New Hope for ACC and CRC Patients: FDA Clears Clinical Trial for Innovative Oral Drug

FDA Gives Green Light to Clinical Trial

In an exciting development, Rgenta Therapeutics has received clearance from the U.S. Food and Drug Administration (FDA) to start a clinical trial for their new drug, RGT-61159. This trial is a significant milestone, marking the first time this innovative oral medication will be tested in humans.

RGT-61159 aims to treat two challenging cancers: adenoid cystic carcinoma (ACC) and colorectal cancer (CRC). The drug is designed to specifically inhibit a protein called MYB, which drives the growth of these cancers.

Details of the Clinical Trial

The upcoming clinical trial, known as Phase 1a/1b, will assess both the safety and effectiveness of RGT-61159. The trial will include adults diagnosed with ACC and CRC to see if this new treatment can offer them a much-needed therapeutic option.

Dr. Simon Xi, co-founder and CEO of Rgenta, expressed enthusiasm about the project's progress, emphasizing the drug's potential to treat previously incurable diseases. The trial will take place in multiple stages to evaluate dosage and response.

Preclinical Success Paves the Way

Before reaching the clinical trial stage, RGT-61159 showed promise in preclinical studies. These studies demonstrated that the drug effectively inhibits the MYB protein, which is often overexpressed in various cancers, including ACC and CRC.

Dr. Travis Wager, co-founder and chief scientific officer, highlighted the difficulties in developing drugs targeting the MYB protein in the past. However, RGT-61159 has shown potent tumor growth inhibition in several preclinical cancer models.

Understanding RGT-61159

RGT-61159 is an oral small molecule designed to alter the splicing of RNA in cells, which results in reduced production of the harmful MYB protein. This modulation can inhibit cancer cell growth or even lead to cancer cell death in tumors where MYB is overactive.

The MYB protein is a key factor in the proliferation and survival of cancer cells, and its aberrant expression has been linked to various deadly cancers, making it an important target for new treatments.

Impact on Adenoid Cystic Carcinoma (ACC)

Although ACC is rare, affecting about 200,000 people worldwide, it presents a significant challenge. The cancer is notorious for spreading into surrounding tissues and metastasizing to distant sites. Current treatment often involves surgery and radiation, which may not be sufficient to control the disease long-term.

With over 90% of ACC cases showing heightened MYB activity, a targeted therapy like RGT-61159 offers a potential breakthrough for patients who have few other options.

Addressing Colorectal Cancer (CRC)

CRC is more common, being the third most prevalent cancer globally. In 2022, there were over 1.9 million new cases. Despite improvements in treatment, the survival rate for patients with advanced CRC remains low, especially for those who have exhausted traditional therapies.

RGT-61159 targets MYB, which is overexpressed in 80-85% of CRC cases, suggesting this drug could be particularly effective in combating aggressive and difficult-to-treat tumors.

About Rgenta Therapeutics

Rgenta Therapeutics is a company focused on developing oral small molecules that target RNA and RNA regulation. Their innovative platform examines genomics data to find new ways to modulate RNA processes, aiming to transform treatments for various diseases, particularly cancers and neurological disorders.

By advancing RGT-61159 into clinical trials, Rgenta is making significant strides in the fight against cancers that have so far eluded effective treatments.