OV101 (gaboxadol) as therapy of Angelman syndrome

The randomised phase 2 trial STARS showed clinical benefit of OV101 (gaboxadol) in patients with Angelman syndrome versus placebo [1]. Improvements were seen in sleep, motor, and behavioural function, using the Clinical Global Impressions - Improvement scale (CGI-I). The authors think CGI-I may be particularly well suited for assessing clinical improvements in a highly heterogeneous disorder like Angelman syndrome.

Angelman syndrome is a rare, genetic, neurodevelopmental disorder, characterised by intellectual disability, seizures, and severe impairments in speech, behaviour, motor skills, and sleep. In the STARS trial, OV101 once-daily (15 mg) and twice-daily (10 mg, 15 mg) were tested versus placebo. The primary objective was safety and tolerability over 12 weeks.

In the 78 patients who completed the study, most adverse events were mild, with similar frequencies in all 3 groups. Global improvement was observed after 12 weeks with OV101 once-daily versus placebo (P=0.0006), with additional post-hoc analyses showing improved sleep onset latency and overall sleep and motor function. The Parent Global Impressions suggests that patients who show a clinically meaningful improvement on CGI-I (>2), also demonstrate improvements in communication, challenging behaviour, and anxiety.

1. Bird L, et al. AAN 2019, emerging science 004.



Posted on 30 July 201915 March 2021