Galactosemia patients are in urgent need of treatment options and the company is committed to advancing AT-007 through clinical development

Reports confirm that Applied Therapeutics Inc. has recently unveiled the initiation of a Phase 1/2 clinical trial of AT-007 in Galactosemia. The study, named ACTION-Galactosemia, will investigate pharmacokinetics and safety of AT-007, which is a central nervous system penetrant Aldose Reductase inhibitor in healthy participants.

The trial will apparently then explore safety, PK and biomarker-based effects in adult subjects with Galactosemia.

Seemingly, Applied Therapeutics is a US-based clinical-stage biopharmaceutical company engaged in developing new drugs against validated molecular targets in indications of huge unmet medical needs.

Sources close to the matter mentioned that the multicenter study will mainly register patients with Classic Galactosemia, the most familiar form of Galactosemia in the US. It will also look to recruit for a single cohort of GALK deficient patients, which is commonly observed in Europe. 

According to Chief Medical Officer of Applied Therapeutics, Riccardo Perfetti, MD, PhD, dietary constraints alone cannot prevent long term complications of disease as the body endogenously creates galactose. Galactosemia patients are in urgent need of treatment options and the company is committed to advancing AT-007 through clinical development as fast as possible owing to this urgent unmet medical requirement. 

Supposedly, Applied Therapeutics is planning to optimize recent FDA guidance that allows biomarker-based development in low prevalence, gradually progressing rare metabolic diseases, like Galactosemia. 

Aldose Reductase is allegedly responsible for creation of a toxic metabolite of galactose, termed galactitol, which has been found to be responsible for several of the long-term complications in Galactosemia.  Initially, patients will be treated with a single dose of AT-007, followed by continuous 27 days of dosing. The clinical trial endpoints will remain tolerability, safety, PK and biomarker-based analyses.

Sources commented that the clinical trial will evidently determine the capability of AT-007 to repress toxic accumulation of galactitol in patients and is looking to explore viability of measuring galactitol levels in the brain through MRI.  

Source Credits: https://ir.appliedtherapeutics.com/news-releases/news-release-details/applied-therapeutics-announces-initiation-phase-12-study-007