A window-of-opportunity trial reveals mechanisms of response and resistance to navtemadlin in patients with recurrent glioblastoma
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Inhibitors of murine double minute homolog 2 (MDM2) represent a promising therapeutic approach for the treatment of TP53 wild-type glioblastomas (GBMs), reactivating p53 signaling to induce cancer cell death. We conducted a surgical window-of-opportunity trial (NCT03107780) of the MDM2 inhibitor navtemadlin (KRT-232) in 21 patients with TP53 wild-type recurrent GBM to determine achievable drug concentrations within tumor tissues and biological mechanisms of response and resistance. Participants received navtemadlin at 120 mg ( n = 10) or 240 mg ( n = 11) for 2 days before surgical resection and after surgery until progression or unacceptable toxicity. Both 120 and 240 mg daily dosing achieved a pharmacodynamic impact, but median progression-free survival was 3.1 months. DNA sequencing of three recurrent tumors revealed an absence of TP53 -inactivating mutations, indicating alternative mechanisms of resistance. To understand the mechanisms of response and resistance associated with navtemadlin, we conducted functional and spatial analyses of human tissue and patient-derived GBM neurosphere models. Navtemadlin induced partial tumor cell death as monotherapy, and combination with temozolomide enhanced apoptosis in GBM neurospheres while sparing normal bone marrow cells in vitro. We also observed up-regulation of oligodendrocyte differentiation genes with navtemadlin treatment and enrichment of oligodendrocyte transcription factor 2 (OLIG2)–positive cells at relapse, suggesting an unexplored mechanism of navtemadlin tolerance in GBM. Overall, these results indicated that clinically achievable doses of navtemadlin exert pharmacodynamic effects on GBM and suggest that combined treatment with temozolomide may be a route to more durable survival benefits.
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In GBM, the navtemadlin phase 0 study completed enrollment, and total intra-tumoral drug concentrations at the 240 mg dose level exceeded the predefined target of >25 nmol/L, which was defined based on the IC50 in cell culture.
“In GBM, the navtemadlin phase 0 study completed enrollment, and total intra-tumoral drug concentrations at the 240 mg dose level exceeded the predefined target of >25 nmol/L, which was defined based on the IC50 in cell culture (16).”
From Preclinical Modeling of Navtemadlin Pharmacokinetics, Pharmacodynamics, and Efficacy in IDH–Wild-type Glioblastoma (2025) · cites this paper as [16]
The Results (Trial outcomes) describe the phase 0 cohort and report 21 patients enrolled. The Results (Pharmacokinetic and pharmacodynamic analysis) report that all 10 assessed patients in the 240 mg cohort had contrast-enhancing tumor concentrations ≥25 nM. However, the Introduction says the threshold was based on the lowest concentration showing benefit with radiation in preclinical in vitro models, not on a cell-culture IC50; the paper also reports ≥25 nM rather than strictly >25 nM for every patient. (LLM, conf=0.88)
Experimental — accuracy not yet measuredThe phase 0 component of the navtemadlin GBM trial met the prespecified target for measured intra-tumoral drug levels with evidence of p53 pathway modulation in tumor tissue.
“the phase 0 component met the prespecified target for measured intra-tumoral drug levels with evidence of p53 pathway modulation in tumor tissue (16).”
From Preclinical Modeling of Navtemadlin Pharmacokinetics, Pharmacodynamics, and Efficacy in IDH–Wild-type Glioblastoma (2025) · cites this paper as [16]
Although both 120 and 240 mg navtemadlin achieved PD impact in patient tumors, progression-free and overall survival were similar to historical controls.
“Although both 120 and 240 mg navtemadlin achieved PD impact in patient tumors, progression-free and overall survival were similar to historical controls.”
From Preclinical Modeling of Navtemadlin Pharmacokinetics, Pharmacodynamics, and Efficacy in IDH–Wild-type Glioblastoma (2025) · cites this paper as [16]
A disconnect between PD and patient survival was also observed in the human phase 0 trial.
