Biogen Initiates Phase 3 Study of Felzartamab for the Treatment of Late Antibody-Mediated Rejection (AMR) in Kidney Transplant Patients
Biogen (BIIB) has initiated the Phase 3 TRANSCEND study to evaluate felzartamab for treating late antibody-mediated rejection (AMR) in kidney transplant patients. The study will enroll approximately 120 kidney transplant recipients and compare the drug's efficacy against placebo.
The trial is structured in two parts over 52 weeks: Part A involves nine intravenous infusions over 6 months, with the primary endpoint measuring AMR resolution by biopsy at 6 months. Part B provides all participants with felzartamab for an additional 6 months to evaluate longer-term effects.
AMR affects approximately 23,000 patients in the U.S. and is a leading cause of kidney transplant loss. The study follows promising Phase 2 results, and Biogen plans additional Phase 3 trials for felzartamab in IgA nephropathy and primary membranous nephropathy in 2025. As part of the trial initiation, MorphoSys will receive a $35 million milestone payment from Biogen.
Biogen (BIIB) ha avviato lo studio di Fase 3 TRANSCEND per valutare felzartamab nel trattamento del rigetto mediato da anticorpi tardivo (AMR) nei pazienti trapiantati di rene. Lo studio arruolerà circa 120 riceventi di trapianto di rene e confronterà l'efficacia del farmaco rispetto al placebo.
Il trial è strutturato in due parti nell'arco di 52 settimane: la Parte A prevede nove infusioni endovenose nell'arco di 6 mesi, con l'obiettivo primario che misura la risoluzione dell'AMR tramite biopsia a 6 mesi. La Parte B fornisce a tutti i partecipanti felzartamab per ulteriori 6 mesi per valutare gli effetti a lungo termine.
AMR colpisce circa 23.000 pazienti negli Stati Uniti ed è una delle principali cause di perdita del trapianto di rene. Lo studio segue risultati promettenti della Fase 2, e Biogen prevede ulteriori studi di Fase 3 per felzartamab nella nefropatia da IgA e nella nefropatia membranosa primaria nel 2025. Come parte dell'avvio dello studio, MorphoSys riceverà un pagamento di 35 milioni di dollari da Biogen.
Biogen (BIIB) ha iniciado el estudio de Fase 3 TRANSCEND para evaluar felzartamab en el tratamiento del rechazo mediado por anticuerpos tardío (AMR) en pacientes trasplantados de riñón. El estudio inscribirá aproximadamente a 120 receptores de trasplante de riñón y comparará la eficacia del fármaco frente al placebo.
El ensayo está estructurado en dos partes durante 52 semanas: la Parte A implica nueve infusiones intravenosas durante 6 meses, con el objetivo primario de medir la resolución del AMR mediante biopsia a los 6 meses. La Parte B proporciona a todos los participantes felzartamab durante 6 meses adicionales para evaluar los efectos a largo plazo.
AMR afecta a aproximadamente 23,000 pacientes en EE.UU. y es una de las principales causas de pérdida del trasplante de riñón. El estudio sigue resultados prometedores de la Fase 2, y Biogen planea ensayos adicionales de Fase 3 para felzartamab en nefropatía por IgA y nefropatía membranosa primaria en 2025. Como parte del inicio del ensayo, MorphoSys recibirá un pago de hitos de 35 millones de dólares de Biogen.
Biogen (BIIB)은 신장 이식 환자의 후기 항체 매개 거부반응(AMR) 치료를 위해 felzartamab의 효과를 평가하기 위한 3상 TRANSCEND 연구를 시작했습니다. 이 연구는 약 120명의 신장 이식 수혜자를 모집하며, 약물의 효능을 위약과 비교합니다.
이 시험은 52주 동안 두 부분으로 구성되어 있습니다: A 부분은 6개월 동안 9회의 정맥 주사를 포함하며, 주요 목표는 6개월 후 생검을 통해 AMR의 해결을 측정하는 것입니다. B 부분은 모든 참가자에게 추가로 6개월 동안 felzartamab을 제공하여 장기적인 효과를 평가합니다.
AMR은 미국에서 약 23,000명의 환자에게 영향을 미치며, 신장 이식 손실의 주요 원인 중 하나입니다. 이 연구는 2상에서의 유망한 결과를 따르며, Biogen은 2025년에 IgA 신병증 및 원발성 막신병증에 대한 felzartamab의 추가 3상 시험을 계획하고 있습니다. 시험 시작의 일환으로 MorphoSys는 Biogen으로부터 3,500만 달러의 이정표 지급을 받을 것입니다.
Biogen (BIIB) a lancé l'étude de Phase 3 TRANSCEND pour évaluer felzartamab dans le traitement du rejet médié par anticorps tardif (AMR) chez les patients ayant subi une greffe de rein. L'étude recrutera environ 120 receveurs de greffe de rein et comparera l'efficacité du médicament par rapport à un placebo.
L'essai est structuré en deux parties sur 52 semaines : la Partie A comprend neuf perfusions intraveineuses sur 6 mois, avec l'objectif principal mesurant la résolution de l'AMR par biopsie à 6 mois. La Partie B fournit à tous les participants du felzartamab pendant 6 mois supplémentaires pour évaluer les effets à long terme.
