Rgenta Therapeutics Showcases Promising Anti-Cancer Drug RGT-61159 at ASH Annual Meeting

Introduction

Rgenta Therapeutics, a pioneering clinical-stage biotechnology company focused on developing innovative oral small molecules targeting RNA for oncology and neurological conditions, unveiled exciting preclinical findings regarding their drug candidate, RGT-61159, at the 66th Annual Meeting of the American Society of Hematology (ASH) held from December 7-10, 2024 in San Diego, California. This groundbreaking research indicates significant anti-tumor activity of RGT-61159 as a potential treatment for acute myeloid leukemia (AML).

Anti-Tumor Activity of RGT-61159

RGT-61159 is a selective oral small molecule inhibitor targeting MYB, a pivotal oncogene implicated in several malignancies. Presented data highlighted its capacity to eliminate MYB RNA and protein in cancer cells, showcasing its potent anti-tumor effects. Dr. Simon Xi, CEO and co-founder of Rgenta, asserted, "RGT-61159 has demonstrated elimination of MYB RNA and protein, leading to considerable anti-tumor activity across various AML models that often exhibit genetic alterations responsible for treatment resistance."

Implications for AML Treatment

Acute myeloid leukemia is an aggressive blood cancer characterized by the rapid increase of abnormal cells in the bone marrow and bloodstream. Despite potential for remission in around 70% of newly diagnosed patients, long-term survival rates remain low, underscoring the critical need for new therapeutic approaches. RGT-61159 has shown significant promise not only in AML but also in broader applications for MYB-dependent hematologic malignancies.

Evidence presented at ASH demonstrated that RGT-61159's mechanism works through inducing the inclusion of a cryptic exon into MYB RNA transcripts. This action enhances the degradation of MYB mRNA and ultimately leads to the reduction of the MYB protein. The data showcased an impressive dose-dependent elimination of MYB RNA and protein within AML cell lines, exhibiting the drug’s ability to promote differentiation in THP-1 AML cells, further supporting its development as a therapeutic option for AML patients.

RGT-61159's Future Development

In addition to AML, Rgenta plans to explore RGT-61159's efficacy in other hematologic tumors such as adenoid cystic carcinoma (ACC) and colorectal cancer (CRC). The ongoing Phase 1a/b clinical trials focus on assessing the safety, tolerability, and pharmacokinetics of this promising drug. Dr. Travis Wager, president and chief scientific officer, emphasized the significance of their genomic analyses in unraveling key oncogenes regulated by MYB and how these findings reinforce RGT-61159's potential as a best-in-class MYB inhibitor.

Conclusion

The emergence of RGT-61159 enhances the landscape of treatment possibilities for AML, potentially intervening in therapeutic resistance posed by current methods. As Rgenta Therapeutics moves forward with expanded clinical studies, the scientific community is keenly observing this breakthrough. With the potential to reshape the treatment paradigm for both AML and other MYB-related malignancies, RGT-61159 is indeed a beacon of hope for patients battling these challenging cancers.

For additional updates, follow Rgenta Therapeutics or check ClinicalTrials.gov for ongoing trials related to RGT-61159.