AnaMar has announced results from its Phase I study, and the completion of Phase II enabling pre-clinical studies for its initial orphan indication in systemic sclerosis (SSc) characterized by lung and skin fibrosis.
AM1476, a highly selective small molecule, delivered as an orally administered tablet, was found to be safe and well-tolerated at all relevant doses in a trial of approximately 100 healthy subjects, states the company.
“Our Phase II ready compound AM1476 for the treatment of the orphan disease systemic sclerosis will be a significant milestone in the development of novel medicines to prevent, heal and slow organ scarring from fibrotic diseases. We hold a broad, patent-protected, portfolio of selective and high affinity 5-HT2B receptor antagonists. We have seen great potential from our pre-clinical and phase I studies, and now look forward to working with a pharma partner to turn our research into life-changing treatments for patients with fibrosis,” says AnaMar’s CEO, Ulf Ljungberg.
A Phase II study has been designed to evaluate the treatment effects in SSc-ILD with a proposed dosing regimen for 60 patients in a double-blinded, placebo-controlled randomized trial over 12 months with lung function (forced vital capacity) and skin thickness (modified Rodnan skin score) as primary efficacy readouts. Active product ingredients are being made ready as cGMP materials for this Phase II trial. The company is planning regulatory submissions, in 2023, to the FDA and EMA for Orphan Drug Designation for the rare disease SSc. Drawing on its heritage in diagnostics, the company is also developing biomarkers and gene signatures to identify patients most likely to respond to treatment and ensure the best outcomes for patients with fibrosis.
In several well-known preclinical state-of-the-art animal models of fibrosis AM1476 halted and reversed the progression of pre-established fibrosis in skin and lung tissue, clearly demonstrating the anti-fibrotic efficacy of AM1476.
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