Biotechnology company Certa Therapeutics has revealed positive Phase II clinical trial data for FT011 in patients with scleroderma.
The results were presented at ACR Convergence 2023, the annual scientific meeting of the American College of Rheumatology in San Diego, California (10-15 November 2023).
Treatment with FT011 for 12 weeks resulted in a clinically meaningful improvement in 60% of patients treated with FT011 400mg and 20% of patients in the FT011 200mg group compared with 10% in the placebo group.
In totality, treatment with FT011 led to significant improvements across multiple efficacy measures, including the American College of Rheumatology Combined Response Index in diffuse cutaneous Systemic Sclerosis (CRISS) score, three skin thickness (defined by the modified Rodnan Skin Score, mRSS), lung function (%FVC), physician-reported assessment and quality of life evaluations.
Transcriptomic analysis indicated that the systemic fibrotic disease gene signature is modulated after 12 weeks, with an increased fibrosis signature score in the placebo group but significantly, a decrease in the same fibrosis signature score following FT011 400mg treatment. This suggests that FT011 may have a positive effect for scleroderma patients by reducing the inflammation and fibrosis associated with the disease.
The US Food and Drug Administration (FDA) recently granted Orphan Drug Designation for FT011 for the treatment of systemic sclerosis (scleroderma).
Professor Dinesh Khanna, Frederick GL Huetwell Professor of Rheumatology and Director, Scleroderma Program at the University of Michigan said: “The mechanism of action for FT011 offers an important point of difference for the scleroderma research field as the upstream inhibition of inflammation and fibrosis may offer meaningful clinical outcomes. It is important to advance safe and effective treatments through clinical development, given the limited therapeutic options for scleroderma patients. I look forward to collaborating with the company in the FT011 development program ahead.”