Certa Therapeutics, a Melbourne-based biotech company, announced the results of its Phase 2 clinical trial for FT011, a novel oral therapy for chronic fibrosis.
The trial showed clinically meaningful improvements in more than 60% of scleroderma patients after 12 weeks of treatment.
In the Phase 2 study, 60% of patients treated with a 400 mg dose of FT011 showed a significant clinical improvement at 12 weeks while 20% of patients in the 200 mg did.
Several dimensions of improvement
Clinical trial investigators noted significant advances in lung function, patient self-assessment, and physician evaluation as measured by American College of Rheumatology Combined Response Index in diffuse cutaneous Systemic Sclerosis (CRISS) score, skin thickness, lung function, physician-reported assessment and quality of life evaluations.
The drug candidate was well tolerated in the study.
FT011 targets a G protein-coupled receptor (GPCR), which is inactive in healthy tissue but activated in the presence of injury or illness. GPCR is involved in various pathways that trigger inflammation and fibrosis. FT011 is designed to block this GPCR, potentially stemming chronic fibrosis in various organs.
Traditional scleroderma drug therapies are generally immunosuppressive or cytotoxic in nature.
Scleroderma is a rare chronic autoimmune disease associated with fibrosis in multiple organs, including the skin and organs. The disease involves hardening and thickening of the skin and tissues, potentially leading to symptoms such as joint pain, digestive problems and impaired blood flow to the hands and feet. In severe cases, scleroderma can also damage internal organs such as the heart, lungs and kidneys.
Targeting fibrotic disease
Certa has identified the gene profile that drives fibrotic disease. Fibrosis is involved in a range of chronic diseases. Outside of scleroderma, examples include lupus, chronic kidney disease, inflammatory bowel disease and rheumatoid arthritis.
Approximately 45% of deaths in developed countries are linked to some form of chronic fibroproliferative illness, according to a 2008 study in The Journal of Pathology.
Certa Therapeutics plans to conduct Phase 2 studies of FT011 in chronic kidney disease (CKD) and focal segmental glomerulosclerosis (FSGS).