About Glioblastoma (GBM)
Glioblastoma (GBM) is the most common and deadliest primary malignant brain tumor in adults. It is characterized by highly invasive growth, significant genetic heterogeneity, and resistance to standard treatments. GBM tumors often infiltrate surrounding brain tissue, making complete surgical removal difficult and leading to high recurrence rates. The standard treatment involves maximal safe surgical resection followed by radiotherapy and combined with adjuvant temozolomide (TMZ) chemotherapy. Despite this, the prognosis remains poor, with a median overall survival of 12 to 18 months and a 5-year survival rate of less than 10%. A significant obstacle to effective treatment is the development of resistance to TMZ, primarily due to factors such as the overexpression of MGMT (O6-methylguanine-DNA methyltransferase), mutations in TP53, and epigenetic dysregulation. Preclinical studies suggest that inhibiting HDAC8 may enhance the sensitivity of GBM cells to chemotherapy and slow tumor growth by restoring wild-type p53 function and reducing MGMT expression. Our experimental therapy has shown promise in overcoming TMZ resistance, representing a potential new approach to improve outcomes in patients with recurrent or newly diagnosed GBM.
About Metastatic Uveal Melanoma (mUM)
Uveal melanoma arises from melanocytes within the uveal tract of the eye. While localized uveal melanoma can often be treated with surgery or radiation, up to 50% of patients eventually develop metastatic disease, most commonly to the liver. Metastatic uveal melanoma (mUM) is a rare but aggressive cancer with limited treatment options and a poor prognosis—median overall survival after metastasis is typically less than one year. Unlike cutaneous melanoma, mUM is genetically distinct and generally unresponsive to conventional immunotherapies, highlighting the urgent need for novel targeted treatments.In our Phase 1 clinical trial, a complete response was observed in a patient with metastatic uveal melanoma, demonstrating encouraging early signs of anti-tumor activity. We are advancing NBM-BMX through clinical development to address this significant unmet medical need.
