Mitazalimab has received orphan drug designation status by the European Medicines Agency (EMA) for the treatment of patients with pancreatic cancer, its manufacturer Alligator Bioscience has announced.
A human CD40 agonistic antibody targeting CD40, mitazalimab kickstarts the cancer-immunity cycle by priming and activating tumour-specific T cells. Targeting CD40 with mitazalimab has the potential to augment responses to chemotherapy.
Pancreatic ductal adenocarcinoma (PDAC) is known to be the fourth-leading cause of cancer related mortality in the world and has a poor prognosis, with a five-year survival rate of below 5%.
To qualify for the EMA’s orphan designation, a medicine must be intended for the treatment, prevention or diagnosis of rare, life-threatening or chronically debilitating diseases that affect fewer than five in 10,000 persons in the EU. Medicines that meet these criteria are eligible for financial and regulatory incentives that include 10 years of marketing exclusivity in the EU after product approval.
The EMA orphan drug designation follows a similar approval by the FDA in May 2023.
Commenting on the EMA approval for orphan drug designation status, Søren Bregenholt, CEO of Alligator Bioscience, said: ‘We are very pleased that the European Medicines Agency has granted orphan designation to our lead asset mitazalimab in the treatment of pancreatic cancer.
‘It is our second orphan designation this year following the FDA‘s decision to grant us [orphan drug designation] 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.‘
Mitazalimab and OPTIMIZE-1
Mitazalimab is currently being evaluated in the phase 1b/2 OPTIMIZE-1 trial in combination with mFOLFIRINOX chemotherapy for adult patients with previously untreated metastatic PDAC.
In the trial, participants receive mitazalimab and mFOLFIRINOX via intravenous infusions following a 14-day cycle schedule. Mitazalimab is administered two days after mFOLFIRINOX, except for the first cycle of 21 days, where the drug is administered on days one and 10 with infusion of mFOLFIRINOX starting on day eight.
Interim results from OPTIMIZE-1 released in June 2023, showed a deepening of tumour response and an increase in the objective response rate (ORR) from 52% to 57% in a cohort of 23 patients.
In the full study cohort of 57 patients, there was an interim ORR of 44%, and this is expected to further improve with longer follow-up. A median duration of response of 8.7 months was also reported.