WOBURN, Mass., March 25, 2025 /PRNewswire/ — 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 American Association for Cancer Research (AACR) 2025 Annual Meeting, which will be held from April 25-30, 2025, in Chicago, IL.
Title: RGT-61159, Best-in-class Oral Small Molecule Inhibitor of MYB via Selective RNA Splicing Alteration, Synergistic Anti-Tumor Activity When Combined with Standards of Care in Leukemia Disease Models Harboring AML Common Genetic Lesions and with NOTCH Inhibitors in ACC Disease Models
Authors: Norman Lu, Patricia Soulard, Kai Li, Xiubin Gu, Ibrahim Kay, Sam Hasson, Chris Yates, Zhiping Weng, Simon Xi, Travis Wager
Session category: Chemistry
Session title: Targeted Protein Degradation
Session date and time: April 30, 2025, 9:00 -12:00 PM CT
Abstract #: 7013 (Poster section 26 / Poster board #13)
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 and target engagement and clinical efficacy of RGT-61159 in patients with ACC or CRC. Additional information about the Phase 1a/b clinical trial can be accessed at ClinicalTrials.gov (NCT06462183).
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. Learn more at: http://www.rgentatx.com.
Contacts
Investors:
Sylvia Wheeler
Wheelhouse Life Science Advisors
[email protected]
Elizabeth Wolffe, Ph.D.
Wheelhouse Life Science Advisors
[email protected]
Media
Aljanae Reynolds
Wheelhouse Life Science Advisors
[email protected]
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SOURCE Rgenta Therapeutics
