Date: 2015-01-23
Type of information: Presentation of results at a congress
phase: 2
Announcement: presentation of results at the 28th Annual European Association of Nuclear Medicine (EANM) Congress being held in Hamburg, Germany
Company: Progenics Pharmaceuticals (USA - NY)
Product: Azedra™ (Ultratrace® iobenguane I 131)
Action
mechanism: radiopharmaceutical. Azedra® is a radio-therapeutic with FDA Fast Track status currently being studied in a phase 2 registrational trial under a SPA with the FDA using a surrogate marker as registrational endpoint. In addition to potentially treating pheochromocytoma and paraganglioma, Azedra® may also have utility in treating neuroblastoma and other neuroendocrine tumors. Clinical trials for Azedra have not been completed and regulatory approvals for Azedra remain subject to the successful completion of all required studies. Progenics acquired Azedra, a novel targeted cancer radiotherapy, in conjunction with its 2013 acquisition of Molecular Insight Pharmaceuticals (MIP). Azedra has received Orphan Drug and Fast Track designations from the FDA.
Disease: pheochromocytoma
Therapeutic area: Cancer - Oncology - Rare diseases
Country:
Trial details:
Latest
news: * On October 12, 2015, Progenics Pharmaceuticals announced that data from its Phase 2 study of Azedra® for the treatment of ultra-orphan indications pheochromocytoma and paraganglioma were highlighted during an oral presentation at the 28th Annual European Association of Nuclear Medicine (EANM) Congress being held in Hamburg, Germany. The study was designed to evaluate the efficacy and safety of two therapeutic doses of Azedra® given 3 months apart, with dosimetric evaluation prior to the first therapeutic dose to ascertain avidity and tissue distribution. 44 patients had received a dosimetry dose before Molecular Insight suspended patient enrollment due to lack of funding. In this study, 44 patients underwent dosimetric evaluation, of which 41 (93.2%) demonstrated tumor avidity. 38 (86.4%) were eligible to receive up to 37 GBq based on dosimetry, while 6 (13.6%) required dose reduction based on pre-specified limits for the kidneys, liver, and lungs. Azedra was generally well tolerated with gastrointestinal and hematologic toxicity most frequently reported. * On January 23, 2015, Progenics Pharmaceuticals announced that it has dosed the first subject in the resumed pivotal Phase 2 clinical study of Azedra™ in patients with malignant pheochromocytoma and paraganglioma. The trial is being conducted under a Special Protocol Assessment (SPA) with the FDA. The study 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. In late 2010, MIP suspended enrollment in the trial to seek additional funding. The trial has now resumed to fulfill enrollment requirements under the SPA. The trial has treated 41 patients and 32% of those patients have achieved the primary endpoint. The most common adverse events observed have been gastroenterological and hematologic disorders.
Progenics also presented updated preliminary long-term survival data from the first 41 patients dosed before enrollment in the trial was suspended in 2010. Seven of those patients had received one and 34 had received two therapeutic doses of Azedra. The study's primary endpoint, a reduction of all anti-hypertensive medications for at least 50% and for at least six months, was achieved by 31.7% of patients. As of April 2015, the 34 patients receiving two therapeutic doses of Azedra had a median survival of 48.5 months.
