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12:00 AM
May 14, 2007
 |  BioCentury  |  Regulation

A roll of the dice


A roll of the dice

There may be better days to come for the cancer immunotherapy space, but last Wednesday was not one of them. The Oncologic Drugs Advisory Committee's thumbs-down for IDM Pharma Inc.'s Junovan mifamurtide for osteosarcoma and the approvable letter for Dendreon Corp.'s Provenge sipuleucel-T prostate cancer vaccine underlined that FDA is in no mood to relax its demand for well-designed trials that meet their primary endpoint.

Indeed, the Junovan panel, which voted 12-2 that IDMI failed to demonstrate substantial evidence of efficacy, is a reminder that asking for approval based on a non-registration study is a roll of the dice, at best.

Junovan and Provenge are two of the first in a wave of cancer immunotherapies moving through the clinic that act by stimulating the immune system to reject or destroy tumor cells.

For IDMI (San Diego, Calif.), ODAC's issues related mainly to flaws in the design of the trial used to support the company's efficacy claim for Junovan, a liposomal formulation of muramyl tripeptide phosphatidyl ethanolamine that works by stimulating tumoricidal activity by macrophages. This may come as no surprise, given that the data package was based on pooled analyses of data from a single cooperative trial that began in 1993.

The early preclinical and clinical work on Junovan was conducted by Ciba-Geigy Corp., a predecessor of Novartis AG (NVS; SWX:NOVN, Basel, Switzerland), under an IND originally submitted in 1988. However, the pharma company dropped the program in 1996 after deciding that the osteosarcoma market would be too small to justify its R&D costs. Junovan was picked up by Jenner Biotherapies Inc., which subsequently went out of business and sold the program to IDMI in 2003 - long after the clinical trial supporting the NDA had completed enrolling patients.

While the IND was changing hands, a pediatric cooperative group called Children's Oncology Group began an open-label Phase III trial (Study INT-0133) that enrolled 678 patients with resectable, non-metastatic, high-grade osteosarcoma. It was by far the largest study ever conducted in a patient population where only 400 cases are diagnosed each year in the U.S.

The trial was never meant for registration purposes. While it was powered to measure disease-free survival, DFS was not clearly defined as the primary endpoint, although the company and...

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