Phase 2 trial of orelabrutinib for NMOSD underway in China
Researchers testing the safety, effectiveness of InnoCare Pharma's experimental oral med
The trial (NCT05284175), being conducted at the Peking Union Medical College Hospital, in Beijing, China, was cleared by the country’s regulatory agency last year. InnoCare announced in a recent press release that the study is ongoing.
In NMOSD, the immune system launches an inflammatory attack that damages healthy cells in the brain and spinal cord. This attack is driven in large part by self-reactive antibodies, particularly those against AQP4, a protein found at the surface of neuron-supporting cells.
Orelabrutinib is designed to reduce the inflammatory activity of B-cells, the immune cells responsible for producing antibodies. It specifically works by blocking a protein called Bruton tyrosine kinase or BTK, which B-cells require in order to activate and survive.
As such, the therapy is expected to lower antibody production, ultimately dampening the autoimmune attack that drives NMOSD.
The Phase 2 trial is evaluating the safety and effectiveness of orelabrutinib in up to 23 adults with NMOSD who are positive for anti-AQP4 antibodies. Participants also must have experienced at least two NMOSD attacks in the year prior and at least one in the previous six months.
All participants are being treated with orelabrutinib at a dose of 50 mg once per day, for up to 48 weeks, or nearly one year.
The main goal of the study, sponsored by Peking Union Medical College Hospital and conducted in collaboration with InnoCare, is to evaluate changes in the rate of NMOSD attacks. Secondary goals include safety measures, the proportion of patients free of relapses, and changes in disability, vision, and health-related quality of life.
The researchers also will measure blood levels of anti-AQP4 antibodies, B-cells, and a marker or nerve cell damage.
Orelabrutinib studied in other diseases
In addition to NMOSD, InnoCare is developing orelabrutinib also as a treatment for a range of other disorders that are mediated by B-cells. The therapy is being tested in clinical trials for other autoimmune disorders like multiple sclerosis and lupus, as well as some blood cancers that are driven by the uncontrolled growth of B-cells.
Orelabrutinib was approved earlier this year in China [here] as a treatment for hard-to-treat marginal zone lymphoma, a type of B-cell cancer.