Actuate Wins FDA Rare Pediatric Disease Designation for Ewing Sarcoma Treatment
Actuate Therapeutics, Inc. (NASDAQ: ACTU) has received FDA rare pediatric disease designation for elraglusib, a GSK-3β inhibitor, for treating Ewing sarcoma (EWS). EWS is a highly metastatic bone sarcoma, most prevalent in children and adolescents, with limited effective treatments.
The company’s ongoing Phase 1/2 trial of elraglusib in relapsed/refractory EWS is actively enrolling, with topline Phase 1 data expected in the second half of 2025. Early data has shown promising anti-tumor activity. The FDA designation allows eligibility for a Priority Review Voucher (PRV) upon approval, which can expedite or be monetized by the company for regulatory processes.
Daniel Schmitt, CEO of Actuate commented, “Early clinical data from our ongoing Phase 1/2 trial show promising anti-tumor activity with objective tumor responses, including two ongoing durable Complete Responses (CRs) in the first six patients treated with relapsed/refractory EWS, reinforcing our confidence in elraglusib’s potential impact in this challenging disease setting. We are committed to advancing elraglusib’s clinical development with the ultimate goal of providing new therapeutic options where current approaches are unsatisfactory.”
Recently, Actuate announced its participation in Lantern’s Pharma webinar on AI-driven drug development to discuss how Lantern’s AI platform is being leveraged for the identification and development of enrichment biomarkers for elraglusib.
