ENYO Pharma has secured €39m Series C funding to advance Vonafexor, a synthetic non-steroidal, non-bile acid nuclear receptor subfamily 1 group H member 4 agonist, for the treatment of Alport syndrome.

Alport syndrome is a genetic condition characterised by kidney disease, hearing loss and eye abnormalities.

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Co-led by OrbiMed and Morningside Ventures, the financing round will support the Phase II Alpestria-1 clinical trial and further research and development activities of Vonafexor through to the first half of 2026.

Existing investors including AndEra Partners, Bpifrance InnoBio and Bpifrance Large Ventures also participated in the financing round.

The funding will also facilitate the continued profiling of the therapy for other kidney conditions such as autosomal dominant polycystic kidney disease.

ENYO Pharma also received clearance from the US Food and Drug Administration for its investigational new drug application paving the way for the Phase II clinical study of Vonafexor in Alport syndrome.

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The ALPESTRIA-1 trial is set to commence in the first half of 2024.

Vonafexor is being developed as a once-daily oral treatment with potent fibrolytic and anti-inflammatory properties.

It has shown promise in improving renal function in the Phase II LIVIFY study involving patients with kidney impairment and fibrotic liver disease.

Preclinical studies have also indicated its potential benefits in kidney remodelling and function in Alport syndrome and chronic kidney disease mouse models.

ENYO Pharma CEO Dr Jacky Vonderscher stated: “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.

“The results already obtained with Vonafexor, our highly differentiated anti-inflammatory and fibrolytic lead compound, in patients with moderate kidney impairment and several preclinical protocols, make us confident that it will greatly benefit those patients with rare kidney diseases like Alport syndrome.”

Last year, the company received orphan drug designation from both the European Medicines Agency and the US Food and Drug Administration for Vonafexor.

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