Drug intelligence / Profile preview

GTX-102

Development stage
Discontinued
Lead developer
Grace Therapeutics
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
Administration
Oral
01

Overview

GTX-102 is a novel, concentrated oral-mucosal spray formulation of **betamethasone**, a synthetic glucocorticoid. Developed by **Grace Therapeutics** (a subsidiary of **Acasti Pharma**), it was specifically designed to treat neurological symptoms in patients with **Ataxia Telangiectasia (A-T)**, a rare genetic disorder. The formulation allows for rapid absorption through the tongue and oral mucosa, providing a convenient administration route for pediatric populations who may have difficulty swallowing traditional oral medications. While GTX-102 demonstrated comparable bioavailability to intramuscular and oral solution forms of betamethasone in Phase 1 pharmacokinetic bridging studies, its development was deprioritized by the company in favor of other pipeline candidates, such as GTx-104.

Other names
betamethasone oral spraybetamethasone oral-mucosal spray
02

Targets

UBE3A-ATS (UBE3A antisense transcript)

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