IONS - Ionis says Angelman syndrome drug well tolerated in study

2023-11-13 05:41:04 ET

Ionis Pharmaceuticals ( NASDAQ: IONS ) on Saturday shared positive clinical findings from an ongoing trial of its investigational antisense medicine, ION582, in Angelman syndrome (AS).

ION582, which is being developed under a strategic collaboration with Biogen ( NASDAQ: BIIB ), is designed to inhibit the expression of the UBE3A transcript (UBE3A-ATS) for the potential treatment of AS.

Preliminary findings from Part 1 of the ongoing HALOS Phase 1/2a study suggests that ION582 was generally well tolerated at all dose levels, with encouraging electroencephalogram (EEG) activity trends and early signals of positive clinical improvement observed.

Part 1 of HALOS trial evaluating ION582 has completed enrollment with results expected in mid-2024. Part 2 is a long-term extension study, which will evaluate the drug for an additional 12 months.

Frank Bennett, EVP and chief scientific officer of Ionis, said, "We are encouraged by the completion of enrollment in the HALOS trial in Angelman syndrome as well as the positive preliminary clinical findings. We look forward to reviewing results from the study mid-year next year, which will help us define the next stage of development for ION582."

• Biogen's Uncertain Strategic Pivots Amidst MS Market Challenges
• Biogen Inc. (BIIB) Q3 2023 Earnings Call Transcript
• Biogen Inc. 2023 Q3 - Results - Earnings Call Presentation
• Biogen swings to Q3 growth after Reata acquisition (update)
• Biogen Q3 2023 Earnings Preview

For further details see: