HedgePath Pharmaceuticals, a clinical stage biopharmaceutical company that discovers, develops and plans to commercialize innovative therapeutics for patients with cancer, has filed an IND application with the FDA.

The IND covers HPPI's proposed clinical development of a patented formulation of the currently marketed anti-fungal drug itraconazole (known as SUBA-Itraconazole) for the treatment of basal cell carcinoma (BCC) in patients with basal cell carcinoma nevus syndrome (BCCNS).

The filing follows a pre-IND meeting held with FDA at which HPPI reviewed potential phase IIb trial considerations in order to gain FDA input on its clinical development strategy and protocol which HPPI now believes is reflected in its IND filing. If the IND is approved, HPPI would expect to commence patient recruiting to begin its clinical trial in the first quarter of 2015.

Nicholas J. Virca, HPPI's president and CEO, said, "This is a milestone moment for HPPI as we submit our first IND under which we will seek to repurpose our licensed, patented formulation of the FDA approved anti-fungal drug itraconazole as a candidate for anti-cancer therapy in humans. Based on existing third party data, we believe that itraconazole has the potential to inhibit the Hedgehog (Hh) Pathway, a major regulator of many fundamental cellular processes, which could in turn impact the development and growth of certain cancers. We believe this could serve as a treatment for skin, lung and prostate cancers, and we will begin to develop this approach with human testing for BCC tumor response in patients with BCCNS.

"We believe BCCNS provides us with an ideal first opportunity to test itraconazole's anti-cancer potential," continued Virca. "This is a relatively well-defined patient population that suffers from multiple tumors which can be debilitating and often require disfiguring surgeries to remove cancerous lesions. Existing topical therapies have proven suboptimal due to lack of efficacy, and the only approved oral therapy for BCC has a significant side-effect profile. As such, we look forward to continuing our interactions with FDA, as we work to see this IND approved to study the safety and efficacy of SUBA-Itraconazole in these patients."

Industry sources estimate that there are approximately 10,000 patients in the U.S. with BCCNS, which could potentially qualify SUBA-Itraconazole under the FDA's Orphan Drug designation program for treatment of BCC in this patient population. Nearly all individuals with BCCNS carry a mutation in the PTCH1 gene that results in constitutive activation of the Hh pathway. Itraconazole has been shown to inhibit the Hh pathway which plays an important role in basal cell carcinomas.

Basal cell carcinoma is a skin cancer that commonly develops on sun exposed areas of fair-skinned individuals. BCC is generally a slow-growing tumor that does not metastasize (rate of metastasis estimated at no higher than 0.5%); however, it is locally invasive and can result in severe morbidity, especially in those patients with BCCNS who can develop hundreds or thousands of BCCs requiring frequent surgical intervention.