(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
|
||
|
|
Emerging growth company
|
|
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised
financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.
|
☐
|
Item 2.02 |
Results of Operations and Financial Condition.
|
Item 9.01. |
Financial Statements and Exhibits.
|
Exhibit No.
|
Description
|
Press Release dated August 5, 2020.
|
|
104
|
Cover Page Interactive Data File (embedded within the Inline XBRL document).
|
Ionis Pharmaceuticals, Inc.
|
||
Dated: August 5, 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 $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
|
o |
Commercial revenue from SPINRAZA® (nusinersen) royalties increased to $72 million
|
o |
Product sales from TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen) increased to $16 million
|
o |
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
|
• |
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 second quarter of this year
|
o |
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
|
o |
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
|
o |
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
|
o |
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
|
• |
o |
Commercially available in 15 countries
|
o |
Reimbursement approved in Portugal, Spain, Italy and Austria
|
o |
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
|
o |
Launch progressing in Germany, Austria, Greece and through the ATU in France; launching in additional EU countries this year
|
o |
Filed for marketing approval in Brazil; refiling new drug application for U.S. marketing authorization
|
• |
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
|
o |
Published data from the Phase 1/2 study of tofersen in the New England Journal of Medicine
|
o |
Progressed the IONIS-MAPTRx long-term extension study in patients with Alzheimer’s disease and achieved a $12 million milestone payment
|
o |
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
|
o |
Licensed ION736 to AstraZeneca for the treatment of cancer and achieved a $13 million license fee
|
o |
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
|
• |
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
|
Three months ended,
June 30,
|
Six months ended
June 30,
|
|||||||||||||||
2020
|
2019
|
2020
|
2019
|
|||||||||||||
Revenue:
|
||||||||||||||||
Commercial revenue:
|
||||||||||||||||
SPINRAZA royalties
|
$
|
72
|
$
|
71
|
$
|
138
|
$
|
130
|
||||||||
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
|
$
|
146
|
$
|
164
|
$
|
279
|
$
|
461
|
Three months ended,
June 30,
|
Six months ended,
June 30,
|
|||||||||||||||
2020
|
2019
|
2020
|
2019
|
|||||||||||||
(unaudited)
|
||||||||||||||||
Revenue:
|
||||||||||||||||
Commercial revenue:
|
||||||||||||||||
SPINRAZA royalties
|
$
|
72
|
$
|
71
|
$
|
138
|
$
|
130
|
||||||||
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)
|
$
|
(44
|
)
|
$
|
(10
|
)
|
$
|
(102
|
)
|
$
|
81
|
|||||
Net loss attributable to noncontrolling interest in Akcea Therapeutics, Inc.
|
12
|
9
|
22
|
3
|
||||||||||||
Net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders
|
$
|
(32
|
)
|
$
|
(1
|
)
|
$
|
(80
|
)
|
$
|
84
|
|||||
Basic net income (loss) per share
|
$
|
(0.23
|
)
|
$
|
(0.01
|
)
|
$
|
(0.58
|
)
|
$
|
0.62
|
|||||
Diluted net income (loss) per share
|
$
|
(0.23
|
)
|
$
|
(0.01
|
)
|
$
|
(0.58
|
)
|
$
|
0.61
|
|||||
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
|
Six months ended,
June 30, 2020
(unaudited)
|
||||||||||||||||
Ionis
|
Akcea
|
Eliminations
|
Ionis Consolidated
|
|||||||||||||
Revenue:
|
||||||||||||||||
Commercial revenue:
|
||||||||||||||||
SPINRAZA royalties
|
$
|
$138
|
$
|
-
|
$
|
-
|
$
|
138
|
||||||||
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
|
$
|
(12
|
)
|
$
|
(92
|
)
|
$
|
2
|
$
|
(102
|
)
|
|||||
Net loss attributable to noncontrolling interest in Akcea Therapeutics, Inc.
