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Molecular Insight Pharmaceuticals, Inc. announced the receipt of a SPA letter stating that the U.S. FDA has reached agreement with the Company regarding the design of the pivotal Phase 2 trial for registration of its lead oncology candidate Molecular Insight Pharmaceuticals, Inc. announced the receipt of a Special Protocol Assessment (SPA) letter stating that the U.S. Food and Drug Administration (FDA) has reached agreement with the Company regarding the design of the pivotal Phase 2 trial for registration of its lead oncology candidate, Azedra(TM) (Ultratrace(TM) iobenguane I 131, formerly known as Ultratrace MIBG). Molecular Insight is developing Azedra for the treatment of neuroendocrine tumors, such as pheochromocytoma in adults and neuroblastoma in children. This study will be conducted in adults with pheochromocytoma. If successful, Azedra would be the first anti-cancer therapy in the United States indicated for the treatment of pheochromocytoma.

An SPA is an agreement between the trial sponsor and the FDA covering the major design features such as patient population, choice of control, primary efficacy endpoint(s), safety monitoring plan, and statistical analysis plan of a clinical trial to be used as the pivotal evidence of safety and efficacy in support of regulatory approval. Having secured FDA's agreement to the Phase 2 protocol, the Company plans to proceed immediately with initiation of the study.

"Neuroendocrine cancers can be insidious, aggressive and difficult to diagnose," said John W. Babich, Ph.D., Chief Executive Officer, President and Chief Scientific Officer of Molecular Insight. "The prognosis is poor because of local recurrence or widespread metastasis. There are no known, effective treatments for relapsed refractory pheochromocytoma or neuroblastoma. Azedra has demonstrated enhanced tumor kill in preclinical models of human neuroendocrine cancers, the avoidance of cardiovascular side effects of other iobenguane preparations and excellent tumor targeting in initial clinical trials. FDA's agreement with our pivotal Phase 2 protocol represents an important step in the advancement of our clinical program."

The study (IB-12b) will be a single-arm trial conducted in 58 adults with pheochromocytoma. The primary endpoint of the study will be Azedra's sustained improvement of severe hypertension, a prominent sign of pheochromocytoma due to the abnormal release of large amounts of hormones by the tumor. Secondary endpoints will include demonstration of overall anti-tumor response, and the improvement in patients' daily functioning and performance including improvement in quality of life.

Azedra recognizes a unique molecular target expressed on the surface of certain neuroendocrine cancer cells, which enables the drug to be accumulated selectively by the tumor. Azedra, developed using Molecular Insight's proprietary Ultratrace technology, is designed to maximize the delivery of iobenguane I 131 molecules to targeted neuroendocrine tumors and to enable safe and effective treatment while minimizing the amount of non-radioactive iobenguane molecules administered to the patient. Azedra is based on iobenguane I 131, a radiotherapeutic currently marketed in Europe for treating several types of neuroendocrine cancers. Therapeutic iobenguane I 131 has not previously been approved for marketing in the United States.

In 2006, Azedra was granted Fast Track designation and Orphan Drug status by FDA. Molecular Insight has completed a Phase 1 dosimetry study and a Phase 1 dose-finding study in patients with pheochromocytoma. A Phase 2a dose-ranging and efficacy clinical trial in children with high-risk neuroblastoma is underway and FDA's review of a second SPA for the pivotal trial supporting the pediatric neuroblastoma indication is in progress.

Source: Molecular Insight Pharmaceuticals, Inc.