RGX-104, an investigational drug being developed by Rgenix, Inc., a biotechnology company based in New York, NY received another Orphan Drug Designation (ODD) from the U.S. FDA for the treatment of malignant melanoma stages IIB to IV. This designation is the third ODDs that RGX-104 gathered in two weeks. The two ODDs preceding this one are for the treatment of ovarian cancer and for the treatment of glioblastoma multiforme. RGX-104 is an orally bioavailable small molecule LXR-agonist. 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. Melanoma accounts only for less than 2% of skin cancer but causes a large majority of the skin cancer deaths. 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.
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