IONIS PHARMACEUTICALS (IONS) Company Report

JAMA Neurology has published final results from the Phase 3 VALOR study and its open-label extension (OLE) for QALSODY (tofersen) in SOD1-ALS, with over 3.5 years of follow-up.
Early initiation of QALSODY was associated with a numerically slower decline in clinical function, breathing, and strength, and a reduced risk of death or permanent ventilation.
Over three years, a subset of QALSODY-treated participants regained previously lost function and strength, an outcome not previously reported in the natural history of SOD1-ALS.
For faster-progressing participants, initiating QALSODY just six months earlier was associated with a 3.4-year extension of event-free-survival.
QALSODY, an antisense oligonucleotide (ASO) discovered by Ionis, is approved in 44 countries, including accelerated approval in the US, for adults with a SOD1 gene mutation.

Dec 22, 2025, 4:20 PM

Press Release

Ionis Pharmaceuticals' Donidalorsen Secures Distribution Partnership in Central and Eastern Europe

IONS

Product Launch

GENESIS Pharma has signed an exclusive distribution agreement with Otsuka Pharmaceutical Europe Ltd. for donidalorsen in fourteen Central and Eastern European countries.
Donidalorsen, an experimental RNA-targeted medicine for hereditary angioedema (HAE), was discovered and developed by Ionis Pharmaceuticals, Inc., which licensed exclusive European commercialization rights to Otsuka in December 2023.
In November 2025, the Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending marketing authorization for donidalorsen, with the European Commission currently reviewing this recommendation.

Dec 16, 2025, 12:26 AM

Press Release

Ionis Pharmaceuticals' Donidalorsen to be marketed in Central and Eastern Europe

IONS

Product Launch

New Projects/Investments

GENESIS Pharma announced an exclusive agreement with Otsuka Pharmaceutical Europe Ltd. (OPEL) to distribute and market Donidalorsen in fourteen Central and Eastern European markets, including Bulgaria, Croatia, Cyprus, Czech Republic, Estonia, Greece, Hungary, Latvia, Lithuania, Malta, Poland, Romania, Slovakia, and Slovenia.
Donidalorsen, a drug discovered and developed by Ionis Pharmaceuticals, is designed for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.
In November 2025, the Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending marketing authorization for Donidalorsen, with the European Commission (EC) currently reviewing this for EU approval.
Otsuka Pharmaceutical Europe Ltd. acquired the exclusive rights to market Donidalorsen in Europe through a license agreement with Ionis in December 2023.

Dec 15, 2025, 2:45 PM

Press Release

Ionis-Developed Donidalorsen to be Commercialized in CEE Following Positive CHMP Opinion

IONS

Product Launch

New Projects/Investments

GENESIS Pharma has entered into an exclusive agreement with Otsuka Pharmaceutical Europe Ltd. for the commercialization of donidalorsen in 14 Central and Eastern European markets.
Donidalorsen, an investigational RNA-targeted medicine developed by Ionis Pharmaceuticals, received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in November 2025, recommending marketing authorization for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older in the EU.
Otsuka had previously acquired exclusive rights to commercialize donidalorsen in Europe through a licensing agreement with Ionis in December 2023.

Dec 15, 2025, 9:00 AM

Press Release

Ionis Pharmaceuticals Receives Dual FDA Breakthrough Therapy Designations

IONS

New Projects/Investments

Ionis Pharmaceuticals has been granted FDA Breakthrough Therapy designations for two investigational drugs: zilganersen for Alexander disease (AxD) and olezarsen for severe hypertriglyceridemia (sHTG).
Zilganersen is the first potential disease-modifying treatment for AxD, with a new drug application planned for early 2026.
Olezarsen demonstrated statistically significant reductions in triglyceride levels and acute pancreatitis events in Phase 3 studies, with a supplemental new drug application expected by year-end.
The company has shown strong market momentum, with a 129% total shareholder return over one year and a 138% increase in share price year-to-date.

Dec 1, 2025, 12:10 PM

Morningstar

Ionis Highlights Recent Product Launches, Strong Pipeline Progress, and Upcoming Approvals

IONS

Product Launch

New Projects/Investments

Guidance Update

Ionis successfully launched two independent medicines in the U.S. this year: Tryngolza for FCS (approved December last year) and Donidalorsen for hereditary angioedema (approved August this year), both showing excellent initial performance.
The company announced positive Phase 3 data for Olezarsen in severe hypertriglyceridemia (SHGG), demonstrating an 85% reduction in acute pancreatitis attacks and normalization of triglycerides in over 50% of cases, with approval anticipated in the second half of next year.
Ionis anticipates five Phase 3 readouts and two to three FDA approvals in the coming year, including for Alexander disease and Tryngolza for SHGG, and expects Phase 3 data for Eplontersen in TTR cardiomyopathy in the second half of next year.
A next-generation Spinraza product, offering once-per-year dosing and potentially greater efficacy, will enter a pivotal Phase 3 study in the first half of next year, with significantly more attractive royalty terms for Ionis (mid 20% range).

