Idefirix (imlifidase) / Hansa Biopharma, Sarepta Therapeutics - LARVOL DELTA

Encore presentation: The ConfIdeS study: Imlifidase desensitization results in timelier transplant with superior 1-year eGFR among highly sensitized patients compared to control (TTS 2026) - "Participants were randomized 1:1 to imlifidase desensitization or control (any combination of PLEX, IVIG, anti-CD20, eculizumab, or waiting a more compatible organ). The imlifidase arm received rabbit anti-thymocyte globulin induction and triple maintenance immunosuppression with corticosteroids, mycophenolic acid, and tacrolimus... Imlifidase significantly increased immediate transplant rates and met the primary endpoint by improving 1-year kidney function compared with heterogeneous, institution-specific desensitization strategies. The variability of control treatments highlighted the absence of a standardized alternative approach. Imlifidase was generally well tolerated with no unexpected safety concerns."

Clinical • Chronic Kidney Disease • Transplantation

Short and long-term outcomes of imlifidase-enabled transplantation: ConfIdeS and beyond (TTS 2026) - No abstract available

Transplantation

Imlifidase in highly sensitized kidney transplant recipients with a positive crossmatch against a deceased donor - Results of the French ISKIA Registry (TTS 2026) - "These real-world data show that the use of imlifidase to desensitize patients has an acceptable medium-term efficacy and safety profile in selected patients, despite a high incidence of ABMR."

Clinical • Antibody-mediated Rejection • Infectious Disease • Pneumonia • Respiratory Diseases • Transplantation

Hansa BioPharma Corporate Lunch Symposium on Transplanting the untransplantable – Idefirix® (imlifidase), a breakthrough innovation for highly sensitised kidney transplantation (TTS 2026) - "Presentations will cover long-term efficacy and safety outcomes from controlled clinical studies, real-world experience, and early Australian practice, across both deceased and living donor settings. Together, the faculty will move from evidence to bedside, distilling how this expanding body of data is reshaping the way clinicians identify, transplant, and care for patients once considered untransplantable."

Transplantation

Naturally immunised AAV patients: modeling responder rates and treatable populations using VTX‑PID (imlifidase) dose‑range data to achieve non‑inhibitory AAV neutralizing antibody titers (ESGCT 2026) - No abstract available

Clinical

Overcoming AAV8 Immunity: One-year follow-up of the first seropositive Crigler–Najjar syndrome patient treated with GNT0003 following imlifidase pretreatment (GNT-018-IDES clinical trial) (ESGCT 2026) - No abstract available

Clinical • Genetic Disorders • Metabolic Disorders

Felzartamab, an Anti-CD38 Antibody, in Late AMR: Efficacy Independent of Prior Rejection History or Therapy (ATC 2026) - P1, P2 | "In total, 11 patients underwent immunoadsorption-based desensitization (n=8) and/or anti-humoral therapy (n=6), the latter including plasmapheresis (n=2), rituximab (n=2), IVIG (n=1), tocilizumab (n=1), clazakizumab (n=2), and/or the C1s monoclonal antibody BIVV009. Five patients had previously participated in AMR trials evaluating clazakizumab (NCT03444103; n=2), imlifidase (NCT03897205; n=1; control), bortezomib (NCT01873157; n=2; placebo), or BIVV009 (NCT02502903; n=1). Felzartamab is able to reverse AMR, even in patients with a long-standing rejection history and different forms of previous rejection treatment."

Clinical • Antibody-mediated Rejection • Inflammation

Imlifidase rescue in anti-GBM rapidly progressive glomerulonephritis when plasma exchange was contraindicated by immediate hypersensitivity (ERA 2026) - "Induction was complicated by RSV infection and subsequent neutropenic sepsis after cyclophosphamide given despite pre-dose neutropenia (1.02×10⁹/L), requiring antibiotics and filgrastim...Antibody kinetics are striking: anti- GBM 479 → 4.8 within 24 hours (99% reduction), with rebound managed by rituximab and sustained seronegativity at four-month follow-up, illustrating imlifidase's therapeutic window and limitations. The outcome—dialysis independence from a dialysis-dependent presentation—exceeds historical expectations (~8% dialysis independence at one year). Governance lesson - neutropenic sepsis from cyclophosphamide given despite neutrophils 1.02×10⁹/L demonstrates that immunological rescue can be undermined if cytopenia safeguards are not treated as hard-stops."

