Ionis readies move to Phase III for Angelman syndrome drug hopeful
22 Jul 2024
Phase 3OligonucleotideClinical ResultPhase 2
Ionis Pharmaceuticals is moving forward with plans to initiate Phase III development of ION582 for Angelman syndrome (AS) in the first half of 2025, following the announcement of positive detailed results from the mid-stage HALOS study on Monday.
According to Ionis, the experimental antisense oligonucleotide showed benefits in communication, cognition and motor function across a broad patient population. At six months, 97% of patients in the medium- and high-dose groups exhibited clinically meaningful improvement in overall symptoms of the rare genetic neurological disease, based on the SAS-CGI-C scale, which measures clinicians' impressions of AS symptoms.
Additionally, an objective clinician-administered assessment of clinical functioning known as Bayley-4 revealed improvements in 67% of participants for cognition, 67% for receptive communication, 69% for expressive communication, 46% for gross motor skills and 72% for fine motor function. Clinical improvements were also observed across key functional areas in the Vineland-3 and ORCA parent-reported assessment tools.
In a note to clients at the time, Baird analysts viewed Ionis' topline data positively for Ultragenyx, a key competitor in the Angelman syndrome space with its antisense oligonucleotide GTX-102. "By eliminating a situation where Ultragenyx would have been facing commercial competition from a medication backed by Biogen's substantial commercial resources, we believe this update has readthroughs positively to Ultragenyx position in Angelman."
The company reported early data on GTX-102 in May, with Baird analysts noting that cross-trial comparisons appeared to favour Ultragenyx's drug over ION582. "When looking at Ultragenyx's data we calculate that ~83% (20/24) patients saw improvements on Bayley-4 cognition measures…Similiarly, we estimate ~75% (18/24) patients saw improvements at day 170 in cohorts A&B Ultragenyx's study on Bayley measures of receptive and/or expressive communication."
They added "we believe the initial read here is that GTX-102 appears to be the more efficacious option and expect that the low rate of transient lower limb extremity weakness seen with GTX-102 should be tolerable in the context of greater efficacy in this rare disease indication."
Ultragenyx recently announced successful alignment with the FDA on its Phase III study design for GTX-102. It plans to initiate its trial by the end of this year.
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