This major milestone was achieved with the last-patient-in dosed ahead of schedule. The study timelines were accelerated thanks to a tailored strategy based on a very strong and effective collaboration with our partners Natera and Accelsiors, all clinical sites in the US, France ans Spain with investigators,

ALPESTRIA-1 evaluates the safety and the effects of Vonafexor on kidney function in subjects with at risk of progression Alport syndrome. The total duration of study for a participant will be up to 40 weeks and include a screening period, a treatment period of 24 weeks and a follow-up period of 12 weeks.

The ALPESTRIA-1 Phase 2 clinical study () is an open-label, single arm, fixed dose escalation of the investigational drug Vonafexor. This study is a proof-of-concept trial of Vonafexor safety, its effects on kidney function in subjects with at risk of progression Alport syndrome. The study aims to recruit 20 patients in the US and EU (France, Spain and Germany).

The first site for patient recruitment is now open in Idaho (US) and more sites will open in the coming weeks.

Alport syndrome is a rare kidney disease, a genetic disorder affecting 1 in 5,000-10,000 live births. It is characterized by a progressive loss of kidney function and may result in hearing loss and eye abnormalities.

This article describes the remarkable effects of Vonafexor after only 12 weeks of treatment in these patients: strong liver fat reduction, weight loss, liver enzyme improvements …., and last but not least renal function recovery. These strongly differentiated fibrolytic and anti-inflammatory effects of Vonafexor compared to other FXR agonists correlate very well with those observed in several preclinical models of severe CKD and rare kidney diseases like Alport Syndrome.