Certa Therapeutics Receives EU Orphan Designation for FT011, a GPR68 Inhibitor Being Developed as an Anti-Fibrotic Treatment for Systemic Sclerosis

Melbourne, Australia 22 July 2024: Certa Therapeutics (Certa), a biotechnology company developing innovative precision therapies for patients with inflammatory and fibrotic diseases, today announces that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) for its lead drug candidate, FT011, as a potential treatment of Systemic Sclerosis (SSc). Certa has previously also received ODD and Fast Track designation for FT011 as a potential treatment of SSc from the U.S. Food and Drug Administration (FDA).

FT011 is a novel, first-in-class oral therapy for the treatment of chronic fibrosis in multiple organs. It targets an important but previously undrugged membrane GPCR receptor, GPR68, a master switch of fibrosis. An extensive body of data demonstrates promising efficacy in multiple in vitro and in vivo models of inflammatory and fibrotic disease. Phase I and IIa clinical studies in patients with SSc have also demonstrated favourable efficacy, safety and pharmacokinetics.

SSc is a chronic, progressive, autoimmune disease characterised by inflammation and fibrosis (scar tissue formation) in the skin and in various internal organs - commonly lungs, kidneys and heart.

Skin fibrosis is the distinguishing feature of SSc and is associated with significantly reduced function and disability in patients. Currently, there are no treatments on the market that effectively stop or reverse scarring in the skin and organs.

Professor Darren Kelly Certa Therapeutics CEO and founder said, “GPR68 inhibition modulates the biological pathways causing inflammation and fibrosis to the skin in patients with SSc. With limited treatment options available for patients with SSc, the EMA Orphan Drug Designation and FDA Orphan Drug and Fast Track Designations reflect the potential for FT011 to address a critical need for people living with this debilitating and life-threatening condition.

“These are significant validations for the company and highlight the unmet need for novel medicines to reduce inflammation and scarring in chronic fibrotic diseases, which are often progressive and can have a poor prognosis.”

Certa is planning for a Phase IIb confirmatory SSc clinical trial with FT011, bringing the company closer to providing better treatment options for patients with fibrosis. It is also developing biomarkers and gene signatures to identify patients most likely to respond to treatment to ensure the best outcomes for those with fibrosis.

The FDA and EMA grant orphan status to products intended to treat, diagnose, or prevent life-threatening rare diseases or conditions that affect fewer than five in 10,000 people in Europe, or under 200,000 people in the US, and with either no currently approved method of diagnosis, prevention, or treatment, or with significant benefit to those affected by the disease. Orphan drug designation provides certain benefits, including the potential for extensive marketing exclusivity following regulatory approval, reduction in regulatory fees and, in the case of EU, a centralised approval process.