Enyo Raises $42.5 Million to Advance Development of Experimental Alport Syndrome Therapy

Rare Daily Staff

Enyo Pharma announced a first closing of a $42.5 million (€39 million) series C financing as it said the U.S. Food and Drug Administration cleared it to begin a phase 2 clinical study of its experimental therapy Vonafexor for the treatment of the rare genetic condition Alport Syndrome that affects the kidneys.

OrbiMed and Morningside Ventures co-lead the financing with participation from other existing investors including AndEra Partners, Bpifrance InnoBio and Bpifrance Large Ventures.

The financing will support the phase 2 Alpestria-1 study and further profiling of Vonafexor in other kidney diseases, such as Autosomal Dominant Polycystic Kidney Disease (ADPKD).

The clearance for Enyo to begin its clinical trial represents a significant milestone for the company as it embarks on its first clinical trial focusing on renal disease. Enyo’s lead candidate Vonafexor, as well as its EYP651, are highly specific FXR agonists given as once-daily oral treatments. Both compounds have strong fibrolytic and anti-inflammatory properties that are broadly applicable across several renal diseases.

Vonafexor effect on renal function (eGFR) was already shown in the phase 2 LIVIFY study of patients with both kidney impairment and fibrotic liver disease. Preclinical studies in both Alport syndrome and chronic kidney disease mouse models showed beneficial effects of Vonafexor on kidney remodeling and function in a curative mode. In 2023, Vonafexor was granted as Orphan Drug designation by both the FDA and the European Medicines Agency for Alport syndrome.

“There are huge unmet needs in many kidney diseases with fibrosis or inflammatory components. These patients almost invariably progress to end-stage kidney disease despite current therapies,” said Jacky Vonderscher, CEO at Enyo Pharma. “The results already obtained with Vonafexor, our highly differentiated anti-inflammatory and fibrolytic lead compound, in patients with moderate kidney impairment and in several preclinical protocols, make us confident that it will greatly benefit to those patients with rare kidney diseases like Alport syndrome.”

Photo: Jacky Vonderscher, CEO at Enyo Pharma