CHICAGO and FORT WORTH, Texas - Actuate Therapeutics, Inc. (NASDAQ: ACTU), a biopharmaceutical company with a market capitalization of $161.72 million, announced today that the European Medicines Agency (EMA) has awarded Orphan Medicinal Product Designation (OMPD) to its drug, elraglusib, for the treatment of pancreatic ductal adenocarcinoma (PDAC). According to InvestingPro data, the company maintains a strong cash position relative to debt, positioning it well for drug development initiatives. This designation is a key regulatory milestone that could expedite the drug's development and potential market exclusivity in the European Union.
Elraglusib is a novel inhibitor targeting glycogen synthase kinase-3 beta (GSK-3β), a molecular pathway associated with tumor growth and resistance to conventional chemotherapy. Currently, elraglusib is being evaluated in a randomized Phase 2 trial, comparing its effectiveness in combination with standard chemotherapy to chemotherapy alone in patients with metastatic PDAC.
The trial has shown promising interim results, including statistically significant increases in the 1-year survival rate and median overall survival when elraglusib is added to the treatment regimen. Daniel Schmitt, President & CEO of Actuate, expressed optimism about the drug's potential and the company's plans to report topline data from the ongoing trial in the first half of 2025.
Orphan Designation by the EMA is granted to drugs intended for the treatment of rare, life-threatening, or chronically debilitating diseases that affect no more than two in 10,000 people in the EU and for which no satisfactory therapy exists. Benefits of this designation include potential market exclusivity for ten years post-approval, reduced regulatory fees, and a centralized approval process within the EU.
Actuate's focus remains on developing therapies for high-impact, difficult-to-treat cancers, and this recent development with the EMA is a significant step forward in their mission. While the company maintains a healthy current ratio of 1.68, InvestingPro analysis shows it is not yet profitable, with earnings results next expected on February 26, 2025. The information for this article is based on a press release statement from Actuate Therapeutics.
In other recent news, Actuate Therapeutics has reported significant results in its Phase 2 trial of elraglusib for metastatic pancreatic cancer. The trial met its primary endpoints, showing a notable improvement in survival rates and reduction in the risk of death. The company plans to discuss the topline data and prospects of a Phase 3 registration trial with the FDA in the first half of 2025.
In parallel, Actuate has gained FDA's rare pediatric disease designation for elraglusib for the treatment of Ewing sarcoma, a metastatic form of bone cancer primarily affecting children and adolescents. The company has also reported encouraging results from its Phase 1/2 trial of elraglusib, with two ongoing complete responses and a disease control rate of approximately 62%.
Moreover, Actuate announced a change in its independent registered public accounting firm, with KMJ Corbin & Company LLP being replaced by Crowe LLP. The company confirmed that there were no disagreements with KMJ on any matter of accounting principles or practices, financial statement disclosure, or auditing scope or procedure. These are recent developments that provide a glimpse into Actuate's progress in both its clinical trials and financial operations.
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