On December 03, 2015 Progenics Pharmaceuticals, Inc. (Nasdaq:PGNX) reported that it has completed enrollment in its Phase 2b clinical trial of its ultra-orphan radiotherapeutic candidate Azedra for the treatment of malignant or recurrent pheochromocytoma and paraganglioma in accordance with the Special Protocol Assessment (SPA) agreement with U.S. Food and Drug Administration (FDA) (Press release, Progenics Pharmaceuticals, DEC 3, 2015, View Source [SID:1234508392]). Schedule your 30 min Free 1stOncology Demo! Progenics will allow enrollment to continue through the end of December so that patients already identified as potential candidates may have the opportunity to participate in the trial. The Company expects to report top-line data in the second half of 2016.
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"Achieving the number of enrolled patients required under the SPA is a major milestone for Progenics and the Azedra program, a novel therapeutic candidate that has the potential to be the first approved therapy in the U.S. for the treatment of malignant or recurrent pheochromocytoma and paraganglioma," said Mark Baker, Chief Executive Officer of Progenics. "The trial, if positive, will provide the basis for a regulatory submission to the FDA, and we look forward to building on the impressive data reported to date as we advance Azedra as a treatment option for these very rare and life threatening tumors."
The open-label trial is designed to evaluate the efficacy and safety of the administration of two therapeutic doses of Azedra in patients with malignant relapsed/refractory pheochromocytoma or paraganglioma, ultra-orphan cancers with limited treatment options. The primary objective of the study is to determine the clinical benefit of Azedra based on the proportion of study participants with a reduction of all antihypertensive medication by at least 50% for at least six months. The SPA requires that 25% of 58 evaluable patients achieve the primary endpoint.
Azedra has received Orphan Drug, Fast Track and Breakthrough Therapy designations from the FDA.
About Azedra
Azedra (Ultratrace iobenguane I-131) is a radio-therapeutic with Breakthrough Therapy Designation and FDA Fast Track status currently being studied in a phase 2b registrational trial under a Special Protocol Assessment with the U.S. Food and Drug Administration using a surrogate marker registrational endpoint.
About Pheochromocytoma and Paraganglioma
Pheochromocytomas and paragangliomas are rare neuroendocrine tumors that arise from cells of the sympathetic nervous system. When pheochromocytomas are located outside the adrenal glands, they are called paragangliomas. Standard treatment options for these tumors include surgery, palliative therapy and symptom management. Malignant and recurrent pheochromocytomas and paragangliomas may result in unresectable disease with a poor prognosis, representing a significant management challenge with very limited treatment options and no approved anti-tumor therapies.