MTX-463

Table of contents

• What is MTX-463?
• How does MTX-463 work?
• Which conditions is MTX-463 studied for?
• How is MTX-463 given?
• What do the clinical trials show?
• What side effects are known?

What is MTX-463?

MTX-463 is an investigational substance developed by Mediar Therapeutics for the treatment of idiopathic pulmonary fibrosis (IPF), a chronic lung condition with a poor prognosis and a significant impact on quality of life.^[1] It is not yet an authorised medicine and is currently being evaluated in a single Phase 2 clinical trial.^[1] The substance is given by intravenous infusion.^[1]

How does MTX-463 work?

MTX-463 aims to reduce lung damage by targeting WISP1, a protein that is believed to play a critical role in activating the cells that cause lung scarring in IPF.^[1] By blocking or reducing the activity of WISP1, the drug is intended to slow the scarring process that progressively stiffens the lungs and reduces their ability to take in air.^[1]

Which conditions is MTX-463 studied for?

• Idiopathic pulmonary fibrosis (IPF) — a chronic lung condition in which scarring of the lungs makes it progressively harder to breathe. Symptoms include shortness of breath and cough.^[1] The current treatments, pirfenidone and nintedanib, can slow disease progression but cause side effects such as vomiting and diarrhoea that limit their use.^[1]

How is MTX-463 given?

MTX-463 is administered as a solution for infusion by intravenous use.^[1] In the Phase 2 trial, the dose under investigation is 28 mg per kilogram of body weight.^[1] The trial also includes two placebo arms: a sodium chloride 0.9% solution for infusion and a dextrose 5% solution for infusion.^[1]

What do the clinical trials show?

One Phase 2 trial (NCT06967805) is registered for MTX-463 in idiopathic pulmonary fibrosis.^[1] The study is randomised, double-blind and placebo-controlled, meaning participants are assigned by chance to receive either MTX-463 or a placebo, and neither the participants nor the researchers know who is receiving which treatment during the study.^[1] The trial plans to enrol 164 participants aged 40 years or over.^[1][1]

The primary outcome is the change from baseline to Week 24 in forced vital capacity (FVC), which is a measure of how much air the lungs can take in.^[1][1] As the lungs scar in IPF, their ability to inhale air goes down, so FVC is used as the main measure of whether the drug slows disease progression.^[1] Other goals of the study include assessing how safe MTX-463 is, how much of the dose stays in the blood until the next dose, and how well the drug relieves symptoms of IPF.^[1] The study lasts up to 32 weeks for each participant, including a 4-week screening period followed by a 24-week treatment period.^[1] The trial is currently authorised but not yet recruiting.^[1]

What side effects are known?

The available sources do not report specific side effects of MTX-463. The trial is designed to assess the safety of the drug, and safety data are expected to emerge once the study is underway.^[1] The research summary notes that the currently approved IPF treatments pirfenidone and nintedanib cause side effects such as vomiting and diarrhoea that limit how much they are used, which is part of the rationale for developing new options.^[1]