Ivy Brain Tumor Center Announces Promising Data for Niraparib in Phase 0/2 Glioblastoma Clinical Trial
PHOENIX, ARIZ, November 17, 2023 – The Ivy Brain Tumor Center at Barrow Neurological Institute announces promising results from a Phase 0/2 trial of niraparib in patients with newly diagnosed glioblastoma with unmethylated MGMT. Updated data from this collaborative study, supported by GSK, will be presented in two oral presentations at the Society for Neuro-Oncology (SNO) 28th Annual Meeting being held November 15-19 in Vancouver, Canada. Glioblastoma is an incurable malignant brain tumor and MGMT-unmethylated glioblastoma is the most common and fastest-recurring variant, comprising more than 60 percent of all glioblastoma tumors.
Nineteen patients with MGMT-unmethylated, newly diagnosed glioblastoma were enrolled into the Phase 2 component of the study, where niraparib was combined with standard-of-care radiotherapy followed by niraparib maintenance until disease progression. At the time of data cutoff, patients treated with niraparib achieved a median progression-free survival (PFS) of 11.7 months, comparing favorably to the 5.3 months historically reported with temozolomide, the standard-of-care chemotherapy for this patient population. The median overall survival (OS) was not yet reached and the combination of niraparib plus radiotherapy was well tolerated, consistent with the known safety profile of niraparib, thrombocytopenia being the most common adverse event reported in the trial.
In the Phase 0 component, patients exposed to niraparib demonstrated high drug concentrations in brain tumor tissue. Based on these results, all Phase 0 study patients with unmethylated MGMT promoter tumor status qualified for the Phase 2 component of the study.
Dr. Nader Sanai, Director of the Ivy Brain Tumor Center and Director of Neurosurgical Oncology at Barrow Neurological Institute, said, “This clinical trial identifies niraparib as highly brain penetrant and raises the possibility that PARP inhibition in combination with radiotherapy may be effective for the two-thirds of newly diagnosed glioblastoma patients who are MGMT-unmethylated and insensitive to the standard of care (temozolomide).”
Tania Small, Vice President, Head Global Medical Affairs, Oncology, GSK, said, “The promising data advance our understanding of the impact of niraparib in glioblastoma with unmethylated MGMT status, a very aggressive cancer with limited treatment improvements for decades. We are committed to the development of transformative cancer medicines and are pleased to work with the Ivy Brain Tumor Center to explore the potential role of niraparib for patients with newly diagnosed glioblastoma.”
Based on encouraging pharmacokinetic signals coupled with the pharmacodynamic and survival data from this study, GSK and the Ivy Center are exploring next steps for the clinical development of niraparib in this patient population.
Dr. Sanai will present findings from the Phase 0/2 study of niraparib at the Society for Neuro-Oncology (SNO) Annual Meeting today, Nov. 17, at 8:35 a.m. PST during an oral abstract presentation.
Expert Ivy Center clinicians and scientists will present five oral abstracts and five posters at the meeting. These presentations represent a broader portfolio of pharmacodynamic and pharmacokinetic-driven clinical trials, utilizing industry-supported drug development to test new-in-class therapies for brain tumor patients.
GSK’s drug, niraparib, brand-named Zejula, is an oral, once-daily PARP inhibitor currently being evaluated in multiple pivotal trials. GSK is building a robust clinical development program by assessing activity across multiple tumor types and evaluating several potential combinations of niraparib with other therapeutics. The ongoing development program includes several combination studies, including the FIRST phase 3 trial assessing niraparib in combination with dostarlimab, a programed death receptor-1 (PD-1)-blocking antibody, as a potential treatment for first-line ovarian cancer maintenance and the phase 3 ZEAL trial assessing niraparib in combination with standard of care for the maintenance treatment of first line advanced non-small cell lung cancer.
About Ivy Brain Tumor Center
Ivy Brain Tumor Center at Barrow Neurological Institute in Phoenix, AZ, is a tertiary care and nonprofit translational research program that employs bold, early-phase clinical trial strategies to identify new treatments for aggressive brain tumors, including glioblastoma. Our leading experts in neurosurgical oncology, neuro-oncology, radiation oncology, neuroradiology, neuropathology and neuroscience nursing treat more patients annually than any other brain tumor center in the United States. The Ivy Center’s Phase 0 clinical trials program is the largest in the world and enables personalized care in a fraction of the time and cost associated with traditional drug development. In addition, unlike conventional clinical trials focusing on single drugs, the Ivy Center’s accelerated program tests therapeutic combinations matched to individual patients. We leave no stone unturned in the pursuit of hope and healing. Learn more at IvyBrainTumorCenter.org. Follow the Ivy Brain Tumor Center on Facebook, Instagram, Twitter, and LinkedIn.