What is Glioblastoma (GBM)?
Glioblastoma (also known as Glioblastoma multiforme or GBM) is an aggressive cancerous (malignant) tumor that develops in the brain or spinal cord. Glioblastoma forms from cells called astrocytes that support nerve cells. GBM usually begin as a grade 4 tumor, which means it’s the fastest growing of all brain tumors.
There is currently no cure for glioblastoma. Fewer than 10% of patients live more than five years after diagnosis, and nearly all experience disease relapse following initial treatment.
Current standard-of-care therapy involves surgical resection, radiation therapy (RT), and/or chemotherapy (e.g., temozolomide; TMZ). However, acquired chemoresistance is common, and response rates to a second cycle of TMZ chemotherapy in recurrent gliomas are less than 10%.
Recent studies have highlighted the critical role of mitochondrial dysfunction in GBM progression, including altered protein expression, metabolic reprogramming, mitochondrial-dependent regulation of apoptotic (programmed cell death) pathways as well as a high glycolytic phenotype (Warburg effect).
Symptoms of GBM
GBM symptoms vary from person to person. They may include:
- Headaches
- Seizures
- Nausea and vomiting
- Blurred vision, double vision, or loss of peripheral (side) vision
- Changes in mental function, mood or personality
- Difficulty with balance
- Difficulty with speech
BPM31510 for GBM Clinical Trial
BPGbio recently completed enrollment of a Phase 2b clinical study evaluating BPM31510 in subjects with newly diagnosed glioblastoma (GBM). This single arm, non-randomized, multicenter, US-based open-label trial was designed to assess the safety and efficacy of BPM31510 followed by standard of care therapy (radiation and concurrent temozolomide treatment).
Primary endpoints of the study are progression-free survival at 6 months (PFS6) and 12 months (PFS12); secondary endpoints include overall survival and safety.
Latest Trial-in-Progress Update
Interim data from the Phase 2 study of BPM31510 combined with vitamin K, presented in October 2025 at the European Society of Medical Oncology (ESMO), demonstrated encouraging signs of activity in patients with newly diagnosed GBM receiving standard chemoradiation. Notably, the findings suggest a potential benefit in MGMT-unmethylated patients, a population with urgent need for new therapeutic options due to their poor response to current treatments.
Summary of safety, efficacy, and next steps:
- BPM31510 is well-tolerated and does not exacerbate toxicity of chemoradiation; no new drug related serious adverse events (SAEs) have been observed in treatment naïve patients in a front-line setting.
- Prophylactic vitamin K administration has significantly reduced any grade 1 elevations in PTT/INR, improving the safety profile concerning bleeding risk.
- Enrollment for the BPM31510 trial (NCT04752813) is now complete.
- A contemporaneous control arm is being built with EHR (electronic health records) data and discussions are ongoing with several sites to develop a case-control comparison.
For more information about BPGbio’s BPM31510 GBM trial, please click here.