AMR touche environ 23 000 patients aux États-Unis et est l'une des principales causes de perte de greffe de rein. L'étude fait suite à des résultats prometteurs de la Phase 2, et Biogen prévoit des essais supplémentaires de Phase 3 pour le felzartamab dans la néphropathie à IgA et la néphropathie membranosaire primaire en 2025. Dans le cadre du lancement de l'essai, MorphoSys recevra un paiement d'étape de 35 millions de dollars de Biogen.
Biogen (BIIB) hat die Phase-3-Studie TRANSCEND gestartet, um felzartamab zur Behandlung der späten antikörpervermittelten Abstoßung (AMR) bei Nierentransplantationspatienten zu evaluieren. Die Studie wird etwa 120 Nierentransplantatempfänger einschließen und die Wirksamkeit des Medikaments mit einem Placebo vergleichen.
Die Studie ist in zwei Teile über 52 Wochen strukturiert: Teil A umfasst neun intravenöse Infusionen über 6 Monate, wobei der primäre Endpunkt die AMR-Resolution durch Biopsie nach 6 Monaten misst. Teil B stellt allen Teilnehmern felzartamab für weitere 6 Monate zur Verfügung, um die langfristigen Effekte zu bewerten.
AMR betrifft etwa 23.000 Patienten in den USA und ist eine der Hauptursachen für den Verlust von Nierentransplantaten. Die Studie folgt vielversprechenden Ergebnissen der Phase 2, und Biogen plant zusätzliche Phase-3-Studien für felzartamab bei IgA-Nephropathie und primärer membranöser Nephropathie im Jahr 2025. Im Rahmen des Studienstarts wird MorphoSys eine Meilensteinzahlung von 35 Millionen Dollar von Biogen erhalten.
- Advancement to Phase 3 trial following promising Phase 2 results
- Potential first-in-class treatment for late AMR, addressing an unmet medical need
- Large addressable market with ~23,000 patients in the U.S.
- Pipeline expansion with additional Phase 3 trials planned for 2025
- $35 million milestone payment obligation to MorphoSys
- Extended 52-week trial period before potential results
- Competitive success dependent on meeting primary endpoint of AMR resolution
Insights
Biogen's initiation of the Phase 3 TRANSCEND trial for felzartamab represents a strategic advancement in their immunology portfolio diversification beyond their traditional neuroscience focus. With approximately 23,000 patients affected by antibody-mediated rejection (AMR) in the US alone, this addresses a specialized but significant unmet medical need.
The study design including 120 patients is notably compact for a Phase 3 trial, potentially indicating confidence in the treatment effect size based on previous data. The primary endpoint focusing on biopsy-confirmed AMR resolution at 6 months provides a clear efficacy marker, while the secondary endpoint measuring microvascular inflammation (MVI) scores targets a clinically relevant predictor of long-term transplant survival.
Most compelling is Biogen's broader development strategy for felzartamab across three different immune-mediated indications (AMR, IgA nephropathy, and primary membranous nephropathy), creating a pipeline-in-a-product approach that increases the asset's potential value and reduces program risk through indication diversification.
The
The TRANSCEND trial represents a significant development in transplant medicine where antibody-mediated rejection remains one of our most challenging clinical problems. Current AMR management relies on non-specific immunosuppression with substantial toxicity and efficacy, making targeted approaches desperately needed.
Felzartamab's mechanism targeting CD38 is particularly elegant for AMR management as it addresses two critical pathological processes simultaneously: depleting antibody-producing plasma cells while reducing NK cell activity that contributes to microvascular inflammation. This dual action potentially interrupts the immunological cascade that leads to transplant damage.
The trial's focus on MVI scores as a secondary endpoint is clinically astute since microvascular inflammation strongly correlates with long-term graft survival. By targeting MVI reduction, Biogen is addressing a histologic marker that transplant nephrologists recognize as critical for preserving kidney function.
The two-part study design with a 6-month placebo-controlled period followed by open-label treatment allows assessment of both controlled efficacy and longer-term safety/activity. If successful, this approach could fundamentally change how we manage post-transplant complications, potentially reducing the devastating impact of transplant loss that necessitates patients returning to dialysis. The qualification of felzartamab as potentially "first-in-class" and "disease-modifying" based on Phase 2 results suggests this might represent more than symptomatic management.
- Global Phase 3 TRANSCEND study will evaluate the efficacy and safety of felzartamab, as compared to placebo, in adults with late AMR
- AMR is a leading cause of kidney transplant loss, with approximately ~23k patients living with all forms of AMR in the U.S1
- Felzartamab, with demonstrated proof of concept in multiple immune-mediated diseases, represents a key asset in Biogen’s late-stage immunology portfolio
CAMBRIDGE, Mass., March 11, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) – announced the initiation of dosing in the global clinical study, TRANSCEND. The Phase 3 study will evaluate the efficacy and safety of the investigational drug 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.
