Rgenta Therapeutics has showcased its preclinical data at ASCO 2024 from its lead program, RGT-61159, which is being developed for the potential treatment of adenoid cystic carcinoma (ACC), colorectal cancer (CRC) and other solid tumors as well as acute myeloid leukemia (AML).

"The data presented at ASCO 2024 demonstrate efficient and specific modulation of the oncogenic transcription factor MYB, a target that historically has been difficult to drug, by RGT-61159 in a range of preclinical models of ACC and support our planned clinical development program of this novel small molecule designed to modulate RNA splicing," said Simon Xi PhD, Co-Founder and Chief Executive Officer of Rgenta.

"Down-regulation of MYB is a very promising therapeutic strategy to treat ACC and other cancers driven by MYB dysregulation including CRC, breast, small cell lung cancer and AML. We look forward to advancing this novel therapeutic in a first-in-human Phase 1a/1b clinical study in adults with ACC and CRC this year," Xi added.

"The data demonstrate that daily, single agent treatment with RGT-61159 results in significant anti-tumor activity at tolerated doses in all four ACC patient-derived xenograft (PDX) models evaluated," said Travis Wager, PhD, Co-Founder, President and Chief Scientific Officer of Rgenta.  

"Further, RGT-61159's anti-tumor activity correlated with MYB target modulation in a dose-dependent fashion, strongly supporting an on-target anti-tumor effect. Treatment with RGT-61159 was well tolerated at efficacious doses," Wager added.