Alligator Bioscience has announced that the European Medicines Agency (EMA) has granted orphan designation for the company’s lead asset, mitazalimab, for the treatment of pancreatic cancer.

Mitazalimab is a monoclonal antibody targeting CD40 and has been created to sensitise tumours to chemotherapy while also inducing immune-mediated tumour killing by activating dendritic cells, B cells and macrophages.

At present, the drug is being evaluated in OPTIMIZE-1, a phase 2 multi-location, open-label trial to analyse efficacy and safety when combined with chemotherapy (mFOLFIRINOX) in previously untreated patients with metastatic pancreatic ductal adenocarcinoma.

In June, Alligator announced a second set of strong interim results from OPTIMIZE-1, during which mitazalimab combined with mFOLFIRINOX demonstrated a deepening of tumour reaction and an increase in objective response rate (ORR) to 57% (it was initially 52% ORR among the 23 patients involved).

To meet the criteria for the EMA’s orphan designation, a therapy must be delivered for the treatment, prevention or diagnosis of rare, life-threatening or chronically debilitating diseases that impact fewer than five in 10,000 individuals across the EU.
Treatments that qualify are eligible for financial and regulatory incentives that include ten years of marketing exclusivity throughout the EU after a product has been approved.

Søren Bregenholt, chief executive officer at Alligator Bioscience, reflected: “We are very pleased that the European Medicines Agency has granted orphan designation to our lead asset mitazalimab in the treatment of pancreatic cancer.”

He added: “It is our second orphan designation this year following the US Food and Drug Administration’s (FDA) decision to grant us ODD in May, meaning mitazalimab now has stronger commercial protection through market exclusivity in these two key markets. This latest designation adds to the momentum we are building in our efforts to bring this promising drug candidate to market.”

Earlier this year, the FDA provided orphan drug designation to mitazalimab for treating pancreatic cancer.

MSD has revealed that the National Institute for Health and Care Excellence (NICE) has recommended the use of Keytruda.

Also known as pembrolizumab, the therapy treats tumours with microsatellite instability-high (MSI-H) or mismatch repair deficiency (dMMR). It also involves adults with advanced or recurrent endometrial cancer that has progressed during or after platinum-based therapy, as well as certain patients with gastric, small intestine or biliary cancer.

The decision from NICE is the first verdict based on data from a wider immunotherapy basket study. In contrast to a traditional clinical trial, a basket version tests treatments on multiple types of cancer, with the same specific molecular alteration. Using this method can decrease the number of studies required to help bring treatments to patients.

Last year, a UK licence was issued for the indication of pembrolizumab among patients as a monotherapy across five MSI-H/dMMR tumour sites, meeting specific criteria across the basket trial. This included its indication for treating metastatic or unresectable MSI-H/dMMR colorectal cancer following fluoropyrimidine-based combination therapy.

In MSI-H/dMMR unresectable or metastatic gastric, small intestine, or biliary cancer, pembrolizumab was licensed for candidates with disease progression on or following at least one prior therapy.

Julie Harrington, CEO at Guts UK, explained: “We are delighted that some people with metastatic gastric, small intestine, biliary and colorectal cancer have gained access to an additional treatment option.

“As the first immunotherapy multi-tumour basket trial recommended by NICE for people with these types of tumours, this decision will help address this unmet need.”
David Long, Head of Oncology at MSD UK, welcomed the approval from NICE for the basket study for adults with previously treated MSI-H/dMMR tumours, saying: “MSI-H/dMMR tumours are associated with a poorer prognosis in advanced cancers, compared with non-MSI-H/dMMR status, which means there is a high unmet for patients with these gene features.”