Albireo Pharma is planning to initiate a Phase III clinical programme of its product candidate A4250 in the second half of this year for the treatment of patients with progressive familial intrahepatic cholestasis (PFIC).
A4250 is a first-in-class, potent and selective ileal bile acid transporter (IBAT) inhibitor.
Following its discussions with the US Food and Drug Administration (FDA) and European Medicines Agency (EMA), the firm established a study design for the Phase III programme.
The programme will include a single, randomised, double-blind, placebo controlled, multi-centre trial and a long-term extension study.
Albireo president and chief executive officer Ron Cooper said: “We are pleased with the collaborative and constructive dialogue we have had for A4250 with regulatory authorities in the US and Europe, which has guided us as we work to develop our investigational drug for children suffering from PFIC.
“A4250 is a potent IBAT inhibitor that, with Phase III success and regulatory approval, could potentially transform the treatment paradigm for what is a life-altering disease.”
The Phase III trial will evaluate 40mcg/kg a day (d) or 120mcg/kg/d of A4250 in 60 patients aged six months to 18 years at centres in the US, Canada, Western Europe, the Middle East and Australian for six months.
The trial’s FDA primary endpoint and main EMA secondary endpoint is change in pruritus, while the EMA primary endpoint and FDA key secondary endpoint is serum bile acid (sBA) responder rate.
Secondary endpoints such as progression to surgery, change in growth markers and liver biochemistry variables will also be evaluated during the treatment period.
The open-label extension study will examine the long-term safety and durability of response.
Results from the Phase III programme, along with the existing data on A4250, are expected to support the drug approval applications in the US and EU.