Glioblastoma • Glomerulonephritis • Hematological Disorders • Immunology • Infectious Disease • Lupus Nephritis • Nephrology • Neutropenia • Respiratory Diseases • Respiratory Syncytial Virus Infections • Septic Shock

PAES-LTFU: A Long-term Follow-up Trial of Patients Previously Treated With Imlifidase Prior to Kidney Transplantation and a Non-comparative Transplanted Patient Group (clinicaltrials.gov) - P3 | N=150 | Active, not recruiting | Sponsor: Hansa Biopharma AB | Recruiting ➔ Active, not recruiting

Enrollment closed • Transplantation

Superior 1-year eGFR Among Highly Sensitized Patients Desensitized with Imlifidase Compared to Control (ATC 2026) - "As there are no FDA approved agents or interventions for desensitization in the US, the purpose of this trial is to provide efficacy & safety data of imlifidase for desensitization & kidney transplantation in HS patients.* ConfIdeS is a phase 3, pivotal, open-label, randomized, controlled trial in 64 HS patients with cPRA ≥99.9% & a positive crossmatch to a deceased donor kidney, randomized 1:1 to imlifidase desensitization or control (any combination of PLEX, IVIG, anti-CD20, & eculizumab or await a more compatible organ offer). In this trial, imlifidase enabled successful transplantation & resulted in a significantly higher transplant rate compared with varied, institution-specific desensitization, fulfilling the primary endpoint. At 1-year post-transplant, treatment with imlifidase was associated with clinically meaningful & statistically significant improvements in kidney function as measured by eGFR, underscoring its impact on..."

Clinical • Late-breaking abstract

Imlifidase Enables Living Donor Kidney Transplantation in Highly Sensitized Recipients: A Two-Center German Experience (ATC 2026) - "ABMR was managed with multimodal therapy including high-dose corticosteroid pulses, intravenous immunoglobulins (IVIG) and daratumumab; three of four patients required immunoadsorption for rising DSA, and one received short-term eculizumab for rejection-triggered hemolytic uremic syndrome. One recipient remains on quadruple immunosuppression including belatacept, while the others continue standard triple therapy... Off-label use of imlifidase enabled successful living donor kidney transplantation in highly sensitized recipients across two German centers. Although ABMR occurred frequently, graft function remained preserved under intensified immunosuppression including anti-CD38-based maintenance. These findings support further evaluation of imlifidase in living donor desensitization protocols."

Antibody-mediated Rejection • Atypical Hemolytic Uremic Syndrome • Nephrology • Transplantation

GOOD-IDES-02 - a randomized controlled phase 3 trial testing imlifidase treatment in anti-GBM disease – outcomes and post hoc analysis (ERA 2026) - P2, P3 | "All patients received corticosteroids and cyclophosphamide according to a standardized protocol. In older patients there was no observed benefit, which could be explained by the differences in prognostic factors and comorbidities which may lead to diminished renal regenerative capacity. The most likely explanation for the reduced need for red cell transfusions is that imlifidase ameliorated subclinical lung bleedings."

Clinical • P3 data • Retrospective data • Cardiovascular • Coronary Artery Disease • Diabetes • Glioblastoma • Heart Failure • Metabolic Disorders • Nephrology

Imlifidase Enables Living Donor Kidney Transplantation in Highly Sensitized Recipients: A Two-Center German Experience (ERA 2026) - "ABMR was managed with multimodal therapy including high-dose corticosteroid pulses, intravenous immunoglobulins (IVIG) and daratumumab; three of four patients required immunoadsorption for rising DSA, and one received short-term eculizumab for rejection-triggered hemolytic uremic syndrome. One recipient remains on quadruple immunosuppression including belatacept, while the others continue standard triple therapy...Although ABMR occurred frequently, graft function remained preserved under intensified immunosuppression including anti-CD38–based maintenance. These findings support further evaluation of imlifidase in living donor desensitization protocols."

