Acasti Pharma has reported preliminary topline results from the pharmacokinetic (PK) bridging study of its drug candidate GTX-102 to treat ataxia telangiectasia (A-T).

The randomised, open-label, crossover study Phase I PK bridging study was designed to assess the PK, safety, and bioavailability of GTX-102 given as an oral spray compared to intramuscular (IM) betamethasone and to betamethasone oral solution (OS) in healthy volunteers.

Its primary objective was to assess and characterise the GTX-102’s PK profile as an oral spray.

The company stated that 48 healthy adult participants were enrolled in this eight-sequence, five-treatment, two-period, single centre study.

Findings from the study demonstrated good linearity and dose-proportionality for betamethasone blood concentrations at 0.0125 (low), 0.5 (medium) or 0.1 (high) mg/kg dose levels of GTX-102.

The betamethasone blood concentrations were within the same range of exposure as IM and OS betamethasone after receiving a high dose of GTX-102.

Additionally, no significant difference was observed between the fast rate administration of GTX-102 and the slow rate.

Acasti Pharma stated that the Cmax of GTX-102 was within the same range of exposure as OS; however, the Cmax was lower for IM formulation compared to both GTX-102 and the OS.

Acasti CEO Jan D’Alvise said: “The completion of this PK bridging study is an important milestone in the advancement of our GTX-102 program designed to provide a new and convenient therapy for treating the chronic symptoms of A-T in children with this rare genetic disorder.

“We are very pleased to report the results of this study, which we expect will now support the advancement of the program directly into Phase 3.

“Currently there are no drugs approved for A-T, and we are pleased to report the topline findings of this pivotal study as we remain committed to bring this exciting and proprietary treatment to children who suffer from A-T.”

The new formulation of GTX-102 is aimed at improving the neurological A-T symptoms in paediatric patients.