|
$
|
-
|
$
|
-
|
$
|
22
|
$
|
22
|
||||||||
Net loss attributable to Ionis Pharmaceuticals, Inc. common stockholders
|
$
|
(12
|
)
|
$
|
(92
|
)
|
$
|
24
|
$
|
(80
|
)
|
Three months ended,
June 30,
|
Six months ended,
June 30,
|
|||||||||||||||
2020
|
2019
|
2020
|
2019
|
|||||||||||||
(unaudited)
|
||||||||||||||||
As reported research, development and patent expenses according to GAAP
|
$
|
122
|
$
|
106
|
$
|
239
|
$
|
213
|
||||||||
Excluding compensation expense related to equity awards
|
(26
|
)
|
(24
|
)
|
(52
|
)
|
(49
|
)
|
||||||||
Non-GAAP research, development and patent expenses
|
$
|
96
|
$
|
82
|
$
|
187
|
$
|
164
|
||||||||
As reported selling, general and administrative expenses according to GAAP
|
$
|
73
|
$
|
76
|
$
|
147
|
$
|
143
|
||||||||
Excluding compensation expense related to equity awards
|
(22
|
)
|
(18
|
)
|
(37
|
)
|
(39
|
)
|
||||||||
Non-GAAP selling, general and administrative expenses
|
$
|
51
|
$
|
58
|
$
|
110
|
$
|
104
|
||||||||
As reported operating expenses according to GAAP
|
$
|
198
|
$
|
183
|
$
|
392
|
$
|
358
|
||||||||
Excluding compensation expense related to equity awards
|
(48
|
)
|
(42
|
)
|
(89
|
)
|
(87
|
)
|
||||||||
Non-GAAP operating expenses
|
$
|
150
|
$
|
141
|
$
|
303
|
$
|
271
|
||||||||
As reported income (loss) from operations according to GAAP
|
$
|
(52
|
)
|
$
|
(19
|
)
|
$
|
(113
|
)
|
$
|
103
|
|||||
Excluding compensation expense related to equity awards
|
(48
|
)
|
(42
|
)
|
(89
|
)
|
(87
|
)
|
||||||||
Non-GAAP income (loss) from operations
|
$
|
(4
|
)
|
$
|
23
|
$
|
(24
|
)
|
$
|
190
|
||||||
As reported net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders according to
GAAP
|
$
|
(32
|
)
|
$
|
(1
|
)
|
$
|
(80
|
)
|
$
|
84
|
|||||
Excluding compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc. common stockholders
|
(45
|
)
|
(39
|
)
|
(84
|
)
|
(78
|
)
|
||||||||
Income tax effect related to compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc.
common stockholders
|
5
|
14
|
11
|
13
|
||||||||||||
Non-GAAP net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders according to
GAAP
|
$
|
8
|
$
|
24
|
$
|
(7
|
)
|
$
|
149
|
June 30,
2020
|
December 31,
2019
|
|||||||
(unaudited)
|
||||||||
Assets:
|
||||||||
Cash, cash equivalents and short-term investments
|
$
|
2,349
|
$
|
2,500
|
||||
Contracts receivable
|
28
|
63
|
||||||
Other current assets
|
155
|
158
|
||||||
Property, plant and equipment, net
|
173
|
154
|
||||||
Other assets
|
374
|
358
|
||||||
Total assets
|
$
|
3,079
|
$
|
3,233
|
||||
Liabilities and stockholders’ equity:
|
||||||||
Other current liabilities
|
$
|
132
|
$
|
155
|
||||
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 Akcea Therapeutics, Inc.
|
218
|
214
|
||||||
Total stockholders’ equity
|
$
|
1,595
|
$
|
1,685
|
||||
Total liabilities and stockholders’ equity
|
$
|
3,079
|
$
|
3,233
|
June 30, 2020
(unaudited)
|
||||||||||||||||
Ionis
|
Akcea
|
Eliminations
|
Ionis
Consolidated |
|||||||||||||
Assets:
|
||||||||||||||||
Cash, cash equivalents and short-term investments
|
$
|
1,959
|
$
|
390
|
$
|
-
|
$
|
2,349
|
||||||||
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
|
$
|
3,398
|
$
|
528
|
$
|
(847
|
)
|
$
|
3,079
|
|||||||
Liabilities and stockholders’ equity:
|
||||||||||||||||
Other current liabilities
|
$
|
102
|
$
|
35
|
$
|
(5
|
)
|
$
|
132
|
|||||||
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 Akcea Therapeutics, Inc.
|
-
|
-
|
218
|
218
|
||||||||||||
Total stockholders’ equity
|
$
|
1,955
|
$
|
479
|
$
|
(839
|
)
|
$
|
1,595
|
|||||||
Total liabilities and stockholders’ equity
|
$
|
3,398
|
$
|
528
|
$
|
(847
|
)
|
$
|
3,079
|