New Study Begins for Kids With High-Risk Cancer

Molecular Insight Pharmaceuticals, Inc. (NASDAQ: MIPI - News) announced today that it has initiated a Phase 2a clinical trial of Azedra (Ultratrace™ iobenguane I-131 or Ultratrace MIBG) for the treatment of children with high-risk neuroblastoma, a neuroendocrine cancer that primarily affects children. Azedra is already being evaluated in an ongoing dose-ranging clinical trial for pheochromocytoma in adults. Azedra is a targeted radiotherapeutic comprised of the known I-131 MIBG molecule radiolabeled using Molecular Insight’s proprietary Ultratrace technology, which removes unnecessary nonradioactive molecules, or cold contaminants, to maximize therapeutic potential.

The primary objective of the trial is to determine the maximum tolerated dose (MTD) of Azedra in children with neuroblastoma. Secondary goals are to determine tumor and dose responses; determine toxicity; estimate normal organ dosimetry; and describe the effect of Azedra therapy on overall quality of life. The Molecular Insight trial is being coordinated by the New Approaches to Neuroblastoma Therapy (NANT) Consortium, a group of 14 universities and Children’s Hospitals with strong research and treatment programs for neuroblastoma.

“This important Azedra trial in children with neuroblastoma underscores our full commitment to develop small molecule, targeted radiotherapeutics that address significant unmet needs in cancers affecting both adults and children,” said David S. Barlow, Chairman and Chief Executive Officer of Molecular Insight. “Along with our ongoing Azedra dose-ranging clinical trial in adults with pheochromocytoma, we are developing several other targeted radiotherapeutics to treat and detect cancer, including OnaltaTM, which complements Azedra, for the treatment of neuroendocrine tumors; SolazedTM for the treatment of malignant melanoma; and TrofexTM for the detection and staging of metastatic prostate cancer.”

“Neuroblastoma is an aggressive and difficult to treat form of neuroendocrine cancer that usually affects children under five years of age,” said Katherine Matthay, M.D., Chief of Pediatric Hematology and Oncology at the University of California San Francisco Children’s Medical Center. “Unfortunately, the prognosis for many of these children is quite poor. There currently are no FDA-approved treatments available for patients who have progressive, recurrent or refractory disease, and our current treatment options are extremely limited.” Dr. Matthay is chairing the study for the NANT Consortium.

“This trial is the first to assess Azedra’s activity in neuroblastoma patients as part of our larger program to evaluate its therapeutic potential in multiple types of neuroendocrine cancer,” said John W. Babich, Ph.D., President and Chief Scientific Officer of Molecular Insight. “While traditional I-131 MIBG has demonstrated clinical activity in neuroblastoma in the past, it contains cold contaminants that compete with the active radiotherapeutic molecules binding to receptor sites on tumor cells. Our preclinical studies have shown that Azedra, produced with our proprietary Ultratrace technology, enables the highest proportion of MIBG molecules to be radiolabeled, significantly increasing the amount of radiolabeled MIBG uptake in the tumor and reducing the potential for side effects due to cold contaminants.”