Applied Therapeutics Reports Promising Long-Term Results for Govorestat in CMT-SORD Treatment
Applied Therapeutics, Inc. has unveiled encouraging 24-month data from its INSPIRE Phase 2/3 trial of govorestat, a potential treatment for Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD). The findings, presented at the Peripheral Nerve Society 2025 Annual Meeting, highlight a notable reduction in blood sorbitol levels and a deceleration in disease progression, as evidenced by MRI scans. Despite the primary endpoint of the 10-meter walk-run test not reaching statistical significance, the correlation between decreased sorbitol levels and enhanced functional outcomes offers a beacon of hope for patients suffering from this rare neuromuscular disorder.
The trial's results underscore govorestat's ability to maintain improvements in patient-reported outcomes and sorbitol reduction over a two-year period. A statistically significant improvement in the CMT-Health Index (CMT-HI) and a correlation between sorbitol reduction and functional improvements were observed, reinforcing the drug's potential therapeutic value. Applied Therapeutics remains on track for a New Drug Application (NDA) submission in 2025, buoyed by govorestat's consistent safety profile and tolerability in patients.
CMT-SORD, a rare and progressive hereditary neuropathy, stems from genetic mutations affecting the sorbitol dehydrogenase enzyme, leading to mobility and sensory impairments. The INSPIRE trial's design as a randomized, double-blind, placebo-controlled study provides a robust foundation for evaluating govorestat's efficacy and safety. The presentation of full 12-month clinical results alongside new topline data from 18 and 24 months offers a comprehensive view of the drug's long-term benefits and safety.
As Applied Therapeutics advances toward regulatory submission, the medical community watches closely. The potential approval of govorestat could mark a significant milestone in the treatment of CMT-SORD, offering a much-needed therapeutic option for patients grappling with this debilitating condition. The company's commitment to addressing rare diseases through innovative treatments like govorestat exemplifies the strides being made in precision medicine and patient care.
