Chimerix (NASDAQ: CMRX) has announced the publication of a peer-reviewed article entitled, “Phase 2 Study of ONC201 in Neuroendocrine Tumors including Pheochromocytoma-Paraganglioma (PC-PG) and Desmoplastic Small Round Cell Tumor (DSRCT),” in the Clinical Cancer Research journal. In addition, an interim report of specific cohorts from the trial was presented at the American Society of Clinical Oncology (ASCO) annual meeting in 2021.
ONC201 is a dopamine receptor D2
ONC201 is a small compound dopamine receptor D2 (DRD2) blocker and caseinolytic protease (ClpP) activator that is taken orally.
Professor, Department Pediatric Hematology/Oncology/BMT at Cleveland Clinic Children’s and Case Comprehensive Cancer Center and principal study investigator, Peter Anderson, said, “Given ONC201’s demonstrated biological activity with DRD2, we took a deliberate approach to explore its potential utility in neuroendocrine tumors. Metastatic PC-PG and DSRCT patients had meaningful responses by RECIST criteria, and some were treated for years with improvements in their disease-related symptoms.”
Chimerix Chief Medical Officer Allen Melamed, “Patients with metastatic neuroendocrine cancers are in need of more effective and well-tolerated treatment options. This efficacy and safety data supports further investigation of ONC201’s activity beyond recurrent H3 K27M-mutant diffuse midline glioma. We look forward to continuing to explore additional tumors where ONC201 may provide therapeutic benefit to existing standards of care.”
Thirty rare neuroendocrine tumors treated in phase 2
Thirty individuals with rare neuroendocrine tumors were treated in the open-label second Phase investigator-initiated research. Patients with paraganglioma were divided into two groups in the research, each taking ONC201 once or twice per week. Patients with different neuroendocrine tumors, such as desmoplastic small round cell tumor (DSRCT), were dosed each week with ONC201 in a third group. As judged by RECIST criteria, the radiographic reaction was the primary outcome.
In the group of paraganglioma individuals who received ONC201 once per week, 50% of the patients showed a partial response (PR), while two more subjects had stable disease (SD) for more than 3 months. Five of the ten subjects in the group received treatment for more than a year. Seven SD and one PR were recorded among the paraganglioma subjects receiving ONC201 twice per week.
