Benzinga | Dec 9, 2020 07:08AM EST

Applied Therapeutics Announces MRS Data From ACTION-Galactosemia Study

NEW YORK, Dec. 09, 2020 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (NASDAQ:APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced magnetic resonance spectroscopy (MRS) data on reduction of galactitol levels in the brain of Galactosemia patients treated with AT-007 in the ACTION-Galactodsemia adult study. Overall, plasma reduction in galactitol correlated with brain reduction in galactitol. There were two exceptions, which may have resulted from incomplete peak separation on the MRS scans. At the two doses which demonstrated statistically significant reduction in plasma galactitol, 20 and 40mg/kg, 3 out of 4 patients displayed substantial galactitol reduction ranging from 61.94% to 69.80% reduction from baseline.



"We are pleased to share our MRS data from ACTION-Galactosemia and believe this represents an important scientific advancement in the field of Galactosemia research," said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. "We have demonstrated that galactitol can be quantitated in the brain of Galactosemia patients on a restricted diet, and that levels of galactitol in the brain can be reduced through treatment with a AT-007, a CNS penetrant aldose reductase inhibitor."

Additional data on galactitol reduction in ACTION-Galactosemia can be found below. Applied Therapeutics will hold a conference call to discuss the data in more detail at 8:30 a.m. ET, and slides can be downloaded prior to the meeting at https://ir.appliedtherapeutics.com/.

Graphic 1 accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/a3c76cbc-4979-4ad2-8937-24b1c5cf1706