Actuate Therapeutics has gained orphan medicinal product designation (OMPD) from the European Medicines Agency (EMA) for elraglusib to treat pancreatic ductal adenocarcinoma (PDAC).

The glycogen synthase kinase-3 beta (GSK-3β) inhibitor elraglusib is being studied as a first-line therapy in combination with gemcitabine/nab-paclitaxel (GnP) in individuals with metastatic pancreatic cancer (mPDAC). The randomised Actuate-1801 Part 3β trial is comparing this combination to GnP alone.

The Phase II trial’s interim analysis revealed a one-year survival rate increase with a p-value of 0.002 and a median overall survival with a hazard ratio of 0.63 and a p-value of 0.016, favouring the elraglusib combination.

Actuate Therapeutics CEO and president Daniel Schmitt stated: “This regulatory milestone, along with the recently announced positive interim results from our Phase II trial of elraglusib in patients with mPDAC that showed a significant clinical benefit and anti-tumour activity, should expedite our efforts to advance elraglusib as a novel, potential treatment for mPDAC.

“We look forward to reporting topline data from our Phase II trial in 1H 2025 and working closely with the EMA and the FDA [US Food and Drugs Administration] to accelerate elraglusib’s clinical development programme in metastatic pancreatic cancer.”

Elraglusib, the company's lead investigational candidate, targets molecular pathways that promote tumour growth and resistance to conventional cancer treatments, including several DNA damage response (DDR) pathways.

It is tailored to mediate anti-tumour immunity by inhibiting nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kB) and regulating several immune checkpoints and immune cell functions.

EMA orphan designation is awarded to therapies for life-threatening or chronically debilitating diseases that affect no more than two in 10,000 individuals in the European Union (EU) and for which there is no satisfactory therapy.