Glioblastoma Multiforme

About Glioblastoma Multiforme

  • GBM is the most common and lethal form of brain cancer.
  • 15,000 new cases are diagnosed in the United States each year.1
  • Median survival in newly diagnosed patients with best available treatments is 14.6 months.
  • Standard treatment following diagnosis includes surgical resection to remove as much of the tumor as possible (debulking), followed by radiotherapy and chemotherapy with Temodar® (temozolomide, "TMZ").
  • Nearly all patients diagnosed with GBM will relapse following first-line treatment, with a 1-year survival rate of approximately 25% following failure of front-line therapy, with average 5-year survival rate less than 3%.2

Probability of Overall Survival

Probability of Overall Survivle of Glioblastoma Multiforme

Why VAL-083 for GBM

Based on historical data and our own research, we believe that VAL-083 has the potential to offer physicians and patients a new paradigm in the treatment of GBM.

O6-DNA methylguanine methyl-transferase (MGMT) is a DNA repair enzyme that causes resistance to temozolomide, the current standard of care in the treatment of GBM. High expression of the MGMT enzyme is strongly correlated with poor patient outcomes.3

We have demonstrated that VAL-083's cytotoxic mechanism has more potent activity against brain tumor cells in comparison to TMZ and can overcome resistance associated with MGMT, suggesting that VAL-083 has the potential to surpass TMZ as the current standard-of-care in the treatment of GBM.4

Our research is backed by historical clinical trials with VAL-083 that were conducted by the U.S. National Cancer Institute ("NCI") demonstrating activity against GBM that is comparable, or superior, to TMZ and other chemotherapies approved for the treatment of GBM.

We are conducting clinical trials with VAL-083 as a potential treatment for GBM patients that have failed or whose tumors express biological features, such as a high expression of MGMT, that make them unlikely to respond to currently available therapies.


1 CBTRUS Statistical Report: Primary Brain and Central Nervous System Tumors Diagnosed in the United States in 2004-2006 . Hinsdale, IL: Central Brain Tumor Registry of the United States; 2010

2 Johnson, Derek R.; O'Neill, Brian Patrick (2011). "Glioblastoma"

3 Hegi et al. N Eng. J Med (2005)

4 DelMar Abstract, AACR Annual Meeting (2012)