Nov 18, 2025, 1:30 PM

Transcript

Ionis Highlights Recent Product Launches, Upcoming Approvals, and Pipeline Progress at Jefferies Conference

IONS

Product Launch

Guidance Update

New Projects/Investments

Ionis successfully launched Tryngolza for familial chylomicronemia syndrome (FCS) and Donidalorsen for hereditary angioedema independently in the U.S. this year, with both launches off to excellent starts.
The company anticipates two FDA approvals next year for Alexander disease and Tryngolza for severe hypertriglyceridemia (SHGG), with a potential third approval for chronic HPV. Additionally, five Phase 3 readouts are expected next year.
Positive Phase 3 data for Olezarsen in severe hypertriglyceridemia demonstrated an 85% reduction in acute pancreatitis attacks and normalization of triglycerides in over 50% of cases, with physicians expressing eagerness to prescribe it.
A next-generation, once-per-year Spinraza is advancing to a Phase 3 study in the first half of next year, with significantly more attractive mid-20% royalty rates for Ionis compared to the current compound's mid-teens.

Nov 18, 2025, 1:30 PM

Transcript

Ionis Highlights Recent Product Launches and Anticipated 2026 Approvals and Pipeline Milestones

IONS

Product Launch

New Projects/Investments

Guidance Update

Ionis launched two medicines independently in the U.S. this year: Tryngolza for FCS (approved December last year) and DAWNZERA for hereditary angioedema (approved August this year), both of which are off to excellent starts.
The company announced positive Phase III data for severe hypertriglyceridemia (SHGG) from its CORE and CORE2 programs, demonstrating substantial reductions in triglycerides and acute pancreatitis, with approval anticipated in 2026. Positive Phase III data was also announced for a wholly-owned neurology drug for Alexander disease, with approval planned for 2026.
Ionis expects a significant year ahead with five Phase III readouts and two to three FDA approvals in 2026.
A next-generation, yearly dosed SPINRAZA product, licensed to Biogen, is expected to enter a pivotal Phase III study in the first half of 2026, offering significantly more attractive royalties in the mid-20% range compared to the current mid-teens.

Nov 18, 2025, 1:30 PM

Transcript

Ionis Pharmaceuticals Prices $770 Million Convertible Notes Offering to Refinance 2026 Notes

IONS

Debt Issuance

Convertible Preferred Issuance

Ionis Pharmaceuticals, Inc. (IONS) priced an offering of $700.0 million aggregate principal amount of 0.00% Convertible Senior Notes due 2030 in a private placement.
The initial purchasers exercised their option in full, increasing the total aggregate principal amount of notes to $770.0 million, with the sale expected to close on November 17, 2025.
The net proceeds from the offering, estimated at approximately $682.8 million (or approximately $751.2 million if the option was fully exercised), are expected to be used primarily to repurchase or repay its 0% Convertible Senior Notes due 2026.
Ionis expects to use approximately $267.6 million of the net proceeds to repurchase $200.0 million in aggregate principal amount of its 2026 notes concurrently with the offering.
The initial conversion price for the new notes is approximately $98.10 per share, representing a 35.0% premium over the last reported sale price of $72.67 per share on November 12, 2025.

Nov 17, 2025, 9:06 PM

8-K

Ionis Pharmaceuticals Updates on Commercial Performance, Pipeline, and Financial Outlook

IONS

Product Launch

Guidance Update

New Projects/Investments

Ionis Pharmaceuticals reported that Tryngolza, approved in December last year for FCS, has been beating expectations, with full-year guidance raised to $85-$95 million. The drug generated $57 million through the end of September.
Donidalorsen for HAE received on-time approval in August and is now on the market, with an expectation of greater than $500 million in peak sales and $8 million in sales by the end of this year.
The company announced "incredibly impressive" Phase 3 data for olezarsen (SHTG), projecting a potential billion-dollar opportunity and anticipating sNDA filing by year-end with approval in October next year. Additionally, impressive Phase 3 data for zilganersen (Alexander disease) was reported, with plans to bring it to market next year.
Ionis laid out a path to positive cash flow break-even in 2028. The company anticipates annual peak sales of $5 billion or more, comprising $3 billion from its wholly owned pipeline and greater than $2 billion in royalties from partnered programs.

Nov 13, 2025, 1:00 PM

Transcript

Earnings Transcripts

Quarterly earnings call transcripts for IONIS PHARMACEUTICALS.

Q3 2025

Oct 29, 2025

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Q2 2025

Jul 30, 2025

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Q1 2025

Apr 30, 2025

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IONIS PHARMACEUTICALS (IONS)

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Elizabeth L. Hougen

Frank Bennett

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Kyle Jenne

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Richard S. Geary

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B. Lynne Parshall

Joan E. Herman

Joseph H. Wender

Joseph Klein, III

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Michael Yang

Spencer R. Berthelsen

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Gina Wang

Huidong Wang

Jacob Roberge

Jason Gerbery

Jeroen Werber

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Mani Foroohar

Steven Eynav

Zaki Molvi

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