The U.S. FDA has granted RGX-104 two Orphan Drug Designations for the treatment of ovarian cancer and glioblastoma multiforme. RGX-104 is an orally bioavailable small molecule LXR-agonist under development by Rgenix, Inc., a biotechnology company based in New York, NY. Preclinical studies has demonstrated robust efficacy of RGX-104 in inhibiting tumor proliferation in established animal models of various cancer types including melanoma, ovarian cancer and glioblastoma. This efficacy includes, in some cases, complete tumor regression and the stopping of the growth of metastatic disease. Ovarian cancer accounts for only about 3% of cancers among women; however it causes more deaths than any other gynecology cancers representing an urgent and unmet medical need. Glioblastoma multiforme is a rare form of brain cancer and yet the most aggressive form. According to the company’s website, Rgenix is currently developing RGX-104 to be a first-in-class agent for melanoma and several other cancer types.