CARLSBAD, Calif. and CAMBRIDGE, Mass. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Biogen Inc. (Nasdaq: BIIB) announced the decision to terminate development of BIIB105 (ION541) an investigational antisense oligonucleotide (ASO) for amyotrophic lateral sclerosis (ALS) based on topline results from the Phase 1/2 ALSpire study. BIIB105 was designed to reduce expression of...
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CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. today announced that it has entered into a license agreement with Otsuka Pharmaceutical Co., Ltd. under which Otsuka obtains exclusive rights in Europe to commercialize donidalorsen, an investigational prophylactic treatment for hereditary angioedema (HAE). Ionis will maintain responsibility for the non-clinical and clinical development...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it has entered into a license agreement with Recordati (“Recordati”), under which Recordati obtained exclusive rights to develop and commercialize zilganersen, an investigational RNA-targeted medicine for Alexander disease (AxD), in all countries outside the U.S. Ionis will maintain sole commercial responsibility...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced additional positive results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments. These findings, which will be presented today at the 2026 American...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline data from the HALOS Phase 1/2a open-label study of ION582 in Angelman syndrome. ION582 was safe and well tolerated in the study and showed encouraging and consistent benefits in individuals living with Angelman syndrome, with the most robust improvements observed...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments. Zilganersen 50 mg demonstrated statistically significant and clinically meaningful stabilization...
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...
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...