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With Positive Data From Ionis, Angelman Syndrome Race Intensifies

 
• By Jessica Merrill

Ionis plans to initiate a Phase III trial for ION582 in the first half of 2025, positioning it just behind Ultragenyx in the race to bring a treatment to market for the rare neurologic condition.

Progress is being made in the treatment of Angelman syndrome • Source: Shutterstock

Ionis Pharmaceuticals, Inc. is looking to initiate a Phase III trial for ION582 as quickly as possible based on positive data announced on 22 July from the Phase I/II open-label HALOS study in people with the rare neurological condition Angelman syndrome (AS).

“Our top priority is to gain alignment with the FDA rapidly and to move ION582 into Phase III development as...

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