Antibody-mediated Rejection • Atypical Hemolytic Uremic Syndrome • Nephrology • Transplantation

Two Cases of Anti-GBM Disease : A Temporal Cluster and Clinical Insights (ERA 2026) - "Discussion We initiated daily plasmapheresis and immunosuppressive therapy with high dose steroids and cyclophosphamide...The clinical course was so rapid that we did not have the opportunity to introduce imlifidase, a new promising therapy specific for Goodpasture syndrome...Why this case is exeptional The simultaneous presentation of two cases of such a rare disease represents a rarity in itself. Remarkably, our experience was not isolated: during the same time frame, six additional similar cases were reported in neighboring provinces, all sharing an exceptionally elevated antibody titer, aggressive course and previous airway infection as a possible trigger."

Clinical • Acute Kidney Injury • Cough • Glioblastoma • Glomerulonephritis • Immunology • Infectious Disease • Inflammation • Lupus Nephritis • Nephrology • Rare Diseases • Renal Disease • Respiratory Diseases • Respiratory Syncytial Virus Infections

Impact of pre-existing neutralizing antibodies and IdeS treatment on AAV9 biodistribution in liver, heart and muscle in non-human primates (ASGCT 2026) - "While multiple solutions are being developed, we recently reported the efficacy of a pretreatment with an IgG degrading enzyme (IdeS, Imlifidase) for the treatment of a seropositive individual affected by Crigler-Najjar syndrome...Investigations on the effect of the presence of antibodies on biodistribution at cellular levels in muscle as well as evaluation of the presence of neutralizing factors other than antibodies in the same tissue are ongoing. To conclude, results shown reveal the complexity of targeting the muscle in seropositive primates but also suggest new avenues for the optimization of clinical protocols adapted to the doses and the target tissue in AAV seropositive individuals."

CNS Disorders • Gene Therapies • Genetic Disorders • Hematological Disorders • Hemophilia • Inherited Retinal Dystrophy • Metabolic Disorders • Movement Disorders • Muscular Atrophy • Rare Diseases • ACACA

Lowering pre-existing immunity to adeno-associated virus-based gene therapy: Pre‑treatment with imlifidase or plasmapheresis prior to administration of delandistrogene moxeparvovec in Duchenne muscular dystrophy (ASGCT 2026) - P1 | "Transduction and micro-dystrophin expression were observed in both studies, though at reduced levels relative to values observed in patients without pre-existing AAVrh74 antibody titers ≥1:400 in previous clinical studies. Results suggest that pre-treatment with imlifidase or plasmapheresis are potential approaches to lower pre- existing anti-AAV antibodies prior to treatment with AAV-based gene therapies such as delandistrogene moxeparvovec, though further studies are needed to better understand the mechanism of reduced gene therapy transduction and expression."

Gene therapy • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Immunology • Muscular Dystrophy

Modeling of VTX-PID-driven AAV3B Neutralizing Antibody Depletion To Expand Treatable Populations Across Gene Therapy Platforms : From Mechanism and Clinical data to Prediction. (ASGCT 2026) - "VTX-PID (imlifidase-), a bacterial IgG-specific endopeptidase, that cleaves all subclasses of circulating immunoglobulins G, enables a temporary permissive window for efficient AAV-based transduction...Conclusion NAVIgATE study results support VTX-PID as a promising pre-treatment approach to broaden eligibility of patients for systemic AAV-based gene therapies. The model’s modular structure supports projection to alternative AAV capsids through the incorporation of serotype-specific NAb-transduction relationships, enabling forward-looking assessment of VTX-PID utility across evolving AAV platforms and guides the translational clinical design of next-generation AAV-based therapies."

Clinical data • First-in-human • Gene therapy • Gene Therapies • Musculoskeletal Diseases

A novel human IgG-producing mouse model reveals limitations of IdeS efficacy in overcoming pre-existing AAV immunity (ASGCT 2026) - "In recent years, immunoglobulin G (IgG)-degrading enzymes such as imlifidase (IdeS) have been investigated as a strategy to enable treatment in AAV seropositive individuals by transiently reducing AAV antibody titers...Conclusion Using a new mouse model producing fully human IgG, we found that although IgG-degrading enzymes can reduce anti-AAV IgG titers by ~90% or more, these titer reductions enabled partial AAV transduction only when starting antibody titers were relatively low. Overall, our findings suggest that while IdeS may confer some benefit for individuals with low anti-AAV antibody titers, individuals with higher titers (including those observed post-AAV gene Therapy are not likely to be addressed by this approach."

