Benzinga - by Vandana Singh, Benzinga Editor.
Applied Therapeutics Inc (NASDAQ:APLT) shares are trading higher after the company, on Thursday, released interim 12-month results from the ongoing Phase 3 INSPIRE trial, in which the primary and several key secondary endpoints were achieved.
The INSPIRE Phase 3 trial evaluates the effect of once-daily (QD) oral govorestat (AT-007) in 56 patients aged 16-55 with SORD Deficiency.
SORD Deficiency is a hereditary axonal neuropathy caused by mutations in the Sorbitol Dehydrogenase gene, leading to an inability to metabolize the sugar sorbitol and resulting in the accumulation of high levels of toxic sorbitol, which causes motor neuron degeneration and loss of mobility and motility.
Govorestat is a central nervous system penetrant Aldose Reductase Inhibitor, which blocks the conversion of glucose to sorbitol and has previously been shown to reduce sorbitol levels in patients with SORD Deficiency.
The data demonstrated a statistically significant correlation between sorbitol level and the prespecified CMT-FOM composite clinical endpoint (10-meter walk-run test, 4 stair climb, sit-to-stand test,6-minute walk test, and dorsiflexion) (p=0.05).
Govorestat treatment provided a sustained reduction in sorbitol levels in patients with SORD Deficiency over 12 months of treatment, which was statistically significant compared to the placebo (p
Govorestat treatment also resulted in a statistically significant effect (p=0.01) on the CMT Health Index (CMT-HI), an important patient-reported outcome measure of disease severity and well-being, a secondary endpoint in the study.
Aspects of the CMT-HI that demonstrated a treatment effect included lower limb function, mobility, fatigue, pain, sensory function, and upper limb function.
Govorestat was safe and well tolerated, with a similar incidence of adverse events between active and placebo-treated groups.
Based on current data, the company plans to request a pre-NDA meeting with the FDA’s neurology division regarding potential approval.
Price Action: APLT shares are up 34.70% at $3.61 on the last check Thursday.
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