Rgenta Therapeutics, a US-based biotechnology company involved in the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, announced on Wednesday that the Investigational New Drug application (IND) for RGT-61159 has received approval from the US Food and Drug Administration (FDA).

RGT-61159 is being developed for the potential treatment of adenoid cystic carcinoma (ACC), colorectal cancer (CRC), other solid tumours and acute myeloid leukaemia (AML). 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.

Simon Xi, Ph.D., Rgenta co-founder and chief executive officer, said, '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. 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.'