AS is a rare neurodevelopmental disorder caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide.
The data demonstrated consistent and encouraging clinical improvement on assessing all functional domains, including communication, cognition, and motor function measures.
Overall, 97% of people in the medium and high dose groups saw an improvement in overall AS symptoms.
In the 51-patient HALOS study, ION582 showed favorable safety and tolerability at all dose levels.
Ionis plans to meet with regulators later this year to review and confirm its Phase 3 study design. This puts the company on track for a pivotal study initiation in the first half of 2025.
William Blair writes that the update is a clear positive for Ionis, positioning ION582 as a key asset in its wholly owned portfolio after recently reacquiring sole rights from Biogen Inc (NASDAQ:BIIB).
William Blair reiterates the Outperform rating on Ionis.
The analyst notes that the consistent improvements across various metrics in the robust dataset, which will likely be used as a primary endpoint for approval, boost confidence in the drug’s potential benefits.
While slightly behind a peer program from Ultragenyx Pharmaceutical Inc (NASDAQ:RARE), Ionis has largely closed the development timeline gap and could be differentiated from a safety perspective, making ION582 an attractive asset for future growth given lack of approved therapies for the indication currently.
Price Action: IONS stock is up 4.50%, trading at $49.97 at last check Monday.
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