ArQule Gets Orphan Drug Designation For ARQ 092 In Proteus Syndrome

(RTTNews) - ArQule, Inc. (ARQL) and the National Human Genome Research Institute (NHGRI) of the National Institutes of Health (NIH) announced enrollment of the first patient in the phase 1 clinical trial for Proteus syndrome. This trial, with ARQ 092, an orally available, selective pan-AKT inhibitor, marks the first clinical trial in this rare disease. Proteus syndrome is characterized by overgrowth of the skeleton, skin, adipose tissue and central nervous system.

Additionally, ArQule received orphan drug designation from the Federal Drug Administration for ARQ 092 in Proteus syndrome. This marks an important regulatory milestone and the first orphan drug designation for this population. Proteus syndrome impacts fewer than one in a million people worldwide.

The phase 1 trial for ARQ 092 in Proteus syndrome, being conducted by the NHGRI, is a biodynamic dose finding study. ArQule will be providing ARQ 092 for the trial.