Full Press Release Details
Ionis well-positioned for continued momentum and substantial value creation in 2026 with two new independent launches and several pivotal data
- TRYNGOLZA (olezarsen) outperforms expectations, achieved 2025 preliminary U.S. net product sales of $105M* as first FDA-approved treatment for FCS; sNDA for sHTG submitted for review -
- Increasing annual TRYNGOLZA peak net sales
guidance to >$2B for sHTG -
- Positive topline results announced for pivotal
Phase 3 program of bepirovirsen in chronic hepatitis B; first of five Phase 3 readouts from partnered programs expected this year -
CARLSBAD, Calif., January 12 - Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced highlights from the Company's 2025 achievements and provided additional updates on key
milestones expected in 2026. Ionis will provide a business update at the 44th Annual J.P. Morgan Healthcare Conference on
Tuesday, January 13 at 8:15am PT; the presentation is available today on the Ionis website here.
"2025 was a defining year for Ionis, as we successfully executed our first two independent launches as a commercial stage biotech company. We expect 2026 to be another
transformative year, poised for two additional independent launches of breakthrough therapies - olezarsen for severe hypertriglyceridemia, Ionis' first launch in a large patient population, and zilganersen for Alexander disease, Ionis' first
independent launch from our leading neurology pipeline," said Brett P. Monia, Ph.D., chief executive officer, Ionis. "This momentum is further bolstered by the promise of our late-stage Ionis owned and partnered programs, with a total of five Phase
3 readouts and four NDA submissions anticipated this year. With strong execution and multiple key catalysts expected, Ionis is well-positioned to deliver accelerating revenue growth to achieve cash flow breakeven in 2028."
Preliminary TRYNGOLZA (olezarsen) 2025 Full Year U.S. Net Sales
2026 Anticipated Highlights Include:
Ionis Owned Programs
*Indicates preliminary unaudited results.
INDICATION for TRYNGOLZA (olezarsen)
TRYNGOLZA (olezarsen) was approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides in adults with familial
chylomicronemia syndrome (FCS).
IMPORTANT SAFETY INFORMATION
TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions
requiring medical treatment have occurred.
WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills and myalgias) have been reported in patients treated
with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur.
The most common adverse reactions (incidence >5% of TRYNGOLZA-treated patients and >3% higher frequency than placebo) were injection site reactions, decreased
platelet count and arthralgia.
Please see full Prescribing Information for TRYNGOLZA.
INDICATION for DAWNZERA (donidalorsen)
DAWNZERA (donidalorsen) is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older.
IMPORTANT SAFETY INFORMATION
DAWNZERA is contraindicated in patients with a history of serious hypersensitivity reactions, including anaphylaxis, to donidalorsen or any of the excipients in DAWNZERA.
WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with DAWNZERA. If signs and symptoms of serious hypersensitivity reactions
occur, discontinue DAWNZERA and institute appropriate therapy.
Most common adverse reactions (incidence 5%) are injection site reactions, upper respiratory tract infection, urinary tract infection, and abdominal discomfort.
Please see full Prescribing Information for DAWNZERA.
About Ionis Pharmaceuticals, Inc.
For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has marketed medicines and a leading pipeline
in neurology, cardiometabolic disease 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
Ionis Forward-Looking Statements
This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of TRYNGOLZA, DAWNZERA and additional
medicines in development and technologies, and our expectations regarding development and regulatory milestones. Any statement describing Ionis' goals, expectations, financial or other
projections or guidance, 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 but not limited to those related to our
commercial products and the medicines in our pipeline, and particularly 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 Dec. 31, 2024, and most recent Form 10-Q, which are on file with the SEC. Copies of these and other documents are available at www.Ionis.com.
Ionis Pharmaceuticals and TRYNGOLZA are registered trademarks of Ionis Pharmaceuticals, Inc. DAWNZERA is a trademark of Ionis Pharmaceuticals,
Ionis Investor Contact:
D. Wade Walke, Ph.D.
Ionis Media Contact: