Dual-functional immunotherapy! The FDA awarded Merck TGF-β/PD-L1 dual-target therapy M7824 to treat bile duct cancer orphans.

Merck KGaA, the German pharmaceutical giant, recently announced that the U.S. Food and Drug Administration (FDA) has granted M7824 the right to treat bile tube cancer (BTC).
is used to prevent, treat and diagnose rare diseases, which are the general term for a very low incidence of diseases, also known as "orphan diseases". In the United States, rare diseases are the type of disease that affects fewer than 200,000 people. Pharmaceutical companies that develop rare disease drugs will receive incentives, including various clinical development incentives, such as tax credits related to clinical trial costs, FDA user fee waivers, FDA assistance in clinical trial design, and a seven-year market exclusive period after the drug is approved for market.
M7824 is an experimental two-functional immunotherapy that combines a transformational growth factor-β (TGF-β) trap with an anti-PF-L1 mechanism in a fusion protein designed to bind common local blocking of two immunosuppressive pathways, while targeting these two pathways designed to control tumor growth by potentially restoring and enhancing the anti-tumor response.
BTC is a group of rare invasive gastrointestinal cancers, including intra-liver bile tube cancer (ICC), ex-hepatic bile tube cancer (ECC) and gallbladder cancer (GBC). It is estimated that 16,000 BTC cases are diagnosed each year in the United States, and these cancers occur later in most patients. BTC treatment options are limited, the medium survival of late-stage patients is less than 1 year, the objective tumor mitigation rate for common chemotherapy is usually less than 10%, and the remission time is short.
the first clinical data on M7824 treatment BTC, released at the ESMO Conference in October, showed that M7824 was clinically active in Asian BTC patients with progression after first-line treatment with platinum chemotherapy, according to the IRC. In a total of 30 patients treated, the total remission rate (ORR) was 20%, and remission was observed in patients with various PD-L1 levels, with a duration range of 8.3 months - 13.9 months or more. In 10 patients (33.3%) with treatment-related adverse events (TRAEs) of level 3 or above, the most common TRAE was rash (10%) and lipase elevation (10%).
M7824 is an important part of Merck's search for new combinations that harness the immune system's ability and address the extremely complex nature of resolvable tumors. To date, more than 670 patients with various solid tumors have been treated with M7824. In addition to BTC, M7824 has been studied in solid tumor adaptations, including non-small cell lung cancer, HPV-related tumors, gastrointestinal cancers (e.g. stomach cancer, esophageal squamous cell carcinoma, and esophageal adenocarcinoma).
Currently, M7824 treats solid tumors in Phase I clinical trials, and Merck is conducting a multi-center randomized open-label controlled study to assess the efficacy and safety of M7824 and Mercado PD-1 tumor immunoKeytruda (Creda, generic name: pembrolizumab, Pabliju monomabolic) as a single-drug therapy for patients with advanced PD-L1-positive non-small cell lung cancer (NCLC). (Bio Valley)