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

Imlifidase a new treatment for severe minimal change disease? (ERA 2025) - No abstract available

Glomerulonephritis

Urinary Markers of Inflammation and Damage in Anti-GBM Disease Before and After Treatment with Imlifidase (ERA 2025) - No abstract available

Glioblastoma • Inflammation

Depletion of AAV8 Antibodies in Seropositive Individuals Treated with the IgG Cleaving Enzyme Imlifidase (ASGCT 2025) - "Disclaimer : Imlifidase is an investigational product and is not approved in the USA for any use . Disease Focus of Abstract:None"

Clinical • Gene Therapies

Lymphocyte depletion post alemtuzumab administration in imlifidase enabled transplants; a UK wide case series (UKKW 2025) - No abstract available

Clinical • Transplantation

SUCCESSFUL POTENT REDUCTION OF DONOR-SPECIFIC ANTI-HLA ANTIBODIES USING IMLIFIDASE IN A PATIENT UNDERGOING HAPLOIDENTICAL STEM CELL TRANSPLANTATION (EBMT 2025) - "Due to ongoing minimal residual disease (MRD) positivity, she was switched to Decitabine and Venetoclax, with the aim of consolidation by allogeneic stem cell transplant...Desensitisation was performed using 3-weekly IV Rituximab 375mg/m2 and SC Bortezomib 2mg with twice weekly plasmapheresis...Compassionate access to 2 doses of Imlifidase was obtained to further attempt reduction of DSA levels.The patient underwent reduced intensity conditioning with Fludarabine, Cyclophosphamide and 2 Gy TBI and post-transplant Cyclophosphamide, with Ciclosporin and Mycophenolate Mofetil for graft versus host disease prophylaxis... To our knowledge, this was the first use of Imlifidase for this indication. This case demonstrates the potency of Imlifidase to achieve rapid transient reduction in DSA with high MFI. More studies e.g., frequency of infusion and incorporation to desensitisation regimens are required."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Novel Coronavirus Disease • Oncology • Transplantation • DNMT3A • IDH1

Hansa Biopharma publishes 2024 Annual and Sustainability Reports (PRNewswire) - "'2024 was a momentum-building year for Hansa. With year-on-year IDEFIRIX sales growth of 83%, a successful financing round'...Preparation for commercialization in the US is progressing, with complete randomization of the pivotal Phase 3 trial - ConfIdeS - announced in May 2024. Data readout for the trial and submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) are planned in 2H 2025...The company plans to align with regulatory authorities in the first half of 2025 on a clinical development path for HNSA-5487 in MG...In Autoimmune, enrolment was completed for the GOOD-IDES-02 Phase 3 trial in anti-glomerular basement membrane (anti-GBM) disease in December, with data readout expected in the second half of 2025."

Commercial • FDA filing • P3 data • Regulatory • Immunology • Myasthenia Gravis • Rare Diseases • Transplantation

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

Enrollment closed • Transplantation

LIVEDES: Imlifidase in Living Donor Renal Transplantation Highly Sensitized Recipients (clinicaltrials.gov) - P2 | N=0 | Withdrawn | Sponsor: Fundacion Clinic per a la Recerca Biomédica | N=10 ➔ 0 | Trial completion date: Dec 2026 ➔ May 2027 | Not yet recruiting ➔ Withdrawn | Trial primary completion date: Jul 2026 ➔ Dec 2026

Enrollment change • Trial completion date • Trial primary completion date • Trial withdrawal • Nephrology • Transplantation

Hansa Biopharma completes enrolment in European Phase 3 20-HMedIdeS-19 Post Authorization Efficacy and Safety study in highly sensitized kidney transplant patients (PRNewswire) - "Hansa Biopharma AB...announced today that it has completed enrolment of its 20-HMedIdeS-19 Post Authorization Efficacy and Safety (PAES) study, an open-label Phase 3 confirmatory study in Europe investigating the one-year patient and graft survival in highly sensitized patients who have undergone HLA-incompatible kidney transplantation following desensitization treatment with imlifidase...The Company anticipates data readout in the second half of 2026 followed by submission to the European Medicines Agency to seek full authorization....The PAES study is a controlled, open-label post-authorization efficacy and safety study to determine the one-year graft failure-free survival in highly sensitized kidney transplant patients with positive crossmatch against a deceased donor who received desensitization treatment with imlifidase followed by an HLA-incompatible kidney transplantation."

