CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for zilganersen, an investigational RNA-targeted medicine for Alexander disease (AxD), a rare, progressive and often fatal neurological condition. The FDA has set a Prescription Drug User Fee...
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CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it has completed enrollment in the pivotal cohort (Cohort 1) of the global Phase 3 REVEAL study evaluating obudanersen (ION582), an investigational RNA-targeted medicine for people living with Angelman syndrome (AS), a serious and rare neurodevelopmental disorder associated with significant...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the first participant has been dosed in the Phase 1-2 ASCEND study of ION337, an investigational RNA-targeted medicine for people living with Dravet syndrome, a rare, severe and lifelong neurological disorder. Dravet syndrome typically begins in infancy and is associated...
CARLSBAD, Calif. – Ionis Pharmaceuticals (NASDAQ: IONS) today announced that the company has launched a new grant program that provides funding to U.S.-based researchers whose work advances the understanding of transthyretin amyloidosis (ATTR), facilitates early diagnosis and enhances disease management of patients living with ATTR amyloidosis. The Janice Wiesman Young...
CARLSBAD, Calif. – Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapies, announced today initiation of OASIS-HAE, the registrational study in the donidalorsen (formerly IONIS-PKK-LRx) Phase 3 clinical program. Donidalorsen is an investigational antisense medicine designed to reduce the production of prekallikrein, which plays a key role in the activation...
CARLSBAD, Calif. – Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced the initiation of a Phase 3 clinical trial of ION363 in patients with amyotrophic lateral sclerosis (ALS) with mutations in the fused in sarcoma gene (FUS). Patients with a mutation in the FUS gene develop a rare form of ALS, referred...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to the investigational medicine olezarsen for the treatment of familial chylomicronemia syndrome (FCS), a rare, genetic disease characterized by extremely elevated triglyceride levels and recurrent acute pancreatitis (AP)....
Completion of enrollment anticipated in 2026 CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the first participant has been dosed in the global Phase 3 REVEAL study, which is designed to evaluate the efficacy and safety of ION582, an investigational medicine for the treatment of people living with...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Ionis and AstraZeneca’s eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults. The FDA grants development programs Fast Track designation to facilitate...
CARLSBAD, Calif. – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced new long-term data for DAWNZERA™ (donidalorsen), the first and only RNA-targeted prophylactic medicine for hereditary angioedema (HAE), to be presented at the 2025 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting in Orlando, Florida. Results demonstrate the long-term durability and safety...