12:06 PM EDT, 07/22/2024 (MT Newswires) -- Ionis Pharmaceuticals ( IONS ) said Monday that results from the multiple ascending dose part of phase 1/2 study of ION582 showed consistent clinical improvement of all functional domains in patients with Angelman syndrome.

Angelman syndrome is a rare genetic disorder causing developmental delays, speech impairment, seizures and balance issues.

The company said 97% of participants in medium and high-dose groups showed improvement in overall disease symptoms and the drug was safe and tolerable at all dose levels.

The company plans to meet with regulators later this year to review and confirm the design for a phase 3 study, which is expected to start in H1 2025.

Ionis shares were up nearly 5% in recent trading.

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