Niraxostat

{{Drugbox | Verifiedfields = changed | verifiedrevid = 123456789 | IUPAC_name = (2S)-2-[[4-(4-fluorophenyl)-1H-pyrazol-3-yl]amino]-3-methylbutanoic acid | image = Niraxostat_structure.png | width = 250 | alt = Structural formula of Niraxostat | tradename = Niraxostat | legal_status = Investigational | routes_of_administration = Oral | bioavailability = High | protein_bound = 95% | metabolism = Hepatic | elimination_half-life = 12 hours | excretion = Renal }}

Niraxostat is an investigational drug that is currently being studied for its potential use in the treatment of fibrotic diseases. It is a small molecule inhibitor that targets specific enzymes involved in the fibrotic process, aiming to reduce or reverse fibrosis in affected tissues.

Mechanism of Action

Niraxostat functions by inhibiting the activity of the enzyme lysyl oxidase-like 2 (LOXL2), which plays a critical role in the cross-linking of collagen fibers. By inhibiting LOXL2, Niraxostat reduces the formation of stable collagen cross-links, thereby decreasing tissue stiffness and fibrosis. This mechanism is particularly relevant in diseases such as idiopathic pulmonary fibrosis and liver cirrhosis, where excessive collagen deposition leads to organ dysfunction.

Pharmacokinetics

Niraxostat is administered orally and has a high bioavailability. It is extensively bound to plasma proteins, with a binding rate of approximately 95%. The drug undergoes hepatic metabolism, primarily through the cytochrome P450 enzyme system, and has an elimination half-life of about 12 hours. Niraxostat is primarily excreted via the renal route.

Clinical Trials

Niraxostat is currently in Phase II clinical trials for the treatment of idiopathic pulmonary fibrosis. Preliminary results have shown a reduction in fibrotic markers and stabilization of lung function in patients receiving the drug. Further studies are ongoing to evaluate its efficacy and safety in larger patient populations.

Adverse Effects

The most commonly reported adverse effects of Niraxostat include mild gastrointestinal disturbances, headache, and fatigue. In clinical trials, these side effects were generally well-tolerated and did not lead to discontinuation of therapy.

Research and Development

Niraxostat was developed by a collaboration between academic institutions and pharmaceutical companies, with the aim of addressing unmet needs in the treatment of fibrotic diseases. Ongoing research is focused on understanding the broader applications of Niraxostat in other fibrotic conditions, such as systemic sclerosis and chronic kidney disease.

Also see

• Fibrosis
• Lysyl oxidase
• Idiopathic pulmonary fibrosis
• Liver cirrhosis
• Systemic sclerosis