Ionis reports third quarter 2024 financial results
WAINUATM U.S. launch progressing well; approved in UK; positive CHMP opinion
Olezarsen FCS PDUFA December 19, 2024
Donidalorsen HAE PDUFA August 21, 2025; EU regulatory submission in process
On track to achieve 2024 P&L financial guidance; increased 2024 cash guidance
CARLSBAD, Calif., Nov. 6, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) (the "Company") today reported financial results for the third quarter of 2024.
"Today, we stand on the cusp of a new era for Ionis, with our first co-commercialization launch proceeding well with WAINUA, our first planned independent launch fast approaching and continued strong progress across our rich pipeline. With an upcoming December FDA action date, we are ready to independently bring olezarsen to people with familial chylomicronemia syndrome, a serious rare disease with no approved treatments in the U.S. We are similarly well positioned for our second independent launch for donidalorsen, which we believe could become a preferred treatment choice for people with hereditary angioedema, with an FDA action date of August 21, 2025," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "In parallel, we are making great progress across the rest of our rich Phase 3 pipeline. We expect Phase 3 results supporting olezarsen's second indication in severe hypertriglyceridemia and pelacarsen in Lp(a)-driven cardiovascular disease next year, as well as Phase 3 results supporting eplontersen's second indication in ATTR cardiomyopathy in the second half of 2026. We are also advancing our next wave of potentially transformational wholly owned medicines, including ION582 for Angelman syndrome, which we expect to enter Phase 3 development in the first half of next year following our positive end of Phase 2 discussion with the FDA. Our recent achievements, together with our advancing and expanding pipeline, position Ionis to deliver on our goal to bring a steady cadence of transformational medicines to people with serious diseases."
Third Quarter 2024 Summary Financial Results(1):
Three monthsendedSeptember 30,
Nine monthsendedSeptember 30,
2024
2023
2024
2023
(amounts in millions)
Total revenue
$134
$144
$479
$463
Operating expenses
$282
$287
$843
$811
Operating expenses on a non-GAAP basis
$250
$261
$749
$732
Loss from operations
($148)
($143)
($364)
($348)
Loss from operations on a non-GAAP basis
($116)
($117)
($270)
($269)
(1) Reconciliation of GAAP to non-GAAP basis contained later in this release.
Financial Highlights
Revenue for the three and nine months ended September 30, 2024 decreased by 7% and increased by 3% compared to the same periods last year, respectively. Ionis continued to generate revenue from diverse sources, including a new source of royalty revenue with the launch of WAINUA in the U.S in the first quarter
Operating expenses for the three and nine months ended September 30, 2024 increased as planned compared to the same periods last year, excluding certain one-time costs in 2023, reflecting continued investments in late-stage development, including WAINUA for ATTR cardiomyopathy (ATTR-CM) and olezarsen for severe hypertriglyceridemia (sHTG), and commercialization efforts for WAINUA, olezarsen and donidalorsen
Reaffirmed 2024 P&L financial guidance, increased cash guidance to $2.2 billion reflecting proceeds from equity offering
Recent Marketed Medicines Highlights
WAINUA (WAINZUA in Europe) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) achieved multiple commercial and regulatory milestones:
Generated sales of $23 million and $44 million resulting in royalty revenue of $5 million and $10 million in the three and nine months ended September 30, 2024, respectively
Received positive Committee for Medicinal Products for Human Use (CHMP) opinion from European Medicines Agency (EMA) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
Approved in UK by the Medicines and Healthcare products Regulatory Agency (MHRA) with an accelerated National Institute for Health and Care Excellence (NICE) recommendation; earning $30 million from AstraZeneca
Launch underway in Canada, following approval and reimbursement from Health Canada
SPINRAZA® (nusinersen) for the treatment of spinal muscular atrophy (SMA) generated global sales of $381 million and $1.2 billion resulting in royalty revenue of $57 million and $152 million in the three and nine months ended September 30, 2024, respectively
Positive Phase 2/3 DEVOTE study data presented from higher dose nusinersen; global regulatory applications planned
QALSODY® (tofersen) granted marketing approval in China for the treatment of SOD1-ALS
