Biotechnology firm Promethera Biosciences has received authorisation in Belgium to conduct a Phase IIb/III trial (HEP002) of HepaStem to treat paediatric patients with urea cycle disorders (UCD).
The company is developing HepaStem, a cell-based therapy, to treat both orphan liver-based metabolic diseases and acquired liver diseases.
In the prospective, open label, multicentre efficacy and safety HEP002 trial, patients will be enrolled and treated in Cliniques Universitaires Saint-Luc in Brussels.
These rare diseases have limited therapeutic options and affect most young patients who can die at an early age.
UCD are a group of eight inborn errors of metabolism that affect the transfer of nitrogen into urea, with an incidence between one in 8,000 and 1 in 44,000.
Hyperammonemia and hyperglutaminemia are the common biochemical hallmarks of these disorders.
The primary objective of the HEP002 trial is to evaluate the efficacy of HepaStem in patients with urea cycle deficiencies during the year following treatment.
Promethera Biosciences chief medical officer Dr Béatrice de Vos said: "We are excited to pursue the development of this promising disease-modulating therapy. It is urgently needed by the children living with these disabling metabolic diseases."
In the first clinical trial, HEP001 Promethera HepaStem showed to be a safe treatment that can result in functional metabolic improvement in UCD patients.
Promethera Biosciences founder and chief scientific officer Etienne Sokal said: "This is a new important achievement in the process of transferring original clinical research to the industry and giving access to this innovative therapy to the maximum number of children."
The company is currently completing a multicentre Phase I/II HEP001 trial in five countries including Belgium, France, UK, Italy and Israel, involving 20 patients suffering from Crigler-Najjar syndrome and urea cycle disorders and treated with HepaStem.