Nuvation Bio Announces Publication of Positive Phase 2 Study Results for Safusidenib for the Treatment of Grade 2 IDH1-Mutant Glioma in Neuro-Oncology
Safusidenib demonstrated durable responses, with an ORR of 44% and 88% of patients were progression-free at 24 months
Findings supported recent favorable interactions with FDA where alignment was reached on modifying the ongoing G203 into a pivotal trial in high-grade gliomas
Nuvation Bio Inc. (NYSE:NUVB), a global oncology company focused on tackling some of the toughest challenges in cancer treatment, today announced the publication of positive results from a Phase 2 study of safusidenib, a novel, oral, potent, brain-penetrant targeted inhibitor of mutant IDH1, in patients with chemotherapy- and radiotherapy-naïve grade 2 IDH1-mutant gliomas. The findings were first published online on November 8 in the journal of Neuro-Oncology.
"Until recently, standard therapy for most IDH1-mutant gliomas was surgical resection followed by radiotherapy and chemotherapy, with only a subset of grade 2 patients undergoing active observation and delaying adjuvant chemoradiation and associated long-term toxicities," said David Reardon, M.D., Clinical Director of the Center for Neuro-Oncology at Dana-Farber Cancer Institute and a primary investigator for G203, the enrolling pivotal study of safusidenib in high-grade IDH1-mutant gliomas. "While approved IDH-inhibitors can now also be used to delay chemoradiation, the durability of benefit remains inadequate for many patients, leaving a significant unmet need for providers and patients. The high rates and durability of responses seen in this trial suggest the potential for sustained disease control with safusidenib and support its further evaluation in IDH1-mutant gliomas."
As reported in the new publication, the open-label, multicenter, single-arm study evaluated 27 patients with IDH1-mutant grade 2 gliomas who had no prior anticancer therapy except for resection or biopsy. The study met its primary endpoint, demonstrating an objective response rate (ORR) of 44.4%; median duration of response could not be estimated because no progression events had occurred. As of the data cut-off (March 10, 2023), median progression-free survival (PFS) was not yet reached, with a median follow-up time of 28 months, demonstrating the long-term potential of safusidenib to delay disease progression. At 24 months, 87.9% of patients were progression free.
Adverse events were mostly mild to moderate and manageable. Grade 3 or greater treatment-related adverse events occurred in five (18.5%) patients. No grade 5 events were reported. Three (11.1%) patients had treatment-emergent adverse events that led to study treatment discontinuation, two of which were considered related to safusidenib by study investigators and were resolved with dose interruption and/or appropriate medical management.
"The results published in the journal of Neuro-Oncology demonstrate safusidenib's impressive response rate and prolonged progression-free survival that suggest the possibility to delay chemoradiation, potentially improving quality of life for patients," said David Hung, M.D., Founder, President and Chief Executive Officer of Nuvation Bio. "While the published results reflect the March 10, 2023 data cut-off, as of September 15, 2025, 12 patients remained on treatment with safusidenib, further supporting the durability of responses. We look forward to sharing longer-term follow-up data in the future."
It should be noted that a Good Clinical Practice (GCP) noncompliance issue regarding the collection of adverse events was identified during the study. Therefore, all safety data presented in this manuscript was based on a subsequent re-investigation and re-collection of adverse events performed in strict accordance with the study protocol to ensure data integrity.
Nuvation Bio is progressing the ongoing G203 study in high-grade IDH1-mutant gliomas, with a protocol amendment on track to finalize the study as a global Phase 3 to support potential regulatory approvals. The primary endpoint is PFS as assessed by Blinded Independent Central Review per Response Assessment in Neuro-Oncology 2.0, which the FDA agreed could support full approval in this setting. Secondary endpoints include overall survival, PFS as assessed by the investigator, ORR, and duration of response. Nuvation Bio is continuing to evaluate enrollment of grade 2 patients with high-risk features in G203, as these patients currently have very limited options.
A trial in progress poster on G203 was presented at the 30th Annual Meeting of the Society for Neuro-Oncology on Friday, November 21 (abstract #CTNI19).
About IDH1-Mutant Glioma
Gliomas are the most common type of brain cancer in adults worldwide. In the U.S., nearly 2,400 people are diagnosed with IDH1-mutant gliomas each year. Most patients are diagnosed in their 30s and 40s. While patients with IDH1 mutations generally have longer survival times than those with wild-type IDH1, gliomas are not currently curable and prognosis worsens for those with high grade tumors.
About Safusidenib
Safusidenib is a novel, oral, potent, brain-penetrant, targeted inhibitor of mutant IDH1. In a Phase 1 clinical study, safusidenib was well-tolerated and demonstrated anti-tumor activity and high blood-brain barrier penetration.
About Nuvation Bio
Nuvation Bio is a global oncology company focused on tackling some of the toughest challenges in cancer treatment with the goal of developing therapies that create a profound, positive impact on patients' lives. Our diverse pipeline includes taletrectinib (IBTROZI™), a next-generation ROS1 inhibitor; safusidenib, a brain-penetrant IDH1 inhibitor; NUV-868, a BD2-selective BET inhibitor; and an innovative drug-drug conjugate (DDC) program.
Nuvation Bio was founded in 2018 by biopharma industry veteran David Hung, M.D., who previously founded Medivation, Inc., which brought to patients one of the world's leading prostate cancer medicines. Nuvation Bio has offices in New York, San Francisco, Boston, and Shanghai. For more information, visit www.nuvationbio.com or follow the company on LinkedIn and X (@nuvationbioinc).
Forward-Looking Statements
Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as "believe," "may," "will," "estimate," "continue," "anticipate," "intend," "expect," "should," "would," "plan," "predict," "potential," "seem," "seek," "future," "outlook," and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding the need for new therapeutic options in glioma and the therapeutic potential of safusidenib, our plans for G203 study protocol amendment and our expectations that the amended G203 study may support approval of safusidenib for the maintenance treatment of high-grade IDH1-mutant glioma, our plan to evaluate safusidenib for grade 2 IDH1-mutant gliomas with high-risk features, and our plans for future data updates. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of the management team of Nuvation Bio and are not predictions of actual performance. These forward-looking statements are subject to a number of risks and uncertainties that may cause actual results to differ from those anticipated by the forward-looking statements, including but not limited to whether Nuvation Bio is successful in commercializing IBTROZI; the challenges associated with conducting drug discovery and initiating or conducting clinical studies due to, among other things, difficulties or delays in the regulatory process, enrolling subjects or manufacturing or acquiring necessary products; the emergence or worsening of adverse events or other undesirable side effects; risks associated with preliminary and interim data, which may not be representative of more mature data; whether Nuvation Bio meets its post-marketing requirements and commitments for IBTROZI; and competitive developments. Risks and uncertainties facing Nuvation Bio are described more fully in its Form 10-Q filed with the SEC on November 3, 2025, under the heading "Risk Factors," and other documents that Nuvation Bio has filed or will file with the SEC. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date of this press release. Nuvation Bio disclaims any obligation or undertaking to update, supplement, or revise any forward-looking statements contained in this press release.
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