Innovative Oral Small Molecule RGT-61159 Shows Promise Against AML

Rgenta Therapeutics Unveils RGT-61159's Potential

Rgenta Therapeutics has made significant strides in the fight against acute myeloid leukemia (AML) with the presentation of preclinical data showcasing the effectiveness of their lead compound, RGT-61159. This oral small molecule inhibitor specifically targets MYB, a key oncogene implicated in various cancers, including AML. The research was presented at the prestigious American Association for Cancer Research (AACR) 2025 Annual Meeting held from April 25 to 30 in Chicago, IL.

A New Frontier in Cancer Treatment

The data reveal that RGT-61159 not only exhibits robust anti-tumor activity in multiple cell-derived xenograft models of AML but also works synergistically when combined with standard care treatments. Given the challenging nature of AML, these findings come as a beacon of hope. The compound operates by reducing the levels of MYB RNA and MYB protein in a dose-dependent manner, effectively leading to the death of MYB-overexpressing cancer cells. According to Travis Wager, Ph.D., co-founder and chief scientific officer of Rgenta, “These results highlight RGT-61159's potential to treat a wide array of AML patients.”

Targeting a Difficult Oncogene

MYB has long been a considered a challenging target due to its role as an oncogenic driver in various cancers. However, RGT-61159’s mechanism works by modifying RNA splicing to inhibit MYB's activity. Simon Xi, Ph.D., co-founder and CEO of Rgenta, emphasized the significance of this approach during his presentation, stating, “Until now, MYB has been a difficult drug target. RGT-61159's unique action provides us a new avenue in treating cancers where MYB has a vital role.”

As Rgenta advances into clinical trials, including a Phase 1/a/b trial for patients with relapsed or refractory cancers, the excitement around RGT-61159 continues to build. It’s expected that the pharmaceutical landscape will shift as data further confirms RGT-61159's efficacy across various genetic alterations typical in AML.

Clinical Trials in Progress

Rgenta is currently evaluating RGT-61159’s safety and effectiveness in an ongoing multi-center, open-label Phase 1a/b clinical trial, particularly focusing on patients with advanced stages of relapsed or refractory adenoid cystic carcinoma (ACC) and colorectal cancer (CRC). With the success of preliminary testing, the company anticipates launching a new Phase 1/2 study targeting adults suffering from AML and high-risk myelodysplastic syndromes in the latter half of 2025, further establishing RGT-61159's potential medical relevance.

The Science Behind RGT-61159

The oral small molecule RGT-61159 is designed to specifically modulate RNA splicing of MYB. This modulation leads to the inhibition of oncogenic MYB protein, directly facilitating cancer cell death in the process. The implications of RGT-61159 are far-reaching, as MYB is linked to proliferation, self-renewal, and differentiation in cells, making it a critical factor in the development of numerous types of cancer.

The data highlighted at the AACR meeting emphasizes the compelling relationship between MYB’s elimination and the potent anti-cancer activities observed. RGT-61159 successfully downregulated the expression of significant oncogenes controlled by MYB, such as MYC, BCL2, FLT3, and IDH1. With evidence from multiple CDX models and various genetic backgrounds, the robustness of RGT-61159’s anti-tumor activity underscores its therapeutic promise.

Rgenta’s Future Direction

Rgenta Therapeutics is committed to tackling complex oncological and neurological disorders through innovative RNA-targeting small molecules. By leveraging their proprietary platform, they aim to identify previously undruggable targets within human diseases. With RGT-61159 at the forefront, the company is paving the way for significant advancements in cancer therapy. As the Phase 1a/b clinical trial unfolds, the scientific community watches closely, hopeful for a breakthrough in cancer treatment that could transform the lives of many.

To learn more about Rgenta Therapeutics and their promising clinical trials, visit their official website.