"Our investigational ATO, ORH-2014, is a patented, oral capsule that achieved the required target exposure at a dose of 15 mg once daily in a recently completed Phase 1 Study. ORH-2014 has been granted Orphan Drug Designation by both FDA and EMEA, and Orsenix gained alignment with the FDA to proceed with a single registrational study for accelerated approval of ORH-2014 for the treatment of newly diagnosed patients with APL, assuming favorable regulatory review."

"The primary endpoint for the study will be complete molecular and clinical response (MolCR) in comparison to historical control. ORH-2014 also offers physicians the opportunity to further explore the full potential of ATO to treat other myeloid cancers such as Myelodysplastic Syndromes and Myelofibrosis, as well as Glioblastoma."