Peloton Therapeutics, Inc., announced on 6/6/2018 the dosing of the first patient in a Phase 2 trial evaluating the efficacy and safety of lead investigational oncology agent, PT2977, to treat von Hippel-Lindau (VHL) disease-associated kidney cancer. PT2977 is a once-daily, oral inhibitor of HIF-2α, a transcription factor that has been implicated in the development and progression of renal cell carcinoma (RCC).

Von Hippel-Lindau (VHL) disease is a rare, hereditary cancer syndrome in which affected individuals have a mutation and/or deletion of the tumor suppressor VHL gene, and are at risk for the development of multiple tumors including renal cell cancer. The global incidence of VHL is estimated to be one in 36,000 people.

“Unfortunately, there are no approved systemic therapies available for VHL disease. Currently, the only option is surgery, sometimes required multiple times which can be devastating for patients and does not cure the disease,” said Eric Jonasch, M.D., Professor, Department of Genitourinary Medical Oncology, Division of Cancer Medicine; Director, VHL Clinical Center, The University of Texas MD Anderson Cancer Center. “We are excited about the opportunity to study PT2977 which may offer a new option as an orally available agent that could reduce the need and frequency of surgical intervention for patients with VHL-associated kidney disease.”

PT2977 is a once-daily, orally-active agent that blocks hypoxia-inducible factor-2α (HIF-2α). It is a structurally-related compound designed to be more potent with less pharmacokinetics variability compared to PT2385. PT2977 has demonstrated anti-tumor activity with a favorable safety profile in an early-stage clinical study in patients with solid tumors.