Proteinuria

The detailed study results confirmed interim findings, showing stable kidney function and sustained treatment effect more than 18 months after the last dose of felzartamab Felzartamab, an investigational anti-CD38 monoclonal antibody, is a potential first-in-class therapeutic candidate for a range of rare immune-mediated indications with planning underway for Phase 3 development IgA Nephropathy (IgAN) is a leading cause of chronic kidney disease with up to 40% of IgAN patients progressing to end stage kidney disease about 20 years after diagnosis Oct. 26, 2024 -- Biogen Inc. (Nasdaq: BIIB) – today presented complete results from the Phase 2 IGNAZ study evaluating felzartamab, an investigational anti-CD38 monoclonal antibody, in people living with IgA nephropathy (IgAN). The results showed substantial reductions in proteinuria, stabilization of kidney function, and sustained treatment effect more than 18 months after the last dose of felzartamab. The complete results were shared during an oral presentation at Kidney Week 2024, the American Society of Nephrology’s annual meeting, in San Diego, California. "The complete results of the IGNAZ Study reaffirm our interim findings, showing a reduction in proteinuria, stabilization of kidney function, and sustained treatment effect more than 18 months after the last dose of felzartamab,” said Jonathan Barratt, MD, PhD, FRCP, Mayer Professor of Renal Medicine at the University of Leicester. “This is promising news for patients and supports the potential of felzartamab to be a meaningful treatment option for people living with IgA nephropathy, a leading cause of chronic kidney disease.” The Phase 2 IGNAZ study (n=54) explored the efficacy and safety of felzartamab in patients with IgAN and high risk of progressive kidney dysfunction. With respect to efficacy, patients receiving a nine-dose regimen of felzartamab over a six-month treatment period experienced substantial reductions in proteinuria levels as assessed by the urinary protein:creatinine ratio (UPCR) and stabilization of kidney function, as measured by the estimated glomerular filtration rate (eGFR), through 24 months. Notably, patients maintained a mean reduction of approximately 50% in the UPCR through month 24, which was more than 18 months after the last dose was administered. These results suggest that felzartamab may have the potential to preserve kidney function and be administered on treatment cycles instead of continuous dosing. Further analysis revealed that felzartamab administration resulted in selective and durable reductions in IgA antibody levels, while IgG and IgM levels recovered to baseline 3 months off-treatment. This selective reduction may offer maintenance of significant immune functions essential for infection protection. Overall, administration of felzartamab was generally well tolerated with a safety profile consistent with prior studies. “We are encouraged by the overall results of the IGNAZ study, especially given the significant unmet medical need for additional treatments to address high-risk IgA nephropathy,” said Uptal Patel, M.D., Head of Development, HI-Bio at Biogen. “We are grateful to all the participants, investigators and study staff who contributed to this study, whose findings will help us continue to evaluate felzartamab’s role in preserving kidney function as we plan for Phase 3.” 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 for multiple myeloma. 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. Immunoglobulin A nephropathy (IgAN) is the most common primary glomerulonephritis worldwide. It is a leading cause of chronic kidney disease with up to 40% of IgAN patients progressing to end stage kidney disease about 20 years after diagnosis. IgAN accounts for about 40% of all native-kidney biopsies in Japan, 25% in Europe, 12% in the United States, but less than 5% in central Africa.1 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. References: Rajasekaran et al. (2021) IgA nephropathy: An interesting autoimmune kidney disease. Available at https://www-ncbi-nlm-nih-gov.libproxy1.nus.edu.sg/pmc/articles/PMC5198292/. Hastings et al (2018) Clinical Research, Life Expectancy for Patients From the Southeastern United States With IgA Nephropathy. 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Plus, news about Novartis, CombiGene, ProQR and Aardvark Therapeutics: Biogen presents full Phase 2 data for felzartamab: The drug, which was acquired in Biogen’s $1.15 billion buyout of HI-Bio earlier this year, showed patients with IgA nephropathy maintaining a 50% average reduction in a measurement of kidney stabilization after two years. Biogen expects to take the drug into a Phase 3 study but exact details have not yet been disclosed. — Max Gelman Sanofi snags equity in Resalis Therapeutics for undisclosed amount: The new tranche of cash will help the biotech pay for a Phase 2 proof-of-concept study of its lead obesity medicine, called RES-010. Resalis raised €10 million ($10.8 million) in early 2024 to afford the drug’s first obesity trial. — Max Bayer Novartis shares one-year data for Fabhalta in ultra-rare kidney disease: For patients with C3 glomerulopathy, Fabhalta at 12 months sustained reductions in proteinuria that had been seen at six months, when the study reported a 35.1% reduction in protein buildup in the urine compared to placebo. At six months, patients who received placebo in the Phase 3 study were switched over to iptacopan, which Novartis markets as Fabhalta for a rare