Chronic and acute administration of DRI's leading candidate drug ACT01 to animal models of FXS restored normal-behavior and corrected the underlying pathophysiology. Supported by NIH, DRI's research for the first time identified disease-relevant companion biomarkers that are linked to better understanding disease pathology and provided a confirming profile for FXS. Lack of FXS biomarkers for patient segmentation and large placebo effects contributed to previous failures by pharma companies to address this disease. DRI's proprietary FXS biomarkers, when deployed in the clinic, will allow DRI to objectively identify patients that will benefit from ACT01 in clinical trials. Our biomarkers will allow objective assessment of patient responses thus minimizing placebo interference. DRI is advancing its lead candidate toward FXS clinic.

Research & Development