Rgenta Therapeutics Receives FDA Approval for IND Application of RGT-61159, an Oral Small Molecule RNA Modulator Targeting MYB Production in Adenoid Cystic Carcinoma and Colorectal Cancer

11 July 2024

Rgenta Therapeutics, a biotechnology company specializing in developing oral small molecules that target RNA and RNA regulation for oncology and neurological disorders, has achieved a significant milestone with the FDA's clearance of its Investigational New Drug (IND) application for RGT-61159. This compound is being developed to potentially treat adenoid cystic carcinoma (ACC), colorectal cancer (CRC), acute myeloid leukemia (AML), and other solid tumors.

Rgenta, emphasized the IND clearance as a pivotal step in their mission to develop effective treatments for previously untreatable diseases. The company plans to begin clinical trials, starting with a Phase 1a/1b study in adults with ACC and CRC, aiming to offer new therapeutic options for patients facing these challenging cancers.

Dr. Travis Wager, co-founder, president, and Chief Scientific Officer, highlighted the innovative nature of RGT-61159 in targeting the oncogenic MYB protein, which plays a crucial role in promoting cancer cell growth. Promising results from preclinical studies have shown significant tumor growth inhibition in models of ACC and other cancers at tolerated doses.

RGT-61159 is designed to modulate splicing of the MYB transcription factor, thereby inhibiting oncogenic MYB protein production. This mechanism holds potential for halting cancer cell proliferation and inducing cell death in cancers where MYB is overexpressed, including ACC, AML, CRC, as well as T-cell acute lymphoblastic leukemia (T-ALL), small cell lung cancer (SCLC), and breast cancer.

Adenoid cystic carcinoma (ACC), known for its aggressive nature and tendency to metastasize, often proves resistant to conventional treatments such as surgery and radiation. Similarly, colorectal cancer (CRC) presents significant challenges due to its high prevalence and propensity for metastasis even after initial treatment.

The development of RGT-61159 represents a critical advancement in addressing these unmet medical needs, offering hope for improved outcomes in patients with these difficult-to-treat cancers.

 

Source: prnewswire.com