HTL-001 is capable of targeting HOX gene over-expression in glioblastoma by disrupting the interaction between HOX proteins and their co-factor, PBX. HTL-001 induced both caspase-dependent and -independent apoptosis in Glioblastoma cell lines.
In vivo biodistribution studies confirmed that the peptide was able to cross the blood-brain barrier. Systemic delivery of HTL-001 resulted in improved control of subcutaneous murine and human xenograft tumors and improved survival in a murine orthotopic model 1).