Azedra® (Ultratrace® iobenguane I 131) is a later-stage drug candidate being developed for the treatment of malignant pheochromocytoma and paraganglioma. Pheochromocytomas are rare tumors that arise from cells of the sympathetic nervous system. Such tumors are usually found within one or both adrenal glands (85%), but may arise in other areas of sympathetic nerve cells, and are then referred to as paragangliomas. We have taken a unique theranostic approach in treating this ultra-orphan disease. We use a small molecule that specifically targets neuroendocrine tumors (such as pheochromocytoma and paraganglioma) and attach a radioisotope to create Azedra®, which is designed for use as an imaging agent as well as for therapy. The patients are first imaged with this radiopharmaceutical to determine a therapeutic dose that is safe to normal organs and effective in destroying cancer cells. This imaging step is designed to enable the delivery of an individualized therapeutic dose that is optimized for each patient.
Currently, the standard treatment options for metastatic malignant pheochromocytoma or paraganglioma include adrenergic blockade, surgery, chemotherapy, and radiation. Unfortunately, these options are only palliative or generally fail to produce cure or significant remissions. A product chemically similar to Azedra® with a much lower specific activity and less desirable side effect profile has been used with some success; however, it is not approved by the FDA for the treatment of pheochromocytoma and paraganglioma. Therefore, effective alternative therapies are needed, especially in patients who have relapsed following initial treatment or who have tumors refractory to initial therapies.
Azedra® (Ultratrace® iobenguane I 131) is a very high specific activity form of iobenguane l 131, produced using our proprietary Ultratrace® platform. This technology is designed to prevent unlabeled or “cold” iobenguane from being carried through the manufacture process to the final formulation, and to produce a product of a very high specific activity. We are working to make Azedra® deliver more effective tumor destructive power and avoid typical side effects from the use of a low specific activity product, and therefore to provide effective therapy for patients suffering from pheochromocytoma and paraganglioma.