On June 16, 2016 Medivation, Inc. (NASDAQ: MDVN) reported results from a Phase I/II study of pidilizumab, an investigational antibody with immune-mediated anti-tumor effects, that demonstrated potential clinical benefit in pediatric patients with diffuse intrinsic pontine glioma (DIPG) (Press release, Medivation, JUN 16, 2016, View Source [SID:1234513409]). Schedule your 30 min Free 1stOncology Demo! The study, which was exploratory in nature, was designed to assess the safety and tolerability of pidilizumab, as well as key clinical outcomes, such as event-free and overall survival, in this pediatric population. Preliminary data were presented this week in an oral symposium at the International Symposium on Pediatric Neuro-Oncology (ISPNO) by the study’s lead investigator Iris Fried, M.D., Attending Physician, Pediatric Hemato-oncology, Hadassah Medical Center, Jerusalem, Israel.
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Diffuse intrinsic pontine glioma (DIPG) is a rare and aggressive pediatric cancer which is responsible for the highest brain tumor mortality in children.1 Approximately 300-400 pediatric brain stem tumors are diagnosed per year in the United States, approximately 75%-80% of which are DIPGs.2 Children with DIPG experience a median overall survival between 9-12 months and a two-year survival rate of less than 10%.1
Data from nine pediatric patients with DIPG who were treated with pidilizumab following completion of standard radiation therapy were presented. The median age of the study population was 6.5 years (range: 3-19 years): eight patients had intermediate risk features and one patient had high risk features. The reported mean event-free and overall survival estimates were 12 and 15.6 months, respectively. Three patients with DIPG remained progression-free at 16.3, 22, and 24 months following diagnosis, with one patient experiencing a partial response. Adverse events of any grade reported in at least one treatment cycle include neutropenia, fatigue, loss of appetite, hypertension, nausea, and lymphopenia; only neutropenia and hypertension were reported as grade 3 adverse events. The study continues to enroll patients.
"Separate studies have revealed the potential of pidilizumab in hematological malignancies, and these results, while in a small cohort in a rare disease, suggest its potential activity in other conditions," said David Hung, M.D., Founder, President and Chief Executive Officer of Medivation. "We are especially encouraged by these results in a devastating disease that typically leads to such rapid and certain mortality, and we remain committed to advancing the clinical development of pidilizumab as we continue to characterize its unique and differentiated mechanism of action."
Despite more than 30 years of clinical research, there have been no improvements in clinical outcomes and there are no approved treatments for DIPG.1,2