Actuate Therapeutics (NASDAQ:ACTU) reported successful Phase 1 results from its Actuate-1902 study on January 6, 2026.
The trial evaluated elraglusib, a first-in-class small molecule GSK-3β inhibitor, in 40 pediatric patients (ages 3–21) with relapsed or refractory solid tumors.
These patients were heavily pretreated, having failed a median of 4 prior therapies (ranging from 2 to 11).
The data demonstrated significant clinical benefit, particularly in Ewing sarcoma and neuroblastoma.
Out of 40 patients, 15 (37.5%) achieved clinical responses or disease control.
The results were especially robust in the combination arm where elraglusib was paired with cyclophosphamide and topotecan, yielding responses in 10 of 19 patients (52.6%).
Notable findings included two Complete Metabolic Responses (CMRs) in patients with metastatic Ewing sarcoma and one Complete Response (CR) in a patient with metastatic neuroblastoma.
Additionally, one patient experienced a 60% tumor reduction in a target lung lesion.
These "unexpected" responses in highly refractory settings have earned elraglusib Rare Pediatric Disease Designations from the FDA for both Ewing sarcoma and neuroblastoma, positioning the company for a potential Priority Review Voucher (PRV) upon future approval.