US-based biotechnology company Ionis Pharmaceuticals has published positive results from the multiple ascending dose (MAD) portion of the Phase I/II HALOS study of ION582, a drug designed to treat Angelman syndrome (AS).
The international, open-label study enrolled 51 participants aged between two and 50 years across 11 sites in six countries.
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The first part of the trial was a three-month MAD study that assessed three doses of ION582, with the final evaluations conducted at six months.
All eligible patients have since proceeded to the Part II long-term extension of the study, which is currently examining two higher doses of ION582 over an additional 12 months.
The third part of the study will evaluate eligible patients for a further four years or less.
The safety and tolerability of multiple doses of ION582 administered intrathecally is the trial’s primary endpoint.
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By GlobalDataVariations in clinical function, including communication, cognition and motor function, were also assessed in the study.
Data from the trial showed ‘consistent and encouraging’ clinical improvements in all functional domains assessed, including communication, cognition and motor function.
ION582 was found to be well-tolerated across all doses and showed evidence of consistent benefits across all ages and genotypes, with clinical improvements observed in key functional areas.
Ionis Pharmaceuticals CEO Brett Monia said: “Ionis looks forward to collaborating with investigators, regulators and members of the Angelman syndrome community to initiate Phase III development for ION582 in the first half of 2025.
“These encouraging results from the HALOS study position ION582 to be the cornerstone of Ionis’ next wave of transformational, wholly owned medicines for neurological conditions, which currently includes five clinical-stage programmes.”
Ionis Pharmaceuticals is preparing to engage with regulatory authorities to review and confirm the design for its Phase III study later this year.
Earlier this year, the company’s primary candidate olezarsen met the primary endpoint in a trial involving adults with familial chylomicronaemia syndrome.