“Building upon the promising results from the Phase 2 study, which demonstrated felzartamab’s first-in-class potential, the launch of the TRANSCEND trial is a crucial milestone in the advancement of its clinical development,” said Travis Murdoch, Head of HI-Bio at Biogen. “Losing a kidney after receiving a long-awaited transplant is devastating for the patient and the donor. As we enroll this pivotal Phase 3 trial, we look forward to working in collaboration with medical and patient communities worldwide with the hope of bringing felzartamab forward as potentially the first meaningful treatment option, if approved, for people living with late AMR.”
“Antibody-mediated rejection remains a significant challenge in kidney transplantation, with limited safer and effective treatment options currently available. With the potential to be disease-modifying based on the encouraging results observed in the Phase 2 study, I believe felzartamab could be an important new therapeutic treatment option for patients with late AMR,” said Suphamai Bunnapradist, M.D., principal investigator of the study and Professor of Clinical Medicine, Director of Research at Connie Frank Kidney Transplant Center, University of California, Los Angeles. "I am pleased to see Biogen enroll the first patient in the Phase 3 TRANSCEND study of felzartamab in AMR and look forward to the trial’s continued enrollment.”
TRANSCEND is a two-part, 52-week, double-blind, placebo-controlled, multicenter, randomized Phase 3 clinical trial (NCT06685757) to evaluate the efficacy and safety of felzartamab compared with placebo. In Part A, participants will be randomized to receive nine intravenous infusions of felzartamab or placebo over 6 months, and the efficacy and safety of felzartamab compared to placebo will be assessed at 24 weeks. The primary endpoint of TRANSCEND is the percentage of participants who achieve resolution by biopsy of AMR at 6 months. Key secondary endpoints include changes in microvascular inflammation (MVI) score and the percentage of patients achieving an MVI score of zero. MVI is a key histologic feature of AMR and higher MVI scores strongly correlate with reduced kidney allograft survival rates.2 By targeting CD38, felzartamab is designed to reduce pathogenic antibody producing plasma cells and NK cell activity, addressing key pathophysiologic drivers of MVI and AMR. In Part B, all participants will receive felzartamab for an additional open-label period of 6 months through 52 weeks in order to evaluate longer-term activity, safety and tolerability.
In addition to beginning a Phase 3 study of felzartamab in AMR, as previously announced, Biogen plans to initiate Phase 3 trials of felzartamab in IgA nephropathy and primary membranous nephropathy in 2025. Felzartamab was originally developed by MorphoSys AG (now MorphoSys GmbH, a Novartis company) which was acquired by Novartis in May of 2024. As part of the initiation of the Phase 3 trial for felzartamab, MorphoSys will earn a one-time milestone payment of
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 Antibody-Mediated Rejection (AMR) in Kidney Transplant Recipients
Antibody-mediated rejection (AMR) is a major cause of kidney transplant failure. AMR in kidney transplant is caused by the immune system recognizing the donor kidney as foreign. This can result in antibodies being generated against the donor kidney and potentially leading to its destruction and eventual rejection. AMR demonstrates different properties depending on whether it occurs early (<6 months) or late (>6 months) post-transplantation. Late AMR is associated with a greater risk of graft loss versus early.3 Effective treatment options for late AMR are currently limited.4
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.
Biogen Safe Harbor
This press release contains forward-looking statements, relating to: our strategy and plans; potential of, and expectations for the design, timing and results of the TRANSCEND study, the ability of felzartamab to treat AMR, PMN or IgAN, our commercial business and pipeline programs; capital allocation and investment strategy; clinical development programs, clinical trials, and data readouts and presentations; regulatory discussions, submissions, filings, and approvals; the potential benefits, safety, our future financial and operating results. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “forecast,” “goal,” “guidance,” “hope,” “intend,” “may,” “objective,” “plan,” “possible,” “potential,” “predict,” “project,” “prospect,” “should,” “target,” “will,” “would,” and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements. These forward-looking statements are based on management's current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward-looking statements will be realized in whole or in part.
These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our subsequent reports on Form 10-Q and Form 10-K, in each case including in the sections thereof captioned “Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in our subsequent reports on Form 8-K. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise.
References:
- SRTR; National Institute of Diabetes and Digestive and Kidney Diseases; Department of Health; Hart, Singh. Clin Transplant 2021; Crew. Am J. Transplant. 2016
- Sablik et al. (2024) Microvascular Inflammation of Kidney Allografts and Clinical Outcomes. Available at: https://www.nejm.org/doi/full/10.1056/NEJMoa2408835
- Fernando et al. (2023) Early Versus Late Acute AMR in Kidney Transplant Recipients – A Comparison of Treatment Approaches and Outcomes From the ANZDATA Registry. Available at: https://pubmed.ncbi.nlm.nih.gov/37322595/
- 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
| MEDIA CONTACT: Biogen Jack Cox + 1 781 464 3260 public.affairs@biogen.com | INVESTOR CONTACT: Biogen Tim Power +1 781 464 2442 IR@biogen.com |
FAQ
What is the primary endpoint of Biogen's (BIIB) TRANSCEND Phase 3 study for felzartamab?
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What other conditions will Biogen (BIIB) study felzartamab for in 2025?
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