- Felzartamab, an investigational anti-CD38 monoclonal antibody, is a potentially differentiated therapeutic candidate with promise for a broad range of immune-mediated diseases
- Biogen is initiating three pivotal Phase 3 studies of felzartamab across rare kidney indications in 2025, with first readout expected in 2027
CAMBRIDGE, Mass. – Biogen Inc. (Nasdaq: BIIB) will host a virtual investor seminar today at 10:00 a.m. ET focused on the potential of the investigational drug felzartamab in rare kidney diseases and the potential to target CD38 for a range of immune-mediated diseases. Since 2023, Biogen has worked to transform its portfolio, building upon its legacy in multiple sclerosis and neuroscience to expand in immunology and rare diseases, where the company has well-established global development commercialization capabilities.
“We have made important progress to support our long-term growth objective, build a strong portfolio with momentum from our recently launched products, and advance an innovative and compelling pipeline,” said Christopher A. Viehbacher, President and Chief Executive Officer at Biogen. “We are particularly excited about the addition of felzartamab, which gives us the potential to deliver novel treatments across a range of immune-mediated diseases, beginning with three rare kidney diseases.”
Immune-mediated diseases occur when antibodies produced by the immune system mistakenly attack the body’s own tissues. These antibodies are produced mainly by plasmablasts and plasma cells that express CD38. Biogen believes that by selectively targeting CD38-expressing plasma cells, the source of these autoantibodies, it may be possible to more precisely modify the disease course in this class of conditions. Felzartamab has been shown in clinical studies to selectively deplete CD38+ cells, including plasma cells and natural killer, or NK, cells and has established proof of concept in three rare kidney diseases. Biogen plans to investigate felzartamab in three Phase 3 pivotal studies, two of which have already been initiated.
Late antibody-mediated rejection (AMR) in kidney transplant recipients
AMR is a leading cause of kidney loss after receiving a transplant. This disease is driven by antibodies to antigens on the donor cells, as well as by damage from natural killer cells that causes microvascular inflammation in the transplanted kidney. Today there are no approved treatment options for AMR.1
In a positive Phase 2 study, 82% (n=9/11) of patients who received felzartamab experienced resolution of AMR at week 24, compared to 20% (n=2/10) who received placebo. In patients with late AMR, most adverse events were mild or moderate in severity. Mild or moderate infusion reactions, typically on the first infusion, occurred in patients in the felzartamab group (n=8). There were no treatment-related discontinuations.
Biogen initiated a pivotal global Phase 3 study in AMR, TRANSCEND, in March 2025. This Phase 3 study (NCT06685757) will evaluate the efficacy and safety of felzartamab compared to placebo in adult kidney transplant recipients diagnosed with late antibody-mediated rejection (AMR). TRANSCEND is designed to enroll approximately 120 kidney transplant recipients with late AMR.
In the U.S., felzartamab has received Breakthrough Therapy Designation (BTD) for the treatment of late AMR without T-cell mediated rejection in kidney transplant patients and Orphan Drug Designation (ODD) for development in the treatment of AMR in kidney transplant recipients from the U.S. Food and Drug Administration (FDA). In Europe, felzartamab has received ODD for treatment in solid organ transplantation from the European Medicines Agency (EMA).
Immunoglobulin A nephropathy (IgAN)
IgAN is the most common type of primary glomerulonephritis worldwide. Up to 40% of IgAN patients progress to kidney failure within 20 years after diagnosis, which can lead to the need for dialysis or a kidney transplant.2 It occurs when an abnormal immunoglobulin protein deposits in the filtering unit (glomerulus) inside the kidneys, leading to inflammation and progressive kidney damage. It is believed that the damage is caused by immune complexes, molecules formed from the binding of antigens to antibodies, which are believed to be produced by CD38 positive plasma cells and plasmablasts. Most patients who get this disease are typically diagnosed in the second and third decade of life and while treatments are available, there is a real need for a novel therapy that provides durable disease remission.
In October 2024, Biogen presented complete and encouraging results from the Phase 2 IGNAZ study (n=54) evaluating felzartamab in people living with IgAN. The results showed reductions in proteinuria levels as assessed by the urinary protein: creatinine ratio (UPCR) and stabilization of kidney function. Notably, patients maintained a mean reduction from baseline of approximately 50% in the UPCR through month 24, which was more than 18 months after the last dose was administered. Overall, administration of felzartamab had a safety profile consistent with prior studies.
Today, Biogen announced the first patient was dosed in the pivotal global Phase 3 study, PREVAIL. The Phase 3 study (NCT06935357) will evaluate the efficacy and safety of felzartamab compared to placebo on proteinuria and preservation of kidney function in adults diagnosed with IgAN. PREVAIL is designed to enroll approximately 454 patients worldwide.