Enrollment closed • P3 data • Transplantation

Industry sponsored symposium: Clinical Experience with Imlifidase-Enabled Kidney Transplants in UK & France (BTS-NHSBT JAC 2025) - "Sponsored by HANSA BIOPHARMA"

Clinical • Transplantation

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74) (clinicaltrials.gov) - P1 | N=6 | Enrolling by invitation | Sponsor: Sarepta Therapeutics, Inc. | Trial completion date: Sep 2026 ➔ Oct 2027 | Trial primary completion date: Jan 2025 ➔ Jan 2026

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

PAES: Efficacy and Safety in Imlifidase Desensitized Kidney Tx Patients, Including Two Non-Comparative Reference Cohorts (clinicaltrials.gov) - P3 | N=225 | Recruiting | Sponsor: Hansa Biopharma AB | Trial completion date: Dec 2024 ➔ Apr 2026 | Trial primary completion date: Dec 2024 ➔ Apr 2026

Trial completion date • Trial primary completion date • Transplantation

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 | Recruiting | Sponsor: Hansa Biopharma AB | Trial completion date: Dec 2028 ➔ Apr 2030 | Trial primary completion date: Dec 2028 ➔ Apr 2030

Trial completion date • Trial primary completion date • Transplantation

DINKY: Imlifidase Prior to Kidney Transplant in Highly Sensitised Children (clinicaltrials.gov) - P2 | N=10 | Recruiting | Sponsor: Hansa Biopharma AB | Trial completion date: Sep 2029 ➔ Aug 2031 | Trial primary completion date: Sep 2024 ➔ Feb 2027

Trial completion date • Trial primary completion date • Transplantation

Anti-HLA-I IgG Subclass Influences Antibody-Mediated Platelet Activation in the Context of Platelet Transfusion Refractoriness (ASH 2024) - "In some conditions, platelets were incubated either with Eculizumab (100 µg/mL), IV.3 (10µg/mL) or IdeS (Imlifidase, 20 µg/mL) before incubation with the different chimeric antibodies in order to inhibit certain activation pathways. Our research reveals that platelet activation mechanisms in response to anti-HLA-I antibodies depend on the IgG subclass. Cleavage of anti-HLA-I IgG by IdeS abolishes platelet activation mediated by antibodies, which opens the door for developing strategies to safely transfuse platelets despite the limitations imposed by alloantibodies."

Hematological Disorders • ANXA5 • FCGR2A

GOOD-IDES-02: A Study With Imlifidase in Anti-GBM Disease (clinicaltrials.gov) - P3 | N=50 | Active, not recruiting | Sponsor: Hansa Biopharma AB | Recruiting ➔ Active, not recruiting

Enrollment closed • Glomerulonephritis • Renal Disease

Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome (clinicaltrials.gov) - P2 | N=3 | Recruiting | Sponsor: Genethon | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Metabolic Disorders

Depletion of AAV8 antibodies in seropositive healthy subjects and patients treated with the IgG cleaving enzyme imlifidase (ESGCT 2024) - No abstract available

Clinical

Superiority of Imlifidase over Plasma Exchange in Rapid and Efficient Elimination of All IgGs, Including Donor Specific Antibodies (DSAs) Assessed in an Antibody-Mediated Rejection (AMR) Trial (KIDNEY WEEK 2024) - P2 | "In a head-to-head comparison in kidney transplant recipients with AMR, one dose of imlifidase given over a 15-min infusion was more efficacious in reducing all IgG, both in speed and magnitude, compared to several PLEX sessions. Thus, imlifidase offers a rapid and effective option of hindering any IgG-mediated attack momentarily with a beneficial safety profile."

Head-to-Head • Antibody-mediated Rejection

Successful Use Of Imlifidase In A Highly And Broadly Sensitized Fontan Patient: First Reported Use In Heart Transplantation (HFSA 2024) - "Initial desensitization attempts used bortezomib/plasmapheresis, tocilizumab, and lastly a combination of plasmapheresis with weekly immunoglobulin IV. Imlifidase administration immediately prior to transplant effectively reduced pre-existing HLA Ab in a highly sensitized heart/liver candidate who was refractory to conventional desensitization therapies. This novel desensitization strategy is expected to benefit highly sensitized heart/liver recipients, providing a window during which compatible transplant could proceed."

Clinical • Late-breaking abstract • Cardiovascular • Heart Failure • Hepatology • Ischemic stroke • Transplant Rejection • Transplantation

European multi-centre trial with imlifidase prior to kidney transplant in highly sensitised children (ESPN 2024) - P2 | "The successful completion of the trial is aimed at addressing an unmet medical need in highly sensitised kidney transplant paedriatic/child patients and will be presented."

Clinical • Chronic Kidney Disease • Infectious Disease • Nephrology • Pediatrics • Renal Disease • Solid Organ Transplantation • Transplantation

A Study to Investigate DSA Rebound in Patients Treated with Imlifidase Prior to Transplantation (clinicaltrials.gov) - P2 | N=3 | Terminated | Sponsor: Hansa Biopharma AB | Active, not recruiting ➔ Terminated; Company decision, not due to any safety reason

Trial termination • Transplantation

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74) (clinicaltrials.gov) - P1 | N=6 | Enrolling by invitation | Sponsor: Sarepta Therapeutics, Inc. | Active, not recruiting ➔ Enrolling by invitation

Enrollment open • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome (clinicaltrials.gov) - P2 | N=3 | Not yet recruiting | Sponsor: Genethon

New P2 trial • Genetic Disorders • Metabolic Disorders

ConfIdeS: Renal Function in Highly Sensitized Patients 1 Year After Desensitization With Imlifidase Prior to DD Kidney Tx (clinicaltrials.gov) - P3 | N=64 | Active, not recruiting | Sponsor: Hansa Biopharma AB | Recruiting ➔ Active, not recruiting

Enrollment closed • Transplantation