“We are thrilled to report a positive outcome from this POC study. These results provide pharmacological validation of VRK1 as a relevant target in glioblastoma. To our knowledge, this is the first demonstration of anti-tumor effects from selective VRK1 inhibition in vivo. We look forward to presenting these results at upcoming partnering conferences,” said Martin Andersson, Chief Scientific Officer at Sprint Bioscience.

The VRK1 program targets glioblastoma tumors with low expression of VRK2, a state in which tumor cells become uniquely dependent on VRK1 for survival. This creates a therapeutic opportunity to selectively inhibit VRK1 and disrupt essential processes in VRK2-low tumors, while sparing healthy tissue that maintains normal VRK2 levels. By exploiting this tumor‑specific vulnerability, VRK1 inhibition offers a precision‑medicine strategy.

Using a highly potent and selective VRK1 inhibitor developed within the program, Sprint Bioscience demonstrated the selective killing of VRK2-low gliobastoma cells in a panel of cell lines. The in vivo POC study used a xenograft model of VRK2-low glioblastoma, and significant tumor growth inhibition was seen with a VRK1 inhibitor dosed for 21 days. The treatment was well tolerated throughout the study.

About glioblastoma

Glioblastoma remains one of the most aggressive and lethal forms of cancer, with limited therapeutic progress over the past two decades. Standard-of-care treatments, including surgery, radiation and chemotherapy, offer only modest benefits, with median survival remaining approximately 15 months and five‑year survival rates below 10 percent. These outcomes underscore an urgent need for innovative treatment approaches to deliver durable clinical benefit for patients who currently have few effective options.