Reported Sunday, Applied Therapeutics Reports 24-Month Data From INSPIRE Trial Showing Govorestat Slows Disease Progression In CMT-SORD, Reinforces Plans For 2025 NDA

Author: Benzinga Newsdesk | May 19, 2025 03:14am

•  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 the potential NDA submission of govorestat for treatment of CMT-SORD in 2025

Posted In: APLT