On May 30, 2017 HedgePath Pharmaceuticals, Inc. (OTCQX:HPPI), a clinical stage biopharmaceutical company that discovers, develops and plans to commercialize innovative therapeutics for patients with cancer, reported the grant of a Type-C Guidance Meeting Request by the U.S. Food and Drug Administration (FDA) concerning further guidance from FDA for HPPI’s ongoing, open-label Phase 2(b) clinical trial studying the effect of SUBA-Itraconazole (SUBA-Cap) oral capsules in patients with Basal Cell Carcinoma Nevus Syndrome (BCCNS), also known as Gorlin Syndrome (Press release, HedgePath Pharmaceuticals, MAY 30, 2017, View Source [SID1234519323]). Schedule your 30 min Free 1stOncology Demo! Included in HPPI’s meeting request were summary data for 35 patients enrolled in the trial relating to reduction in target tumor burden, safety and time on study, along with specific questions to FDA regarding further steps necessary for completion of the study and reporting of final data. HPPI noted in its meeting request to FDA that all patients on SUBA-Cap therapy had some degree of measurable target tumor burden decrease with a median time on study of 32 weeks and a dropout rate of only 11%.
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As a result of FDA granting HPPI’s meeting request, HPPI is required to file a complete background package for its Phase 2(b) trial results to FDA by mid-June 2017, and FDA has indicated its goal is to provide a written response to HPPI with further guidance before the end of July 2017.
Nicholas Virca, President and CEO of HPPI, stated that, "We reported to FDA that 37% of our patients in our Phase 2(b) trial have demonstrated an equal to or greater than 30% reduction in target tumor burden and there has been a complete disappearance of 28% of all target lesions across all subjects. We are testing SUBA-Cap therapy in BCCNS patients with a significant history of BCC surgeries and intend to further note in our background package that, for the 35 patients being dosed in our trial, the mean number of prior BCCs removed by surgery was 195 per patient, yet 97% of our study group have avoided surgery while on SUBA-Cap therapy. We are very pleased with these results and look forward to FDA’s feedback as we move towards the conclusion and reporting of the results of this trial."
While these data appear to be predictive of the desired final study results while HPPI seeks further guidance from FDA, readers are cautioned that no assurances can be given that (i) the final study results will match these latest results or (ii) the study when and if completed will achieve its primary and secondary endpoints or (iii) that the study will be found by FDA to be sufficient for the filing of a New Drug Application (NDA) or (iv) if an NDA is filed, that it will be approved by FDA. Further, HPPI is not committing to providing further interim updates prior to the reporting of the final study results.
About BCCNS
BCCNS results from a genetic mutation which causes the Hedgehog pathway (a major regulator of processes in cells) to function improperly, leading to the chronic formation of basal cell tumors, including potentially disfiguring lesions on the face. Industry sources estimate that there are approximately 10,000 patients in the United States with BCCNS, which has qualified SUBA-Itraconazole under the FDA’s Orphan Drug Designation Program.
About SUBA-Itraconazole
SUBA-Itraconazole is a patented and proprietary itraconazole formulation that enhances the absorption of itraconazole to improve the bioavailability of orally administered drugs that are poorly soluble. The U.S. rights to SUBA-Itraconazole for the treatment of cancer are exclusively licensed to HPPI by an affiliate of Mayne Pharma Group Limited. SUBA-Itraconazole was developed to improve absorption and significantly reduce variability compared to generic itraconazole. These benefits provide enhancements to patients and prescribers with reduced intra- and inter-patient variability, enabling a more predictable clinical response and a reduction in the active drug quantity to deliver the required therapeutic blood levels.