Investigational Treatment Promising for SORD Deficiency

At the 12-month point of the 24-month prospective trial, patients randomly assigned to receive daily govorestat 20 mg/kg had statistically significant improvements on theFor the primary pharmacodynamic/biomarker endpoint, treated patients also had a sustained reduction in sorbitol levels that was statistically significant vs placebo.

There are currently no approved therapies for SORD deficiency, but the INSPIRE trial is testing govorestat, an aldose reductase inhibitor that prevents the conversion of glucose to sorbitol. "We found it interesting that these are proximal rather than distal components, suggesting — and this is not definitive —perhaps that there's a distal predominance in benefits," Shy said.

The company plans to discuss a potential new drug application submission with the US Food and Drug Administration based on this clinical data, a press statement noted.

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