Actuate Therapeutics Announces Complete Responses and Clinical Trial Update for Elraglusib in Relapsed/Refractory Ewing Sarcoma
Actuate Therapeutics, Inc., a clinical-stage biopharmaceutical firm listed on NASDAQ as ACTU, has provided a significant update on its Phase 1/2 clinical trial involving elraglusib in the treatment of relapsed/refractory Ewing Sarcoma (r/r EWS). Elraglusib, a novel glycogen synthase kinase-3 beta (GSK-3β) inhibitor, has shown encouraging results in early clinical settings.
Ewing sarcoma is a highly metastatic and aggressive cancer, primarily affecting children and adolescents. It is the second most common primary malignant tumor in this demographic. The disease's prognosis remains poor, especially in relapsed or refractory cases, due to the absence of standardized second-line treatments. Daniel Schmitt, the President and CEO of Actuate, emphasized the urgent need for new therapeutic strategies that combine innovative targeted therapies with existing chemotherapy regimens. He highlighted that elraglusib is one of the few novel treatments being explored for this patient population and continues to show promise.
The ongoing Phase 1/2 trial (NCT 04239092) is designed as an open-label, multicenter study. The trial assesses the safety and efficacy of elraglusib in pediatric patients with relapsed/refractory malignancies, including Ewing Sarcoma and related small round cell sarcomas. So far, the study has enrolled eight patients: six with relapsed/refractory EWS, one with a desmoplastic small round cell tumor (DSRCT), and one with a CIC rearrangement. All participants received a combination of elraglusib with cyclophosphamide/topotecan.
Among the enrolled EWS patients, two have achieved ongoing complete responses (CR), two have stable disease (SD), and two withdrew from the study before their tumor responses could be evaluated. Notably, the patient with DSRCT experienced a partial response (PR) with a 52% reduction in tumor size but eventually withdrew from the trial and is now being monitored for survival.
Dr. Andrew Mazar, Actuate’s Scientific Co-Founder and Chief Operating Officer, expressed optimism about the disease stabilization and ongoing complete responses observed in the trial. He pointed out that despite the small sample size, the results are promising, particularly given the significant unmet medical need in recurrent EWS. The findings support the continued development of elraglusib in this rare cancer patient population.
The study remains open exclusively to patients with recurrent EWS, with plans to enroll up to 12 EWS patients. This will provide further rationale for advancing the elraglusib/cyclophosphamide/topotecan combination into a Phase 2 study for recurrent EWS patients. Actuate intends to consult with the FDA to discuss the design of the Phase 2 study and explore options for accelerating the development of elraglusib towards commercial registration for EWS treatment.
Ewing Sarcoma is characterized by its highly metastatic nature, primarily affecting the bone, with peak incidence at around 15 years of age. Approximately 25% of new EWS patients present with metastatic disease at diagnosis, significantly predicting poor survival. Treatment options for relapsed/refractory EWS are limited, and despite intensive therapy, recurrent EWS is often fatal. While the combination of chemotherapy and surgery has improved 5-year survival rates, no standard treatment exists for relapsed/refractory cases.
Actuate Therapeutics is dedicated to developing therapies for difficult-to-treat cancers. Its lead investigational drug, elraglusib, targets molecular cancer pathways that promote tumor growth and resistance to conventional cancer drugs.
How to obtain the latest research advancements in the field of biopharmaceuticals?
In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!
