In mid-September 2022, specialty pharmaceutical company Acasti Pharma Inc. (“Acasti”) shared via a news release that it had initiated a pharmacokinetic study on GTX-102. Within the study, Acasti hopes to explore GTX-102’s safety, bioavailability, and pharmacokinetics in comparison to oral or intramuscular betamethasone. Although GTX-102 is being developed for patients with ataxia-telangiectasia (A-T), no patients will enroll in this study. Rather, Acasti will be enrolling 48 healthy participants. The first participant was dosed on September 13, 2022.
Within the trial, different participant groups will receive either a low, medium, or high dose of GTX-102. Researchers will then use the findings to better understand the impact of this therapy.
Acasti describes GTX-102 as:
a novel and convenient oral mucosal spray formulation of betamethasone intended to potentially improve neurological symptoms of A-T, including clinical assessments of posture and gait disturbance, and kinetic speech and oculomotor functions. Acasti also believes that GTX-102 could ease drug administration…given its application as a more convenient, concentrated and metered betamethasone liquid spray onto the tongue.
At the moment, there are no FDA-approved therapeutic options for those with ataxia-telangiectasia. Therefore, if GTX-102 is found to be beneficial, it could be huge for this patient population.
What is Ataxia-telangiectasia (AT)?
Ataxia-telangiectasia (A-T) is a rare, complex neurodegenerative disorder which affects many bodily systems, from the immune system to the nervous system. ATM gene mutations cause A-T. Normally, ATM helps to create a protein which plays a role in DNA repair, bodily system development, and cell division. However, these mutations mean that the protein is produced either way less frequently or not at all, causing cell instability and death.
Typically, ataxia-telangiectasia manifests in infancy and early childhood. Many of those affected utilize mobility assistance by adolescence. Symptoms and characteristics can include:
- Slurred speech
- Chorea (involuntary jerking movements) and/or muscle twitching
- Difficulty walking
- High blood AFP levels
- Difficulty speaking
- Gray hair and wrinkled skin during adolescence
- High sensitivity to radiation exposure
- Neuropathy
- Weakened immune system
- Chronic sinus and respiratory infections
- Difficulty moving eyes from side-to-side
- Small clusters of enlarged blood vessels in the eyes and on the skin (telangiectasia)
- Problems with balance and coordination
- Delayed physical and sexual development
- Increased risk of developing lymphoma or leukemia
