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(Commission File No.)
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(IRS Employer Identification No.)
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
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Title of each class
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Trading symbol
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Name of each exchange on which registered
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“
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Item 2.02 |
Results of Operations and Financial Condition.
|
Item 9.01. |
Financial Statements and Exhibits.
|
Exhibit No.
|
Description
|
Press Release dated May 6, 2020.
|
|
104
|
Cover Page Interactive Data File (embedded within the Inline XBRL document).
|
Ionis Pharmaceuticals, Inc.
|
||
Dated: May 6, 2020
|
By:
|
/s/ Patrick R. O’Neil
|
Patrick R. O’Neil
|
||
Senior Vice President, Legal, General Counsel and Chief Compliance Officer
|
• |
Growing commercial revenues combined with a substantial base of R&D revenues
|
o |
Commercial revenue from SPINRAZA® (nusinersen) royalties increased by more than 10 percent to $66 million compared to Q1 2019
|
o |
Product sales from TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen) more than doubled to $15 million compared to Q1 2019
|
o |
R&D revenue of $49 million included over $25 million from Ionis’ neurology disease franchise and $15 million from its cardiometabolic franchise
|
• |
First quarter results in line with projections to be meaningfully profitable this year
|
o |
Operating loss and net loss of $61 million and $48 million, respectively, on a GAAP basis
|
o |
Non-GAAP operating loss and net loss of $20 million and $15 million, respectively
|
• |
Cash position of $2.4 billion provides substantial financial strength 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 |
Worldwide sales increased to $565 million in the first quarter, a 9 percent increase compared to the first quarter of 2019
|
o |
Worldwide patients on treatment increased to approximately 10,800 at the end of the first quarter, including patients across commercial, expanded access and clinical trial settings
|
o |
Patient treatment is underway in the Phase 2/3 DEVOTE study evaluating the safety, tolerability and potential to achieve even greater efficacy with a higher dose of SPINRAZA
|
o |
Data from an independent study published in Lancet Neurology demonstrated statistically
significant improvement in motor function with SPINRAZA treatment in teens and adults
|
• |
TEGSEDI: launched in multiple markets for the treatment of hereditary transthyretin amyloidosis (hATTR) with polyneuropathy in adult patients
|
o |
Commercially available in 12 countries
|
o |
Launching in additional EU countries this year and expanding in Latin America through PTC Therapeutics
|
o |
Results from the NEURO-TTR Phase 3 open-label extension study were published in the European
Journal of Neurology
|
• |
WAYLIVRA: launched in the EU as the only approved treatment for adults with genetically confirmed familial chylomicronemia syndrome (FCS) at high risk for pancreatitis
|
o |
Launch progressing in Germany, Austria and through the ATU in France
|
o |
Launching in additional EU countries this year
|
• |
Roche completed enrollment in the global, GENERATION HD1 Phase 3 study in patients with Huntington’s disease
|
• |
Initiated the CARDIO-TTRansform Phase 3 clinical trial for AKCEA-TTR-LRx in patients with TTR-mediated amyloid cardiomyopathy
|
• |
Two medicines granted Fast Track Designation by the U.S. FDA
|
o |
AKCEA-APO(a)-LRx for the treatment of cardiovascular disease due to elevated Lp(a) levels
|
o |
IONIS-C9Rx for the treatment of C9orf72-ALS
|
• |
Ionis generated more than $20 million as numerous partnered medicines advanced
|
o |
$10 million from AstraZeneca for ION532, targeting APOL1 for the treatment of kidney disease
|
o |
$7.5 million from Biogen for IONIS-MAPTRx for the treatment of Alzheimer’s disease
|
o |
$5 million from Dynacure for IONIS-DNM2-2.5Rx for the treatment of centronuclear myopathies
|
• |
Ionis and Akcea reported positive topline results for AKCEA-APOCIII-LRx and vupanorsen (AKCEA-ANGPTL3-LRx)
|
• |
Results from the Phase 2 study of AKCEA-APO(a)-LRx in patients with Lp(a)-driven cardiovascular disease, highlighting the favorable safety and tolerability profile and
