Actuate Therapeutics Advances Clinical Program in Ewing Sarcoma After Positive Phase 1 Trial Demonstrates Complete and Partial Responses in Difficult-to-Treat Pediatric Sarcomas
Phase 1 study of elraglusib in pediatric patients with refractory Ewing sarcoma showed complete responses in two out of ten patients and durable stable disease in two patients, supporting advancement to a Phase 2 trial.
Phase 1 study of elraglusib (alone or with irinotecan/temozolomide, or cyclophosphamide/topotecan) in pediatric patients with refractory or relapsed malignancies, including Ewing sarcoma (EWS). Ten EWS patients were treated, with 2 patients achieving complete responses and 2 patients achieving durable stable disease; a partial response was observed in a patient with desmoplastic small‑round‑cell tumor (DSRCT). The results support progression to a Phase 2 trial of elraglusib in pediatric, adolescent, and adult patients with relapsed/refractory EWS.
The findings demonstrate significant antitumor activity of elraglusib in a disease with very limited treatment options and low survival rates, potentially offering a first‑in‑class targeted therapy for relapsed/refractory Ewing sarcoma.
Evidence level: Számítógépes vagy elméleti. Modellből vagy adatbányászatból származó jel.
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