Lund, Sweden – Alligator Bioscience (Nasdaq Stockholm: ATORX) today announces that the European Medicines Agency (EMA) has granted Orphan Designation to its lead asset mitazalimab for the treatment of pancreatic cancer.
Mitazalimab is a monoclonal antibody targeting CD40 designed to sensitize tumors to chemotherapy and induce immune-mediated tumor killing by activating dendritic cells, B cells, and macrophages. Mitazalimab is currently being evaluated in OPTIMIZE-1, a Phase 2 open-label, multi-center study to assess its safety and efficacy in combination with chemotherapy, mFOLFIRINOX, in previously untreated patients with metastatic pancreatic ductal adenocarcinoma (NCT04888312).
In May 2023, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to mitazalimab for the treatment of pancreatic cancer.
"We are very pleased that the European Medicines Agency has granted orphan designation to our lead asset mitazalimab in the treatment of pancreatic cancer," said Søren Bregenholt, CEO of Alligator Bioscience. "It is our second orphan designation this year following the FDA's 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."
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.
In June 2023, Alligator announced a second set of strong interim results from OPTIMIZE-1, in which mitazalimab combined with mFOLFIRINOX showed deepening of tumor response and increase in objective response rate (ORR) to 57% (from initially reported 52% ORR in 23 patients) and demonstrated an interim ORR of 44% in the full study cohort (57 patients) that is expected to further improve with longer follow up. A median duration of response (DoR) of 8.7 months , as per the Response Evaluation Criteria in Solid Tumors (RECIST 1.1), was also reported. This compares very strongly with an ORR of 31.6% and DOR of 5.9 months reported in a similar patient population treated with standard of care.
OPTIMIZE-1 is on track for top-line readout in early Q1 2024.
 N Engl J Med 2011; 364:1817-1825; DOI: 10.1056/NEJMoa1011923