Ionis Pharmaceuticals Inc.

08/01/2024 | Press release | Distributed by Public on 08/01/2024 09:16

Ionis reports second quarter 2024 financial results Form 8 K

Ionis reports second quarter 2024 financial results

WAINUATM U.S. launch progressing well; approved in Canada; EU approval decisionexpected this year
Olezarsen PDUFA December 19, 2024 for FCS
Positive Phase 3 donidalorsen data for HAE; preparing U.S. and EU regulatory submissions
On track to achieve 2024 financial guidance
CARLSBAD, Calif., August 1, 2024 - Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company"), today reported financial results for the second quarter of 2024.
"Over the first half of this year, we continued to deliver on our goal to bring a steady cadence of medicines to people with serious diseases. The WAINUA launch for hereditary ATTR polyneuropathy (ATTRv-PN) continues to progress well with AstraZeneca. QALSODY is now approved in the EU, expanding the number of patients who can benefit from the first approved treatment for a genetic form of ALS. And we are well positioned for our first independent launch with olezarsen, which was accepted for Priority Review with a December FDA action date for people with familial chylomicronemia syndrome (FCS), a serious and rare disease with no approved treatments in the U.S. Additionally, we completed enrollment in our Phase 3 olezarsen program for the much larger severehypertriglyceridemia (sHTG) patient population, keeping us on track for data in the second half of next year. And based on recent positive Phase 3 results, we believe donidalorsen, our second planned independent U.S. launch, is positioned to be a preferred choice for people with hereditary angioedema (HAE)," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "We also advanced our next wave of potentially transformational medicines, including announcing plans to independently advance ION582 into a Phase 3 study next year, based on positive data in Angelman syndrome; this program is poised to become the cornerstone of our robust wholly owned neurology pipeline. Our recent achievements, together with multiple upcoming catalysts, position Ionis to deliver next-level value for all stakeholders."

Second Quarter 2024 Summary Financial Results(1):

Three months ended
June 30,
Six months ended
June 30,
2024
2023
2024
2023
(amounts in millions)
Total revenue
$
225
$
188
$
345
$
319
Operating expenses
$
291
$
279
$
560
$
523
Operating expenses on a non-GAAP basis
$
260
$
252
$
498
$
469
Loss from operations
$
(66
)
$
(91
)
$
(215
)
$
(204
)
Loss from operations on a non-GAAP basis
$
(35
)
$
(64
)
$
(153
)
$
(150
)

(1)
Reconciliation of GAAP to non-GAAP basis contained later in this release.

1
Financial Highlights
•
Revenue increased for the second quarter and first half of 2024 by 20% and 8% compared to the same periods last year, respectively, primarily driven by an increase in R&D revenue reflecting the value Ionis' pipeline and technology continues to generate

•
Operating expenses increased in the second quarter and first half of 2024 compared to the same periods last year, reflecting continued strategic investments in late-stage development, including WAINUA for ATTR cardiomyopathy and olezarsen for sHTG, and commercialization efforts for WAINUA, olezarsen and donidalorsen

•
Reaffirmed 2024 financial guidance

Recent Marketed Medicines Highlights
•
WAINUA for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) generated sales of $16 million and $21 million resulting in royalty revenue of $4 million and $5 million in the second quarter and first half of 2024, respectively
•
WAINUA for the treatment of adults with ATTRv-PN approved in Canada
•
SPINRAZA for the treatment of spinal muscular atrophy (SMA) generated global sales of $429 million and $770 million resulting in royalty revenue of $57 million and $95 million in the second quarter and first half of 2024, respectively
•
QALSODY for the treatment of SOD1-ALS granted marketing approval in the EU

Recent Late-Stage Pipeline Highlights

•
Olezarsen achieved multiple clinical and regulatory milestones that support pursuit of two patient populations with urgent unmet need, familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG):

o
FDA accepted the NDA for patients with FCS for Priority Review with a PDUFA date of December 19, 2024

o
Presented positive Phase 3 Balance study data in patients with FCS with a simultaneous publication in the New England Journal of Medicine

o
Opened Expanded Access Program (EAP) for FCS in the U.S.

