Actuate Therapeutics disclosed that the Phase 1 portion of its Actuate‑1902 study, which evaluates elraglusib alone or with standard chemotherapy, was completed in July 2025 and that early clinical responses were observed in a cohort of 40 pediatric patients with relapsed or refractory malignancies, including Ewing sarcoma.
The open‑label, multicenter trial enrolled children and adolescents aged 3 to 21 years. In the 40‑patient cohort, 15 patients achieved disease control, and 10 of the 19 patients treated with elraglusib plus cyclophosphamide and topotecan achieved positive responses. Notably, two patients with metastatic Ewing sarcoma achieved complete metabolic responses, and one patient with metastatic neuroblastoma achieved a complete response, underscoring the drug’s potential in high‑unmet‑need pediatric cancers.
Actuate’s leadership highlighted that these results validate the drug’s dual mechanism of action—initially developed as a glycogen synthase kinase‑3β inhibitor and now shown to also destabilize microtubules—providing a broader therapeutic rationale. The FDA has granted rare pediatric disease designations for both Ewing sarcoma and neuroblastoma, a regulatory milestone that could accelerate future approvals and partnership opportunities.
The company plans to advance elraglusib into Phase 2 trials for children, adolescents, and adults with relapsed or refractory Ewing sarcoma, with a launch of the Phase 2 program slated for 2026. Management noted that securing additional capital is essential to support the next development phase, citing the need for funding to sustain the accelerated timeline.
Actuate’s announcement was well received by the market, with analysts noting the significance of the complete responses and the regulatory endorsement. CEO Daniel Schmitt emphasized that “elraglusib continues to exhibit the potential to extend and improve the lives of patients in disease settings where specific treatment options are extremely limited,” while COO Dr. Andrew Mazar remarked that “the early complete responses in Ewing sarcoma are highly unexpected and position elraglusib as a potential first‑in‑class therapy in an indication with no approved targeted treatments.”
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