Nuvation Bio Reports Positive Phase 2 Data and Broad Clinical Expansion for Safusidenib in IDH1-Mutant Glioma

Nuvation Bio Inc. announced updated long-term follow-up data from its Phase 2 (J201) study of safusidenib in patients with chemotherapy- and radiotherapy-naïve grade 2 IDH1-mutant glioma, showing a confirmed objective response rate (ORR) of 51.9% and a 36-month progression-free survival (PFS) rate of 79.1% at a median follow-up of 38.8 months. The company also detailed a significant expansion of its safusidenib clinical development program, including the initiation of two new studies and the expansion of the existing SIGMA trial.

The updated results from the J201 study, conducted in 27 patients in Japan, continued to demonstrate durable efficacy with no new safety signals. Responses to safusidenib deepened over time, with only one previously responding patient experiencing subsequent disease progression. The centrally assessed ORR was based on Response Assessment in Neuro-Oncology (RANO) for low-grade gliomas criteria, and median PFS was not reached.

Nuvation Bio will initiate a pivotal Phase 3 study (G307; NCT07712757) evaluating safusidenib in approximately 140 patients with newly diagnosed grade 2 IDH1-mutant glioma who have not received chemotherapy or radiation. The randomized, placebo-controlled trial will be conducted at sites outside the U.S. in regions where the targeted therapy vorasidenib is not yet approved or accessible. The primary endpoint is PFS assessed by blinded independent central review (BICR).

A second new study, a Phase 2 trial (G209; NCT07703436), will enroll up to 40 patients in the U.S. with grade 2 or 3 IDH1-mutant glioma whose disease has progressed after prior treatment with vorasidenib. This study aims to establish proof-of-concept for safusidenib in the post-vorasidenib setting, with the primary endpoint being ORR by BICR.

Separately, the ongoing SIGMA trial (NCT05303519) is being expanded from a Phase 2 to a pivotal Phase 3 study. Originally designed to test safusidenib against placebo in 100 patients with high-grade IDH1-mutant astrocytoma, the trial will now enroll 300 patients with broader eligibility criteria. This includes patients with grade 2 and 3 IDH1-mutant astrocytoma with high-risk features, and grade 4 IDH1-mutant astrocytoma who have undergone standard treatments such as radiation. Enrollment is underway in the U.S., Australia, and China. Additionally, a new, third portion of the SIGMA trial will evaluate safusidenib in about 40 patients with grade 3 IDH1-mutant oligodendroglioma who have had surgery but not chemotherapy or radiation; the main goal is treatment response, with results expected in 2027.

“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 the Founder, President, and CEO of Nuvation Bio. The company noted that the SIGMA protocol updates reflect alignment with U.S. regulators to support the potential approval of safusidenib.