SM04646 is an investigational therapy being developed by Samumed for the treatment of idiopathic pulmonary fibrosis (IPF). It is a small molecule delivered as an inhaled aerosol.

IPF is a chronic, progressive lung disorder that is usually fatal and typically affects adults over 40. It is the most common interstitial lung disease seen by lung specialists.

How SM04646 works

SM04646 is a Wnt pathway inhibitor. The Wnt/beta-catenin signaling pathway is a biological pathway involved in various developmental processes as well as the maintenance of balance between different tissues. It regulates cell growth, differentiation, migration, stability, and death.

When a problem occurs in the regulation of any of these processes, different diseases may develop, including IPF and cancer.

SM04646 has been shown to inhibit the Wnt pathway, attenuating fibrosis in an in vitro model of pulmonary fibrosis. It has even shown greater anti-fibrotic activity in vitro compared to Esbriet and Ofev, the two main anti-fibrotic therapies used to treat IPF, because it inhibits the expression of the gene that causes fibrosis in healthy lung cells and in IPF lung cells.

SM04646 in clinical trials

A Phase 1 clinical trial in healthy volunteers to assess SM04646’s potential to treat IPF has been successfully completed.

The study assessed the safety and tolerability of one inhalation of SM04646 in 17 healthy participants, ages 18 to 50. Four different doses were tested in four treatment groups to determine the maximum dose that can be inhaled by a healthy person without causing a strong cough.

The safety was evaluated according to the incidence and severity of adverse events caused by SM04646 using clinical laboratory analyses, lung function tests, and electrocardiograms (EEG).

No serious adverse events or dose limiting toxicity were reported in any of the groups tested, and the molecule appeared safe and well-tolerated at all doses studied.

The results supported continuation of the development of the molecule as a potential treatment for people with IPF.

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