Merck KGaA announces FDA orphan drug designation for M7824
Merck KGaA announced that the US Food and Drug Administration has granted orphan drug designation to M7824, the first regulatory designation for the bifunctional immunotherapy, for the treatment of biliary tract cancer. The FDA orphan drug designation follows the recent presentation of the first clinical data for M7824 in BTC at the European Society of Medical Oncology congress in October. M7824 is an investigational bifunctional immunotherapy designed to combine co-localized blocking of the transforming growth factor-beta and anti-PD-L1 immune escape mechanisms. The first clinical data for M7824 in BTC, presented at the ESMO congress in October, demonstrated clinical activity in Asian patients who had progressed after platinum-based first-line treatment. The ORR among the total of 30 patients was 20%, as assessed by IRC, and responses were observed across PD-L1 levels with a duration of response ranging from 8.3 months to 13.9+ months. Grade 3 or higher TRAEs were experienced by 10 patients and the most common Grade 3 TRAEs were rash and lipase increase. M7824 is an investigational bifunctional immunotherapy that combines a TGF-beta trap with the anti-PD-L1 mechanism in one fusion protein. Designed to combine co-localized blocking of the two immunosuppressive pathways, M7824 is thought to control tumor growth by potentially restoring and enhancing anti-tumor responses. M7824 is an important part of a novel combination approach that seeks to harness the power of the immune system and address the tremendously complex nature of difficult-to-treat tumors. To-date, more than 670 patients with various types of solid tumors have been treated across the program with M7824. In addition to BTC, M7824 is being studied in solid tumor indications, including non-small cell lung, HPV associated tumors and gastrointestinal cancers, such as gastric cancer, esophageal squamous cell carcinoma and esophageal adenocarcinoma.