |
16 |
- |
39 |
||||
Other current assets |
150 |
28 |
(9) |
169 |
||||
Property, plant and equipment, net |
176 |
6 |
- |
182 |
||||
Other assets |
1,125 |
92 |
(840) |
377 |
||||
Total assets |
|
|
|
|
||||
Liabilities and stockholders' equity: |
||||||||
Other current liabilities |
100 |
49 |
(9) |
140 |
||||
Current portion of deferred contract revenue |
105 |
- |
- |
105 |
||||
0.125% convertible senior notes |
450 |
- |
- |
450 |
||||
1% convertible senior notes |
289 |
- |
- |
289 |
||||
Long-term obligations, less current portion |
62 |
14 |
- |
76 |
||||
Long-term deferred contract revenue |
432 |
- |
(2) |
430 |
||||
Total stockholders' equity before noncontrolling interest |
1,999 |
445 |
(1,061) |
1,383 |
||||
Noncontrolling interest in |
- |
- |
223 |
223 |
||||
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; Ionis Pharmaceuticals Media Contact: Roslyn Patterson, Vice President, Corporate Communications, 760-603-2681