A novel oral formulation of itraconazole (SUBA-Itraconazole, HedgePath Pharmaceuticals) is providing dramatic benefit for patients with basal cell carcinoma nevus syndrome (BCCNS, also known as Gorlin syndrome) and with a favorable safety profile, according to a recent interim analysis of data collected in a phase IIb study.
Launched in September 2015, the multicenter, open-label, single-arm study has enrolled 37 patients. As summarized in a recent submission to the FDA, the interim analysis was based on 35 patients who had been taking SUBA-Itraconazole daily for a median duration of 32 weeks.
Each patient had at least 10 to 15 pre-existing tumors of a size and location that made them surgically eligible. Together, the enrolled patients had a total of about 475 “target” basal cell carcinomas (BCCs). Prior to the study, the average number of surgically removed BCCs per patient was 195. Overall, they had a total of about 6,800 prior surgical excisions.
Efficacy assessments showed that all patients achieved a measurable decrease in target tumor burden. Overall, 28% of target lesions completely disappeared and another 28% shrunk by ≥30%. Only 4 lesions exhibited >20% growth with the rest remaining stable. Only 1 of about 475 target BCCs required surgical resection.
Data from follow-up visits scheduled at weeks 4, 8, and every 8 weeks thereafter showed treatment can have a rapid onset of benefit. For example, in the first enrolled patient, 7 of 8 facial BCCs had disappeared at the week 16 visit.
Serious adverse events occurred in 3 patients, but all were determined not to be drug related. Side effect data show mostly Grade 1 toxicities that are typical for itraconazole. Higher-grade (Grade 3) toxicities occurred in just a few patients.
“BCCNS is an autosomal dominant condition that affects an estimated 10,000 people in the United States,” said Frank E. O’Donnell, Jr., MD, formerly chairman, Department of Ophthalmology, St. Louis University School of Medicine, St. Louis, MO, and now manager of Hopkins Capital Group LLC, an investment company whose mission is identification and development of disruptive innovations in healthcare.
“These patients can develop hundreds to thousands of BCCs on the head, neck, and body over a lifetime--treatment for which has historically been surgical,” he said. “SUBA-Itraconazole is an exciting advance because it is showing promise for becoming the first oral treatment that can both address existing tumors and be used as chronic therapy to prevent the development of new BCCs for patients with this genetically driven disease.”
“The net clinical impact of SUBA-Itraconazole is that it has stabilized or greatly reduced the tumor burden for patients with BCCNS, while causing minimal side effects and allowing them to avoid disfiguring surgery,” said Nick Virca, president and chief executive officer, HedgePath Pharmaceuticals, Tampa, FL.