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8-K - 8-K - IONIS PHARMACEUTICALS INCbrhc10016584_8k.htm

Exhibit 99.1


Ionis reports third quarter 2020 financial results and recent business achievements

 On track to achieve 2020 financial guidance
 
Akcea acquisition supports Ionis’ commercial strategy
 
Webcast today, November 4, 2020, at 11:30 a.m. Eastern Time
 
CARLSBAD, Calif., November 4, 2020 – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today reported its financial results for the third quarter of 2020 and recent business highlights.
 
“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 Brett P. Monia, Ph.D., chief executive officer at Ionis. “We made significant progress across our pipeline this year. Recently, we advanced inhaled delivery with IONIS-ENAC-2.5Rx, positioning us to bring new treatment options to patients with pulmonary diseases. We also initiated mid-stage studies for vupanorsen in cardiovascular disease patients and ION541, our medicine to treat nearly all forms of ALS. Additionally, our five Phase 3 studies continue to progress, with our sixth expected to begin by the end of this year. We believe our achievements this year move us closer to delivering 10 or more marketing applications through 2025. Our goal is to bring these medicines to millions of patients around the world.”
 
Third Quarter 2020 Financial Results and Highlights
 

On track to achieve financial guidance of being meaningfully profitable this year

o
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

o
Commercial revenue from SPINRAZA® (nusinersen) royalties of $74 million, in line with the prior quarter

o
Product sales from TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen) increased more than 15 percent

o
R&D revenue increased more than 15 percent from advancing several partnered programs


Maintained a strong balance sheet with cash of $2.3 billion

o
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 Elizabeth L. Hougen, chief financial officer of Ionis. “Looking ahead, we are maintaining our 2020 financial guidance driven by the significant revenue and earnings growth we expect in the fourth quarter. We have already earned revenue from multiple sources this quarter, including $75 million from Pfizer for advancing vupanorsen. Importantly, we remain well capitalized with the financial resources to achieve our 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.
 
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Commercial Medicine Highlights
 
SPINRAZA: a global foundation-of-care for the treatment of spinal muscular atrophy (SMA) patients of all ages
 
o
$495 million in worldwide sales in the third quarter

o
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

o
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

o
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

o
Commercially available in 15 countries

o
Secured pricing and reimbursement in multiple new EU markets and in Canada in the largest provinces and with multiple private payers

o
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

o
Commercially available in 4 countries

o
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

o
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

o
IONIS-FXI-LRx advanced into Phase 2b development in patients with end-stage renal disease

o
IONIS-HBVRx advanced into Phase 2b development in patients with hepatitis B virus infection
Advanced inhaled delivery

o
Positive IONIS-ENAC-2.5Rx healthy volunteer results provided support for inhaled antisense medicine delivery

o
Dosing completed in the IONIS-ENAC-2.5Rx Phase 2 study in patients with cystic fibrosis
Advanced the IONIS-PKK-LRx program

o
Proof-of-concept data from the PKK development program in patients with hereditary angioedema were reported in the New England Journal of Medicine

o
Enrollment completed in the IONIS-PKK-LRx Phase 2 study in patients with hereditary angioedema

o
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

o
ION541 advanced into Phase 1/2 development in patients with nearly all forms of ALS

o
ION464 advanced into Phase 1/2 development in patients with multiple system atrophy

o
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:

2


o
Subcutaneous and orally delivered ION449 targeting PCSK9 at the American Heart Association Scientific Sessions 2020

o
IONIS-AGT-LRx Phase 2 studies in patients with hypertension

o
IONIS-ENAC-2.5Rx Phase 2 study in patients with cystic fibrosis

o
IONIS-GHR-LRx Phase 2 study in patients with acromegaly

o
IONIS-PKK-LRx Phase 2 study in patients with hereditary angioedema
Advance the pipeline with numerous study initiations:

o
AKCEA-APOCIII-LRx Phase 3 study in patients with FCS

o
ION363 registrational study in patients with FUS-ALS

o
IONIS-ENAC-2.5Rx Phase 2 study in patients with chronic obstructive pulmonary disease

o
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):
 
   
Three months ended,
September 30,
   
Nine months ended,
September 30,
 
   
2020
   
2019
   
2020
   
2019
 
Revenue:
                 
Commercial revenue:
                       
SPINRAZA royalties
 
$
74
   
$
82
   
$
212
   
$
212
 
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
 
$
160
   
$
168
   
$
439
   
$
629
 

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 October 2020, Ionis owned approximately 76 percent of Akcea. The line titled “Net loss attributable to noncontrolling interest in Akcea” on Ionis’ statement of operations reflects the portion of Akcea’s net income or loss attributable to the other owners of Akcea’s common stock. In October 2020, after the acquisition of Akcea closed, Ionis no longer recognizes any noncontrolling interest in Akcea on its statement of operations.

3

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 $150 million license fee Ionis earned from Novartis. Additionally, Ionis’ operating expenses increased in 2020 compared to the same period last year as described above.

Balance Sheet

Ionis ended September 2020 with cash, cash equivalents and short-term investments of more than $2.3 billion, compared to $2.5 billion at December 31, 2019. In October 2020, Ionis used approximately $545 million of its cash for the Akcea acquisition.

Webcast

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.
 
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 Akcea Therapeutics, Inc., Ionis’ wholly owned subsidiary. 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 but not limited to those related to our commercial products and the medicines in our pipeline, and particularly 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, 2019, 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 subsidiaries.

4

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:
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

5

 IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Millions, Except Per Share Data)

   
Three months ended,
September 30,
   
Nine months ended,
September 30,
 
   
2020
   
2019
   
2020
   
2019
 
   
 
   
(unaudited)
 
Revenue:
                       
Commercial revenue:
                       
SPINRAZA royalties
 
$
74
   
$
82
   
$
212
   
$
212
 
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 collaborative agreements
   
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)
 
$
(43
)
 
$
18
   
$
(145
)
 
$
99
 
Net loss attributable to noncontrolling interest in Akcea Therapeutics, Inc.
   
