IRVINE, Calif., Aug. 27 IDM Pharma, Inc.(Nasdaq: IDMI) announced today that it received a not approvable letter fromthe U.S. Food and Drug Administration (FDA) after completing the review of thenew drug application (NDA) for the investigational drug mifamurtide(L-MTP-PE), formerly known as Junovan, for the treatment of non-metastaticosteosarcoma, a rare and often fatal bone tumor that typically affectschildren and young adults.
The FDA has requested data from additional clinical trials to demonstratethe benefit of L-MTP-PE. Additionally, the FDA has requested information orclarification with respect to other sections of the NDA.
"We continue to strongly believe in the overall survival benefit andsafety profile of L-MTP-PE for the treatment of osteosarcoma and are committedto obtaining regulatory approval to provide L-MTP-PE to the critically illchildren and young adults who have not had a new treatment option in more than20 years," said Timothy P. Walbert, President and Chief Executive Officer ofIDM Pharma, Inc. "In anticipation of a not approvable letter from the FDA, inJuly we announced our intent to amend the current NDA with additional survivaldata with the goal of bringing L-MTP-PE to market as quickly as possible.While the FDA has asked for data from additional clinical trials, we believethat this decision was made in context of the lack of complete data in thesubmitted NDA and that capturing supplemental data will overcome the need foradditional trials and further confirm the overall survival benefit of L-MTP-PEin osteosarcoma and provide evidence for approvability."
The Company will continue working with the cooperative groups andinvestigative sites involved in the study to collect vital status (informationon whether the subjects remain alive or have died) on patients whoparticipated in the Phase 3 clinical trial and for whom complete data were notavailable at the time of filing of the NDA in October 2006. When theadditional follow up data have been collected, the Company will analyze thedata and expects to submit an amendment to the NDA to the FDA by the firstquarter of 2008. In addition to collecting supplemental Phase 3 data, theCompany will address the other points raised in the FDA action letter.
"While we believe the evidence demonstrating the overall survival benefitof L-MTP-PE exists, we commend IDM Pharma for their continued investment inand commitment to working with investigators to collect the most recent dataavailable," said Matthew Alsante, executive director, Sarcoma Foundation ofAmerica. "As the survival in children with osteosarcoma has not improved inthe last twenty years, gaining approval of investigational agents withdemonstrated improvement in survival is critical."
Conference Call Details
The Company will be holding a conference call on Monday, August 27, 2007at 4:30 p.m. ET. A webcast of the call will be accessible through theCompany's website at http://www.idm-pharma.com. A replay of the call will beavailable on the website for 30 days.
About the mifamurtide (L-MTP-PE) NDA
The L-MTP-PE NDA includes efficacy and safety data from 678 patients withnon-metastatic resectable osteosarcoma, 332 of whom received L-MTP-PE, andfrom 115 patients with metastatic or unresectable osteosarcoma, 39 of whomreceived L-MTP-PE, in the controlled Phase 3 trial conducted by the PediatricOncology Group (POG) and the Children's Cancer Group (CCG), now the Children'sOncology Group (COG), sponsored by the Cancer Therapy Evaluation Program(CTEP) of the National Cancer Institute. Also included are safety andefficacy data from 51 patients with metastatic osteosarcoma treated in earlierPhase 2 studies. The biological effects and safety of L-MTP-PE are furthersupported by data from 7 other Phase 1 and 2 clinical studies performed underIND, in which an additional 197 patients received at least