the potential to address a major area of unmet need, were published in the New England Journal of Medicine
|
• |
Initiated a Phase 1 study of ION224, an Ionis-owned medicine in development for the treatment of NASH
|
• |
Initiate the Phase 3 study of AKCEA-APOCIII-LRx in patients with FCS
|
• |
Refile WAYLIVRA new drug application for U.S. marketing authorization
|
• |
File for WAYLIVRA marketing approval in Brazil with PTC Therapeutics
|
• |
Report clinical proof-of-concept results for four or more programs
|
• |
Initiate a first-in-human study of ION541 in patients with sporadic ALS, conducted by Biogen
|
• |
Advance five or more new medicines into development, including several Ionis-owned medicines
|
Three months ended,
March 31,
|
||||||||
2020
|
2019
|
|||||||
Revenue:
|
||||||||
Commercial revenue:
|
||||||||
SPINRAZA royalties
|
$
|
66
|
$
|
60
|
||||
Product sales, net
|
15
|
7
|
||||||
Licensing and royalty revenue
|
3
|
1
|
||||||
Total commercial revenue
|
84
|
68
|
||||||
R&D Revenue:
|
||||||||
Amortization from upfront payments
|
21
|
36
|
||||||
Milestone payments
|
23
|
40
|
||||||
License fees
|
-
|
150
|
||||||
Other services
|
5
|
3
|
||||||
Total R&D revenue
|
49
|
229
|
||||||
Total revenue
|
$
|
133
|
$
|
297
|
Three months ended
March 31,
|
||||||||
2020
|
2019
|
|||||||
(unaudited)
|
||||||||
Revenue:
|
||||||||
Commercial revenue:
|
||||||||
SPINRAZA royalties
|
$
|
66
|
$
|
60
|
||||
Product sales, net
|
15
|
7
|
||||||
Licensing and royalty revenue
|
3
|
1
|
||||||
Total commercial revenue
|
84
|
68
|
||||||
Research and development revenue under collaborative agreements
|
49
|
229
|
||||||
Total revenue
|
133
|
297
|
||||||
Expenses:
|
||||||||
Cost of products sold
|
3
|
1
|
||||||
Research, development and patent
|
116
|
107
|
||||||
Selling, general and administrative
|
75
|
68
|
||||||
Total operating expenses
|
194
|
176
|
||||||
Income (loss) from operations
|
(61
|
)
|
121
|
|||||
Other income (expense):
|
||||||||
Investment income
|
10
|
12
|
||||||
Interest expense
|
(11
|
)
|
(12
|
)
|
||||
Income (loss) before income tax benefit (expense)
|
(62
|
)
|
121
|
|||||
Income tax benefit (expense)
|
3
|
(31
|
)
|
|||||
Net income (loss)
|
$
|
(59
|
)
|
$
|
90
|
|||
Net (income) loss attributable to noncontrolling interest in Akcea Therapeutics, Inc.
|
11
|
(6
|
)
|
|||||
Net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders
|
$
|
(48
|
)
|
$
|
84
|
|||
Basic net income (loss) per share
|
$
|
(0.35
|
)
|
$
|
0.63
|
|||
Diluted net income (loss) per share
|
$
|
(0.35
|
)
|
$
|
0.62
|
|||
Shares used in computing basic net income (loss) per share
|
139,429
|
138,582
|
||||||
Shares used in computing diluted net income (loss) per share
|
139,429
|
141,537
|
Three months ended,
March 31, 2020
(unaudited)
|
||||||||||||||||
Ionis
|
Akcea
|
Eliminations
|
Ionis
Consolidated
|
|||||||||||||
Revenue:
|
||||||||||||||||
Commercial revenue:
|
||||||||||||||||
SPINRAZA royalties
|
$
|
$66
|
$
|
-
|
$
|
-
|
$
|
66
|
||||||||
Product sales, net
|
-
|
15
|
-
|
15
|
||||||||||||
Licensing and royalty revenue
|
3
|
-
|
-
|
3
|
||||||||||||
Total commercial revenue
|
69
|
15
|
-
|
84
|
||||||||||||
Research and development revenue under collaborative agreements
|
48
|
1
|
-
|
49
|
||||||||||||
Intercompany revenue
|
1
|
-
|
(1
|
)
|
-
|
|||||||||||
Total revenue
|
118
|
16
|
(1
|
)
|
133
|
|||||||||||
Expenses:
|
||||||||||||||||
Cost of products sold
|
-
|
5
|
(2
|
)
|
3
|
|||||||||||
Research, development and patent expenses
|
99
|
17
|
-
|
116
|
||||||||||||
Selling, general and administrative
|
29
|
46
|
-
|
75
|
||||||||||||
Profit/ loss share for TEGSEDI commercialization activities
|
7
|
(7
|
)
|
-
|
-
|
|||||||||||
Total operating expenses
|
135
|
61
|
(2
|
)
|
194
|
|||||||||||
Loss from operations
|
(17
|
)
|
(45
|
)
|
1
|
(61
|
)
|
|||||||||
Other income (expense):
|
||||||||||||||||
Investment income
|
9
|
1
|
-
|
10
|
||||||||||||
Interest expense
|
(11
|
)
|
-
|
-
|
(11
|
)
|
||||||||||
Loss on early retirement of debt
|
-
|
-
|
-
|
-
|
||||||||||||
Loss before income tax benefit
|
(19
|
)
|
(44
|
)
|
1
|
(62
|
)
|
|||||||||
Income tax benefit
|
2
|
1
|
-
|
3
|
||||||||||||
Net loss
|
$
|
(17
|
)
|
$
|
(43
|
)
|
$
|
1
|
$
|
(59
|
)
|
|||||
Net loss attributable to noncontrolling interest in Akcea Therapeutics, Inc.
