New Medicines

Orbcel-CPrimary sclerosing cholangitis


New molecular entity
Orbsen Therapeutics
Orbsen Therapeutics

Development and Regulatory status

Phase II Clinical Trials


An allogeneic CD362 enriched mesenchymal stromal cells, isolated from human umbilical cord
PSC is a rare disease with a prevalence of 0.2-16 per 100,000. PSC commonly affects males with a median age at diagnosis of 35 years. PSC accounts for 10% of all UK liver transplants [1].
Primary sclerosing cholangitis
Intravenous infusion

Trial or other data

Apr 22PIIa basket POLARISE trial is recruiting at the Queen Elizabeth Hospital in Birmingham and Nottingham University Hospital. The trial started in Jan 22 and aims to investigate the safety and activity of the use of ORBCEL-C™ in the treatment of adults with primary sclerosing cholangitis, rheumatoid arthritis, lupus nephritis and Crohns disease. Consenting patients will receive two doses of ORBCEL C, containing a total of 200 million cells. This will be the first time this form of ORBCEL C has been used in a clinical trial. Primary outcome is the relative proportion of M1- and M2-like monocyte/macrophage cells in peripheral blood, determined from cell counts quantified using flow cytometry, after ORBCEL-C™ administration. The trial is due to complete Sep 25 [3].
Feb 19PIIa MERLIN trial is recruiting; collection of these data is due to complete Dec 21 [2].
Dec 18University of Birmingham, in collaboration with European Union, initiate the PIIa MERLIN trial to assess the safety and efficacy of ORBCEL-C for primary sclerosing cholangitis and autoimmune hepatitis (NCT02997878; RG-13-124). The open-label, non-randomised trial intends to enrol 56 patients in the UK at University hospitals Birmingham NHS foundation Trust. The trial has two main stages: Stage 1 will determine the maximum tolerated dose that can be administered by observing for occurrence of dose limiting toxicity (DLT); Stage 2 will use the maximum tolerated dose found in stage 1 to and further determine safety and activity outcomes of Orbcel-C. Orbcel-C will be given in a single dose of 0.5, 1.0,2.5 million cells/kg (3 dose levels) as an IV single-infusion. Primary outcomes are adverse events and change in ALP and ALT [2].