o
Completed enrollment for all Phase 3 sHTG studies: CORE pivotal study, CORE2 confirmatory pivotal study and ESSENCE supportive exposure study; on track for data across all three studies in H2:2025

o
Presented positive Phase 2b Bridge study data in patients with HTG and sHTG with a simultaneous publication in the New England Journal of Medicine
•
Donidalorsen achieved multiple clinical milestones positioning it to become the first RNA-targeted prophylactic treatment for people with hereditary angioedema (HAE):

o
Preparing to submit NDA

o
Otsuka preparing to submit MAA; expanded Otsuka EU commercial licensing agreement to include Asia Pacific

o
Presented positive Phase 3 OASIS-HAE study data in patients treated every four weeks or every eight weeks with a simultaneous publication in the New England Journal of Medicine


o
Presented positive Phase 3 OASISplus open-label extension study data in patients treated every four weeks or every eight weeks

o
Presented positive Phase 3 OASISplus switch study data in patients previously treated with other prophylactic therapies
•
Zilganersen (GFAP) Phase 3 study for the treatment of patients with Alexander disease fully enrolled; on track for data in 2025
2
•
Bepirovirsen Phase 3 studies for the treatment of patients with chronic hepatitis B (CHB) fully enrolled; on track for data in 2026

Recent Other Pipeline Updates

•
Presented positive Phase 2 data for ION582 (UBE3A), our wholly owned medicine, in patients with Angelman syndrome; preparing for meetings with global regulators ahead of planned Phase 3 study start in H1:2025
•
Presented positive Phase 2 data for ION224 (DGAT2) in patients with metabolic dysfunction-associated steatohepatitis (MASH)
•
Initiated the Phase 1/2 Orbit study of ION356 (PLP1) in patients with Pelizaeus-Merzbacher disease (PMD)
•
Discontinued development of IONIS-FB-LRx for geographic atrophy (GA) and ION541 for amyotrophic lateral sclerosis (ALS) following completion of Phase 2 studies showing favorable safety profiles and good target engagement, but insufficient efficacy to advance into Phase 3 development

Second Quarter 2024 Financial Results

"Ionis is at a critical inflection point. We have achieved important development and regulatory milestones for WAINUA, olezarsen and donidalorsen, all of which have significant potential to help patients in need. In parallel, we continue to advance our next wave of potentially transformational medicines," said Elizabeth L. Hougen, chief financial officer of Ionis. "To drive next-level of value creation for all stakeholders, we remain focused on strategically investing our capital to fully unlock the potential of our promising near-and longer-term portfolio. Our investments are focused on go-to-market preparations for our upcoming planned olezarsen and donidalorsen launches. And with our increased confidence in the potential of WAINUA and olezarsen to address broader patient populations, we are planning additional investments to scale our capabilities in line with the significant potential that these important medicines represent. Additionally, we are investing in our next wave of medicines, including pre-commercialization activities and Phase 3 development for ION582 for Angelman syndrome, which we plan to start in the first half of next year. We expect our investments today and in the years ahead will position Ionis for sustainable growth for years to come."

Revenue

Ionis' revenue was comprised of the following:

Three months ended
Six months ended
June 30,
June 30,
2024
2023
2024
2023
Revenue:
(amounts in millions)
Commercial revenue:
SPINRAZA royalties
$
57
$
61
$
95
$
111
WAINUA royalties
4
-
5
-
Other commercial revenue:
TEGSEDI and WAYLIVRA revenue, net
8
11
17
17
Licensing and other royalty revenue
3
6
15
18
Total commercial revenue
72
78
132
146
Research and development revenue:
Amortization from upfront payments
35
15
77
29
Milestone payments
53
51
60
74
License fees
38
20
38
20
Other services
15
4
16
6
Collaborative agreement revenue
141
90
191
129
WAINUA joint development revenue
12
20
22
44
Total research and development revenue
153
110
213
173
Total revenue
$
225
$
188
$
345
$
319

3
Commercial revenue in the second quarter and first half of 2024 included a new source of royalty revenue with the launch of WAINUA in the U.S. in late January 2024. Ionis' commercial revenue in the second quarter and first half of 2024 also included royalties from the net sales of QALSODY, which Biogen launched in the U.S. in the second quarter of 2023 and in the EU in the second quarter of 2024.

