New top-line data from Phase II/III trial of govorestat in CMT-SORD

19 May 2025

USA-based rare disease company Applied Therapeutics (Nasdaq: APLT) saw its shares rise nearly 23% to $0.60 on Friday, ahead of a planned presentation on its lead candidate, govorestat, at the Peripheral Nerve Society (PNS) 2025 Annual Meeting, being held May 17-20 in Edinburgh, Scotland.

Applied Therapeutics presented full 12-month clinical results and new top-line data from 18 and 24 months from the INSPIRE Phase II/III trial of govorestat (AT-007) for the treatment of sorbitol dehydrogenase (SORD) deficiency, a subtype of Charcot-Marie-Tooth (CMT) disease.

A reverified, interim analysis of 49 patients evaluable for efficacy as of February 2024, conducted at 12 months of active treatment. Full clinical results from 12 months include:

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