Rgenta Therapeutics Announces First Patients Dosed in Phase 1a/b Clinical Trial of RGT-61159, an Oral Small Molecule Targeting MYB, in Adenoid Cystic Carcinoma (ACC) and Colorectal Cancer (CRC)

First-in-human study in patients with solid tumors initially evaluating the safety, tolerability pharmacokinetics and target engagement of RGT-61159

WOBURN, Mass., Oct. 8, 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 today that the first patients have been dosed in the Phase 1a/b clinical trial of RGT-61159. This novel drug candidate 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).

"Currently, there are no effective systemic treatment options for ACC. Patients with this rare and aggressive cancer, which carries a high risk of recurrence and metastasis, face an urgent need for innovative treatment approaches," said Lillian Siu, M.D., director of the Phase I Program and co-director of the Bras and Family Drug Development Program at the Princess Margaret Cancer Centre, Toronto, Canada. Enrique Sanz-Garcia, M.D., staff medical oncologist and the principal investigator on the trial at the Princess Margaret Cancer Centre added, "The MYB inhibitor, RGT-61159, represents one such innovative approach designed to address the root cause of ACC and we are excited to evaluate this novel drug candidate in this first-in-human Phase 1 clinical trial."

"Patients with colorectal cancer whose disease has failed initial therapy also have poor outcomes," observed Alexander Spira, M.D., Ph.D., chief executive officer and clinical director of NEXT Oncology-Virginia. "RGT-61159 has a novel mechanism of action and targets a known oncogenic driver. We are pleased to collaborate with Rgenta in exploring the potential of this new agent."

"We are excited to move our first asset into the clinic which is a significant milestone for Rgenta, and to work with a group of world-class clinicians on this trial," said Simon Xi, Ph.D., co-founder and chief executive officer of Rgenta. "At Rgenta we have established a platform to discover and develop small molecule RNA targeting medicines with the goal of unlocking the therapeutic potential of historically undruggable targets in human diseases and we look forward to establishing a robust pipeline of clinical assets."

Rgenta's Phase 1a/b clinical trial of RGT-61159 is a multi-center, open-label study dose escalation and expansion study in patients with advanced relapsed or refractory ACC or CRC. The Phase 1a/b study will evaluate safety, tolerability, pharmacokinetics and target engagement and clinical efficacy of RGT-61159 in patients with ACC or CRC. Additional information about the Phase 1a/b clinical trial can be accessed at ClinicalTrials.gov (NCT06462183).

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. MYB acts as a master regulator of cell proliferation differentiation processes and its aberrant expression has been demonstrated in multiple forms of human cancer including adenoid cystic carcinoma (ACC), acute myeloid leukemias (AML), T-cell acute lymphoblastic leukemias (T-ALL), colorectal cancer (CRC), small cell lung cancer (SCLC) and breast cancer.

About Adenoid Cystic Carcinoma (ACC)
It is estimated that approximately 200,000 people are living with ACC throughout the world including 11,000 in the US. While it is a rare cancer, ACC is the second most common cancer type arising in the salivary gland and is an aggressive malignancy with a tendency to infiltrate surrounding nerves and metastasize to distant sites. Overactivation of the MYB oncogene has been described as a hallmark of ACC and is noted in over 90% of ACC. Treatment for ACC is extremely challenging and may include surgery and/or radiation, which often fails to control local tumor recurrence and distant metastases. There are no effective targeted therapies available for patients with recurrent and/or metastatic disease. There is thus an unmet medical need for new therapeutic targets and treatment strategies for patients with this fatal cancer.

About Colorectal Cancer (CRC)
CRC is the third most prevalent cancer and the second leading cause of cancer-related mortality worldwide. According to the World Health Organization, in 2022, more than 1.9 million cases of CRC were diagnosed. Despite the improved early detection of CRC and the recent success of targeted therapeutics, approximately 15%-30% of patients present with metastases and 20%-50% of patients with initially localized disease will develop metastases. Patients with relapsed or refractory CRC who have exhausted all the available standard of care therapy options, have a very poor prognosis. MYB is significantly overexpressed in 80-85% of CRC and has been frequently found to be a predictive biomarker of tumor aggressiveness and poor prognosis. Developing novel therapies to treat patients with metastatic CRC remains a major unmet medical need.

About Rgenta Therapeutics
Rgenta Therapeutics is developing a pipeline of oral, small-molecule RNA-targeting medicines with an initial focus on oncology and neurological disorders. Our proprietary platform mines the massive genomics data to identify targetable RNA processing events and design small-molecule glues to modulate the interactions among the spliceosome, regulatory proteins, and RNAs. Our lead programs and unique approach are unlocking the therapeutic potential of historically undruggable targets in human diseases. Learn more at http://www.rgentatx.com.

Contacts
Investors: 
Sylvia Wheeler
Wheelhouse Life Science Advisors
[email protected]

Elizabeth Wolffe, Ph.D.
Wheelhouse Life Science Advisors
[email protected]

Media
Aljanae Reynolds
Wheelhouse Life Science Advisors
[email protected]

SOURCE Rgenta Therapeutics