A Phase 2 Study of PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma

This open-label Phase 2 study will evaluate the efficacy and safety of PT2977 in patients
with VHL disease who have at least 1 measurable RCC tumor. PT2977 will be administered orally
and treatment will be continuous. Patients will be evaluated radiologically approximately 12
weeks after initiation of treatment and every 12 weeks thereafter while continuing in the
study. Changes in VHL disease-associated non-RCC tumors will also be evaluated.

Inclusion Criteria:

- Has a diagnosis of von Hippel Lindau disease, based on a germline VHL alteration

- Has at least 1 measurable solid RCC tumor and no RCC tumor that requires immediate
surgical intervention. The diagnosis of RCC can be radiologic (histologic diagnosis
not required). Patients may have VHL disease-associated tumors in other organ systems

Exclusion Criteria:

- Has received prior treatment with PT2977 or another HIF-2α inhibitor

- Has had any systemic anti-cancer therapy (includes anti-VEGF therapy or any systemic
investigational anti-cancer agent)

- Has an immediate need for surgical intervention for tumor treatment

- Has evidence of metastatic disease on screening imaging