Rgenta Therapeutics Gets FDA Nod for RGT-61159 IND in ACC and CRC

Rgenta Therapeutics, a biotechnology firm dedicated to developing innovative oral small molecules that target RNA and RNA regulation for treating cancer and neurological disorders, has announced a significant milestone. The U.S. Food and Drug Administration (FDA) has approved the company's Investigational New Drug application (IND) for RGT-61159. This drug 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).

Simon Xi, Ph.D., the co-founder and CEO of Rgenta, expressed his enthusiasm for this advancement. "Clearing our first IND application marks a pivotal moment in Rgenta's mission to develop RNA-targeting oral medicines to treat diseases that were previously considered incurable," he said. The company aims to begin clinical studies with a first-in-human Phase 1a/1b trial in adults with ACC and CRC, hoping to offer a new therapeutic option for these challenging cancers.

Travis Wager, Ph.D., co-founder, president, and chief scientific officer, highlighted the difficulties of developing small molecule drugs to target oncogenic drivers like MYB. He noted that RGT-61159 effectively inhibits the production of oncogenic MYB protein and demonstrates significant tumor growth reduction in preclinical models. This progress has paved the way for the novel therapeutic to enter clinical evaluation.

RGT-61159 is a small molecule drug designed to modulate the splicing of the MYB transcription factor, thereby inhibiting the production of the oncogenic MYB protein. MYB acts as a master regulator of cell proliferation and differentiation, and its abnormal expression is implicated in various cancers, including ACC, AML, T-cell acute lymphoblastic leukemias (T-ALL), CRC, small cell lung cancer (SCLC), and breast cancer. The planned Phase 1a/1b clinical trial for RGT-61159 is registered under clinicaltrials.gov (NCT06462183).

ACC is a rare but aggressive cancer that predominantly arises in the salivary gland. Approximately 200,000 individuals worldwide, including 11,000 in the U.S., are living with ACC. The cancer is aggressive, often infiltrating surrounding nerves and metastasizing to distant sites. Overactivation of the MYB oncogene occurs in more than 90% of ACC cases, making the disease particularly challenging to treat. Standard treatments, such as surgery and radiation, often fail to control tumor recurrence and metastasis. There are currently no effective targeted therapies for recurrent or metastatic ACC, emphasizing the urgent need for new therapeutic strategies.

Colorectal cancer (CRC) ranks as the third most prevalent cancer and the second leading cause of cancer-related deaths worldwide. According to the World Health Organization, over 1.9 million cases of CRC were diagnosed in 2022. Despite advancements in early detection and targeted therapies, a significant proportion of patients present with metastases or develop metastases after initial treatment. MYB is overexpressed in 80-85% of CRC cases and serves as a predictive biomarker for tumor aggressiveness and poor prognosis. Developing new therapies for metastatic CRC remains a critical unmet medical need.

Rgenta Therapeutics is building a pipeline of oral, small-molecule RNA-targeting medicines, initially focusing on oncology and neurological disorders. Their proprietary platform identifies targetable RNA processing events and designs small-molecule agents to modulate interactions among the spliceosome, regulatory proteins, and RNAs. By targeting these historically undruggable targets, Rgenta aims to unlock new therapeutic possibilities for treating human diseases.