Updated data from Phase 2 study show confirmed objective response rate of 52% and 36-month progression-free survival rate of 79% with nearly 40 months of follow-up

New Phase 3 study to evaluate safusidenib in newly diagnosed grade 2 glioma patients outside the U.S.

New Phase 2 study to evaluate safusidenib in patients whose glioma has progressed following treatment with vorasidenib in U.S.

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.