Peloton Therapeutics Initiates Patient Dosing In Phase II Study Of PT2385 For Von Hippel-Lindau Disease-Associated Kidney Cancer

Study conducted in collaboration with the National Cancer Institute

DALLAS--(BUSINESS WIRE)--Peloton Therapeutics, Inc., a drug discovery and development company focused on advancing novel small molecule cancer therapies, announced today initiation of patient dosing in a Phase 2 study of PT2385, the Company’s investigational first-in-class small molecule drug targeting hypoxia-inducible factor 2a (HIF-2a), for patients with von Hippel-Lindau (VHL) disease-associated kidney cancer. The primary objective of the study is to assess the overall response rate (ORR) of VHL disease-associated clear cell renal cell carcinoma (ccRCC) tumors in untreated VHL patients who received PT2385. The study is being conducted in collaboration with the National Cancer Institute (NCI).

“There is a significant need for new treatment options for VHL disease, a rare disease with serious and life-long consequences for patients, and for which there are no approved systemic therapies,” said John A. Josey, Ph.D., Peloton’s Chief Executive Officer. “The current standard of care for patients with VHL disease-associated kidney cancer is surgery, which commonly does not result in a cure for these patients.”

At the recent ASCO Genitourinary Cancers Symposium, W. Marston Linehan, M.D., Chief of the Urologic Oncology Branch of the NCI, had noted “We are getting ready to start a trial of a drug targeting the HIF-2 pathway with the Peloton PT2385 drug, which we are very encouraged about.”

PT2385 is a selective, orally active agent that blocks HIF-2a with potent anti-cancer activity in preclinical models of ccRCC. This open-label Phase 2 study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of PT2385 in patients with VHL disease who have at least one measurable VHL disease-associated ccRCC tumor (as defined by RECIST 1.1). PT2385 will be administered orally and treatment will be continuous unless there is disease progression. Changes in VHL disease-associated non-ccRCC lesions will also be evaluated.

“Patients with VHL disease-associated kidney cancer look forward to having the opportunity to participate in this first-ever study of a drug in patients with VHL disease that targets the immediate downstream effect of the VHL mutation,” said Ilene Sussman of the VHL Alliance, a patient advocacy group for individuals with VHL disease. “If the drug is shown to be effective, it may reduce the number or frequency of surgeries needed. Overall, having an oral medication that could halt or reverse the progression of this disease would greatly benefit patients.”

Further information on the clinical trial of PT2385 in VHL disease-associated kidney cancer can be found on (Study identifier: NCT03108066).

About VHL Disease

Von Hippel-Lindau disease is a hereditary cancer syndrome caused by a germline mutation in or deletion of the VHL gene, and patients are at risk for developing tumors and fluid-filled sacs (cysts) in a number of organs. Renal cell carcinoma occurs in about 70 percent of individuals with VHL disease and is the leading cause of mortality. Approximately 6,000 people have VHL disease in the U.S.

About Peloton Therapeutics

Peloton Therapeutics, Inc., is a clinical-stage biotechnology company that discovers and develops novel small molecule cancer therapies targeting unexploited molecular vulnerabilities. Peloton Therapeutics’ lead programs are small molecule inhibitors targeting hypoxia inducible factor-2a (HIF-2a), a transcription factor implicated in the development and progression of kidney and other cancers. To learn more about Peloton Therapeutics, visit

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Phone: +1-972-629-4100
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