EMA Grants OMPD to Actuate's Pancreatic Cancer Therapy

Actuate Therapeutics has recently achieved a significant milestone with the European Medicines Agency (EMA) granting orphan medicinal product designation for their investigational drug, elraglusib, aimed at treating pancreatic ductal adenocarcinoma (PDAC). This designation is particularly important as it is awarded to treatments for severe or chronically debilitating diseases affecting a small number of people across the European Union, with no existing satisfactory therapies available.

Elraglusib, a glycogen synthase kinase-3 beta (GSK-3β) inhibitor, is currently being evaluated as a first-line treatment. It is being tested in combination with the standard chemotherapy drugs gemcitabine and nab-paclitaxel (GnP) in individuals with metastatic pancreatic cancer (mPDAC). The efficacy of this combination is being assessed in a randomized trial known as Actuate-1801 Part 3β, which compares the elraglusib and GnP combination to the traditional GnP regimen alone.

The ongoing Phase II trial has shown promising interim results, highlighting a significant increase in the one-year survival rate for patients receiving the elraglusib combination, with a p-value of 0.002. Additionally, the data indicate a median overall survival advantage, as evidenced by a hazard ratio of 0.63 and a p-value of 0.016, both statistically significant in favor of the combination therapy.

Daniel Schmitt, CEO and President of Actuate Therapeutics, emphasized the importance of this regulatory achievement alongside the positive interim findings from their Phase II trial. Schmitt expressed optimism that these developments will support efforts to advance elraglusib as a novel and potentially effective treatment option for individuals with metastatic pancreatic cancer. He also mentioned the company’s plans to continue working closely with both the EMA and the US Food and Drug Administration (FDA) to accelerate the clinical development of elraglusib. The company anticipates reporting the topline data from the ongoing Phase II trial in the first half of 2025.

Elraglusib is positioned as the leading investigational candidate for Actuate Therapeutics. It targets specific molecular pathways that facilitate tumor growth and resistance to standard cancer therapies, including several DNA damage response pathways. The drug is designed to enhance anti-tumor immunity by inhibiting nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB), while also regulating various immune checkpoints and immune cell functionalities.

The orphan designation from the EMA not only underscores the potential of elraglusib as a treatment for PDAC but also provides Actuate Therapeutics with various developmental incentives. These may include market exclusivity, fee reductions, and advisory support, which are crucial for advancing their innovative therapy through the clinical development and regulatory processes. As pancreatic ductal adenocarcinoma remains one of the most challenging forms of cancer to treat, the progress of elraglusib in clinical trials offers hope for improving outcomes for patients affected by this aggressive disease.