“Notably, a disconnect between PD and patient survival was also observed in the human phase 0 trial (16).”
From Preclinical Modeling of Navtemadlin Pharmacokinetics, Pharmacodynamics, and Efficacy in IDH–Wild-type Glioblastoma (2025) · cites this paper as [16]
Navtemadlin has completed a phase 0 window-of-opportunity study in glioblastoma (NCT03107780).
“Navtemadlin is a potent small-molecule inhibitor of MDM2, which has completed a phase 0 window-of-opportunity study in glioblastoma (GBM).”
From Preclinical Modeling of Navtemadlin Pharmacokinetics, Pharmacodynamics, and Efficacy in IDH–Wild-type Glioblastoma (2025) · cites this paper as [16]
Patient data from the navtemadlin phase 0 clinical trial have been previously reported.
“Patient data from the navtemadlin phase 0 clinical trial have been previously reported;”
From Preclinical Modeling of Navtemadlin Pharmacokinetics, Pharmacodynamics, and Efficacy in IDH–Wild-type Glioblastoma (2025) · cites this paper as [16]
Two dose levels were tested (120 and 240 mg) and drug levels were measured in tumor tissues sampled from CE and NCE regions.
“Two dose levels were tested (120 and 240 mg) and drug levels were measured in tumor tissues sampled from CE and NCE regions.”
From Preclinical Modeling of Navtemadlin Pharmacokinetics, Pharmacodynamics, and Efficacy in IDH–Wild-type Glioblastoma (2025) · cites this paper as [16]
The GBM phase 0 study (NCT03107780) evaluated tumor tissue levels in patients with GBM who received two doses of navtemadlin, with resection 3 to 6 hours later.
“The GBM phase 0 study (NCT03107780) evaluated tumor tissue levels in patients with GBM who received two doses of navtemadlin, with resection 3 to 6 hours later (16).”
From Preclinical Modeling of Navtemadlin Pharmacokinetics, Pharmacodynamics, and Efficacy in IDH–Wild-type Glioblastoma (2025) · cites this paper as [16]
MDM2 inhibitors can enhance response to both radiation therapy and temozolomide, the standard of care for GBM.
“MDM2 inhibitors can enhance response to both radiation therapy (RT) and temozolomide,7-9 the standard of care for GBM.”
From Pharmacokinetic-pharmacodynamic-efficacy modeling of the MDM2 inhibitor brigimadlin in glioblastoma patient-derived xenografts (2026) · cites this paper as [9]
In the navtemadlin phase 0/1 clinical study, PD impact was observed, but progression-free survival was similar to historical controls.
“Notably, in the navtemadlin phase 0/1 clinical study, PD impact was observed, but progression-free survival was similar to historical controls.9”
From Pharmacokinetic-pharmacodynamic-efficacy modeling of the MDM2 inhibitor brigimadlin in glioblastoma patient-derived xenografts (2026) · cites this paper as [9]
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- Authors
- Rendo, V., Lee, E. Q., Bossi, C., Khuu, N., Rudek, M. A., Pal, S., Azazmeh, N., Rashid, R., Lin, J.-R., Cusick, M., Reynolds, A. R. N., Fassinou, A. C. R., Ayoub, G., Malinowski, S., Lapinskas, E., Pisano, W., Jeang, J., Stopka, S. A., Regan, M. S., Spetz, J., Desai, A., Lieberman, F., Palanichamy, K., Fisher, J. D., Pelton, K., Huang, R. Y., Sarosiek, K. A., Nabors, L. B., Holdhoff, M., Danda, N., Strowd, R., Desideri, S., Walbert, T., Ye, X., Chakravarti, A., Sorger, P. K., Santagata, S., Agar, N. Y. R., Grossman, S. A., Alexander, B. M., Wen, P. Y., Ligon, K. L., & Beroukhim, R.
