Applied announces INSPIRE Phase 2/3 trial primary endpoint not significant

Applied Therapeutics (APLT) presented full 12-month clinical results and new topline data from 18 and 24 months from the INSPIRE Phase 2/3 trial of govorestat for the treatment of Sorbitol Dehydrogenase Deficiency, a subtype of Charcot-Marie-Tooth disease, in an oral presentation at the Peripheral Nerve Society 2025 Annual Meeting, being held May 17-20, 2025 in Edinburgh, Scotland. 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: Statistically significant correlation between absolute reduction in sorbitol and change in the 10MWRT and the CMT-FOM Lower Limb domain were observed. The primary endpoint, the 10MWRT, was not statistically significant. Since initiation of the INSPIRE study, the 10MWRT was removed from the CMT-FOM, which now only consists of 11 components. Govorestat treatment demonstrated a statistically significant improvement on key secondary endpoint, CMT-HI at 12 months. A statistically significant correlation between percent change in sorbitol and change in CMT-HI was observed at 12 months for the total score as well as in the mobility and balance domains. Statistically significant lowering of blood sorbitol levels were observed. Govorestat continued to be generally safe and well tolerated, with similar incidence of adverse events between active and placebo-treated groups