|
$
|
-
|
$
|
-
|
$
|
11
|
$
|
11
|
||||||||
Net loss attributable to Ionis Pharmaceuticals, Inc. common stockholders
|
$
|
(17
|
)
|
$
|
(43
|
)
|
$
|
12
|
$
|
(48
|
)
|
Three months ended,
March 31,
|
||||||||
2020
|
2019
|
|||||||
(unaudited)
|
||||||||
As reported research, development and patent expenses according to GAAP
|
$
|
116
|
$
|
107
|
||||
Excluding compensation expense related to equity awards
|
(26
|
)
|
(25
|
)
|
||||
Non-GAAP research, development and patent expenses
|
$
|
90
|
$
|
82
|
||||
As reported selling, general and administrative expenses according to GAAP
|
$
|
75
|
$
|
68
|
||||
Excluding compensation expense related to equity awards
|
(15
|
)
|
(21
|
)
|
||||
Non-GAAP selling, general and administrative expenses
|
$
|
60
|
$
|
47
|
||||
As reported operating expenses according to GAAP
|
$
|
194
|
$
|
176
|
||||
Excluding compensation expense related to equity awards
|
(41
|
)
|
(46
|
)
|
||||
Non-GAAP operating expenses
|
$
|
153
|
$
|
130
|
||||
As reported income (loss) from operations according to GAAP
|
$
|
(61
|
)
|
$
|
121
|
|||
Excluding compensation expense related to equity awards
|
(41
|
)
|
(46
|
)
|
||||
Non-GAAP income (loss) from operations
|
$
|
(20
|
)
|
$
|
167
|
|||
As reported net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders according to
GAAP
|
$
|
(48
|
)
|
$
|
84
|
|||
Excluding compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc. common stockholders
|
(39
|
)
|
(42
|
)
|
||||
Income tax effect related to compensation expense related to equity awards attributable to Ionis Pharmaceuticals, Inc.
common stockholders
|
6
|
(1
|
)
|
|||||
Non-GAAP net income (loss) attributable to Ionis Pharmaceuticals, Inc. common stockholders according to
GAAP
|
$
|
(15
|
)
|
$
|
127
|
March 31,
2020
|
December 31,
2019
|
|||||||
(unaudited)
|
||||||||
Assets:
|
||||||||
Cash, cash equivalents and short-term investments
|
$
|
2,384
|
$
|
2,500
|
||||
Contracts receivable
|
29
|
63
|
||||||
Other current assets
|
145
|
158
|
||||||
Property, plant and equipment, net
|
164
|
154
|
||||||
Other assets
|
369
|
358
|
||||||
Total assets
|
$
|
3,091
|
$
|
3,233
|
||||
Liabilities and stockholders’ equity:
|
||||||||
Other current liabilities
|
$
|
137
|
$
|
155
|
||||
Current portion of deferred contract revenue
|
121
|
118
|
||||||
0.125% convertible senior notes
|
440
|
435
|
||||||
1% convertible senior notes
|
280
|
275
|
||||||
Long-term obligations, less current portion
|
75
|
75
|
||||||
Long-term deferred contract revenue
|
468
|
490
|
||||||
Total Ionis stockholders’ equity
|
1,360
|
1,471
|
||||||
Noncontrolling interest in Akcea Therapeutics, Inc.
|
210
|
214
|
||||||
Total stockholders’ equity
|
1,570
|
1,685
|
||||||
Total liabilities and stockholders’ equity
|
$
|
3,091
|
$
|
3,233
|
March 31, 2020
(unaudited)
|
||||||||||||||||
Ionis
|
Akcea
|
Eliminations
|
Ionis
Consolidated |
|||||||||||||
Assets:
|
||||||||||||||||
Cash, cash equivalents and short-term investments
|
$
|
1,963
|
$
|
421
|
$
|
-
|
$
|
2,384
|
||||||||
Contracts receivable
|
17
|
12
|
-
|
29
|
||||||||||||
Other current assets
|
121
|
26
|
(2
|
)
|
145
|
|||||||||||
Property, plant and equipment, net
|
159
|
5
|
-
|
164
|
||||||||||||
Other assets
|
1,116
|
96
|
(843
|
)
|
369
|
|||||||||||
Total assets
|
$
|
3,376
|
$
|
560
|
$
|
(845
|
)
|
$
|
3,091
|
|||||||
Liabilities and stockholders’ equity:
|
||||||||||||||||
Other current liabilities
|
104
|
35
|
(2
|
)
|
137
|
|||||||||||
Current portion of deferred contract revenue
|
120
|
1
|
-
|
121
|
||||||||||||
0.125% convertible senior notes
|
440
|
-
|
-
|
440
|
||||||||||||
1% convertible senior notes
|
280
|
-
|
-
|
280
|
||||||||||||
Long-term obligations, less current portion
|
61
|
14
|
-
|
75
|
||||||||||||
Long-term deferred contract revenue
|
470
|
-
|
(2
|
)
|
468
|
|||||||||||
Total stockholders’ equity before noncontrolling interest
|
1,901
|
510
|
(1,051
|
)
|
1,360
|
|||||||||||
Noncontrolling interest in Akcea Therapeutics, Inc.
|
-
|
-
|
210
|
210
|
||||||||||||
Total stockholders’ equity
|
$
|
1,901
|
$
|
510
|
$
|
(841
|
)
|
$
|
1,570
|
|||||||
Total liabilities and stockholders’ equity
|
$
|
3,376
|
$
|
560
|
$
|
(845
|
)
|
$
|
3,091
|