HPN-100 is a pro-drug of phenylbutryrate and a pre-pro-drug of phenylacetic acid, the active moiety of Buphenyl, the only therapy currently FDA-approved as adjunctive therapy for the chronic management of patients with the most prevalent urea cycle disorders – carbamylphosphate synthetase, ornithine transcarbamylase, and argininosuccinic acid synthetase.
Bruce Scharschmidt, chief medical officer of Hyperion, said: “Completing enrollment in this Phase I/II study is an important milestone in our development program. This represents the first trial of HPN-100 in patients with urea cycle disorders and the results will be important in assessing its potential to improve care for these patients.”