12
     
8
     
34
     
11
 
Net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders
 
$
(31
)
 
$
26
   
$
(111
)
 
$
110
 
                                 
Basic net income (loss) per share
 
$
(0.22
)
 
$
0.19
   
$
(0.80
)
 
$
0.81
 
Diluted net income (loss) per share
 
$
(0.22
)
 
$
0.18
   
$
(0.80
)
 
$
0.79
 
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
 

6

IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidating Statement of Operations
(In Millions)
 
   
Nine months ended,
September 30, 2020
(unaudited)
 
   
Ionis
   
Akcea
   
Eliminations
   
Ionis Consolidated
 
Revenue:
     
Commercial revenue:
                       
SPINRAZA royalties
 
$
$212
   
$
-
   
$
-
   
$
212
 
Product sales, net
   
-
     
51
     
-
     
51
 
Licensing and royalty revenue
   
6
     
-
     
-
     
6
 
Total commercial revenue
   
218
     
51
     
-
     
269
 
Research and development revenue under collaborative agreements
   
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 commercialization activities
   
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
 
$
(7
)
 
$
(142
)
 
$
4
   
$
(145
)
Net loss attributable to noncontrolling interest in Akcea Therapeutics, Inc.
 
$
-
   
$
-
   
$
34
   
$
34
 
Net loss attributable to Ionis Pharmaceuticals, Inc. common stockholders
 
$
(7
)
 
$
(142
)
 
$
38
   
$
(111
)

7

IONIS PHARMACEUTICALS, INC.
Reconciliation of GAAP to Non-GAAP Basis:
Condensed Consolidated Operating Expenses, Income (Loss) From Operations, and Net Income (Loss)
(In Millions)
 
   
Three months ended,
September 30,
   
Nine months ended,
September 30,
 
   
2020
   
2019
   
2020
   
2019
 
   
(unaudited)
 
As reported research, development and patent expenses according to GAAP
 
$
125
   
$
104
   
$
364
   
$
317
 
Excluding compensation expense related to equity awards
   
(25
)
   
(24
)
   
(77
)
   
(72
)
                                 
Non-GAAP research, development and patent expenses
 
$
100
   
$
80
   
$
287
   
$
245
 
As reported selling, general and administrative expenses according to GAAP
 
$
69
   
$
60
   
$
215
   
$
204
 
Excluding compensation expense related to equity awards
   
(20
)
   
-
     
(57
)
   
(39
)
                                 
Non-GAAP selling, general and administrative expenses
 
$
49
   
$
60
   
$
158
   
$
165
 
As reported operating expenses according to GAAP
 
$
197
   
$
165
   
$
588
   
$
524
 
Excluding compensation expense related to equity awards
   
(46
)
   
(24
)
   
(135
)
   
(112
)
                                 
Non-GAAP operating expenses
 
$
151
   
$
141
   
$
453
   
$
412
 
                                 
As reported income (loss) from operations according to GAAP
 
$
(37
)
 
$
3
   
$
(149
)
 
$
105
 
Excluding compensation expense related to equity awards
   
(46
)
   
(24
)
   
(135
)
   
(112
)
Non-GAAP income (loss) from operations
 
$
9
   
$
27
   
$
(14
)
 
$
217
 
As reported net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders according to GAAP
 
$
(31
)
 
$
26
   
$
(111
)
 
$
110
 
Excluding compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc. common stockholders
   
(42
)
   
(25
)
   
(126
)
   
(104
)
Income tax effect related to compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc. common stockholders
   
6
     
12
     
18
     
25
 
Non-GAAP net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders according to GAAP
 
$
5
   
$
39
   
$
(3
)
 
$
189
 

8

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 shareholders were adjusted from GAAP to exclude compensation expense related to equity awards and the related tax effect. Compensation expense related to equity awards are non-cash. 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.
 
9

IONIS PHARMACEUTICALS, INC.
Condensed Consolidated Balance Sheets
(In Millions)
 
   
September 30,
2020
   
December 31,
2019
 
   
(unaudited)
       
Assets:
           
Cash, cash equivalents and short-term investments
 
$
2,329
   
$
2,500
 
Contracts receivable
   
39
     
63
 
Other current assets
   
169
     
158
 
Property, plant and equipment, net
   
182
     
154
 
Other assets
   
377
     
358
 
Total assets
 
$
3,096
   
$
3,233
 
                 
Liabilities and stockholders’ equity:
               
Other current liabilities
 
$
140
   
$
155
 
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 Akcea Therapeutics, Inc.
   
223
     
214
 
Total stockholders’ equity
 
$
1,606
   
$
1,685
 
Total liabilities and stockholders’ equity
 
$
3,096
   
$
3,233
 

10

IONIS PHARMACEUTICALS, INC.
Condensed Consolidating Balance Sheet
(In Millions)

   
September 30, 2020
(unaudited)
 
   
Ionis
   
Akcea
   
Eliminations
   
Ionis
Consolidated
 
                         
Assets:
                       
Cash, cash equivalents and short-term investments
 
$
1,963
   
$
366
   
$
-
   
$
2,329
 
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
 
$
3,437
   
$
508
   
$
(849
)
 
$
3,096
 
                                 
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 Akcea Therapeutics, Inc.
   
-
     
-
     
223
     
223
 
Total stockholders’ equity
 
$
1,999
   
$
445
   
$
(838
)
 
$
1,606
 
Total liabilities and stockholders’ equity
 
$
3,437
   
$
508
   
$
(849
)
 
$
3,096
 


11