- Affiliations
- Department of Medical Oncology and Center for Neuro-Oncology, Dana-Farber Cancer Institute, Boston, MA 02215, USA.
- Brigham and Women's Hospital (US)
- Dana-Farber Cancer Institute (US)
- Broad Institute (US)
- Johns Hopkins University (US)
- Harvard University (US)
- University of Gothenburg (SE)
- University of Pennsylvania (US)
- University of Pittsburgh (US)
- Wake Forest University Health Sciences (US)
- Department of Neurology, University of Alabama at Birmingham, Birmingham, AL 35233, USA.
- Montefiore Medical Center (US)
- Department of Neurology, Wake Forest University, Winston-Salem, NC 27157, USA.
- Henry Ford Health (US)
- The Ohio State University (US)
- Corresponding author
- Lee, Eudocia Q.
- Rudek, Michelle A.
- Wen, Patrick Y.
- Ligon, Keith L.
- Beroukhim, Rameen
- ORCID
- Rendo, Veronica
- Lee, Eudocia Q.
- Bossi, Connor
- Khuu, Nicholas
- Rudek, Michelle A.
- Pal, Sangita
- Azazmeh, Narmen
- Rashid, Rumana
- Lin, Jia-Ren
- Reynolds, Abigail R. N.
- Fassinou, Auriole C. R.
- Ayoub, Georges
- Malinowski, Seth
- Lapinskas, Emily
- Pisano, William
- Stopka, Sylwia A.
- Regan, Michael S.
- Spetz, Johan
- Desai, Arati
- Lieberman, Frank
- Palanichamy, Kamalakannan
- Fisher, Joy D.
- Pelton, Kristine
- Huang, Raymond Y.
- Sarosiek, Kristopher A.
- Nabors, Louis B.
- Holdhoff, Matthias
- Danda, Neeraja
- Strowd, Roy
- Desideri, Serena
- Walbert, Tobias
- Ye, Xiaobu
- Chakravarti, Arnab
- Sorger, Peter K.
- Santagata, Sandro
- Agar, Nathalie Y. R.
- Grossman, Stuart A.
- Alexander, Brian M.
- Wen, Patrick Y.
- Ligon, Keith L.
- Beroukhim, Rameen
- Venue
- Science Translational Medicine
- Published
- 2025-02-19
- Volume
- 17
- Issue
- 786
- Pages
- eadn6274-eadn6274
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- PMC
- PMC11994224
- OpenAlex
- W4407736502
- Cited by
- 19
- References
- 47
- Citations by year
- 2026: 11 · 2025: 8
- FWCI
- 12.21
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institutes of Health (UL1TR003098)
- National Cancer Institute (R01CA219943, UM1CA137443, U54CA210180, R01CA262462, R50CA274277, R01CA188228, P30CA006973, U19CA264504)
- National Institute of Biomedical Imaging and Bioengineering (P41EB028741)
- U.S. National Library of Medicine (T15LM007059)
- National Center for Research Resources (S10RR026824)
- National Center for Advancing Translational Sciences
- Topics
- Health Sciences › Medicine › Genetics › Glioma Diagnosis and Treatment
- Health Sciences › Medicine › Oncology › Cancer-related Molecular Pathways
- Health Sciences › Medicine › Pathology and Forensic Medicine › Cancer Mechanisms and Therapy
- Keywords
- recurrent glioblastoma, MDM2 inhibitors, wild-type p53, p53 reactivation, drug resistance mechanisms, temozolomide combination therapy, cancer cell death, oligodendrocyte differentiation, Olig2, pharmacodynamics, tumor tissue drug concentration, progression-free survival
- MeSH terms
- Adult, Aged, Brain Neoplasms, Female, Glioblastoma, Humans, Male, Middle Aged, Neoplasm Recurrence, Local, Tumor Suppressor Protein p53, Drug Resistance, Neoplasm, Cell Line, Tumor, Proto-Oncogene Proteins c-mdm2
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