Rgenta Therapeutics to Present at 66th ASH Annual Meeting

Rgenta Therapeutics, a biotechnology company specializing in the development of innovative oral small molecules targeting RNA and its regulation for cancer and neurological conditions, has announced that preclinical data from its leading program, RGT-61159, will be presented at the 66th American Society of Hematology Annual Meeting and Exposition. This event will take place from December 7-10, 2024, in San Diego, California.

The presentation, titled "RGT-61159, Best-in-Class Small Molecule Inhibitor of MYB Via Selective RNA Splicing Alteration, Showed Robust Anti-Tumor Activity across AML Tumor Models Harboring Common Genetic Lesions," features contributions from researchers Norman Lu, Patricia Soulard, Ibrahim Kay, Kai Li, Xiubin Gu, Heather Sadlish, Jay Lee, Mariam Elhawary, Michael Rotelli, Sam Hasson, Thomas Wynn, Chris Yates, Zhiping Weng, Simon Xi, and Travis Wager. This session, categorized under Molecular Pharmacology and Drug Resistance for Myeloid Neoplasms, will be held on December 9, 2024, from 6:00 to 8:00 PM PT at the San Diego Convention Center, Halls G-H. The abstract is numbered 209135 with poster 4141.

RGT-61159 is an innovative, orally administered small molecule specifically engineered to alter the splicing of the MYB transcription factor, thereby inhibiting the production of the oncogenic MYB protein. This inhibition can potentially induce the death of cancer cells that overexpress the MYB protein. MYB is a crucial regulator for cell proliferation, self-renewal, and differentiation. Abnormal MYB expression is linked to several cancers, such as adenoid cystic carcinoma (ACC), acute myeloid leukemia (AML), T-cell acute lymphoblastic leukemia (T-ALL), colorectal cancer (CRC), small cell lung cancer (SCLC), and breast cancer.

Rgenta Therapeutics is currently evaluating RGT-61159 in an ongoing multi-center, open-label Phase 1a/b clinical trial involving patients with advanced relapsed or refractory ACC or CRC. This clinical trial aims to assess the safety, tolerability, pharmacokinetics, target engagement, and clinical efficacy of RGT-61159 in these patients. Further details about this Phase 1a/b study are available at ClinicalTrials.gov under the identifier NCT06462183.

Rgenta Therapeutics, headquartered in Woburn, Massachusetts, is a clinical-stage biotechnology firm focused on developing a range of oral RNA-targeting small molecule therapies, primarily targeting cancer and neurological disorders. Using a proprietary platform, Rgenta is able to explore extensive genomics data to pinpoint targetable RNA processing events and design small-molecule agents that modulate interactions among the spliceosome, regulatory proteins, and RNA molecules. Their innovative programs aim to address therapeutic targets previously considered undruggable within human diseases.

By leveraging their unique approach and technological platform, Rgenta Therapeutics is paving the way for the development of novel therapeutic solutions that target RNA regulation and splicing, offering potential new treatments for various malignancies and neurological conditions. Their work exemplifies the cutting-edge advancements in the field of RNA-targeting small molecules and highlights the potential to significantly improve patient outcomes in oncology and other disease areas.