|
||
(Commission File No.)
|
(IRS Employer Identification No.)
|
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
|
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
|
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
|
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
|
Title of each class
|
Trading symbol
|
Name of each exchange on which registered
|
||
|
Item 2.02 |
Results of Operations and Financial Condition.
|
Item 9.01. |
Financial Statements and Exhibits.
|
Exhibit No.
|
Description
|
Press Release dated November 4, 2020.
|
|
104
|
Cover Page Interactive Data File (embedded within the Inline XBRL document).
|
Ionis Pharmaceuticals, Inc.
|
||
Dated: November 4, 2020
|
By:
|
/s/ Patrick R. O’Neil
|
Patrick R. O’Neil
|
||
Executive Vice President, Legal, General
Counsel and Chief Compliance Officer
|
• |
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
|
• |
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
|
• |
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
|
• |
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
|
• |
Report clinical data, potentially enabling key programs to advance towards the market:
|
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
|
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
|
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
|
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
|
)
|
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
|
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
|
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
|