Nuvation Bio announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to NUV-422, a cyclin-dependent kinase (CDK) 2/4/6 inhibitor, for the treatment of patients with malignant gliomas.

The FDA's Office of Orphan Drug Products grants Orphan Drug Designation to support drug candidates in development for underserved patient populations or rare disorders that affect fewer than 200,000 people in the United States. Orphan Drug Designation qualifies a candidate for various development incentives, including tax credits for eligible clinical trials, waiver of application fees and market exclusivity for seven years upon FDA approval.

"We are pleased to receive the FDA's Orphan Drug Designation, which underscores the potential of NUV-422 to address the significant unmet need of patients with high-grade gliomas," said David Hung, M.D., founder and chief executive officer of Nuvation Bio. "We look forward to continuing the development of NUV-422 through our ongoing Phase 1/2 study."

Patient enrollment and dosing is ongoing in the Phase 1/2 study of NUV-422 in adult patients with recurrent or refractory high-grade gliomas, including glioblastoma multiforme (GBM). The Phase 1 dose escalation part of the study is designed to evaluate safety and tolerability, as well as to determine a recommended Phase 2 dose based on the tolerability profile and pharmacokinetic properties of NUV-422. The Phase 2 dose expansion part of the study is expected to initially focus on patients with high-grade gliomas and is designed to evaluate overall response rate, duration of response and survival. Data from the Phase 1 portion of this study is expected in 2022.

NUV-422 is a selective small molecule resulting from Nuvation Bio's cyclin-dependent kinase (CDK) inhibitor program. CDK4/6 inhibitors are known clinical entities with proven efficacy, but cancer cells can evade these treatments by increasing signaling through CDK2. Inhibition of CDK2 in addition to CDK4/6 cuts off the tumor's natural escape route. NUV-422 is a potent inhibitor of CDK 2, 4 and 6. Preclinical studies have shown that NUV-422 has favorable blood-brain barrier penetration.

Primary tumors of the central nervous system (CNS) remain among the most difficult to treat, with a 5-year overall survival of approximately 35%. Gliomas, which begin in the glial or supportive tissue, represent 75% of malignant primary brain tumors in adults. Glioblastoma multiforme (GBM) accounts for 50% to 70% of all gliomas. More than 10,000 people in the United States each year are diagnosed with this aggressive, difficult-to-treat brain tumor. No treatment advances have been made in GBM since 2009 when bevacizumab was approved by the FDA. Temozolomide and radiation are considered the current standard of care for newly diagnosed patients with glioblastoma.