Wednesday, November 01, 2023
Human Immunology Biosciences (HI-Bio™), a clinical-stage biotechnology company, has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for felzartamab, a drug in development for the treatment of primary membranous nephropathy (PMN), a rare autoimmune kidney disease. This designation acknowledges the promising data obtained thus far and the pressing need for more effective therapies for PMN patients.
Felzartamab's Breakthrough Therapy designation is a result of its potential to significantly improve the treatment of PMN, a condition characterized by thickening of the glomerular basement membrane in the kidneys due to the deposition of immune complexes. The drug's mechanism of action involves targeting CD38+ plasma cells, which produce harmful antibodies in PMN and other immune-mediated diseases. HI-Bio is also exploring felzartamab's use in conditions such as IgA nephropathy, antibody-mediated rejection, and lupus nephritis.
Breakthrough Therapy designation expedites the development and review of drugs aimed at serious or life-threatening conditions with promising clinical evidence for substantial improvements over existing therapies. It allows for increased regulatory interactions to ensure the collection of necessary data for drug approval, and it provides the potential for accelerated approval, priority review, and rolling review of a Biologics License Application (BLA).
The FDA's decision was based on clinical data, including results from the M-PLACE study, a Phase 1b/2a proof-of-concept open-label trial. The final analysis of this study has been accepted for presentation at the American Society of Nephrology (ASN) Kidney Week 2023 Annual Meeting.
PMN is a rare autoimmune kidney disease and a leading cause of nephrotic syndrome in adults. It typically manifests between the ages of 40 and 50, presenting with symptoms like edema, significant proteinuria, and hypoalbuminemia. Most cases of PMN involve autoantibodies targeting the phospholipase A2 receptor (PLA2R) antigen expressed on podocytes.
Currently, there are no approved therapies for PMN, and the standard of care involves off-label use of supportive measures, conventional immunosuppressive treatments, or B-cell depleting agents. These treatments are not universally effective and are associated with significant toxicity.
Felzartamab is an investigational human monoclonal antibody that targets CD38, a protein expressed on mature plasma cells. By selectively depleting CD38+ plasma cells, felzartamab has the potential to improve outcomes in a range of autoantibody-driven diseases. It received Orphan Drug Designation (ODD) from the FDA for PMN in May, based on promising data from Phase 2 studies.
HI-Bio in-licensed felzartamab from MorphoSys in June 2022 and holds worldwide rights, except for Greater China. In 2017, MorphoSys entered into a licensing agreement with I-Mab Biopharma for felzartamab's development in Greater China, where it is being evaluated for use in oncology and autoimmune diseases.
It is important to note that felzartamab is an investigational drug and has not yet received approval from regulatory authorities.