Actuate Announces Results From Phase 1 Portion Of Phase 1/2 Clinical Study Evaluating Elraglusib As Monotherapy Or In Combination With Irinotecan, Irinotecan Plus Temozolomide, Or With Cyclophosphamide Plus Topotecan In Pediatric Patients With Refractory Malignancies

• Two Complete Metabolic Responses (CMRs) observed in patients with relapsed/refractory metastatic Ewing sarcoma and one Complete Response (CR) observed in a patient with relapsed/refractory metastatic neuroblastoma
• Clinical responses and disease control observed in 15 of 40 patients with difficult-to-treat refractory pediatric cancers, including 10 of 19 patients treated with elraglusib plus cyclophosphamide/topotecan
• Data support advancing clinical development of elraglusib in Ewing sarcoma and potentially neuroblastoma in 2026; Company has been granted Rare Pediatric Designations from the FDA for both indications

CHICAGO and FORT WORTH, Texas, Jan. 06, 2026 (GLOBE NEWSWIRE) -- Actuate Therapeutics, Inc. (NASDAQ:ACTU) ("Actuate" or the "Company"), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), today announced results from the phase 1 portion of its phase 1/2 clinical study evaluating elraglusib as a monotherapy or in combination with irinotecan, irinotecan plus temozolomide, or with cyclophosphamide plus topotecan in pediatric patients with refractory malignancies (Actuate-1902; NCT 04239092).

The Actuate-1902 trial was an open-label, multicenter phase 1/2 study evaluating the safety and efficacy of elraglusib in 40 pediatric patients ages 3 – 21 with relapsed (>2 remissions)/refractory cancers, including Ewing sarcoma (EWS), neuroblastoma, Central Nervous System (CNS) tumors, non-EWS sarcomas, and other refractory pediatric malignancies.

Importantly, data from the Phase 1 trial showed clinical responses in relapsed/refractory EWS, which is viewed as positive evidence of clinical activity in this difficult-to-treat indication. Following initial responses observed in the Actuate-1902 clinical study, the Company plans to advance clinical development of elraglusib in children, adolescents, and adults with relapsed/refractory EWS, while assessing collaborative development programs in neuroblastoma and other pediatric indications with leading pediatric consortia and Key Opinion Leaders to align future studies with patient needs and regulatory expectations.