Rgenta Therapeutics Receives Orphan Drug Designation from the U.S. FDA for RGT-61159 for the Treatment of Adenoid Cystic Carcinoma (ACC)
RGT-61159 is being evaluated in an ongoing multi-center, Phase 1a/b clinical trial in patients with advanced relapsed or refractory ACC
WOBURN, Mass., Sept. 17, 2025 (GLOBE NEWSWIRE) -- Rgenta Therapeutics, a clinical-stage 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 U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RGT-61159 for the treatment of adenoid cystic carcinoma (ACC). RGT-61159 is a potent and selective orally available small molecule inhibitor of MYB through RNA splicing modulation that is currently being evaluated in a Phase 1a/b clinical trial in patients with advanced relapsed or refractory ACC or colorectal cancer.
The FDA's Orphan Drug Designation (ODD) program provides orphan status to therapies intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. ODD status is given to medicinal products that represent a significant benefit over existing treatments and are intended for the treatment of a disease that is life-threatening or chronically debilitating. This designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved.
“Granting of ODD by the FDA highlights RGT-61159’s potential to provide an innovative therapeutic option for patients with ACC which is primarily driven by overexpression of the MYB oncogene and carries a high risk of recurrence and metastasis,” said Travis Wager, Ph.D., co-founder and chief scientific officer. “As a potent and selective inhibitor of MYB, RGT-61159 is designed to unlock the therapeutic potential of this historically undruggable target and address the root cause of ACC and several other aggressive cancers.”
“Receiving this designation for RGT-61159 is an important milestone for Rgenta, underscoring the significant unmet medical need for treatments for ACC and validating our ongoing commitment to pursuing potential first- and best-in-class medicines for life altering diseases,” said Simon Xi, Ph.D., co-founder and chief executive officer of Rgenta. “Our ongoing Phase1a/b clinical trial of RGT-61159 is designed to evaluate its safety and tolerability as well as pharmacokinetics, target engagement, and clinical efficacy in patients with relapsed or refractory ACC or colorectal cancer. We also plan to broaden this program and initiate a new Phase 1/2 study of RGT-61159 in adults with AML/high risk myelodysplastic syndromes.”
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 the oncogenic MYB protein and potential cell death of the cancer cells that overexpress the MYB protein. MYB acts as a master regulator of cell proliferation, self-renewal, and differentiation processes and its aberrant expression has been demonstrated in multiple forms of human cancer including adenoid cystic carcinoma (ACC), acute myeloid leukemia (AML), T-cell acute lymphoblastic leukemia (T-ALL), colorectal cancer (CRC), small cell lung cancer (SCLC) and breast cancer. Rgenta is evaluating RGT-61159 in an ongoing multi-center, open-label Phase 1a/b clinical trial in patients with advanced relapsed or refractory ACC or CRC. The Phase 1a/b study is designed to evaluate safety, tolerability, pharmacokinetics, 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 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 Rgenta Therapeutics
Rgenta Therapeutics is a clinical stage biotechnology company developing a pipeline of oral RNA-targeting small molecule 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
Swheeler@wheelhouselsa.com
Elizabeth Wolffe, Ph.D.
Wheelhouse Life Science Advisors
Lwolffe@wheelhouselsa.com
Media
Aljanae Reynolds
Wheelhouse Life Science Advisors
Areynolds@wheelhouselsa.com
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