Mirum Pharmaceuticals has secured $120 million in Series A financing to support the development of its lead drug candidate maralixibat for rare cholestatic liver diseases.
The financing was led by New Enterprise Associates (NEA) with participation from Deerfield Management, Frazier Healthcare Partners, Novo Holdings A/S, Pappas Capital, RiverVest Venture Partners and Rock Springs Capital.
Mirum has also entered into an agreement with Shire for the exclusive global rights to develop and market maralixibat, an oral inhibitor of the apical sodium dependent bile acid transporter (ASBT).
Shire will receive an upfront payment, an equity position in Mirum and potential future milestone payments and royalties. Maralixibat is being developed for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).
Maralixibat is under investigation to evaluate its ability to prevent accumulation of excess bile acids and control extreme itching associated with cholestatic liver diseases such as ALGS and PFIC.
Maralixibat could be a first-in-class oral drug for these conditions, pending regulatory authority approval.
While Mirum plans to initially focus its efforts in these two paediatric indications, there is also potential to develop it for additional paediatric and adult cholestatic liver disease indications.
Mirum plans to initiate Phase 3 confirmatory studies in patients with ALGS and PFIC in 2019.