China-based Betta Pharmaceuticals Co., Ltd (SHE: 300558) announced that it has received approval from the National Medical Products Administration (NMPA) to conduct a clinical study assessing its BPI-452080 in von Hippel-Lindau (VHL) syndrome-related tumors and solid tumors. This marks a significant step forward in the development of new treatments for these challenging conditions.
Mechanism of Action and Preclinical Results
BPI-452080 is a novel oral small molecule inhibitor targeting hypoxia inducible factor 2 alpha (HIF-2α). In the clinical study, it is designed to specifically block the heterodimerization of HIF-2α and HIF-1β (also known as aryl hydrocarbon receptor nuclear transporter), thereby inhibiting the transcription and translation of downstream genes. This mechanism of action helps suppress tumor angiogenesis, cell proliferation, survival, and metastasis. Preclinical studies have demonstrated its excellent in vitro activity, in vivo efficacy, good pharmacokinetics, and safety profile.
Global Landscape and Unmet Needs
Currently, Welireg (belzutifan) from US-based Merck, Sharp & Dohme (MSD, NYSE: MRK) is the only commercially available HIF-2α inhibitor globally. Welireg received marketing approval in China in November of last year. Betta’s BPI-452080 aims to address the significant unmet medical needs in treating VHL syndrome-related tumors and solid tumors, offering a potential new therapeutic option for patients.
Understanding VHL Syndrome
Von Hippel-Lindau (VHL) syndrome, also known as VHL disease, is a rare genetic disorder that causes the development of multiple benign and malignant tumors throughout the body. Key features of the condition include retinal angiomas, hemangioblastomas of the cerebellum and spinal cord, and renal cell carcinoma (RCC). The approval of BPI-452080 for clinical trials highlights the ongoing efforts to improve treatment outcomes for patients affected by this rare disorder.-Fineline Info & Tech
