Abstract Reports Curtana's CT-179 Extends Survival as a Single Agent in a Patient-Derived Model of Diffuse Midline Glioma

AUSTIN, Texas - Amzeal -- Curtana Pharmaceuticals, a clinical-stage biotechnology company focused on improving outcomes for brain cancer patients, today announced that an abstract published in the online program of the 31st Annual Meeting of the Society for Neuro-Oncology (SNO), to be held November 12 to 15 in Philadelphia, reports that CT-179 significantly extended survival as a single agent in an orthotopic patient-derived xenograft model of H3 K27M-mutant diffuse midline glioma (DMG). CT-179 is a first-in-class, orally bioavailable, brain-penetrant small-molecule inhibitor of the transcription factor OLIG2.

DMG, which includes diffuse intrinsic pontine glioma (DIPG), is a lethal pediatric brain tumor with median overall survival under one year, and radiation remains the only standard of care. ONC201 (dordaviprone) received FDA accelerated approval in 2025 for H3 K27M-mutant DMG that has progressed after prior therapy, but acquired resistance and limited durability of benefit have created a need for combination strategies. OLIG2 is overexpressed in DMG and has been linked to proliferation, treatment resistance, and tumor growth.

Key findings reported in the abstract:

• Nanomolar activity across patient-derived lines. CT-179 reduced the viability of five patient-derived DMG spheroid lines, with IC50 values of 394 to 865 nM in the four H3 K27M-mutant lines and approximately 903 nM in an H3 wild-type line.
• Single-agent survival benefit in an orthotopic model. In mice bearing orthotopic HSJD-DIPG-007 xenografts, oral CT-179 extended median survival to 65 days, compared with 57.5 days for vehicle (P = 0.0148), with a modest imaging-based reduction in tumor growth. ONC201 alone (median 60.5 days) did not significantly extend survival in this model.
• Combination with imipridones. CT-179 combined with ONC201 or the related imipridone ONC206 produced additive effects in vitro by ZIP analysis, without synergy. In vivo, median survival with CT-179 plus ONC201 was 66.5 days, which was not significantly different from ONC201 alone (P = 0.13).

Analysis of tumor tissue for target engagement, DNA damage, and apoptosis is ongoing, and the investigators are pursuing dose optimization and additional combination regimens. Curtana co-founders Gregory Stein, M.D., and Santosh Kesari, M.D., Ph.D., are co-authors of the abstract. The in vitro studies were conducted in the laboratory of Javad Nazarian, Ph.D., at the University of Zurich.

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"Diffuse midline glioma is a priority indication for Curtana, and a significant single-agent survival benefit in an orthotopic H3 K27M model is an important step for the program," said Gregory Stein, M.D., M.B.A., Chief Executive Officer of Curtana Pharmaceuticals. "Single-agent activity is the foundation for any combination strategy in this disease. The tissue analyses now under way will tell us whether CT-179 is engaging OLIG2 in these tumors and will guide dose selection as we evaluate rational combinations, including radiation, the standard of care in DMG."

About the Abstract

Mizoguchi SF, Yu J, Yadavilli S, Stein G, Kesari S, Packer R, Nazarian J. In vitro and in vivo evaluation of CT-179 in combination with ONC201 and ONC206 in patient-derived models of diffuse midline glioma. Published in the online program of the 31st Annual Meeting of the Society for Neuro-Oncology, Philadelphia, PA, November 12 to 15, 2026. SNO Annual Meeting abstracts are published in a supplement to Neuro-Oncology.

About CT-179

CT-179 is a first-in-class, orally bioavailable, brain-penetrant small-molecule inhibitor of OLIG2, a master transcription factor in diffuse gliomas and OLIG2-positive medulloblastoma. CT-179 is designed to eliminate the cancer stem cells that drive recurrence, and published preclinical work shows that OLIG2 inhibition also relieves tumor-driven suppression of CXCL10 and sensitizes GBM to anti-PD-L1 therapy. The FDA has granted CT-179 Fast Track Designation, Orphan Drug Designation for gliomas, and Rare Pediatric Disease Designation for medulloblastoma and for pediatric high-grade glioma. Curtana's multi-site Phase 1 OPAL trial of CT-179 in glioblastoma will be conducted in Australia in partnership with the Cooperative Trials Group for Neuro-Oncology (COGNO).

About Curtana Pharmaceuticals

Curtana Pharmaceuticals, founded in 2013, is a privately held, clinical-stage biopharmaceutical company headquartered in Austin, Texas. Current investors include Thynk Capital, angelMD, Biosense Global, DEFTA Partners, the Musella Foundation, the Hasso Serrano Foundation, the Uncle Kory Foundation, and other anonymous investors. The company was also awarded a $7.6 million grant from the Cancer Prevention and Research Institute of Texas (CPRIT). Curtana focuses on the development of novel, first-in-class small-molecule therapeutics targeting cancer stem cells in the central nervous system for the treatment of glioblastoma, low-grade glioma, medulloblastoma, diffuse midline glioma, and other brain cancers. For more information, visit https://www.curtanapharma.com/.

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Forward-Looking Statements

This press release contains forward-looking statements regarding the preclinical and clinical development of CT-179, including its potential in diffuse midline glioma and in combination with radiotherapy and other agents, and the design and conduct of the multi-site Phase 1 OPAL study in glioblastoma. Results in preclinical models may not be predictive of results in patients, and data presented in abstract form are preliminary and subject to change. These forward-looking statements involve risks and uncertainties, including the complexities of clinical trial execution and patient accrual and the need to secure additional capital to fund operations and clinical milestones. Curtana undertakes no obligation to update these statements.