Rgenta Therapeutics Unveils Preclinical Data on RGT-61159 at ASCO 2024

Rgenta Therapeutics, a biotechnology company based in Cambridge, Massachusetts, has recently presented promising preclinical data at ASCO 2024 for its leading program, RGT-61159. This innovative oral small molecule has shown potential as a treatment for adenoid cystic carcinoma (ACC), colorectal cancer (CRC), and other solid tumors, including acute myeloid leukemia (AML).

RGT-61159 works by targeting the oncogenic transcription factor MYB, a challenging target for drug development. MYB is known to regulate cell proliferation and differentiation, and its dysregulation is implicated in various cancers such as ACC, AML, T cell acute lymphoblastic leukemias (T-ALL), CRC, and breast cancer. The drug modulates RNA splicing to selectively degrade MYB RNA, thereby inhibiting MYB protein production. This approach represents a novel strategy in cancer therapy.

Dr. Simon Xi, co-founder and CEO of Rgenta, highlighted the significance of the data presented at ASCO 2024. The results demonstrated RGT-61159’s ability to efficiently and specifically modulate MYB in preclinical models of ACC. This supports the clinical development of RGT-61159 and its potential to treat cancers driven by MYB dysregulation, including ACC and CRC. The company plans to advance RGT-61159 into a first-in-human Phase 1 clinical trial in adults with these cancers within the year.

Dr. Travis Wager, co-founder, president, and chief scientific officer at Rgenta, provided further insights into the preclinical findings. The data showed that daily administration of RGT-61159 as a single-agent treatment resulted in significant anti-tumor activity in all four ACC patient-derived xenograft (PDX) models evaluated. The drug’s efficacy was correlated with dose-dependent MYB target modulation, underscoring its on-target anti-tumor effects. Importantly, RGT-61159 was well tolerated at effective doses.

The preclinical success of RGT-61159 sets the stage for its upcoming clinical trials. The Phase 1a/1b study aims to evaluate the drug’s safety, tolerability, and preliminary efficacy in adults with ACC and CRC. This trial marks a critical step towards bringing a new therapeutic option to patients with these challenging cancers.

Rgenta Therapeutics is at the forefront of developing RNA-targeting medicines, leveraging a proprietary platform to identify targetable RNA processing events and design small-molecule modulators. This approach aims to unlock therapeutic potential for historically undruggable targets in oncology and neurological disorders.

In summary, the innovative oral small molecule RGT-61159 has demonstrated significant preclinical anti-tumor activity and specific inhibition of MYB synthesis. With plans for imminent clinical trials, Rgenta Therapeutics is poised to advance this promising therapeutic towards potential approval and availability for patients with ACC, CRC, and other MYB-driven cancers.