R&D revenue in the second quarter and first half of 2024 increased compared to the same periods last year primarily due to the amortization of upfront payments from the new collaborations with Roche and Novartis that Ionis entered into during the second half of last year. In addition, license fees increased year over year as a result of new collaborations Ionis entered into during the second quarter of 2024, including the expanded donidalorsen licensing agreement with Otsuka, which now includes the Asia-Pacific region in addition to Europe. These increases were partially offset by the decrease in WAINUA joint development revenue, which decreased as development activities relating to ATTRv-PN wound down with the launch of WAINUA for this indication.

Operating Expenses
Ionis' operating expenses increased in the second quarter and first half of 2024 compared to the same periods in 2023, consistent with expectations. SG&A expenses increased year over year primarily due to the launch of WAINUA in the U.S. and launch preparation activities for olezarsen and donidalorsen, including establishing the field team for olezarsen. R&D expenses decreased in the second quarter and were essentially flat in the first half of 2024 compared to the same periods last year as several late-stage studies have ended.

Balance Sheet

As of June 30, 2024, Ionis' cash, cash equivalents and short-term investments decreased to $2.1 billion compared to $2.3 billion at December 31, 2023. The Company plans to continue deploying its capital resources toward growth opportunities, and as previously guided, projects to end 2024 with $1.7 billion in cash, cash equivalents and short-term investments. Ionis' working capital also decreased over the same period primarily due to the Company's lower cash and short-term investments balance. We expect to make increased strategic investments in the years ahead, with a focus on late-stage programs, wholly owned assets, and our next wave of innovative medicines.

Webcast

Management will host a conference call and webcast to discuss Ionis' second quarter 2024 results at 11:30 a.m. Eastern time on Thursday, August 1, 2024. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address. To access the Company's second quarter 2024 earnings slides click here.

For more information about SPINRAZA and QALSODY, visit https://www.spinraza.com/ and https://www.qalsody.com/, respectively. QALSODY is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval may be contingent upon verification of clinical benefit in confirmatory trial(s).

INDICATION for WAINUA™ (eplontersen)
WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

IMPORTANT SAFETY INFORMATION for WAINUA™(eplontersen)

4
WARNINGS AND PRECAUTIONS
Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.

ADVERSE REACTIONS
Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%).

Please see link to U.S. Full Prescribing Information for WAINUA.

About Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter) and LinkedIn.

Ionis' Forward-looking Statement
This press release includes forward-looking statements regarding Ionis' business, financial guidance and the therapeutic and commercial potential of our commercial medicines, additional medicines in development and technologies. 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 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. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. 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, 2023, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. 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" all refer to Ionis Pharmaceuticals and its subsidiaries.

Ionis Pharmaceuticals® is a registered 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® and QALSODY® are registered trademarks of Biogen. WAINUATM is a registered trademark of the AstraZeneca group of companies.

Ionis Investor Contact:
D. Wade Walke, Ph.D.
760-603-2331

Ionis Media Contact:
Hayley Soffer
760-603-4679

5
IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Millions, Except Per Share Data)

Three months ended
Six months ended
June 30,
June 30,

2024
2023
2024
2023
(unaudited)
Revenue:
Commercial revenue:
SPINRAZA royalties
$
57
$
61
$
95
$
111
WAINUA royalties
4
-
5
-
Other commercial revenue
11
17
32
35
Total commercial revenue
72
78
132
146
Research and development revenue:
Collaborative agreement revenue
141
90
191
129
WAINUA joint development revenue
12
20
22
44
Total research and development revenue
153
110
213
173
Total revenue
225
188
345
319
Expenses:
Cost of sales
4
3
6
4
Research, development and patent
222
230
436
428
Selling, general and administrative
65
46
118
91
Total operating expenses
291
279
560
523
Loss from operations
(66
)
(91
)
(215
)
(204
)
Other income (expense):
Interest expense related to the sale of future royalties
(18
)
(18
)
(36
)
(33
)
Other income, net
18
32
42
47
Loss before income tax expense
(66
)
(77
)
(209
)
(190
)
Income tax expense
-
(8
)
-
(20
)
Net loss
$
(66
)
$
(85
)
$
(209
)
$
(210
)
Basic and diluted net loss per share
$
(0.45
)
$
(0.60
)
$
(1.43
)
$
(1.47
)
Shares used in computing basic and diluted net loss per share
146
143
146
143

6
IONIS PHARMACEUTICALS, INC.
Reconciliation of GAAP to Non-GAAP Basis:
Condensed Consolidated Operating Expenses, Loss From Operations, and Net Loss
(In Millions)

