FDA Grants Orphan Drug Status to Actuate's Elraglusib for Soft Tissue Sarcomas

Actuate Therapeutics, Inc., a clinical-stage biopharmaceutical company, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for elraglusib, a pioneering GSK-3β inhibitor aimed at treating soft tissue sarcoma (STS). This designation highlights the potential of elraglusib to address significant unmet medical needs for patients with advanced cancers. Daniel Schmitt, President & CEO of Actuate, expressed satisfaction with the FDA's recognition, which underscores the drug's promising safety profile and antitumor efficacy observed in several solid tumors, including melanoma, Ewing sarcoma, colorectal, and pancreatic cancers.

The American Cancer Society projects that approximately 13,590 new cases of soft tissue sarcoma will be diagnosed in the United States in 2024, with an estimated 5,200 fatalities. Soft tissue sarcomas, although rare, comprise a diverse group of tumors with more than 70 histological subtypes, making them particularly challenging to treat. For localized cancer, surgery remains the most effective option, offering a median survival rate close to 50%. However, for patients with metastatic disease, median overall survival drops drastically to less than 6-12 months, posing a significant therapeutic challenge. Dr. Steven D. Reich, Senior VP of Clinical Development and Acting Chief Medical Officer of Actuate, emphasized the dire need for new treatments, noting that doxorubicin, the current standard systemic therapy, offers minimal antitumor activity despite its approval nearly 50 years ago. Preclinical studies have shown that elraglusib induces significant apoptosis in STS cells and exhibits synergistic effects with chemotherapy, providing a strong rationale for its clinical evaluation in metastatic STS.

The FDA’s Orphan Drug Designation is granted to investigational therapies designed to treat rare medical conditions affecting fewer than 200,000 people in the U.S. This status provides several benefits to drug developers, including assistance in the development process, tax credits for clinical costs, exemptions from certain FDA fees, and seven years of post-approval marketing exclusivity.

Actuate Therapeutics is dedicated to developing treatments for hard-to-treat cancers. The company’s lead investigational drug, elraglusib, targets molecular pathways in cancer that contribute to tumor growth and resistance to conventional cancer treatments, including chemotherapy. Elraglusib is designed to act as a mediator of anti-tumor immunity through the inhibition of NF-kB and regulates various immune checkpoints and immune cell functions.

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