Rgenta Therapeutics Announces FDA Clearance of IND Application for RGT-61159, an Oral Small Molecule RNA Modulator Designed to Halt Disease-Driver MYB Production in Adenoid Cystic Carcinoma (ACC) and Colorectal Cancer (CRC)

First-in-human Phase 1a/1b clinical trial will evaluate orally available, RGT-61159 designed to selectively target MYB RNA and inhibit oncogenic MYB protein production

Preclinical data supporting the clinical trial were recently presented at the American Society of Clinical Oncology Annual Meeting (ASCO 2024)

Excerpt from the Press Release:

WOBURN, Mass., July 10, 2024 /PRNewswire/ — Rgenta Therapeutics, a biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, announced the clearance of its Investigational New Drug application (IND) by the U.S. Food and Drug Administration (FDA) for 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).

“Clearance of our first IND application is a significant milestone in Rgenta’s mission to develop oral, small molecule RNA-targeting medicines to treat previously incurable diseases,” said Simon Xi, Ph.D., co-founder and chief executive officer of Rgenta. “We look forward to initiating clinical studies of RGT-61159 with a first-in-human Phase1a/1b clinical study in adults with ACC and CRC to potentially provide a new therapeutic option for patients with these difficult-to-treat cancers.”

“Development of small molecule drugs targeting oncogenic drivers such as MYB, has proven challenging in the past,” said Travis Wager, Ph.D., co-founder, president and chief scientific officer. “RGT-61159 demonstrates potent inhibition of oncogenic MYB protein production and significant inhibition of tumor growth at tolerated doses in preclinical models of ACC and other cancers, and we are excited to move this novel therapeutic into clinical evaluation.”

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 oncogenic MYB protein production, which has the potential to inhibit proliferation or induce cell death of cancer cells that overexpress MYB protein.

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