Biotechnology company Alligator Bioscience AB (STO:ATORX) announced on Friday that it has been granted Orphan Drug Designation (ODD) by the the US Food and Drug Administration (FDA) for mitazalimab, its lead asset, for the treatment of pancreatic cancer.

Mitazalimab is a monoclonal antibody that targets CD40 and has the potential to sensitise tumours to chemotherapy and activate immune-mediated tumour killing. Currently, mitazalimab is being evaluated in the OPTIMIZE-1 Phase 2 study, which is designed to assess its safety and efficacy in combination with chemotherapy (mFOLFIRINOX) for metastatic pancreatic ductal adenocarcinoma in previously untreated patients with metastatic pancreatic ductal adenocarcinoma.

Interim results from OPTIMIZE-1 showed an objective response rate (ORR) of 52% in evaluable patients, surpassing the ORR of around 32% reported for patients treated with FOLFIRINOX alone. The disease control rate, the proportion of patients with objective response or stabilisation of disease, exceeded 90%. Additional interim data, including Progression Free Survival, will be available in mid-2023, with full top-line data expected in Q1 2024.

The orphan designation for mitazalimab and the positive interim results will play a crucial role in discussions with regulatory authorities regarding the drug's clinical development and approval pathway for pancreatic cancer. The ODD qualifies Alligator Bioscience for incentives such as market exclusivity for seven years after approval, exemption from user fees, and a tax credit for qualified clinical trials.