Company profile
Ionis Pharmaceuticals, Inc. logo

Ionis Pharmaceuticals, Inc.

Pipeline19 assets

  • ZilganersenION373 / GFAP / RNA-Targeted Therapeutic / Neurology
    Alexander DiseasePhase 1/3ActiveGlobal
  • ION582ION582 / UBE3A-ATS / RNA-Targeted Therapeutic / Neurology
    Angelman SyndromeClinicalActive
  • ION269ION269 / APP / RNA-Targeted Therapeutic / Neurology
    Alzheimer's DiseaseClinicalActive
  • ION717ION717 / PRNP / RNA-Targeted Therapeutic / Neurology
    Prion DiseaseClinicalActive
  • ION356ION356 / PLP1 / RNA-Targeted Therapeutic / Neurology
    Pelizaeus-Merzbacher DiseaseClinicalActive
  • ION859ION859 / LRRK2 / RNA-Targeted Therapeutic / Neurology
    Parkinson's DiseaseClinicalActive
  • ION464ION464 / SNCA / RNA-Targeted Therapeutic / Neurology
    Multiple System AtrophyClinicalActiveParkinson's DiseaseClinicalActive
  • ION440ION440 / MECP2 / RNA-Targeted Therapeutic / Neurology
    MECP2 Duplication SyndromeClinicalActive
  • OlezarsenApoC-III / RNA-Targeted Therapeutic / Cardiology
    Severe HypertriglyceridemiaClinicalActive
  • DonidalorsenPKK / RNA-Targeted Therapeutic / Specialty/Rare
    Hereditary AngioedemaClinicalActive
  • SapablursenTMPRSS6 / RNA-Targeted Therapeutic / Specialty/Rare
    Polycythemia VeraClinicalActive
  • UlefnersenFUS / RNA-Targeted Therapeutic / Neurology
    Amyotrophic Lateral SclerosisClinicalActive
  • TofersenSOD1 / RNA-Targeted Therapeutic / Neurology
    Amyotrophic Lateral Sclerosis (Presymptomatic SOD1)ClinicalActive
  • IONIS-MAPTRxBIIB080 / TAU / RNA-Targeted Therapeutic / Neurology
    Alzheimer's DiseaseClinicalActive
  • TominersenIONIS-HTTRx / HTT / RNA-Targeted Therapeutic / Neurology
    Huntington's DiseasePhase 2Active
  • ION306ION306 / SMN2 / RNA-Targeted Therapeutic / Neurology
    Spinal Muscular AtrophyClinicalActive
  • EplontersenTTR / RNA-Targeted Therapeutic / Cardiology
    Transthyretin-Mediated Amyloid CardiomyopathyPhase 3Active
  • ION904ION904 / Angiotensinogen / RNA-Targeted Therapeutic / Cardiology
    Treatment-Resistant HypertensionClinicalActive
  • BepirovirsenHepatitis B Virus / RNA-Targeted Therapeutic / Other Medicines
    Hepatitis B Virus InfectionPhase 3Active

Latest news15 items

All news

Ionis partner GSK announces bepirovirsen achieves unprecedented functional cure rates with potential to redefine treatment for chronic hepatitis B

– Pivotal B-Well data show significant 19% functional cure rate in the overall study population and 26% in patients with lower viral activity, compared to 0% with standard of care – – 49% of bepirovirsen recipients achieved a surface antigen level of ≤100 IU/mL one year after end of treatment in

Trial Results • Phase 3B-Well 1 and 2 pivotal data readout Thu, May 28, 2026

Ionis partner Biogen announces topline results from Phase 2 CELIA study of diranersen (BIIB080): first study to show reduction in tau pathology and cognitive benefit in patients with early Alzheimer’s disease

- Based on the strength of the biomarker and efficacy data, Biogen plans to advance diranersen to registrational development; CELIA did not meet its primary endpoint assessing dose response - - Robust reductions in tau pathology were observed across all studied doses, with results generally

Trial Results • Phase 2Results event Thu, May 14, 2026

Ionis to host 2026 virtual Annual Meeting of Stockholders

CARLSBAD, Calif. --(BUSINESS WIRE)--May 4, 2026-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will conduct its 2026 virtual Annual Meeting of Stockholders followed by a general corporate update on Thursday, June 4, 2026.

Ionis to present at upcoming investor conferences

CARLSBAD, Calif. --(BUSINESS WIRE)--May 1, 2026-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences: Bank of America Securities 2026 Health Care Conference on Tuesday, May 12, 2026 2026 RBC Capital

Ionis to hold first quarter 2026 financial results webcast

Webcast scheduled for Wednesday, April 29 at 8:30 a.m. Eastern Time CARLSBAD, Calif. --(BUSINESS WIRE)--Apr. 15, 2026-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, April 29 at 8:30 a.m. Eastern Time to discuss its first quarter 2026

Ionis announces changes to Board of Directors

– Lynne Parshall and Joseph Wender to retire at end of term in June 2026 and Peter Reikes to join Ionis Board – CARLSBAD, Calif. --(BUSINESS WIRE)--Mar. 9, 2026-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced upcoming transitions on its Board of Directors. B.

Ionis to present at upcoming investor conferences

CARLSBAD, Calif. --(BUSINESS WIRE)--Feb. 19, 2026-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in fireside chats at the following investor conferences: Oppenheimer 36 th Annual Healthcare Life Sciences Virtual Conference on Thursday, February 26,

Ionis Pharmaceuticals, Inc. • Endpoint Arena