ABM Therapeutics has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for ABM-1310 to treat patients with glioblastoma (GBM) harbouring the BRAF V600 mutation.

A small-molecule BRAF inhibitor, ABM-1310 is an orally administered medicine with high BRAF mutation selectivity, blood-brain barrier permeability and water solubility.

The inhibitor is currently in Phase I trials at clinical sites in China and the US, targeting advanced solid tumours with the BRAF V600 mutation.

Interim results from the US Phase I trial showed that ABM-1310 exhibited promising anti-cancer activity and a good safety profile in patients with advanced solid tumours, including primary brain tumours such as GBM and other gliomas.

In China, a new Phase I clinical study focused specifically on GBM has recently commenced.

GBM is a hard-to-treat and extremely aggressive brain tumour. Its treatment remains a significant challenge owing to tumorigenesis complexities and resistance to the therapy.

Traditional treatments such as surgical resection, radiation therapy and chemotherapy have limitations.

ABM Therapeutics, a clinical-stage biopharmaceutical company, focuses on small molecule research and the development of new medicines to treat cancer, specifically brain cancer and brain metastases from solid tumours.

ABM has been developing a pipeline for the construction of a brain medicine research and development platform known as BPKdd. The pipeline comprises several programmes at different stages of discovery and development.