Certa Therapeutics announces positive results in phase 2 scleroderma study

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Australian company Certa Therapeutics (Certa) has announced clinical trial data for its novel therapy for the treatment of serious inflammatory and fibrotic diseases.

The company said it has recently completed a phase 2, multi-centre, randomised, double-blind, placebo-controlled study of the pharmacokinetics, pharmacodynamic effects, and safety, of oral FT011 in patients with scleroderma.

Scleroderma is a debilitating and potentially life-threatening autoimmune condition characterised by inflammation and fibrosis of the skin and other organs.

The condition can result in high morbidity with substantial detriment to the quality of life, with patients commonly experiencing loss of mobility and function, pain, and fatigue, often accompanied by a significant impact on their mental health. Scleroderma has the highest mortality among rheumatic diseases.

Certa has announced that the results of the phase 2 study indicate that compared to placebo, treatment with FT011 led to significant improvements across multiple efficacy measures, including skin thickness, lung function and quality of life evaluations.

The company said that a clinically meaningful improvement was demonstrated in 60 per cent of patients treated with FT011 400mg and 20 per cent of patients in the FT011 200mg group.

"Three patients in the pooled FT011 group achieved a maximum CRISS score of 1.0, representing the greatest probability of clinical improvement," it said.

Certa Therapeutics CEO and founder Professor Darren Kelly said, “These exceptional trial results demonstrate the potential of this novel treatment for patients with scleroderma. The changes seen in CRISS score, lung function, and physician-reported outcomes in addition to the patient-reported outcomes within such a short treatment timeframe of 12 weeks, is unprecedented and paves the way for a confirmatory global Phase III study.

“We know that this debilitating and life-threatening disease can severely impact the lives of patients and to date existing treatments only focus on the relief and management of symptoms, whereas FT011 precisely targets the root cause of fibrosis and has the potential to offer treatment across multiple organs within these patients.”

The trial recruited a total of 30 adults who were randomly assigned to three treatment arms - FT011 400mg or FT011 200mg or placebo daily, in addition to standard of care, for 12 weeks.

The company said an open-label extension phase of the trial is ongoing where a subset of patients who completed the main study have elected to remain on treatment (FT011 400 mg) for an additional nine months.

Dr Wendy Stevens is a leading rheumatologist at St Vincent’s Hospital in Melbourne and a principal investigator in the FT011.

“The trial demonstrated that FT011 was safe and well tolerated.  Scleroderma can lead to severe and life-threatening issues and patients with the condition often face daily struggles as a result of their symptoms. These results are a significant step towards helping patients with this debilitating disease. Further longer and larger trials of this medication are now needed to assess its potential to improve this debilitating condition,” said Dr Stevens.