On July 20, 2026 Nuvation Bio Inc. (NYSE: NUVB), a global oncology company focused on tackling some of the toughest challenges in cancer treatment, reported updated positive long-term follow-up data from the Phase 2 (J201) study of safusidenib in patients with chemotherapy- and radiotherapy-naïve grade 2 IDH1-mutant glioma. With an additional year of follow-up, responses to safusidenib increased and further deepened, and its safety profile remained consistent and manageable.
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Nuvation Bio also announced a significant expansion of the clinical development program for safusidenib, its selective investigational inhibitor of mutant IDH1, supported by the updated long-term follow-up data from the Phase 2 (J201) study. The company will initiate two new studies to evaluate safusidenib across the broader landscape of IDH1-mutant glioma: a pivotal Phase 3 study in patients with grade 2 IDH1-mutant glioma outside the U.S. (G307; NCT07712757) and a Phase 2 study in patients with IDH1-mutant glioma that has progressed after prior treatment with vorasidenib in the U.S. (G209; NCT07703436).
"We continue to be very encouraged by the longer-term data from our Phase 2 J201 study, and today’s announcement marks a pivotal step forward in our mission to bring safusidenib as a comprehensive treatment option for patients with all types of IDH1-mutant glioma," said David Hung, M.D., Founder, President, and Chief Executive Officer of Nuvation Bio. "These two new studies are designed to evaluate safusidenib across a broader range of patients with IDH1-mutant glioma, with the ultimate goal of providing an effective therapy for nearly every patient with this disease."
Updated long-term data highlights durable efficacy
The updated results from the Phase 2 (J201) study, from 27 patients in Japan, further support this clinical expansion. Highlights of the findings, at a median of 38.8 months of follow-up, include the following:
The centrally assessed confirmed overall response rate (ORR), per Response Assessment in Neuro-Oncology (RANO) for low grade gliomas (LGG) criteria, was 51.9%.
Median progression-free survival (PFS) was not reached, and the 36-month PFS rate was 79.1%.
Responses were durable, with only one patient who had previously responded experiencing subsequent disease progression.
No new safety signals were identified.
Expanding the safusidenib clinical development program
The two new studies are designed to broaden the potential number of patients with IDH1-mutant glioma who could benefit from safusidenib:
G307: A Phase 3, randomized, placebo-controlled study that will enroll approximately 140 patients with newly diagnosed grade 2 IDH1-mutant glioma who have not yet received chemotherapy or radiation. The study will be conducted at sites outside the U.S. in regions where vorasidenib is not yet approved or accessible, providing this clinical trial as a critical option for patients in need in these regions. The primary endpoint is PFS as assessed by blinded independent central review (BICR). Secondary endpoints include ORR, time to next intervention, duration of response, and time to response.
G209: A Phase 2, multicenter study that will enroll up to 40 patients in the U.S. with grade 2 or 3 IDH1-mutant glioma who have experienced disease progression after treatment with vorasidenib and who remain in need of another option to delay radiation or chemotherapy. This study seeks to establish proof-of-concept for safusidenib in a setting of high unmet need. The primary endpoint is ORR by BICR, with a number of secondary endpoints, including tumor growth rate (TGR), an emerging way of assessing early anti-tumor activity.
"While the introduction of targeted therapies has transformed the treatment landscape for IDH1-mutant glioma, a critical question remains regarding sequencing of treatments once a patient progresses on a first-line inhibitor," said Macarena de la Fuente, M.D., Chief of the Neuro-Oncology Division and Co-Director of Clinical Neuro-Oncology for the Brain Tumor Institute at Sylvester Comprehensive Cancer Center, part of the University of Miami Miller School of Medicine. "The G209 study is a vital step in addressing this clinical gap by evaluating the potential role of safusidenib in patients who have progressed on prior targeted therapy."
These studies build upon Nuvation Bio’s safusidenib clinical development program, which includes the currently enrolling pivotal Phase 3 SIGMA study, evaluating safusidenib compared to placebo as a maintenance therapy after standard-of-care in IDH1-mutant astrocytoma with high-risk features, and a separate, exploratory, non-pivotal cohort evaluating safusidenib in participants with grade 3 IDH1-mutant oligodendroglioma who have not yet received chemotherapy or radiotherapy.
"For patients living with an IDH1-mutant glioma, a historically under-studied disease, questions about what to do when a first-line treatment stops working are a major source of anxiety," said Kelly Sitkin, President and Chief Executive Officer of the American Brain Tumor Association. "We are encouraged to see a clinical program of this scale that not only includes a post-vorasidenib trial but also looks comprehensively across different stages of the disease. Broad, expansive evaluation like this is among the most comprehensive programs our community has seen and represents an important step forward in addressing significant clinical gaps."
About IDH1-mutant Glioma
Gliomas are the most common type of brain cancer in adults worldwide. In the U.S., nearly 2,500 people are diagnosed with IDH-mutant gliomas each year, of which more than 95% harbor a mutation in the IDH1 gene. 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-risk features, including high grade tumors.
About Safusidenib
Safusidenib is an investigational, oral, brain-penetrant, selective inhibitor of mutant IDH1. It is being studied in patient populations with significant unmet medical need, including settings where there are limited or no approved targeted treatment options. In Phase 1 and Phase 2 clinical studies, safusidenib demonstrated encouraging clinical activity, including delayed disease progression and durable responses across a range of tumor grades and risk groups, with a favorable risk-benefit profile. These early findings support further investigation of safusidenib in the currently enrolling Phase 3 SIGMA study, as well as in the Phase 3 G307 study outside the U.S. where vorasidenib is not yet approved or accessible and the Phase 2 G209 study in a post-vorasidenib setting.
About the SIGMA (G203) Study
SIGMA is a pivotal Phase 3 study that will evaluate safusidenib compared to placebo as a maintenance therapy after standard-of-care in IDH1-mutant astrocytoma with high-risk features. The pivotal portion of the study will enroll approximately 300 patients.
A separate, exploratory, non-pivotal cohort will evaluate safusidenib in participants with grade 3 IDH1-mutant oligodendroglioma who have not yet received chemotherapy or radiotherapy. The primary endpoint is objective response rate. This cohort is expected to enroll approximately 40 patients.
(Press release, Nuvation Bio, JUL 20, 2026, View Source [SID1234669327])