blood disorder and an autoimmune kidney disease. Novartis submitted the drug for approval in C3G in the EU, China and Japan, and it expects to be in the US by the end of this year. — Lei Lei Wu CombiGene trims pipeline, headcount: The Swedish drugmaker is ending work on its gene therapy candidate for epilepsy, called CG01, and instead prioritizing its chronic pain program, dubbed COZY. CombiGene is also reducing the number of its employees and external consultants by 45% in a bid to extend its cash runway through the second quarter of 2026. It had 11 employees, as of December 2023. — Ayisha Sharma Eli Lilly invests in ProQR as it nabs $75.3M: The company is selling shares for $3.50 apiece, getting $63 million. In addition, the funding includes a private placement from Eli Lilly that totals $12.3 million. — Max Gelman Aardvark Therapeutics buys MEI Pharma’s solid tumor drug: The San Diego-based biotech paid $500,000 upfront for the rights to MEI’s ME-344, an inhibitor of mitochondrial oxidative phosphorylation. MEI is eligible for up to $62 million in milestones. — Ayisha Sharma

2D illustration of kidneys iStock, Elena Nechaeva William Blair analyst Myles Minter in a Monday note to investors said that Vertex’s povetacicept “has maintained its potential to be a best-in-class asset” in the IgA nephropathy space and could become a “multibillion-dollar pipeline-in-a-drug product” for autoimmune disorders, while “outstanding questions” remain for Biogen’s felzartamab before moving into pivotal studies. At the American Society of Nephrology’s Kidney Week Congress last week, Biogen and Vertex Pharmaceuticals unveiled mid-stage data for their respective IgA nephropathy programs, touting strong clinical efficacy and promising benefits. On Saturday, Biogen presented complete data from the Phase II IGNAZ study showing that its investigational anti-CD38 antibody felzartamab could help stabilize kidney function and reduce proteinuria in patients with IgA nephropathy (IgAN). Felzartamab’s treatment effects were also sustained through more than 18 months after the last dose. Biogen noted that felzartamab could sustain an approximately 50% reduction in patients’ urinary protein:creatinine ratios (UPCR) through 24 months of follow-up. Felzartamab also selectively lowered IgA antibody concentrations, whereas IgG and IgM returned to baseline levels after 3 months off-treatment. Overall, these findings suggest that felzartamab can help preserve kidney function in IgAN patients without the need for continuous dosing, according to Biogen. IgAN is a rare autoimmune disease that develops when antibodies attack the kidneys’ glomeruli, leading to inflammation that—when left unchecked—can result in organ damage and compromise its ability to remove waste from the blood. Uptal Patel, head of Development at HI-Bio, said in a statement that the company is “encouraged” by Saturday’s findings. Biogen, which gained ownership of felzartamab from its $1.8 billion acquisition of Hi-Bio in May 2024 , is currently gearing up for the Phase III program of the antibody. Analysts, however, were lukewarm about the readout. While BMO Capital Markets’ Evan Seigerman called IGNAZ’s findings “promising” and a validation of the value of the HI-Bio deal, he said the results were “unlikely to be significantly share moving.” Meanwhile, Jefferies’ Michael Yee wrote in an investor note that the findings were “nice” but Biogen will need to do more business development work in 2025 “to build the pipeline and get investors further engaged.” William Blair analyst Myles Minter in a note to investors said that “there are likely outstanding questions on dosing regimen and reconciling proteinuria reductions with eGFR efficacy to answer prior to moving into pivotal studies.” Vertex also presented IgAN data at ASN , demonstrating that an 80-mg dose of its investigational BAFF/APRIL dual antagonist povetacicept elicited a 66% mean reduction in UPCR at 48 weeks. This effect was associated with stable renal function over the same time frame, as evaluated by estimated glomerular filtration rate. Similar efficacy figures were reported for the 240-mg povetacicept dose. Both dose levels were given subcutaneously every four weeks. Povetacicept was well-tolerated in IgAN, with majority of the adverse events being mild or moderate in severity. There were no serious toxicities reported. Vertex has kicked off the Phase III RAINIER study, a global trial designed to assess the safety and clinical efficacy of 80-mg povetacicept in IgAN. In a separate investor note, Seigerman said that the povetacicept readout “continues to support Vertex’s evolving kidney portfolio” as the company tries to expand its pipeline beyond pain and cystic fibrosis. Friday’s data also “support the product’s potentially best-in-class profile,” Seigerman wrote. Yee in an investor note said that the povetacicept‘s efficacy “continues to look promising vs competitors with its rapid and deep response.” IgAN remains an attractive target for biopharma and in recent months several companies have succeeded in bringing innovative therapies to market. These include Travere’s Filspari, which won full approval last month , and Novartis’ Fabhalta, which the FDA granted accelerated approval in August 2024 . Minter in a note to investors said that although the IgAN space remains competitive, Vertex’s povetacicept “has maintained its potential to be a best-in-class asset” and could become a “multibillion-dollar pipeline-in-a-drug product” for autoimmune disorders.