Investigator-Initiated Trials for AXL driven cancers with high unmet clinical need

BerGenBio is a clinical-stage biopharmaceutical company focused on developing first-in-class drugs to treat multiple aggressive cancers, and the company's lead drug candidate bemcentinib (BGB324) targets a protein on the cancer cell surface which drives cancer spread, immune evasion and drug resistance. This project aims at supporting several investigator-initiated trials with bemcentinib, proposed and conducted by leading doctors worldwide. The clinical trials in this project are including patients with different cancers with high unmet medical need, where there is a strong scientific rationale for treatment with bemcentinib. Five of these clinical trials, in lung cancer, melanoma, pancreatic cancer, mesothelioma and glioblastoma multiforme are underway and recruiting patients while a clinical trial in AML and MDS has completed recruitment.

BGB has collaborated with several world class academic institutions to test bemcentinib clinically in several new indications. Several trials has have been initiated in myelodysplastic syndrome (MDS), Acute Myeloid Leukemia (AML), Non-Small Cell Lung Cancer (NSCLC), Melanoma, Pancreatic Cancer, Mesothelioma and Glioblastoma Multiforme. The AML and MDS trial has completed recruitment and early analysis show encouraging results as reported at ASH in December 2020. Blood and tissue samples are being collected across all studies for biomarker analysis as these become available. Interim analysis from both the melanoma trial and the AML and MDS trial identified promising markers of bemcentinib activity allowing for further exploration of these markers in samples from other studies.

BGB324 is a first-in-class experimental drug inhibiting the hallmarks of aggressive cancers including acquired drug resistance, immune evasion and metastasis; this represents the most significant unmet need in cancer treatment. BGB324 is a highly selective inhibitor of the cancer cell signalling protein called AXL. Increased AXL expression correlates significantly with poor prognosis in many cancers and therefore represents a very large addressable market.  BGB324 is in phase II clinical trials in patients with leukaemia and lung cancer. Encouraging clinical data already published confirms BGB324 is well tolerated by patients and a proportion of patients have reported clinical benefit and long term disease control. This funding will support Investigator initiated clinical trials (IITs) in Norway and overseas. IITs are clinical trials led by patient facing doctors who act as the regulatory sponsor and are partially supported by BerGenBio in bespoke clinical development collaborations. IITs represent good value additional clinical research that benefits patients and expands the potential clinical utility of BGB324. The IITs in this project will include patients with different cancers with high unmet medical need, where there is a strong scientific rationale for treatment with BGB324. The data generated from these IITs will enlarge our experience with BGB324 in cancer patients, complement our safety database as well as inform future clinical studies and BGB324s route to market.