Actuate Therapeutics announces end of Phase 1 portion of elraglusib study

Actuate Therapeutics (ACTU) announced the end of the Phase 1 portion of its clinical study evaluating elraglusib monotherapy or in combination with irinotecan, irinotecan plus temozolomide, or with cyclophosphamide plus topotecan in pediatric patients with refractory malignancies. Following encouraging signals of activity, particularly in treatment-refractory Ewing Sarcoma, a small round cell sarcoma that forms in soft tissue and bone, the Company will seek to advance the clinical development program towards a Phase 2 study in children, adolescents, and adults with relapsed/refractory EWS. The Phase 1/2 Trial, also referred to as Actuate-1902, was an open-label, multicenter study evaluating the safety and efficacy of elraglusib in pediatric patients with relapsed/refractory malignancies, including EWS and other pediatric sarcomas. To date, the study has enrolled ten patients with relapsed/refractory EWS treated with the combination of elraglusib and topotecan/cyclophosphamide. Of the ten EWS patients enrolled, two patients achieved complete responses by CT and/or complete metabolic responses, while two had confirmed stable disease. A partial response was also observed in one patient with a desmoplastic small-round-cell tumor, a small round cell sarcoma that forms in soft tissue. While the sample size specific to Ewing Sarcoma in the 1902 study was small, the responses are viewed as positive evidence of anti-tumor activity in this difficult-to-treat indication, and Actuate plans to further investigate elraglusib’s ability to positively change patients’ potential for successful treatment in a Phase 2 trial in pediatric, adolescent, and adult patients with relapsed/refractory Ewing Sarcoma. The Company is collaborating with key opinion leaders and consortiums with interest in EWS to ensure the design of the upcoming study aligns with both patient needs and regulatory expectations. The Company expects to initiate the trial in 2026, subject to available funding.