Ex-Shire drug improves outcomes in autoimmune disease, setting Certa on path to phase 3

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.