Healthcare Industry News:  fast track designation 

Biopharmaceuticals Oncology FDA

 News Release - March 11, 2009

Molecular Insight Receives Special Protocol Assessment Approval From FDA for Phase 2 Trial of Azedra(TM), a Radiotherapeutic to Treat Neuroendocrine Cancer

Targeted Radiotherapeutic Earlier Received Orphan Drug Designation and Fast Track Status From FDA

CAMBRIDGE, MA--(Healthcare Sales & Marketing Network)--Mar 11, 2009 -- Molecular Insight Pharmaceuticals, Inc. (NasdaqGM:MIPI ) today 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.

About Orphan Drug Designation and Fast Track Status

Under the Orphan Drug Act, the FDA may grant such designation to drugs intended to treat a rare disease or condition that generally affects fewer than 200,000 individuals. Orphan Drugs are granted a seven-year period of market exclusivity during which the FDA may not approve any other application to market the same drug for the same disease except in very limited circumstances.

The Fast Track program, established under the FDA Modernization Act of 1997, provides for expedited regulatory review for investigational drugs demonstrating the potential to address unmet medical needs for the treatment of serious or life-threatening conditions. Under the program, FDA will facilitate and expedite the development and review of Molecular Insight's application for the approval of the product.

About Molecular Insight Pharmaceuticals, Inc.

Molecular Insight Pharmaceuticals (NasdaqGM:MIPI ) is a Cambridge, Massachusetts-based biopharmaceutical company specializing in the emerging field of molecular medicine, applying innovations in the identification and targeting of disease at the molecular level to improve healthcare for patients with life-threatening diseases. The Company is focused on discovering, developing, and commercializing innovative molecular imaging radiopharmaceuticals and targeted molecular radiotherapeutics with initial applications in the areas of cardiology and oncology. Molecular Insight's lead molecular imaging radiopharmaceutical product candidate, Zemiva(TM), is being developed for the diagnosis of cardiac ischemia, or insufficient blood flow to the heart. The Company's imaging candidate, Trofex(TM), is in development for the detection of metastatic prostate cancer. Molecular Insight's lead molecular radiotherapeutic product candidates, Azedra and Onalta(TM), are being developed for detection and treatment of cancer. In addition, the Company has a growing pipeline of product candidates resulting from application of its proprietary platform technologies to new and existing compounds. For more information, visit

Forward-Looking Statements

Statements in this release that are not strictly historical in nature constitute "forward-looking statements." Such statements include, but are not limited to, statements about the development of Azedra(TM), Onalta(TM), Zemiva(TM), Trofex(TM), Solazed(TM) and our other product candidates. Such forward-looking statements involve known and unknown risks, uncertainties, and other factors that may cause the actual results of Molecular Insight to be materially different from historical results or from any results expressed or implied by such forward-looking statements. These factors include, but are not limited to, risks and uncertainties related to the progress, timing, cost, and results of clinical trials and product development programs; difficulties or delays in obtaining regulatory approval for product candidates; competition from other pharmaceutical or biotechnology companies; and the additional risks discussed in filings with the Securities and Exchange Commission (SEC). The Company's SEC filings are available through the SEC's Electronic Data Gathering Analysis and Retrieval system (EDGAR) at Press releases for Molecular Insight Pharmaceuticals, Inc. are available on our website: If you would like to receive press releases via e-mail, please contact: All forward-looking statements are qualified in their entirety by this cautionary statement, and Molecular Insight undertakes no obligation to revise or update this release to reflect events or circumstances after the date hereof.

Source: Molecular Insight Pharmaceuticals

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