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Preclinical + European Hematology Association 2025 Congress

Preclinical  + European Hematology Association 2025 Congress

Rgenta Therapeutics Announces Presentation of Preclinical Data from Lead Program, RGT-61159, a Potent and Selective Small Molecule Inhibitor of MYB RNA, at the European Hematology Association (EHA) 2025 Congress

Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, announced today that preclinical data will be presented on its lead program, RGT-61159, at the European Hematology Association (EHA) 2025 Congress, which will be held from June 12-15, 2025, in Milan, Italy.

Presentation Details:

Title: RGT-61159, a Potent and Selective Small Molecule MYB RNA Inhibitor, Showed Significant Anti-Tumor Activity as Monotherapy or in Combination with Standards of Care in Several Leukemia Disease Models Harboring AML Most Common Genetic Lesions.

Presenting Author: Patricia Soulard

Session title: Poster Session 2

Session date and time: Saturday, June 14 (18:30 - 19:30 CEST)

Abstract #: PS1424

About RGT-61159

RGT-61159 is an orally available small molecule designed to specifically modulate splicing of the transcription factor MYB resulting in the inhibition of the oncogenic MYB protein and potential cell death of the cancer cells that overexpress the MYB protein. MYB acts as a master regulator of cell proliferation, self-renewal, and differentiation processes and its aberrant expression has been demonstrated in multiple forms of human cancer including 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 is evaluating RGT-61159 in an ongoing multi-center, open-label Phase 1a/b clinical trial in patients with advanced relapsed or refractory ACC or CRC. The Phase 1a/b study is designed to evaluate safety, tolerability, pharmacokinetics, target engagement, and clinical efficacy of RGT-61159 in patients with ACC or CRC.

About Rgenta Therapeutics

Rgenta Therapeutics is a clinical stage biotechnology company developing a pipeline of oral RNA-targeting small molecule medicines with an initial focus on oncology and neurological disorders. Our proprietary platform mines the massive genomics data to identify targetable RNA processing events and design small-molecule glues to modulate the interactions among the spliceosome, regulatory proteins, and RNAs. Our lead programs and unique approach are unlocking the therapeutic potential of historically undruggable targets in human diseases.

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