Onco-Summaries: Daily Oncology Updates at a Glance

17/09/2025

Rgenta Therapeutics' RGT-61159 received the FDA Orphan Drug Designation for ACC (Ref)

The US FDA granted the Orphan Drug Designation to Rgenta Therapeutics' RGT-61159 (small molecule inhibitor of MYB) for the treatment of adenoid cystic carcinoma (ACC).

• The asset is currently being evaluated in a Phase 1a/b clinical trial in patients with advanced relapsed or refractory ACC or colorectal cancer

  
  

• Travis Wager, Co-founder and CSO, Rgenta: “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. 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.”

  
  

• Simon Xi, Co-founder and CEO, Rgenta: “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. 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.”