Adrenocortical carcinoma (ACC) is a rare tumor in adults and children with an estimated 700 new cases per year in the U.S. Most adult ACC patients are diagnosed with advanced disease that is not amenable to surgery and median survival is less than one year. Tragically, advanced ACC in children is practically untreatable. The sole FDA-approved drug for late-stage ACC, a derivative of the pesticide DDT, is poorly tolerated and has questionable efficacy. We propose to start clinical development of OR-449, a potent blocker of a tissue-selective gene regulator that is strongly linked to tumor formation and is considered a highly promising target for treatment of ACC. This same target is also high...

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Adrenocortical carcinoma (ACC) is a rare tumor in adults and children with an estimated 700 new cases per year in the U.S. Most adult ACC patients are diagnosed with advanced disease that is not amenable to surgery and median survival is less than one year. Tragically, advanced ACC in children is practically untreatable. The sole FDA-approved drug for late-stage ACC, a derivative of the pesticide DDT, is poorly tolerated and has questionable efficacy. We propose to start clinical development of OR-449, a potent blocker of a tissue-selective gene regulator that is strongly linked to tumor formation and is considered a highly promising target for treatment of ACC. This same target is also highly expressed in 10% of head and neck squamous carcinomas (HNSC) and 3% of lung squamous carcinomas (LUSC), showing it may have broader therapeutic potential. With CPRIT support, we will complete a first-in-human Phase 1 clinical trial in adults centered at MD Anderson, the leading U.S. clinical center for ACC patients. MDACC is also a center for HNSC and LUSC and could lead subsequent expansion trials. Ultimate sales of OR-449, a first-in-class oral small molecule targeted therapy, will take place through narrow specialist channels, enhancing the opportunity for Orphagen to become a successful commercial organization in Texas and achieving our objective of significantly improving survival not only for adult and pediatric ACC patients, but also for certain HNSC and LUSC patients.

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