Orally available RGT-61159 demonstrates potent inhibition of MYB synthesis and significant anti-tumor activity in a range of xenograft models of adenoid cystic carcinoma (ACC)

Data support planned first-in-human Phase 1 clinical trial of RGT-61159 in both ACC and colorectal cancer (CRC)

CAMBRIDGE, Mass., May 31, 2024 /PRNewswire/ — Rgenta Therapeutics, a biotechnology company pioneering the development of a new class of oral small molecules for therapeutic RNA modification for oncology and CNS disorders, today announced the presentation of preclinical data at ASCO 2024 from its lead program, RGT-61159, which is being developed for the potential treatment of adenoid cystic carcinoma (ACC), colorectal cancer (CRC) and  other solid tumors as well as acute myeloid leukemia (AML).

“The data presented at ASCO 2024 demonstrate efficient and specific modulation of the oncogenic transcription factor MYB, a target that historically has been difficult to drug, by RGT-61159 in a range of preclinical models of ACC and support our planned clinical development program of this novel small molecule designed to modulate RNA splicing,” said Simon Xi Ph.D., co-founder and chief executive officer of Rgenta. “Down-regulation of MYB is a very promising therapeutic strategy to treat ACC and other cancers driven by MYB dysregulation including CRC, breast, small cell lung cancer and AML. We look forward to advancing this novel therapeutic in a first-in-human Phase 1a/1b clinical study in adults with ACC and CRC this year.”

“The data demonstrate that daily, single agent treatment with RGT-61159 results in significant anti-tumor activity at tolerated doses in all four ACC patient derived xenograft (PDX) models evaluated,” said Travis Wager, Ph.D., co-founder, president and chief scientific officer.  “Further, RGT-61159’s anti-tumor activity correlated with MYB target modulation in a dose-dependent fashion, strongly supporting an on-target anti-tumor effect. Treatment with RGT-61159 was well tolerated at efficacious doses.”

Details of Rgenta’s ASCO2024 Presentation
Abstract Number: 6107
Title: Effect of RGT-61159 on inhibition of oncogene c-MYB synthesis and tumor growth inhibition in a broad range of ACC PDX models, at well tolerated doses in rodents and non-human primates.
Poster Board #: 423
Session Date and Time: 6/2/2024, 9:00 AM-12:00 PM (CDT)
Location: Hall A

About RGT-61159
RGT-61159 is an orally available small molecule designed to specifically modulate splicing of the transcription factor MYB and was identified and optimized using Rgenta’s novel integrative RNA-targeting platform. Treatment with RGT-61159 leads to the selective degradation of MYB RNA message and inhibition of MYB protein production. MYB acts as a master regulator of cell proliferation differentiation processes and its aberrant expression has been demonstrated in multiple forms of human cancer including adenoid cystic carcinoma (ACC), acute myeloid leukemias (AML), T cell acute lymphoblastic leukemias (T-ALL), colorectal cancer (CRC), and breast cancer.

About Rgenta Therapeutics
Rgenta Therapeutics is developing a pipeline of oral, small-molecule RNA-targeting 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]

SOURCE Rgenta Therapeutics