Applied therapeutics presents full 12-month clinical results and new topline data from inspire phase 2/3 trial of govorestat in cmt-sord in late-breaking oral presentation at the peripheral nerve society 2025 annual meeting

Slowing of progression of disease observed via mri at 24 months improvements in outcomes and reduction in sorbitol maintained through 24 months statistically significant correlation between absolute reduction in sorbitol and change in 10mwrt and cmt-fom lower limb domain at 12 months, though the primary endpoint of 10mwrt at 12 months was not statistically significant (the measure has since been removed from the cmt-fom clinical outcomes assessment based upon the results of the act-cmt study*) statistically significant improvement in cmt-hi and statistically significant correlation between percent change in sorbitol and change in cmt-hi at 12 months treatment with govorestat in cmt-sord-null rats demonstrated a highly statistically significant 37% (p=0.005) reduction of sorbitol in the sciatic nerve, the tissue of interest, after treatment with govorestat.reflective of the peripheral neuropathy experienced by patients with cmt-sord. govorestat remains generally safe and well tolerated company remains committed to potential nda submission of govorestat for treatment of cmt-sord in 2025 new york, may 18, 2025 (globe newswire) -- applied therapeutics, inc. (nasdaq: aplt), a clinical-stage biopharmaceutical company dedicated to creating transformative treatments for rare disease, today presented full 12-month clinical results and new topline data from 18 and 24 months from the inspire phase 2/3 trial of govorestat (at-007) for the treatment of sorbitol dehydrogenase (sord) deficiency, a subtype of charcot-marie-tooth (cmt) disease, in an oral presentation at the peripheral nerve society (pns) 2025 annual meeting, being held may 17-20, 2025 in edinburgh, scotland.
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