“In this pivotal study of AZEDRA in pheo and para patients, the overall tumor biomarker response correlated significantly with responder status with both the primary and secondary endpoints,” said Dr.
Dr. Jimenez reviewed the data in an oral presentation titled, “AZEDRA® (iobenguane I 131) in Patients with Metastatic and/or Recurrent and/or Unresectable Pheochromocytoma or Paraganglioma: Biochemical Tumor Marker Results of a Multicenter, Open-Label Pivotal Phase 2 Study.”
Tumor biomarkers were analyzed in patients who had individual tumor biomarkers above 1.5x the upper limit of normal (ULN) at baseline. The overall tumor biomarker response correlated with responder status for those who met the study’s primary endpoint (r = 0.31, p = 0.011) and objective tumor response (r=0.35, p=0.006). The biomarkers evaluated were Chromogranin A (CgA, serum), Normetanephrine (NM, serum and urine) and Norepinephrine (NE, serum and urine). At 12 months following the first therapeutic dose of AZEDRA, urine and serum NE showed best response (CR/PR) rates of 42.1% and 31.0%, respectively, and urine and serum NM responder rates of 36.0% and 44.0%, respectively. In addition, serum CgA showed best response rate of 67.9% at 12 months.
“The compelling results from this pivotal trial formed the basis of our New Drug Application for AZEDRA, which was accepted for review by the
The pivotal phase 2 open-label, multi-center trial was conducted under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (
AZEDRA (iobenguane I 131) is a high-specific-activity radiotherapeutic product candidate in development as a treatment for malignant, recurrent, or unresectable pheochromocytoma and paraganglioma, which are rare neuroendocrine tumors of neural crest origin. AZEDRA is a substrate for norepinephrine reuptake transporter which is highly expressed on the cell surface of neuroendocrine tumors. AZEDRA has been granted Orphan Drug designation, Fast Track status, and Breakthrough Therapy designation in the U.S. Under a SPA agreement with the FDA, a Phase 2 pivotal study has been completed in patients with malignant, recurrent, or unresectable pheochromocytoma and paraganglioma. The FDA granted Priority Review of Progenics’ New Drug Application and has set an action date of
About Pheochromocytoma and Paraganglioma
Pheochromocytoma and paraganglioma are rare neuroendocrine tumors that arise from cells of the autonomic nervous system. Pheochromocytoma forms in the adrenal medulla, whereas paragangliomas form outside the adrenal gland. Standard treatment options for these tumors include surgery, palliative therapy and symptom management. Pheochromocytoma and paraganglioma tumors frequently secrete high levels of hormones that can lead to life-threatening hypertension, heart failure, and stroke in these patients. Malignant and recurrent pheochromocytoma and paraganglioma 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.
Progenics develops innovative medicines and other technologies to target and treat cancer. Progenics' pipeline includes: 1) therapeutic agents designed to precisely target cancer (AZEDRA®, 1095, and PSMA TTC), 2) PSMA-targeted imaging agents for prostate cancer (1404 and PyL™), and 3) imaging analysis technology. Progenics' first commercial product, RELISTOR® (methylnaltrexone bromide) for opioid-induced constipation, is partnered with
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Source: Progenics Pharmaceuticals Inc.