Preclinical • Gene Therapies

Broadly cross-reactive anti-capsid IgA is an overlooked barrier to AAV-mediated gene transfer (ASGCT 2026) - "Current screening focuses almost exclusively on anti-capsid IgG, and degrading enzymes with IgG-specificity (IdeS/IdeZ/Imlifidase) are being explored to overcome this limitation...Notably, the marked interspecies differences in serum IgA structure and concentration as well as the absence of FcαRI in mice limit the ability of preclinical models to predict IgA-mediated innate immune activation in humans. Together, these findings necessitate a pressing need for systematic evaluation of IgA responses in human gene therapy and support the integration of IgA screening protocols and targeted depletion strategies into clinical practice."

Gene Therapies • Hepatology • Immune Modulation • Immunology • Infectious Disease • Inflammation

Imlifidase as a desensitization strategy in kidney transplantation at CHU de Liège, Belgium: up to one-year outcomes in two high-risk recipients (EFI-BSHI 2026) - No abstract available

Transplantation

PAES: Efficacy and Safety in Imlifidase Desensitized Kidney Tx Patients, Including Two Non-Comparative Reference Cohorts (clinicaltrials.gov) - P3 | N=113 | Completed | Sponsor: Hansa Biopharma AB | Active, not recruiting ➔ Completed | N=225 ➔ 113

Enrollment change • Trial completion • Transplantation

IMLIFIDASE EFFICIENTLY ABROGATES THE EFFECT OF ATLG AND THYMOGLOBULIN ON T- AND NK-CELL VIABILITY AND FUNCTION (EBMT 2026) - "Background: ATLG (Grafalon) and Thymoglobulin are long-established agents in allogeneic hematopoietic stem cell transplantation (allo-HSCT), effectively preventing both graft rejection and graft-versus-host disease (GvHD)... Imlifidase markedly attenuates the impact of ATLG/Thymoglobulin on NK-cell function and abrogates ATLG-/Thymoglobulin-mediated cytotoxicity on T-/NK-cells. This effect of imlifidase is to be evaluated in a clinical study in the context of T-/B-cell-depleted allo-HSCT in order to determine whether pre-transplant application of imlifidase (e.g. on day-2) results in effective inactivation of ATLG/Thymoglobulin in vivo and thereby accelerates post-transplant immune reconstitution."

Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Transplant Rejection • IL2 • NCAM1

IMLIFIDASE EFFICIENTLY ABROGATES THE EFFECT OF ATLG AND THYMOGLOBULIN ON T- AND NK-CELL VIABILITY AND FUNCTION (EBMT 2026) - "Background: ATLG (Grafalon) and Thymoglobulin are long-established agents in allogeneic hematopoietic stem cell transplantation (allo-HSCT), effectively preventing both graft rejection and graft-versus-host disease (GvHD)... Imlifidase markedly attenuates the impact of ATLG/Thymoglobulin on NK-cell function and abrogates ATLG-/Thymoglobulin-mediated cytotoxicity on T-/NK-cells. This effect of imlifidase is to be evaluated in a clinical study in the context of T-/B-cell-depleted allo-HSCT in order to determine whether pre-transplant application of imlifidase (e.g. on day-2) results in effective inactivation of ATLG/Thymoglobulin in vivo and thereby accelerates post-transplant immune reconstitution."

Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Transplant Rejection • IL2 • NCAM1

NL80681.078.22: lmlifidase treatment tor acute inflammation in AQP4-lgG associated neuromyelitis optica spectrum disorder (clinicaltrialsregister.eu) - P1/2 | N=5 | Recruiting | Sponsor: Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC) | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Inflammation • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

GOOD-IDES-02: A Study With Imlifidase in Anti-GBM Disease (clinicaltrials.gov) - P3 | N=50 | Terminated | Sponsor: Hansa Biopharma AB | Trial completion date: Nov 2026 ➔ Feb 2026 | Active, not recruiting ➔ Terminated; Company decision, not due to any safety reason.

Trial completion date • Trial termination • Glomerulonephritis • Renal Disease