FT011 showed promising effects on disability, lung function in trial

Efficacy, safety data measured during Phase 2 testing for experimental therapy

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Clinical trial results are shown with a bar graph, with a stethoscope, pills and hands surrounding the graph.

Treatment with FT011 led to clinically meaningful improvements in physical disability and lung function for some scleroderma patients given the experimental therapy in a Phase 2 clinical trial.

Christopher Denton, MD, a professor at University College London, shared the results at the American College of Rheumatology (ACR) Convergence 2023 annual meeting, in a presentation titled, “FT011 for the Treatment of Systemic Sclerosis. Results from a Phase II Study.”

“The Phase 2 trial demonstrates promising efficacy and safety data for FT011 after 12 weeks of treatment and certainly warrants a further study to assess the potential of FT011 to improve clinical outcomes for scleroderma patients. These results are very encouraging for the scleroderma community and a significant step towards helping patients with this debilitating disease,” Denton said in a press release.

The work was funded by Certa Therapeutics, the company developing FT011.

“We are pleased to have these exceptional clinical trial results presented to the scientific community at ACR Convergence, with FT011 demonstrating clinically important differences in multiple efficacy measures on top of standard of care in a short treatment timeframe,” said Darren Kelly, PhD, founder and CEO of Certa.

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Scleroderma, also called systemic sclerosis or SSc, is marked by abnormal scarring (fibrosis) that can affect the skin and internal organs. FT011 is designed to block the activity of a protein receptor that’s involved in fibrosis called GPR68 (G protein-coupled receptor 68). The experimental therapy was recently named an orphan drug in the U.S.

“As the biological mechanism by which FT011 works precisely targets the root cause of fibrosis, we believe that FT011 is notably differentiated from previously unsuccessful clinical candidates,” Kelly said.

Certa sponsored a Phase 2 clinical trial (NCT04647890) that enrolled 30 adults with SSc. The mean age of the participants was 51 years, about three-quarters were women, and all were white.

Participants were given FT011 at one of two daily doses (200 or 400 mg) or a placebo, in addition to standard treatments, for about three months.

Data show improvements for those taking higher dose of FT011

Top-line results announced earlier this year showed that six of the 10 patients given the higher dose of FT011 reported clinically significant improvements, as measured by the American College of Rheumatology Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (ACR-CRISS) score. By comparison, only one patient in the placebo group experienced a meaningful improvement in ACR-CRISS score.

The mean ACR-CRISS score was significantly higher in the high-dose FT011 group compared with the placebo group (0.542 vs. 0.131 points), suggestive of less severe disease for patients given the experimental treatment.

More than half (60%) of patients given high-dose FT011 experienced clinically meaningful improvements in the Scleroderma Health Assessment Questionnaire-Disability Index, an assessment of physical disability. The percentage of patients on placebo with such index improvement was 22%.

Five of the 10 patients given the higher dose of FT011 experienced clinically significant improvements in Forced Vital Capacity, a measure of lung function based on how much air someone can blow out in a forceful breath. By comparison, none of the patients given a placebo reported notable improvements in the same measure.

Patient-rated measures of health tended to favor FT011 over the placebo, but differences did not reach statistical significance. This study was primarily designed to test the pharmacological properties and safety profile of FT011; future larger studies will be needed to verify the experimental treatment’s efficacy.

Safety data from the Phase 2 study were overall positive: no serious safety issues were reported, and overall rates of safety-related events were similar for patients given FT011 or a placebo. Collectively the findings “warrants a confirmatory phase III study to assess [FT011’s] potential,” the researchers wrote in their abstract.

“It is imperative that effective, safe, and well-tolerated therapeutics are efficiently developed, and which are truly beneficial for a scleroderma patient’s quality of life. … With the benefit of input from world-leading experts in the field, we are highly focused on advancing the clinical development program for FT011 towards the pivotal efficacy study,” Kelly said.