Rgenta Therapeutics Presents Preclinical Data from Lead Program, RGT-61159, a Potent and Selective Small Molecule Inhibitor of MYB RNA, Supporting its Application in the Treatment of Acute Myeloid Leukemia (AML)

Data are being presented at the 30th Annual Congress of the European Hematology Association 2025 (EHA2025)

/EIN News/ -- WOBURN, Mass., June 12, 2025 (GLOBE NEWSWIRE) -- 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 the presentation of preclinical data from its lead program, RGT-61159 highlighting its potential for the treatment of a broad AML patient population. The data are being presented at the European Hematology Association (EHA) 30^th Annual Congress, which is being held from June 12-15, 2025, in Milan, Italy.

“These data clearly show a strong reduction of MYB RNA and MYB protein with RGT-61159, with effective killing activity across various AML cell lines and disease models, highlighting its promising therapeutic potential,” said Travis Wager, Ph.D. co-founder and chief scientific officer. “MYB is known to be crucial for AML, as it is required for the survival and proliferation of leukemic cells and is a key part of complexes that sustain abnormal gene expression, all of which contribute to disease progression. However, traditionally targeting MYB has been challenging. In RGT-61159, Rgenta has developed a powerful oral small molecule that targets MYB RNA, which has the potential to effectively address this important cancer target and offer a new treatment option for a wide range of AML patients.”

“The presentation at EHA highlights data that support our advancement of RGT-61159 as a new therapeutic option for the potential treatment of AML,” said Simon Xi, Ph.D., co-founder and chief executive officer of Rgenta. “Our ongoing Phase 1a/b clinical trial of RGT- 61159 in patients with relapsed or refractory ACC or CRC is advancing well and we look forward to broadening that program and initiating a new Phase 1/2 study of RGT-61159 in adults with AML/high risk myelodysplastic syndromes in the second half of 2025.”

A presentation titled 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 details data demonstrating the on-target therapeutic activity of RGT-61159. RGT-61159 demonstrated potent dose-dependent cell killing of AML derived cell lines that overexpressed MYB, while sparing normal cells. Importantly, the data show a correlation between the elimination of MYB RNA and protein by RGT-61159 and its potent killing activity. RGT-61159 also induced a robust increase in expression of CD11b and CD14 cell differentiation markers and downregulation of the expression of master oncogenes controlled by MYB, including MYC, BCL2, FLT3 and IDH1 supporting the hypothesis that RGT-61159 anti-tumor activity is driven by MYB signaling inhibition and illustrating MYB dependency across a broad AML patient population. In several CDX models of disease with genetic alterations seen commonly in patients with AML, the data demonstrated the robust anti-tumor activity of tolerated doses of RGT-61159 as a single agent and its synergistic activity both in vitro and in vivo when administered in combination with other agents currently used in AML standard of care.

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. 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
Swheeler@wheelhouselsa.com

Elizabeth Wolffe, Ph.D.
Wheelhouse Life Science Advisors
Lwolffe@wheelhouselsa.com

Media
Aljanae Reynolds
Wheelhouse Life Science Advisors
Areynolds@wheelhouselsa.com