Ionis Pharmaceuticals

Ionis Pharmaceuticals To Expedite Development Of Rare Neurological Disease Candidate With FDA Breakthrough Status

FDA grants Breakthrough Therapy status to ION582 based on strong Phase 1/2 results in Angelman syndrome. Phase 3 REVEAL study targeting Angelman syndrome patients is set to enroll until 2026.

On Tuesday, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to Ionis Pharmaceuticals Inc.’s  ION582 for Angelman syndrome (AS), a rare neurological disorder.

This designation follows promising results from the Phase 1/2 HALOS study, which showed significant clinical improvements in communication, cognition, and motor function, along with favorable safety data.

The Breakthrough Therapy designation aims to speed up the review of treatments for serious conditions. ION582 has also shown potential for substantial improvement over existing therapies. Ionis launched the global Phase 3 REVEAL study earlier this year, expected to enroll children and adults with AS by 2026.

William Blair noted that the BTD marks a win for Ionis's neurology division, with analyst Myles Minter highlighting that the HALOS data impressed regulators, particularly given the challenges in assessing communication in AS patients.

In the competitive landscape, ION582 now tracks closely with Ultragenyx Pharmaceutical's (RARE) apazunersen (GTX-102), which remains ahead after completing recruitment for the Aspire study.

Price Action: IONS stock is up 4.68% at $63.86 at the last check on Tuesday.

ION582 is a second-generation antisense oligonucleotide (ASO) drug that targets UBE3A-ATS to treat Angelman syndrome by increasing the production of UBE3A protein. As an ASO, it's a type of synthetic nucleic acid designed to block or modify the expression of specific genes. While the exact chemical structure isn't publicly detailed, it's known to be a 2'-MOE ASO that activates the RNase H cleaving mechanism.