Financial Update: Progress in CMT-SORD Treatment Initiatives

August 14, 2025 - 08:18

In a notable advancement for the treatment of Charcot-Marie-Tooth disease with sorbitol accumulation (CMT-SORD), a company has reported significant developments in its ongoing initiatives. A meeting is scheduled with the FDA in the third quarter of 2025 to discuss govorestat, a promising therapeutic option for CMT-SORD.

At the recent PNS 2025 Annual Meeting, the company presented comprehensive 12-month clinical results and new topline data from the INSPIRE Phase 2/3 trial. The findings highlighted a meaningful slowdown in disease progression, as observed through MRI scans at the 24-month mark. This data underscores the potential effectiveness of govorestat in managing the debilitating effects of CMT-SORD.

Additionally, the company has launched a new sponsored Sorbitol Assay aimed at aiding in the identification of patients who may be suspected of having CMT-SORD. This initiative is expected to enhance diagnostic accuracy and facilitate timely treatment interventions. Furthermore, the company has entered into an out-licensing agreement, marking a strategic move in its efforts to expand the reach of its innovative therapies.