Applied Therapeutics Reports AT-007 SAD Data

Applied Therapeutics (NASDAQ:APLT) has announced it has finished the single ascending dose (SAD) healthy volunteer part of the Phase 1/2 study of AT-007 in galactosemia.

As quoted in the press release:

The results show that AT-007 was well tolerated, with no drug-related adverse events or dose-limiting toxicities reported.  The SAD study treated 4 cohorts of 8 subjects each and explored doses from 0.5mg/kg to 20mg/kg.  In addition to safety, AT-007 demonstrated a linear pharmacokinetic (PK) profile, favorable exposure, and half-life consistent with once-daily dosing.

“We are encouraged by the favorable safety profile in healthy volunteers, and we look forward to advancing AT-007 through the MAD study in healthy volunteers and to the Phase 2 portion of the study in patients with Galactosemia,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “Developing drugs for high unmet indications, such as Galactosemia, is critical to our mission at Applied, and we are committed to developing a potential new therapy for patients with no available treatment options.”

AT-007 will advance in parallel to a Phase 1b Multiple Ascending Dose (MAD) study in healthy volunteers (up to 7 consecutive days of treatment), and a Phase 2 study in adults with Galactosemia.  Galactosemia patients will be eligible for treatment up to 28 days total (single dose followed by 27 consecutive days of dosing).  In addition to safety and PK, the study will determine the ability of AT-007 to suppress toxic accumulation of galactitol in Galactosemia patients. Applied Therapeutics plans to employ recent FDA guidance permitting biomarker-based development in low prevalence, slowly progressing rare metabolic diseases, such as Galactosemia.

Click here to read the full press release.