AM1476, a first-in-class, peripheral-acting serotonin receptor antagonist, has been granted US and EU Orphan Drug Designation as an anti-fibrotic treatment for systemic sclerosis.
AM1476 is a selective peripheral-acting 5-HT2B receptor small molecule antagonist that can be delivered orally. It has demonstrated efficacy in in vitro and in vivo models of fibrosis, as well as favourable safety and pharmacokinetic profiles in Phase I clinical studies.
AM1476 can treat both skin and lung manifestations of systemic sclerosis. Systemic sclerosis is a chronic, progressive, autoimmune disease characterised by inflammation and fibrosis, such as uncontrolled scar tissue formation, in skin and various internal organs. Skin fibrosis is the distinguishing hallmark of SSc, associated with significant disability.
Around 100,000 people in the EU are affected by SSc and up to 80% of these may develop interstitial lung disease (ILD). ILD causes progressive lung scaring, known as fibrosis. There are currently no treatments on the market that effectively stop or reverse scarring in both skin and lung tissue.
AnaMar’s Chief Executive Officer, Dr Ulf Ljungberg, said: “We are delighted with the FDA’s and EMA’s decisions to grant orphan drug designation to AM1476 for SSc. This is a significant milestone and underscores the significant unmet need for novel medicines to prevent, heal and slow organ scarring from fibrotic diseases, which are often progressive and can have a poor prognosis.
“There is great potential in AM1476 as a unique dual-action approach to treat skin and lung manifestations of systemic sclerosis, especially as it represses both macrophage and fibroblast activity. We look forward to commercialising our product with a pharma partner to bring better treatment options to patients with fibrosis.”
The company has designed a Phase II study to evaluate the treatment effects in SSc-ILD over 12 months.