Ionis Pharmaceuticals, Inc.NASDAQ: IONS

Ionis reports first quarter 2025 financial results

Apr 30, 2025 · Issued by Ionis Pharmaceuticals, Inc. via Business Wire

Watch Ionis Pharmaceuticals explain itIonis Pharmaceuticals, Inc. (IONS) Q2 2026 Earnings Call · Replay · Jul 29

- Encouraging start to first independent launch with TRYNGOLZATM -

- On track for second independent launch with donidalorsen PDUFA August 21, 2025 -

- Increasing 2025 financial guidance by more than 20% -

CARLSBAD, Calif.--(BUSINESS WIRE)-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”) today reported financial results for the first quarter ended March 31, 2025.

“With an encouraging start to the TRYNGOLZA launch for familial chylomicronemia syndrome, the first of four independent launches expected over the next two years, Ionis’ new chapter as a fully integrated, commercial-stage biotechnology company is well underway,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “We look forward to continued momentum this year, including our second independent launch for donidalorsen in hereditary angioedema and Phase 3 results for olezarsen for severe hypertriglyceridemia in the third quarter. We also continue to advance our next wave of wholly owned neurology medicines, including ION582 for Angelman syndrome, which is on track to start Phase 3 development shortly. Our advancing pipeline of transformational medicines, together with strong commercial and financial execution, position Ionis to deliver increasing value for all stakeholders.”

First Quarter 2025 Summary Financial Results(1):

	Three months ended March 31,
		2025			2024
	(amounts in millions)
Total revenue	$	132		$	119
Operating expenses	$	278		$	269
Operating expenses on a non-GAAP basis	$	249		$	238
Loss from operations	$	(146	)	$	(150	)
Loss from operations on a non-GAAP basis	$	(117	)	$	(119	)

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

Recent Financial Highlights

Revenue increased 10% in the first quarter of 2025 compared to the same period last year, driven by higher commercial revenue including new TRYNGOLZA product revenue following approval in late December and higher SPINRAZA and WAINUA royalty revenue
Operating expenses increased slightly in the first quarter of 2025 driven by commercialization efforts for TRYNGOLZA, donidalorsen and WAINUA
Increased 2025 financial guidance reflects recent successful licensing transactions:

Full Year 2025 Guidance

Previous Guidance

New Guidance

Revenue

>$600 million

$725-750 million

Operating loss on a non-GAAP basis

3% higher frequency than placebo) were injection site reactions, decreased platelet count and arthralgia.

Please see full Prescribing Information for TRYNGOLZA.

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)

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.

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).

About Ionis Pharmaceuticals, Inc.

For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six marketed medicines and a leading pipeline in neurology, cardiology and select 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), LinkedIn and Instagram.

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, 2024, 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. TRYNGOLZATM is a 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 PHARMACEUTICALS, INC. SELECTED FINANCIAL INFORMATION Condensed Consolidated Statements of Operations (In Millions, Except Per Share Data)

	Three months ended March 31,
		2025			2024
	(unaudited)
Revenue:
Commercial revenue:
Product sales, net	$	6		$	-
Royalty revenue		64			49
Other commercial revenue		6			10
Total commercial revenue		76			59
Research and development revenue:
Collaborative agreement revenue		46			49
WAINUA joint development revenue		10			11
Total research and development revenue		56			60
Total revenue		132			119
Expenses:
Cost of sales		1			2
Research, development and patent		201			214
Selling, general and administrative		76			53
Total operating expenses		278			269
Loss from operations		(146	)		(150	)

Other income (expense):
Interest expense related to the sale of future royalties		(19	)		(18	)
Other income, net		18			25
Loss before income tax expense		(147	)		(143	)

Income tax expense		-			-

Net loss	$	(147	)	$	(143	)

Basic and diluted net loss per share	$	(0.93	)	$	(0.98	)
Shares used in computing basic and diluted net loss per share		159			146


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 March 31,
		2025			2024
	(unaudited)
As reported research, development and patent expenses according to GAAP	$	201		$	214
Excluding compensation expense related to equity awards		(20	)		(22	)
Non-GAAP research, development and patent expenses	$	181		$	192

