IDM Pharma Announces Updated Phase 3 Mifamurtide (L-MTP-PE) Data Confirms Statistically Superior Overall Survival in Osteosarcoma Patients
Thursday November 1, 4:30 pm ET
IRVINE, Calif., Nov. 1 /PRNewswire-FirstCall/ -- IDM Pharma, Inc. (Nasdaq: IDMI - News) today announced the presentation of updated survival data from the Phase 3 mifamurtide (L-MTP-PE) clinical trial (INT-0133). The trial was part of a National Cancer Institute (NCI) funded cooperative group study conducted by the Children's Oncology Group (COG) and the data is based on long-term follow up of patients originally treated in the Phase 3 trial. The results demonstrated that the addition of L-MTP-PE, formerly known as Junovan, to chemotherapy during treatment for osteosarcoma, a rare and often fatal bone tumor that typically affects children and young adults, achieved statistically superior Overall Survival (OS) rates.
"The statistically significant survival findings of L-MTP-PE in combination with chemotherapy in this study are very encouraging, particularly when compared to the outcome of these patients for the last 20 years," said Dr. Meyers, vice chair, department of pediatrics at Memorial Sloan Kettering Cancer Center and principal investigator of the Phase 3 trial. "These results are especially encouraging for the critically ill children and young adults with osteosarcoma who need an innovative treatment option for this devastating disease."
The data were presented during an oral presentation at the Connective Tissue Oncology Society (CTOS) annual meeting today in Seattle.
"We are very pleased to see that the overall survival benefit shown in the primary analysis of the Phase 3 clinical trial data was further strengthened by the statistically superior findings in long-term patient follow-up," said Timothy P. Walbert, president and chief executive officer, IDM Pharma. "As we announced on August 27, 2007 we are currently in the process of amending the L-MTP-PE New Drug Application with more complete long-term survival data from the same study, with the goal of obtaining regulatory approval and bringing L-MTP-PE to the patients who haven't had an improvement in treatment outcomes for the last 20 years."
Study design and findings
The multicenter, open label, randomized, factorial, four parallel treatment group Phase 3 study evaluated the effects of the addition of L-MTP-PE to chemotherapy in patients with resectable osteosarcoma without metastatic disease. There were 672 with newly diagnosed, non-metastatic, resectable osteosarcoma included in the COG analyses.
In the analyses by the COG investigators, Event Free Survival (EFS), including secondary malignancies, after six years in patients treated with chemotherapy and L-MTP-PE was 67%, compared to 61% in patients treated only with chemotherapy and no L-MTP-PE (p=0.08). Overall Survival (OS) after six years in patients treated with chemotherapy and L-MTP-PE was 78%, compared to 70% in patients treated only with chemotherapy and no L-MTP-PE (p=0.03).
Treatment with L-MTP-PE was generally well tolerated in all phases of study. Adverse events were mild to moderate in severity and included chills, fever, nausea, vomiting, myalgia, headache, tachycardia (fast heart rate), hypo- and hypertension, fatigue and shortness of breath, all of which are consistent events with the activation of monocytes and macrophages by L-MTP-PE and the flu-like symptoms that follow cytokine release. These side effects are readily prevented or treated with acetaminophen or ibuprofen.
L-MTP-PE Regulatory Status
The L-MTP-PE New Drug Application (NDA) includes efficacy and safety data from 678 patients with non-metastatic resectable osteosarcoma, 332 of whom received L-MTP-PE, and from 115 patients with metastatic or unresectable osteosarcoma, 39 of whom received L-MTP-PE, in the controlled Phase 3 trial conducted by the Pediatric Oncology Group (POG) and the Children's Cancer Group (CCG), now the Children's Oncology Group (COG), sponsored by the Cancer Therapy Evaluation Program (CTEP) of the National Cancer Institute. Also included are safety and efficacy data from 51 patients with metastatic osteosarcoma treated in earlier Phase 2 studies. The biological effects and safety of L-MTP-PE are further supported by data from 7 other Phase 1 and 2 clinical studies performed under IND, in which an additional 197 patients received at least one dose of L-MTP-PE.
L-MTP-PE was granted orphan drug status in the United States in 2001. The NDA for L-MTP-PE was submitted to the U.S. Food and Drug Administration (FDA) in October 2006 and was accepted for review in December 2006.
The FDA's Oncologic Drugs Advisory Committee, or ODAC, met in May 2007 and voted 12 to 2 that the results of the Company's Phase 3 trial do not provide substantial evidence of effectiveness of L-MTP-PE in the treatment of patients with non-metastatic, resectable osteosarcoma receiving combination chemotherapy. In July 2007, following a meeting with the FDA, the Company announced that it would collect, analyze and submit additional data for L-MTP-PE to the FDA, in an amendment to the NDA, by the first quarter of 2008. In August 2007 the FDA, considering ODAC's recommendation, issued a not approvable letter to the Company after completing the review of the NDA for L-MTP-PE. In this letter, the FDA requested data from additional clinical trials to demonstrate the benefit of L-MTP-PE, as well as information or clarification with respect to other sections of the NDA.
IDM Pharma also is seeking marketing approval from the European Medicines Agency (EMEA) for the use of L-MTP-PE, or MEPACT as it is known in Europe. L-MTP-PE was granted orphan drug status in Europe in 2004. The Marketing Authorization Application (MAA) for L-MTP-PE was submitted to the EMEA and accepted for review in November 2006. The EMEA application is currently under review and the Company continues to work closely with the regulatory body to ensure it has the information needed to approve L-MTP-PE. |
