Ionis Pharmaceuticals (IONS) Begins Pivotal Clinical Study of Novel Antisense Medicine to Treat Patients with Alexander Disease

Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced it has initiated a pivotal clinical study of ION373 in patients with Alexander disease, a rare and generally fatal neurological disorder. Alexander disease patients experience progressive deterioration, leading to severe disability and loss of independence. Childhood onset is associated with more serious disease, with patients rarely surviving beyond adolescence. Alexander disease is caused by a genetic mutation that leads to overproduction and toxic accumulation of glial fibrillary acidic protein (GFAP) in the brain. ION373 is an investigational antisense medicine designed to reduce the level of GFAP.

ION373 is one of Ionis' wholly owned medicines that the company plans to commercialize as part of its strategy to develop and commercialize rare disease medicines primarily focused on its core neurological and cardiometabolic franchises.

"Initiation of a pivotal study of ION373 marks an important step toward bringing a novel disease modifying therapy to Alexander disease patients afflicted by this devastating disorder. The ability to begin our clinical program with a pivotal study serves as a testament to how Ionis' long-standing collaboration with the patient, medical and research communities can lay the foundation for an aggressive clinical path," said C. Frank Bennett, Ph.D., Ionis' chief scientific officer and franchise leader for neurological programs. "This study is yet another example of the power of Ionis' antisense technology to target the root causes of neurological diseases, delivering potentially transformative treatments to patients, and futures to families who previously had little hope."

Alexander disease has been estimated to occur in about one in one million births. ION373 has received orphan drug designation and rare pediatric disease designation from the U.S. Food and Drug Administration (FDA). The European Medicines Agency (EMA) has also granted orphan drug status to ION373.

The Phase 2/3 study of ION373 is a multi-center, double-blind, placebo-controlled, multiple-ascending dose (MAD) study in up to 58 patients with Alexander disease. Patients will receive ION373 or placebo for a 60-week period, after which all patients in the study will receive ION373 for a 60-week open-label treatment period.

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