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Alligator Bioscience receives FDA Orphan Drug Designation

Søren Bregenholt

The U.S. Food and Drug Administration has granted Orphan Drug Designation (ODD) to the company’s lead asset mitazalimab for the treatment of pancreatic cancer.

“This designation is a key milestone for our lead asset mitazalimab, which is producing outstanding clinical results in its Phase 2 trial in pancreatic cancer,” says Søren Bregenholt, CEO of Alligator Bioscience. “Orphan designation confers significant benefits in the form of cost savings during development and marketing exclusivity following approval, and we are very pleased to see the potential of mitazalimab being recognized with the award of this designation.”


Mitazalimab is a monoclonal antibody targeting CD40 with the potential 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.


In January 2023, Alligator announced strong interim results from OPTIMIZE-1, in which mitazalimab combined with mFOLFIRINOX demonstrated an objective response rate (ORR) of 52% in 23 evaluable patients, as per the Response Evaluation Criteria in Solid Tumors (RECIST 1.1). In comparison, a similar patient population treated only with FOLFIRINOX reported an ORR of around 32%[1]. Disease control rate, the proportion of patients with objective response or stabilization of disease, was more than 90%. In April 2023, Alligator announced that OPTIMIZE-1 had been fully enrolled.

The orphan designation for mitazalimab and the positive interim results from OPTIMIZE-1 will be a key component of discussions with regulatory authorities regarding subsequent clinical development and approval pathway for mitazalimab in pancreatic cancer, states the company. Additional interim data from OPTIMIZE-1, including Progression Free Survival, are due in mid-2023 and full top-line data are expected in the beginning of Q1 2024.

Photo of Søren Bregenholt: Alligator Bioscience