In Europe, felzartamab has received ODD for the treatment of IgAN from the EMA.
Primary membranous nephropathy (PMN)
PMN is a severe antibody-mediated disease of the kidney that is a leading cause of nephrotic syndrome and carries a significant risk of kidney failure. Patients with nephrotic syndrome often present with very severe swelling and fatigue related to high-grade proteinuria, and they are also at an increased risk of infection. Importantly for felzartamab, it is estimated that up to 80% of PMN patients have autoantibodies against PLA2R (aPLA2R) generated by CD38-expressing plasma cells. There are no approved treatments for PMN and the current standard of care includes treatments ranging from immunosuppressants to chemotherapy.3
Two Phase 2 studies, M-PLACE (n=31) and NewPLACE (n=24), enrolled patients with aPLA2R-positive PMN. In the final analysis of M-PLACE, reductions in aPLA2R titers were observed in most patients as early as one week (median reduction of 45%), with responses (>50% reduction) in most patients at six months at end-of-treatment. In addition, improvements in proteinuria and serum albumin levels were observed with administration of felzartamab. Across both studies, the majority of treatment emergent adverse events (TEAEs) reported were mild to moderate and consistent with the known mechanism of action of felzartamab in the PMN population. The most common TEAE was infusion-related reactions on the first infusion that were mostly mild to moderate in intensity.
Biogen plans to initiate dosing in the pivotal global Phase 3 study, PROMINENT, in 2025. The Phase 3 study (NCT06962800) will evaluate the efficacy and safety of felzartamab compared to tacrolimus in adults diagnosed with PMN. PROMINENT is designed to enroll approximately 180 patients worldwide.
In the U.S., felzartamab has received BTD and ODD for development in the treatment of PMN from the FDA.
Potential Indication Expansion
Biogen is evaluating additional expansion indications for felzartamab. Biogen believes the selective targeting of CD38 positive immune cells may provide felzartamab with a potentially differentiated profile across other indications. One example is lupus nephritis (LN), where the company has an ongoing Phase 1 trial. The first data from the LN trial (NCT06064929) is expected in 2026 and will inform the potential for a registrational study.
West Coast Innovation Hub
Biogen obtained felzartamab through the acquisition of Human Immunology Biosciences (HI-Bio) in 2024 as part of the expansion of its immunology portfolio. Biogen established the West Coast Innovation Hub upon the acquisition of HI-Bio. Biogen was able to retain the vast majority of HI-Bio’s employees who are now part of this new innovation hub, which is focused on expanding Biogen efforts in immune-mediated diseases.
About Felzartamab
Felzartamab is an investigational therapeutic human monoclonal antibody directed against CD38, a protein expressed on mature plasma cells. Felzartamab is a potential first-in-class therapeutic candidate with promise as a pipeline-in-a-product across a range of immune-mediated diseases. Felzartamab has been shown in clinical studies to selectively deplete CD38+ plasma cells, which may allow applications that ultimately improve clinical outcomes in a broad range of diseases driven by pathogenic antibodies. Felzartamab was originally developed by MorphoSys AG (now MorphoSys GmbH, a Novartis company). Human Immunology Biosciences (HI-Bio) exclusively licensed the rights to develop and commercialize felzartamab across all indications in all countries and territories excluding China (including Macau and Hong Kong and Taiwan). Biogen acquired HI-Bio in July 2024.
Felzartamab is an investigational therapeutic candidate that has not yet been approved by any regulatory authority and its safety and effectiveness have not been established.
About Biogen
Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients’ lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth.
We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media – Facebook, LinkedIn, X, YouTube.
References
- Schinstock et al. (2018) Kidney Transplant with Low Levels of DSA or Low Positive B-Flow Crossmatch: An Underappreciated Option for Highly-Sensitized Transplant Candidates (Page 8). Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5481511/pdf/nihms837168.pdf#page=8; Ciancio et al. 2018 Antibody-Mediated Rejection Implies a Poor Prognosis in Kidney Transplantation: Results From a Single Center. Available at: https://onlinelibrary.wiley.com/doi/10.1111/ctr.13392
- Yexin Liu, et. Al, Prediction of ESRD in IgA Nephropathy Patients from an Asian Cohort: A Random Forest Model. Kidney Blood Press Res 20 December 2018; 43 (6): 1852–1864
- Rajasekaran et al. (2021) IgA nephropathy: An interesting autoimmune kidney disease. Available at https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5198292/.Hastings et al (2018) Clinical Research, Life Expectancy for Patients From the Southeastern United States With IgA Nephropathy. Available at https://www.kireports.org/article/S2468-0249(17)30362-5/fulltext
Media Contact
Jack Cox
+ 1 781 464 3260
Investor Contact
Tim Power
+1 781 464 2442