Core Insights - The company presented full 12-month clinical results and new topline data from 18 and 24 months of the INSPIRE Phase 2/3 trial for govorestat, aimed at treating CMT-SORD, at the Peripheral Nerve Society Annual Meeting in May 2025 [2][4][10] Group 1: Clinical Trial Results - Govorestat treatment demonstrated a statistically significant 37% reduction in sorbitol levels in the sciatic nerve of CMT-SORD rats, indicating its potential effectiveness [1] - MRI data at 24 months showed a slowing of disease progression in patients treated with govorestat compared to 12 months [8][9] - Statistically significant improvements were observed in the CMT-Health Index (CMT-HI) at 12 months, correlating with reductions in blood sorbitol levels [11][12] Group 2: Safety and Tolerability - Govorestat remained generally safe and well tolerated throughout the 24 months of treatment, with similar adverse event rates between active and placebo groups [12][11] Group 3: Regulatory Pathway and Future Plans - The company is committed to submitting a New Drug Application (NDA) for govorestat for CMT-SORD treatment in 2025 [2][10] - 90% of remaining patients from the INSPIRE trial have transitioned to an open-label extension study, indicating strong patient retention and interest in continued treatment [10] Group 4: Disease Background - CMT-SORD is a rare, progressive neuromuscular disease caused by genetic mutations affecting the sorbitol dehydrogenase enzyme, leading to high levels of sorbitol in blood and tissues [3][14] - The disease was officially recognized in 2020, and there are now commercially available tests for sorbitol and genetic testing specific to CMT-SORD [3][4]
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