Beijing Avistone Biotechnology Co., Ltd. (also referred to as Avistone Biotechnology or Avistone), an innovative biotechnology company focused on precision oncology therapeutics, announced the approval to expand the use of vebreltinib (also referred to as PLB1001, APL-101, bozitinib, CBT-101) from the Chinese National Medical Products Administration (NMPA) for the treatment of adult patients with isocitrate dehydrogenase (IDH) mutant Astrocytoma (World Health Organization grade 4) with the PTPRZ1-MET fusion gene or glioblastoma with a history of low-grade disease who have the PTPRZ1-MET fusion (ZM fusion) gene and have failed previous treatments.

This supplemental New Drug Application (sNDA) approval makes vebreltinib the world’s first c-Met inhibitor approved for treatment of Central Nervous System (CNS) tumor with c-Met alteration. In November 2023, vebreltinib received conditional approval for the treatment of metastatic patients with MET Exon14 Skipping non-small cell lung cancer (NSCLC) from the NMPA.

Glioma is a refractory primary malignant intracranial tumor, accounting for approximately 46% of intracranial tumors [1]. Surgery, radiotherapy and chemotherapy are current standard treatment strategies for gliomas, and the prognosis is poor.

The 5-year overall survival (OS) rate of patients with malignant glioma is less than 10% [2]. In previous studies in Chinese patients, about 12% of brain gliomas were found to have MET fusion [3]. Among the representative type PTPRZ1-MET fusion gene (hereinafter referred to as ZM fusion) occurs in about 14% of glioblastomas with a history of lower-grade disease, often co-occurring with MET Exon 14 skipping mutations and is associated with poor prognosis [3-4].

The approval of vebreltinib for the ZM fusion-positive glioma indications is based on the positive results of the FUGEN study (NCT06105619). The FUGEN study is a randomized controlled, open label, multicenter clinical registration phase II/III study. This 84-patient study compared the efficacy and safety of vebreltinib with the dose-dense regimen of temozolomide or the combination of etoposide and cisplatin, with OS as the primary endpoint. The median OS for the vebreltinib monotherapy regimen was 6.31 months, compared to 3.38 months for the control group, reducing the risk of death by 48% and significantly improving the survival of patients with recurrent relapsing ZM fusion glioma, with an acceptable safety profile.

"The development of drugs for indications related to MET targets has always been a difficult one. This is not only a victory for translational medicine, but also marks the advent of the era of targeted therapy in the field of brain glioma,” said Dr. Hepeng Shi, Chairman, CEO, and Founder of Avistone.

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