Recent Late-Stage Pipeline Highlights
Olezarsen positioned to potentially treat two patient populations with urgent unmet need, familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG):
NDA for patients with FCS under FDA Priority Review with a PDUFA date of December 19, 2024
Marketing authorization application (MAA) under regulatory review by the EMA
Ongoing pivotal development program for sHTG on track for data in H2:2025
Donidalorsen Phase 3 data position it to potentially launch next year as the first RNA-targeted prophylactic treatment for people with hereditary angioedema (HAE):
FDA accepted the NDA for patients with HAE with a PDUFA date of August 21, 2025; Otsuka, Europe and Asia Pacific partner, preparing to submit MAA
Presented positive Phase 2 open label extension (OLE) study data in patients treated up to three years with every four weeks or every eight weeks dosing
Zilganersen Phase 3 study fully enrolled as a potential treatment for Alexander disease; on track for data in 2025
Granted Fast Track designation by the FDA
ION582 achieved important clinical and regulatory milestones enabling initiation of Phase 3 development in Angelman syndrome (AS) in H1:2025:
Presented positive Phase 1/2 data in patients with AS at Angelman Syndrome Foundation (ASF) Family Conference
Completed positive End-of-Phase 2 discussion with FDA, included alignment on Phase 3 design
Reported positive data from the Phase 2 study of IONIS-FB-LRx in patients with immunoglobulin A nephropathy (IgAN); Roche continues to advance IONIS-FB-LRx in the Phase 3 IMAGINATION study
Recent Other Pipeline Updates
Sapablursen for the treatment of polycythemia vera granted orphan drug designation by FDA; enrollment complete in Phase 2 IMPRSSION study with data expected in 2025
IONIS-MAPTRx (BIIB080) enrollment complete in Phase 2 CELIA study in patients with early Alzheimer's disease (AD); data expected in 2026
Initiated first in human studies with multiple medicines from neurological disease pipeline:
Phase 1/2 Orbit study of ION356 (PLP1) in patients with Pelizaeus-Merzbacher disease (PMD)
Phase 1/2 HERO study of ION269 (APP), for the potential treatment of Alzheimer's disease (AD), which is initially being evaluated in patients with Down syndrome (DS) who have a genetic risk for developing AD
Phase 1/2 ATTUNE study of ION440 (MECP2) in patients with MECP2 duplication syndrome
Third Quarter 2024 Financial Results
"This year has been marked by strong delivery on our pipeline and business goals, which position Ionis to deliver on our vision of bringing a steady cadence of innovative medicines to patients in need. Fully realizing these significant opportunities requires substantial investment. As a result, we recently executed an equity offering that extends our cash runway, enabling us to continue to invest in the numerous attractive opportunities ahead of us, including our near-term commercial launches with multi-billion-dollar revenue potential and our rich late and mid-stage pipeline," said Elizabeth L. Hougen, chief financial officer of Ionis. "Looking beyond this year, we will continue to invest in go-to-market preparations for our planned olezarsen and donidalorsen launches. Additionally, with our increased confidence in the potential of WAINUA and olezarsen to address broader patient populations, we plan to scale our capabilities in line with the significant potential of these important medicines. At the same time, we are investing in our next wave of medicines, including pre-commercialization activities and Phase 3 development for ION582 for Angelman syndrome and zilganersen for Alexander disease. We expect our investments today will position Ionis for sustainable growth for years to come."
Revenue
Ionis' revenue was comprised of the following:
Three months endedSeptember 30,
Nine months endedSeptember 30,
2024
2023
2024
2023
Revenue:
(amounts in millions)
Commercial revenue:
SPINRAZA royalties
$57
$67
$152
$179
WAINUA royalties
5
-
10
-
Other commercial revenue:
TEGSEDI and WAYLIVRA revenue, net
9
8
26
25
Licensing and other royalty revenue
5
9
19
26
Total commercial revenue
76
84
207
230
Research and development revenue:
Amortization from upfront payments
28
18
105
47
Milestone payments
16
16
76
90
License fees
-
5
38
25
Other services
1
5
18
11
Collaborative agreement revenue
45
44
237
173
WAINUA joint development revenue
13
16
35
60
Total research and development revenue
58
60
272
233
Total revenue
$134
$144
$479
$463
Commercial revenue for the three and nine months ended September 30, 2024 included a new source of royalty revenue with the launch of WAINUA in the U.S. in ...
