CHICAGO, Ill. and FORT WORTH, Texas — Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), today 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 (Actuate-1902). Following encouraging signals of activity, particularly in treatment-refractory Ewing Sarcoma (EWS), 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.
“Refractory and resistant EWS is one of the most aggressive and underserved cancers affecting children and young adults with very low survival rates,” said Dr. Andrew Mazar, Actuate’s Scientific Co-founder and Chief Operating Officer. “To observe complete responses in this setting is highly unexpected, and these exceptional early results position elraglusib as a potential first-in-class therapy option in an indication where no approved targeted therapies currently exist.”
The Phase 1/2 Trial (NCT 04239092), 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 (10) patients with relapsed/refractory EWS (>1 remission) treated with the combination of elraglusib and topotecan/cyclophosphamide.
“We are highly encouraged by the responses observed in refractory and resistant EWS, a disease where treatment options are extremely limited beyond frontline chemotherapy,” said Daniel Schmitt, President & Chief Executive Officer of Actuate. “With these promising clinical signals in hand, we are advancing our development plans focused on EWS by pursuing a Phase 2 trial and plan to meet with regulators to discuss potential registration path in this indication.”
Of the ten (10) EWS patients enrolled, two (2) patients achieved complete responses by CT and/or complete metabolic responses (CR/CMR), while two (2) had confirmed stable disease. A partial response (PR) was also observed in one patient with a desmoplastic small-round-cell tumor (DSRCT), 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.
About Ewing Sarcoma
Ewing sarcoma (EWS) is a highly metastatic form of sarcoma with a peak incidence at the age of 15, that ranks as the second most prevalent primary malignant tumor of childhood and adolescence. Approximately 25% of new EWS patients have metastatic disease when first diagnosed, which is the most significant predictor of poor survival.
About Actuate Therapeutics, Inc.
Actuate is a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers. Actuate’s lead investigational drug, elraglusib (a novel GSK-3β inhibitor), targets molecular pathways in cancer that are involved in promoting tumor growth and resistance to conventional cancer drugs such as chemotherapy through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and DNA Damage Response (DDR). Elraglusib may also mediate anti-tumor immunity through the regulation of multiple immune checkpoints and immune cell function. For additional information, please visit the Company’s website at http://www.actuatetherapeutics.com.
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