Three months ended
June 30,
Six months ended
June 30,
2024
2023
2024
2023
(unaudited)
As reported research, development and patent expenses according to GAAP
$
222
$
230
$
436
$
428
Excluding compensation expense related to equity awards
(23
)
(19
)
(45
)
(39
)
Non-GAAP research, development and patent expenses
$
199
$
211
$
391
$
389
As reported selling, general and administrative expenses according to GAAP
$
65
$
46
$
118
$
91
Excluding compensation expense related to equity awards
(8
)
(7
)
(17
)
(14
)
Non-GAAP selling, general and administrative expenses
$
57
$
39
$
101
$
77
As reported operating expenses according to GAAP
$
291
$
279
$
560
$
523
Excluding compensation expense related to equity awards
(31
)
(27
)
(62
)
(54
)
Non-GAAP operating expenses
$
260
$
252
$
498
$
469
As reported loss from operations according to GAAP
$
(66
)
$
(91
)
$
(215
)
$
(204
)
Excluding compensation expense related to equity awards
(31
)
(27
)
(62
)
(54
)
Non-GAAP loss from operations
$
(35
)
$
(64
)
$
(153
)
$
(150
)
As reported net loss according to GAAP
$
(66
)
$
(85
)
$
(209
)
$
(210
)
Excluding compensation expense related to equity awards and related tax effects
(31
)
(27
)
(62
)
(54
)
Non-GAAP net loss
$
(35
)
$
(58
)
$
(147
)
$
(156
)

Reconciliation of GAAP to Non-GAAP Basis
As illustrated in the Selected Financial Information in this press release, non-GAAP operating expenses, non-GAAP loss from operations, and non-GAAP net loss were adjusted from GAAP to exclude compensation expense related to equity awards and the related tax effects. Compensation expense related to equity awards are non-cash. 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.
7
IONIS PHARMACEUTICALS, INC.
Condensed Consolidated Balance Sheets
(In Millions)

June 30,
2024
December 31,
2023
(unaudited)
Assets:
Cash, cash equivalents and short-term investments
$
2,079
$
2,331
Contracts receivable
27
98
Other current assets
223
213
Property, plant and equipment, net
76
71
Right-of-use assets
167
172
Other assets
119
105
Total assets
$
2,691
$
2,990
Liabilities and stockholders' equity:
Current portion of deferred contract revenue
$
94
$
151
0.125% convertible senior notes, net - short-term
44
44
Other current liabilities
168
253
1.75% convertible senior notes, net
564
562
0% convertible senior notes, net
627
625
Liability related to sale of future royalties, net
534
514
Long-term lease liabilities
166
171
Long-term obligations, less current portion
41
42
Long-term deferred contract revenue
189
241
Total stockholders' equity
264
387
Total liabilities and stockholders' equity
$
2,691
$
2,990

8
Key 2024 Value Driving Events(1)
New Product Launches
Program

Indication
Achieved
WAINUA

ATTRv-PN
Olezarsen

FCS
QALSODY (EU)

SOD1-ALS

Regulatory Actions
Program

Indication

Regulatory Action
Achieved
Eplontersen

ATTRv-PN

Additional OUS filings

EMA approval decision

Additional OUS approval decision(s)
Olezarsen

FCS

NDA filing

FDA approval decision

EU filing

Canada filing
Donidalorsen

HAE

NDA filing
QALSODY

SOD1-ALS

EMA approval decision

Key Phase 3 Clinical Data Events
Program

Indication

Event
Achieved
Olezarsen

FCS

Balance study full data
Donidalorsen

HAE

OASIS-HAE topline data
Donidalorsen

HAE

OASIS-HAE full data
Donidalorsen

HAE

OASIS-Plus: OLE + Switch data
SPINRAZA

SMA

DEVOTE study data (high dose)

Key Phase 2 Clinical Data Events
Program

Indication

Event
Achieved
Donidalorsen

HAE

3-year Phase 2 OLE data
IONIS-FB-LRx

IgAN

Phase 2 data
IONIS-FB-LRx

GA

GOLDEN study data
-
ION224 (DGAT2)

NASH

Phase 2 data
ION582 (UBE3A)

Angelman syndrome

HALOS study data
ION541 (ATXN2)

ALS

ALSpire study data
-

(1)
Timing expectations based on current assumptions and subject to change.

# # #

9