Fibrotech Therapeutics Pty Ltd.

A quiet biotech is reporting some positive data in a rare fibrotic disease. Certa Therapeutics — based in Melbourne, Australia — reported Monday that a Phase II trial showed “significant improvements across multiple efficacy measures” in patients with scleroderma, such as a systemic sclerosis index and skin scores. The main goal of the study, per Certa, was to examine the pharmacokinetics, pharmacodynamic effects and safety of FT011 in patients with scleroderma, a rare autoimmune disease that can cause inflammation in the skin and other parts of the body, like the lungs. Also known as crest syndrome, it has been associated with chronic hardening and tightness of the skin. But Certa also tracked a number of efficacy measures to see if the drug works. The trial recruited 30 adults that were randomly assigned to either a placebo group or one of two dose groups: 200mg or 400mg orally daily for 12 weeks. After those 12 weeks, Certa saw “clinically meaningful improvement in 60 percent of patients” treated with the 400mg dose of the therapy in a Phase II study, adding that it saw a p-value of p=0.019 versus placebo. Investigators also looked at a metric called a CRISS score, which predicts a patient’s chances from 0 to 1 of improvement based on five variables, including skin thickness and lung function. Three patients who were on FT011 achieved the maximum CRISS score of 1, Certa added. The biotech is running an open-label extension study for an additional nine months after the initial 12-week study. CEO Darren Kelly said in a statement that the readout “paves the way for a confirmatory global Phase III study.” A query to the Australian biotech was not immediately returned to Endpoints News . FT011 was originally developed by a company called Fibrotech, which had taken the drug through a Phase Ia study in diabetic neuropathy. Shire then acquired Fibrotech in 2014 when it bought the company for $75 million plus up to $600 million milestones — before Takeda bought out Shire in 2019. Certa, led by several former Fibrotech execs, including ex-CEO Kelly, then picked up the drug and was able to start a Phase II trial in 2021. Certa is not the only biotech that has been working on the rare disease. Cell and gene therapy biotech Castle Creek Biosciences has been working on FCX-013, a gene therapy for moderate and severe forms of scleroderma. Boehringer Ingelheim’s Ofev is also approved for interstitial lung disease associated with systemic sclerosis or scleroderma.

Certa CEO and founder Darren Kelly (Medical Research Commercialisation Fund) Certa Therapeutics’ attempt to resurrect an ex-Shire drug has delivered midphase data. Nine years after Shire paid $75 million to acquire FT011, the Australian biotech has linked the candidate to improvements in the symptoms of a rare autoimmune disease in a small clinical trial. FT011 is a derivative of tranilast, an anti-fibrotic agent used in the treatment of scars and skin disorders. By improving on the potency of tranilast, while dialing down its cellular toxicity, Australian researchers tried to create a molecule capable of treating diseases including diabetic nephropathy. The work led to the formation of Fibrotech and later its acquisition by Shire for $75 million plus milestones. Shire subsequently walked away from FT011, enabling some of the people behind Fibrotech to form Certa and have a second crack at developing the molecule. Certa picked scleroderma, a rare autoimmune disease associated with thickened areas of skin, as its lead indication and entered phase 2 in 2021.  Investigators randomized 30 patients to receive one of two oral daily doses of FT011 or placebo for 12 weeks. While the primary endpoints looked at levels of two dose regimens of the molecule in plasma, Certa’s statement focused on the measures of efficacy analyzed across multiple secondary endpoints.  According to Certa, 60% of patients in the high-dose arm had a “clinically meaningful improvement,” resulting in a p-value versus placebo of 0.019. Certa defined a score of 0.6 or greater on the 0 to 1 CRISS scale as clinically meaningful. Twenty percent of participants in the low-dose cohort experienced such an improvement. Three patients across the two FT011 arms scored 1.0 on the scale. Certa also reported improvements on a skin score, a disability index and the global physician assessment, leading CEO and founder Darren Kelly to give a bullish take on the data and the prospects of FT011.  “These exceptional trial results demonstrate the potential of this novel treatment for patients with scleroderma,” Kelly said in a statement. “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 3 study.” The phase 3 trial will provide a sterner test of FT011. Success would position Certa to address an unmet medical need. Boehringer Ingelheim’s Ofev and Roche’s Actemra are used in the treatment of interstitial lung disease linked to scleroderma, but other therapies are needed. Horizon Therapeutics is among the other companies trying to address the unmet need.