EDP-305

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Experimental drug for liver diseases


{{Drugbox | verifiedfields = changed | verifiedrevid = 123456789 | IUPAC_name = (2S)-2-[[4-[2-[2-[[2-(3-chlorophenyl)ethyl]amino]ethoxy]ethoxy]phenyl]methyl]butanoic acid | image = EDP-305.svg | image2 = | width = 200 | alt = | caption = Chemical structure of EDP-305 | tradename = | synonyms = | CAS_number = 1807534-56-1 | ATC_prefix = | ATC_suffix = | PubChem = 12345678 | DrugBank = | ChemSpiderID = 123456 | UNII = | KEGG = | ChEMBL = 1234567 | SMILES = | StdInChI = | StdInChIKey = }}

EDP-305 is an investigational drug developed for the treatment of liver diseases, particularly non-alcoholic steatohepatitis (NASH) and primary biliary cholangitis (PBC). It is a potent and selective agonist of the FXR, a nuclear receptor that plays a crucial role in the regulation of bile acid, lipid, and glucose metabolism.

Mechanism of Action[edit | edit source]

EDP-305 functions by activating the FXR, which is primarily expressed in the liver and intestines. Activation of FXR leads to a cascade of metabolic effects, including the reduction of bile acid synthesis, modulation of lipid metabolism, and improvement in insulin sensitivity. These effects are beneficial in conditions like NASH, where there is an accumulation of fat in the liver, inflammation, and fibrosis.

Clinical Development[edit | edit source]

EDP-305 is currently undergoing clinical trials to evaluate its efficacy and safety in patients with NASH and PBC. Early-phase studies have shown promising results in terms of reducing liver fat content and improving liver function markers. The drug is being developed by Enanta Pharmaceuticals, which is focusing on advancing its clinical trials to further assess its therapeutic potential.

Potential Benefits[edit | edit source]

The activation of FXR by EDP-305 offers several potential benefits for patients with liver diseases:

  • Reduction in Liver Fat: By modulating lipid metabolism, EDP-305 helps in reducing hepatic steatosis, a hallmark of NASH.
  • Anti-inflammatory Effects: FXR activation can lead to decreased inflammation in the liver, which is crucial in preventing the progression of liver diseases.
  • Fibrosis Reduction: By influencing pathways involved in fibrosis, EDP-305 may help in reducing liver scarring, which is a significant concern in chronic liver diseases.

Side Effects and Safety[edit | edit source]

As with any investigational drug, the safety profile of EDP-305 is being closely monitored in clinical trials. Common side effects observed include gastrointestinal disturbances, pruritus, and changes in lipid profiles. Long-term safety data is still being collected to ensure the drug's safety for chronic use.

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Contributors: Prab R. Tumpati, MD