FT011 safely eases SSc symptoms, aids lung health in Phase 2 Trial

Clinically meaningful gains in patients given the scleroderma treatment vs placebo

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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FT011, a treatment candidate from Certa Therapeutics, was safe and led to clinically meaningful improvements for more than 60% of systemic sclerosis (SSc) patients in a Phase 2 trial.

After 12 weeks of treatment, patients given the investigational therapy showed gains in lung function and in physician- and patient-reported health outcomes, as well as reductions in skin thickness.

“These exceptional trial results demonstrate the potential of this novel treatment for patients with scleroderma,” Darren Kelly, PhD, CEO and founder of Certa and a professor and director of biomedical research at the University of Melbourne, Australia, said in a company press release.

Patients’ positive response also “paves the way for a confirmatory global Phase III study,” he said.

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FT011 designed to target root cause of fibrosis

Fibrosis occurs when normal wound-healing processes grow out of control, leading to inflammation and scar tissue buildup. It’s one of the key features of SSc, where fibrosis is observed in the skin, but can also develop systemically in the internal organs.

Certa reported that its potential antifibrotic therapies target a receptor linked to signaling pathways that promote fibrosis. While the receptor is normally silent, it is activated after injury or during disease.

FT011, an oral first-in-class treatment, is designed to inhibit activity at this receptor.

“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,” Kelly said.

FT011 demonstrated efficacy in preclinical models of fibrotic diseases, and it has shown ability to reverse activation of genetic markers of fibrosis, Certa previously reported. It’s also demonstrated a good safety profile in Phase 1 studies.

The Phase 2 trial (NCT04647890) aimed to investigate FT011’s safety, pharmacokinetics (movement into, through and out of the body), and pharmacodynamics (effects on the body) in 30 adults with diffuse cutaneous SSc, ages 18 to 75, who were randomly assigned to either oral FT011 at 200 or 400 mg or to a placebo once daily for 12 weeks, in addition to standard of care treatments.

FT011 was safe and well-tolerated, Certa reported, and its use led to significant improvements across efficacy measures compared with placebo.

Patients report easing in pain and finger ulcers, better breathing

Specifically, FT011-treated patients saw clinical improvements, as assessed by the American College of Rheumatology Combined Response Index in diffuse cutaneous Systemic Sclerosis (ACR-CRISS) score, a composite measure of disease severity and organ damage that’s commonly used in SSc clinical trials.

ACR-CRISS predicts a patient’s probability of clinical improvement, taking into account five clinical components including modified Rodnan skin scores (mRSS; a measure of skin fibrosis), lung function, physician-reported outcomes, patient-reported outcomes, and a measure of functional disability. Scores range from 0–1, with any score of at least 0.6 indicating a clinically meaningful probability of improvement.

Results showed that 60% of patients treated at the 400 mg FT011 dose experienced clinically meaningful improvements, as did 20% of the 200 mg FT011 group. Compared with placebo, both groups exhibited clinically meaningful improvements as early as eight weeks after starting treatment, reaching maximum gains at week 12.

Across all FT011-treated patients, three people achieved a CRISS score of 1, representing the highest possible probability of clinical improvement.

Lower mRSS scores also were reported at either dose of FT011, reflecting a reduction in skin thickness, as was better lung function.

Both patient- and physician-reported measures of health status showed significant gains among patients in 400 mg treatment group compared with the placebo group over the 12 weeks. Patients specifically reported an easing in pain symptoms, breathing problems, finger ulcers, and overall disease severity.

Rates of side effects were similar between treatment groups, and no serious adverse events were reported.

Some participants elected to continue treatment in an open-label extension phase of the trial, in which all are receiving the 400 mg dose of FT011 for nine months.

“These results are a significant step towards helping patients with this debilitating disease,” said Wendy Stevens, MD, the study’s principal investigator and a rheumatologist at St. Vincent’s Hospital, Melbourne. “Further longer and larger trials of this medication are now needed to assess its potential to improve this debilitating condition.”