EPCT-04. RESULTS OF A PHASE 1 STUDY OF THE ONCOLYTIC ADENOVIRUS DNX-2401 WITH RADIOTHERAPY FOR NEWLY DIAGNOSED DIFFUSE INTRINSIC PONTINE GLIOMA (DIPG)

Abstract Background A Phase 1, single center study is ongoing to evaluate the conditionally replicative oncolytic adenovirus, DNX-2401 (tasadenoturev), followed by radiotherapy (RT) in pediatric patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG). Methods Patients 1–18 years DIPG no prior treatment, Lansky/Karnofsky performance score ≥ 70, and adequate organ function were enrolled. tumor biopsy was performed a intratumoral injection of 1e10-5e10 virus particles (vp) DNX-2401. Conventional initiated within 1 month administration. Results Enrolled subjects (n=12) had median age 9 (range 3–18) scores 90–100 (n=4; 33%) or 70–80 (n=8; 67%). As part dose escalation design, treated 1e10 vp (n=4) 5e10 (n=8), which then standard RT 11 12 (92%). No dose-limiting toxicities observed treatment regimen well-tolerated. Adverse events (AEs) have been primarily mild moderate consistent underlying disease. The most commonly reported AEs (≥ 5 subjects), regardless drug relationship, include headache, asthenia, vomiting, anemia, leukocytosis, fever. Two SAEs including grade 3 lymphopenia abdominal pain. Tumor reductions efficacy evaluations are ongoing. 09Dec2020, 12-month survival (OS-12) 71% 4 survived > 20 months. Four continue be for survival. Correlative analysis peripheral samples Conclusions can safely administered DIPG; clinical activity preliminary encouraging.