Basal Cell Carcinoma
Newer formulations of itraconazole may offer a significant alternative therapy to vismodegib, which is an oral drug marketed by Roche/Genentech, for treatment of locally advanced and metastatic basal cell carcinoma (BCC) or sonidegib, which is another oral drug marketed by Sun Pharma for locally advanced BCC.
Vismodegib was the first FDA-approved Hedgehog inhibitor-based therapy. However, it has many reported toxicities and is associated with side effects that can result in suspension of chronic dosing. As a result, basal cell tumors reoccur and patients are faced with the choice of returning to vismodegib therapy or, if and when possible, surgical alternatives. Sonidegib, the second FDA-approved Hedgehog inhibitor based-therapy, is also associated with significant toxicities, based on clinical trial reports, and is associated with side effects similar to those of vismodegib that may also result in suspension of dosing after only short periods of treatment or reluctance to use the drug at all.
INHIBITOR first targeted testing of SUBA-Itraconazole for treatment of BCC tumors in patients with Gorlin Syndrome, specifically BCCNS (a genetic disease which causes chronic BCC tumors) to address an unmet need in this patient population since neither vismodegib or sonidegib appear to be tolerable for other than short term therapy due to side-effects. We focused on the BCCNS population of approximately 10,000 patients in the United States, which has qualified SUBA-Itraconazole oral capsules for orphan drug designation as of May 2016. After the successful completion of the Phase 2b study in BCCNS, further clinical development of SUBA-Itraconazole was licensed to our corporate partner, Mayne Pharma with plans to expand developmental efforts and initiate global testing.