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 with prolonged seizures, developmental delays, cognitive impairments and an increased risk of sudden death.
“The first participant to receive ION337 in the ASCEND study marks an important step toward advancing a potential disease modifying therapy for people living with Dravet syndrome,” said Holly Kordasiewicz, Ph.D., executive vice president, chief development officer, Ionis. “ION337 is our first wholly owned medicine developed using Ionis’ advanced NMA technology, which is designed to enhance the potency of our medicines and potentially support infrequent intrathecal dosing of every six months or less. This milestone reflects a new wave of scientific innovation across Ionis’ advancing neurology pipeline for people living with serious neurological conditions. We look forward to advancing the development of ION337 alongside the Dravet syndrome community.”
ASCEND (NCT07531745) is an open-label, Phase 1-2 study evaluating the safety and tolerability of ION337 in children aged 2 to 12 years with a clinical diagnosis of Dravet syndrome. The study consists of two parts: an initial 6-month single ascending dose (SAD) component, followed by a 24-month multiple ascending dose (MAD) component with dosing of ION337 every 6 months, and an additional 7-month safety follow-up period.
ION337 uses next-generation N-Methylacetamide modifications (NMA technology) designed to enhance the potency of splice modulating antisense oligonucleotides (ASOs). This advanced molecular design is intended to provide sustained activity, supporting infrequent dosing.
About ION337
ION337 is an investigational RNA-targeted medicine designed to increase production of the NaV1.1 protein, which is reduced in people with Dravet syndrome (DS) caused by certain SCN1A gene variants. The U.S. Food and Drug Administration (FDA) has granted ION337 Fast Track designation for the treatment of Dravet syndrome.
About Dravet Syndrome (DS)
Dravet syndrome is a rare, severe and lifelong developmental epileptic encephalopathy that impacts 1 in 30,000 individuals globally. Dravet syndrome typically presents in infancy and is commonly associated with prolonged, treatment-resistant seizures. People living with this condition may also experience developmental delays, speech and language difficulties, movement or balance problems and behavioral or attention challenges. Dravet syndrome is typically caused by a change in one copy of the SCN1A gene that leaves the brain with insufficient quantities of a protein called NaV1.1, disrupting signaling and leading to excessive brain activity. Current therapies are primarily focused on symptomatic management of seizures, with generally poor long-term prognosis. There are currently no approved disease modifying therapies.
About Ionis Neurology
Ionis has been at the forefront of discovering and developing leading neurological disease medicines, including SPINRAZA® (nusinersen), the first approved treatment for spinal muscular atrophy, WAINUA® (eplontersen), a medicine to treat hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), and QALSODY® (tofersen) for SOD1-ALS. The clinical-stage neurology portfolio includes 13 investigational medicines, of which six are wholly owned by Ionis. Ionis’ investigational portfolio includes medicines for which there are few or no disease modifying treatments, such as rare diseases including Alexander disease, Angelman syndrome, prion disease, multiple system atrophy and Huntington’s disease, as well as more common conditions such as Alzheimer’s disease.
About Ionis Pharmaceuticals, Inc.
For more than 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 additional 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 and follow us on X (Twitter), LinkedIn and Instagram.
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