FDA Grants Rare Pediatric Disease Designation to Actuate's Elraglusib for Ewing Sarcoma

CHICAGO and FORT WORTH, Texas, Nov. 12, 2024 — Actuate Therapeutics, Inc. (NASDAQ: ACTU) has announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to its drug, elraglusib, for the treatment of Ewing sarcoma (EWS). Actuate is a clinical-stage biopharmaceutical company developing therapies to combat difficult-to-treat cancers by inhibiting glycogen synthase kinase-3 beta (GSK-3β).

Daniel Schmitt, President & CEO of Actuate, emphasized the significance of this designation by the FDA, highlighting the urgent need for new treatment options for EWS patients. Schmitt noted that early clinical data from their Phase 1/2 trial have shown promising anti-tumor activity, including two ongoing durable Complete Responses (CRs) among the first six patients treated with relapsed/refractory EWS. Schmitt reaffirmed the company's commitment to advancing elraglusib's clinical development to provide new therapeutic options for this challenging disease.

Ewing sarcoma is a highly metastatic cancer that often begins in the bones and peaks in incidence at around 15 years of age. It is the second most common primary malignant tumor in children and adolescents. Approximately 25% of new EWS patients present with metastatic disease at diagnosis, significantly reducing survival rates. The ongoing Phase 1/2 trial, Actuate-1902 (NCT 04239092), is an open-label, multicenter study assessing the safety and efficacy of elraglusib in pediatric patients with relapsed/refractory malignancies, including EWS. The study has enrolled eight patients with relapsed/refractory EWS who have been treated with a combination of elraglusib and the chemotherapeutic agents topotecan and cyclophosphamide.

The FDA grants Rare Pediatric Disease Designation to serious or life-threatening diseases that affect fewer than 200,000 people in the U.S., primarily affecting individuals under 18 years of age. If a New Drug Application (NDA) for elraglusib is approved by the FDA, Actuate will be eligible to receive a Priority Review Voucher (PRV). This voucher can be used by the company or sold to another entity.

Actuate Therapeutics, Inc. is focused on developing innovative treatments for high-impact cancers that are difficult to treat. Their lead investigational drug, elraglusib, a novel GSK-3β inhibitor, targets molecular pathways that promote tumor growth and resistance to conventional cancer therapies. Elraglusib also acts as a mediator of anti-tumor immunity by inhibiting nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB), regulating multiple immune checkpoints, and modulating immune cell function.

Actuate’s commitment to advancing clinical development for challenging cancers remains steadfast, with the ultimate goal of providing effective new therapies where current treatments are inadequate.

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