HedgePath gets FDA nod to begin proposed cancer drug development

The IND covers the company’s clinical development plan for a patented formulation of itraconazole (SUBA-Itraconazole) with improved bioavailability to study the treatment of basal cell carcinoma (BCC) in patients with basal cell carcinoma nevus syndrome (BCCNS) also called as Gorlin’s syndrome.

BCCNS results from a genetic mutation which causes the Hedgehog pathway to function improperly, leading to the chronic formation of basal cell tumors, including potentially disfiguring lesions on the face.

The company holds the exclusive US license rights to SUBA-Itraconazole to treat cancer, and this is the first cancer indication the company is seeking to develop.

HPPI president and chief executive officer Nicholas Virca said: "We now hope to establish a track record of achieving positive milestones as we progress this product through clinical development."

The FDA-cleared single arm, multi-center, open-label, non-placebo-controlled Phase IIb trial is expected to enroll around 40 patients starting in January 2015.

The trial’s primary endpoint will be the objective response rate of BCC lesions to SUBA-Itraconazole in subjects with BCCNS, under which a meaningful response will be defined as a 30% or greater reduction in the cumulative size of target tumors, with an appropriate sample size to be 33 of the 40 patients in order to maintain a 90% power.

The company expects that if there is evidence of substantial clinical benefit to these patients, the company would seek to have further discussions with FDA as to whether results from this trial could potentially serve as the basis for the submission of a new drug application (NDA) to FDA for this product candidate.