As reported selling, general and administrative expenses according to GAAP	$	76		$	53
Excluding compensation expense related to equity awards		(9	)		(9	)
Non-GAAP selling, general and administrative expenses	$	67		$	44

As reported operating expenses according to GAAP	$	278		$	269
Excluding compensation expense related to equity awards		(29	)		(31	)
Non-GAAP operating expenses	$	249		$	238

As reported loss from operations according to GAAP	$	(146	)	$	(150	)
Excluding compensation expense related to equity awards		(29	)		(31	)
Non-GAAP loss from operations	$	(117	)	$	(119	)

As reported net loss according to GAAP	$	(147	)	$	(143	)
Excluding compensation expense related to equity awards and related tax effects		(29	)		(31	)
Non-GAAP net loss	$	(118	)	$	(112	)

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.


IONIS PHARMACEUTICALS, INC. Condensed Consolidated Balance Sheets (In Millions)

	March 31, 2025		December 31, 2024
	(unaudited)
Assets:
Cash, cash equivalents and short-term investments	$	2,145	$	2,298
Contracts receivable		40		92
Other current assets		234		230
Property, plant and equipment, net		103		94
Right-of-use assets		159		162
Other assets		132		127
Total assets	$	2,813	$	3,003

Liabilities and stockholders’ equity:
Current portion of deferred contract revenue	$	81	$	79
Other current liabilities		169		229
1.75% convertible senior notes, net		566		565
0% convertible senior notes, net		629		629
Liability related to sale of future royalties, net		536		542
Long-term lease liabilities		159		162
Long-term obligations, less current portion		56		52
Long-term deferred contract revenue		141		157
Total stockholders’ equity		476		588
Total liabilities and stockholders’ equity	$	2,813	$	3,003

Key 2025 and 2026 Value Driving Events(1)

New Product Launches
Program	Indication	2025	2026
Donidalorsen (U.S.)	HAE	•
TRYNGOLZA (U.S.)	FCS	Achieved
WAINZUA (EU)	ATTRv-PN	Achieved
Olezarsen (U.S.)	sHTG		•
Zilganersen (U.S.)	Alexander disease		•

Regulatory Actions
Program	Indication	Regulatory Action	2025	2026
Donidalorsen	HAE	U.S. approval decision	•
Donidalorsen	HAE	EU approval decision		•
TRYNGOLZA	FCS	EU approval decision	•
Olezarsen	sHTG	U.S. submission	•
Olezarsen	sHTG	U.S. approval decision		•
Zilganersen	Alexander disease	U.S. submission		•
Zilganersen	Alexander disease	U.S. approval decision		•
Nusinersen (higher dose)	SMA	U.S. and EU submissions	Achieved
Nusinersen (higher dose)	SMA	U.S. approval decision	•
WAINZUA	ATTRv-PN	EU approval decision	Achieved
Pelacarsen	Lp(a)- CVD	U.S. submission		•
Bepirovirsen	HBV	Regulatory submission(s)		•
Bepirovirsen	HBV	Regulatory decision(s)		•

Key Phase 3 Clinical Events
Program	Indication	Event	2025	2026
Olezarsen	sHTG	CORE, CORE2 and Essence data	•
Zilganersen	Alexander disease	Phase 3 data	•
ION582	Angelman syndrome	Phase 3 study start	•
ION582	Angelman syndrome	Phase 3 enrollment completion		•
Pelacarsen	Lp(a)-CVD	Lp(a) HORIZON data		•
Bepirovirsen	HBV	B-Well data		•
Eplontersen	ATTR-CM	CARDIO-TTRansform data		•
Sefaxersen	IgAN	IMAGINATION data		•
Ulefnersen	FUS-ALS	FUSION data		•

(1)	Timing expectations based on current assumptions and subject to change.
•	Indicates that the milestone is anticipated in the respective year.

View source version on businesswire.com: https://www.businesswire.com/news/home/20250430859857/en/

Ionis Investor Contact: D. Wade Walke, Ph.D. IR@ionis.com 760-603-2331

Ionis Media Contact: Hayley Soffer media@ionis.com 760-603-4679

Source: Ionis Pharmaceuticals, Inc.