Glioblastoma (GBM) is the most common and most lethal form of brain cancer. GBM affects an estimated 12,000 new patients each year in the US alone1. The median survival in newly diagnosed patients with the best available treatments is 20.5 months2.

After diagnosis, today's standard treatment includes surgical resection of the tumor followed by radiotherapy and chemotherapy with temozolomide (TMZ). Nearly all GBM patients relapse following first-line treatment, and those patients have a one-year survival rate of approximately 25%. The average five-year survival rate is less than 3%3.

VAL-083 for the Treatment of Glioblastoma

New Hope Against Unmethylated-MGMT GBM

Based on historical data and our own scientific and clinical research, we believe VAL-083 can potentially offer physicians and patients a new paradigm in the treatment of GBM.

The DNA damage inflicted on cancer cells by TMZ, the current standard of care against GBM, is repaired by the DNA repair enzyme O6-DNA methylguanine methyltransferase (MGMT). Our data indicate that VAL-083 forms inter-strand crosslinks at the N7 position of guanine on the DNA of cancer cells. Our data also indicate that this crosslink forms rapidly and is not easily repaired by the cancer cell, resulting in cell-cycle arrest and lethal double-strand DNA breaks in cancer cells. High expression of MGMT is strongly correlated with resistance to TMZ and poor patient outcomes4.

We have demonstrated that VAL-083 can overcome MGMT-related treatment resistance and has a more potent activity than TMZ against human brain tumor cells. This suggests that VAL-083 has the potential to establish a new standard of care for MGMT-unmethylated patients unlikely to benefit from TMZ.

VAL-083 has been granted orphan drug designations for the treatment of GBM from both the FDA and EMA. VAL-083 has also been granted orphan drug designations for the treatment of medulloblastoma, ovarian cancer, and diffuse intrinsic pontine glioma (DIPG) from the FDA. In addition, the FDA also granted fast track designations for VAL-083 in recurrent and newly-diagnosed unmethylated GBM.

Current Clinical Trials

We are currently conducting clinical trials for VAL-083. Because the mechanism of action of VAL-083 is unique, we are offering a new hope for patients where other treatments may not be effective.

View Our Ongoing Clinical Trials

Explore Our Scientific Publications & Presentations

Dive deeper into our work by reviewing our robust catalog of scientific publications and presentations.

View Publications and Presentations


  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. Optune Instructions For Use. Novocure 2016
  3. Johnson, Derek R.; O'Neill, Brian Patrick (2011). "Glioblastoma"
  4. Hegi et al. N Eng. J Med (2005)