Applied Therapeutics Starts Phase 1/2 Study of AT-007

Applied Therapeutics (NASAQ:APLT) has announced it has started its Phase 1/2 study of AT-007 in galactosemia.

As quoted in the press release:

The study, termed ACTION-Galactosemia, will investigate safety and pharmacokinetics (PK) of AT-007, a central nervous system (CNS) penetrant Aldose Reductase inhibitor in healthy volunteers, and will then explore safety, PK and biomarker effects in adult subjects with Galactosemia.

The multicenter study will primarily enroll patients with Classic Galactosemia, the most common form of Galactosemia in the US, but will also seek to enroll a single cohort of GALK deficient (GALKD) patients, which is more common in Europe.

“Dietary restriction alone does not prevent long term complications of disease because the body makes galactose endogenously,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “Galactosemia patients are in desperate need of treatment options, and we are committed to advancing AT-007 through clinical development as quickly as possible due to the urgent unmet medical need.”

Applied Therapeutics plans to leverage recent FDA guidance permitting biomarker-based development in low prevalence, slowly progressing rare metabolic diseases, such as Galactosemia.

Aldose Reductase (AR) is responsible for formation of a toxic metabolite of galactose, called galactitol, which has been shown to be responsible for many of the long-term complications in Galactosemia.  Patients will be treated initially with a single dose of AT-007, followed by 27 days of consecutive dosing. The study endpoints will be safety, tolerability, PK and biomarker-based analyses. The trial will determine the ability of AT-007 to suppress toxic accumulation of galactitol in patients and will explore feasibility of measuring galactitol levels in the brain via MRI.

Click here to read the full press release.