Actuate Therapeutics' elraglusib, a novel glycogen synthase kinase-3 beta (GSK-3β) inhibitor, has been granted Orphan Medicinal Product Designation (OMPD) by the European Medicines Agency (EMA) for the treatment of pancreatic ductal adenocarcinoma (PDAC). This regulatory milestone highlights the potential of elraglusib to address a critical unmet need in patients with this aggressive and difficult-to-treat cancer.
Clinical Trial and Interim Results
Elraglusib is currently being evaluated in a randomized Phase 2 trial (Actuate-1801 Part 3β study, NCT03678883) as a first-line therapy for metastatic pancreatic cancer (mPDAC). The trial compares the combination of elraglusib with gemcitabine/nab-paclitaxel (GnP) to GnP alone. Interim data from the Phase 2 trial demonstrated statistically significant improvements in the one-year survival rate (p = 0.002) and median overall survival (hazard ratio = 0.63, p = 0.016) for patients treated with the elraglusib combination compared to those treated with GnP alone.
Significance of Orphan Drug Designation
The EMA's orphan drug designation is granted to therapies intended for the treatment, prevention, or diagnosis of life-threatening or chronically debilitating diseases that affect no more than two in 10,000 people in the European Union (EU) and for which no satisfactory therapy is available. This designation provides several benefits, including the potential for 10 years of market exclusivity following regulatory approval in the EU, reduction in regulatory fees, and a centralized EU approval process.
Actuate Therapeutics' Perspective
Daniel Schmitt, President & Chief Executive Officer of Actuate, stated, "With limited treatment options available for patients with PDAC, the EMA Orphan Drug Designation and FDA Orphan Drug Designation granted to elraglusib for the same indication reflect elraglusib’s potential to address a critical need for people living with this aggressive disease with a notably poor prognosis. This regulatory milestone, along with the recently announced positive interim results from our Phase 2 trial of elraglusib in patients with mPDAC that showed a significant clinical benefit and anti-tumor activity, should expedite our efforts to advance elraglusib as a novel, potential treatment for mPDAC."
The company anticipates reporting topline data from its Phase 2 trial in the first half of 2025 and plans to work closely with the EMA and the FDA to accelerate elraglusib’s clinical development program in metastatic pancreatic cancer.
About Elraglusib
Elraglusib, Actuate's lead investigational drug, targets molecular pathways involved in tumor growth and resistance to conventional cancer drugs, including DNA Damage Response (DDR) pathways. It is designed to mediate anti-tumor immunity through the inhibition of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and regulates multiple immune checkpoints and immune cell function.
