Table of Contents

• Clinical trials overview
• Conditions being studied
• Trial phases and study designs
• Who may take part
• Main endpoints and what they measure
• Special study types and extension studies
• Key trials at a glance

Clinical trials overview

The trial program for Ianalumab includes studies in several autoimmune diseases, with many of them already authorised and some completed.^[1] These trials are designed to evaluate efficacy (how well the treatment works), safety, and tolerability in different patient groups.^[1]

Most of the studies are interventional trials, which means participants receive a study treatment and the results are compared with placebo or standard care.^[1] The trial data show repeated testing across diseases such as Sjögren’s syndrome, systemic lupus erythematosus, lupus nephritis, immune thrombocytopenia, warm autoimmune haemolytic anaemia, diffuse cutaneous systemic sclerosis, hidradenitis suppurativa, and autoimmune hepatitis.^[1]

Conditions being studied

Sjögren’s syndrome is one of the main conditions studied, including both active disease and long-term extension follow-up.^[2] The studies in this condition look at clinical improvement, long-term safety, and tissue changes in the salivary glands.^[2][3]

Systemic lupus erythematosus and lupus nephritis are also major focus areas.^{[4][5] In lupus nephritis, the trials look at kidney response and whether treatment can be maintained over time without flare or treatment escalation.[5][6]}

Other conditions include primary immune thrombocytopenia, warm autoimmune haemolytic anaemia, diffuse cutaneous systemic sclerosis, hidradenitis suppurativa, and autoimmune hepatitis.^{[7][8][9][10]}

Trial phases and study designs

The studies include Phase 2, Phase 3, and one Phase 4 trial.^[1] Phase 2 studies in the data are used to explore early effectiveness, tissue changes, and pharmacokinetic comparability, which means how the body handles the treatment in different device formats.^[3][11]

Phase 3 studies are the largest group and usually compare Ianalumab with placebo, often on top of standard-of-care therapy.^{[4][5][6] The Phase 4 study in autoimmune hepatitis was completed and focused on confirming efficacy and safety in a smaller group after incomplete response or intolerance to standard therapy.[10]}

Several trials are randomized and double-blind.^[1] Randomized means participants are assigned by chance to different groups, and double-blind means neither the participant nor the study team knows who receives the active treatment or placebo during the blinded part of the study.^[1]

Who may take part

The trial data show that many studies enroll adults, and some include adult and adolescent participants with moderate-to-severe or active disease.^[4][6]

Some studies are for people who have already tried treatment before, such as participants with warm autoimmune haemolytic anaemia who failed at least one line of treatment, or people with primary immune thrombocytopenia who failed steroids.^{[7][8] Other studies are extension studies for people who already finished a core trial and either benefited from treatment or continued treatment in an open-label setting, meaning both the participant and the study team know what treatment is being given.[2][12]}

Some studies also focus on people with specific disease activity profiles, such as moderate-to-severe disease, active disease, or anti-nuclear antibodies-positive systemic lupus erythematosus.^[4][6]

Main endpoints and what they measure

The endpoints are different because each disease needs a different way to measure improvement.^[1] In Sjögren’s syndrome, the studies measure change in the ESSDAI score, which is a disease activity score used to track how active the illness is.^[4][6]

In systemic lupus erythematosus, the main endpoint is SRI-4, a responder index that shows whether a person has improved by a defined set of criteria.^{[4][12] In lupus nephritis, the studies measure stable complete renal response, renal flare, and the need to increase immunosuppressive medication.[5][6]}

In primary immune thrombocytopenia, the studies measure time to treatment failure, which means how long it takes before the treatment no longer works well enough or the person needs rescue treatment or another ITP treatment.^[8] In warm autoimmune haemolytic anaemia, the key outcome is a durable hemoglobin response, meaning hemoglobin stays improved for at least 8 weeks without rescue or prohibited treatment.^[7][13]

In diffuse cutaneous systemic sclerosis, the key endpoint is the rCRISS25 response at Week 52, which is a composite response measure combining several signs of improvement.^[1] In hidradenitis suppurativa, the main outcome is the simplified HiSCR response after 16 weeks, and in autoimmune hepatitis the main endpoint is ALT normalization at Week 24, with ALT being a liver enzyme measured in blood tests.^[9][10]

Several studies also track treatment-emergent adverse events, often shortened to TEAEs, and serious adverse events, often shortened to SAEs.^{[2][11] These are standard trial safety measures that help researchers see what happens during treatment, without making claims beyond the trial data.[2]}

Special study types and extension studies

Some trials are extension studies, which follow people after they finish a core study.^{[2][12] These studies help researchers learn about long-term safety and what happens if treatment is withdrawn or continued.[2][12]}

One Sjögren’s syndrome study is a biopsy-based study, meaning it includes tissue samples from the salivary glands to look at histopathology, which is the study of tissue changes under a microscope.^[3] Another study in adults with autoimmune disease compares two device formats for subcutaneous Ianalumab and looks at pharmacokinetics, meaning how the drug concentration changes in the body over time.^[11]

There is also a crossover study, where participants receive treatments in more than one period so the different formats can be compared in the same person.^[11] This design can help researchers compare the 2 mL auto-injector, 2 mL pre-filled syringe, and 1 mL pre-filled syringe formats under similar conditions.^[11]

Key trials at a glance

The following trials are the main studies in the data and show the range of diseases and goals being tested.^[1]

• 2022-502966-26-01: Phase 3 extension study in Sjögren’s syndrome, focused on long-term safety and efficacy, with TEAEs and SAEs as the main safety outcome.^[2]

• NCT05639114 and NCT05624749: Phase 3 studies in systemic lupus erythematosus, both using SRI-4 at Week 60 as the main endpoint.^[4][12]

• NCT05126277 and NCT06711887: Phase 3 studies in lupus nephritis, measuring stable complete renal response and kidney-related outcomes over time.^[5][6]

• NCT05653219 and NCT05648968: Phase 3 studies in immune thrombocytopenia and warm autoimmune haemolytic anaemia, both focused on treatment failure or durable blood response.^[7][8]

• 2024-511933-36-00: Phase 2 study in diffuse cutaneous systemic sclerosis, measuring rCRISS25 response at Week 52.^[1]

• NCT05350072 and NCT05349214: Phase 3 studies in active Sjögren’s syndrome, both centered on change in ESSDAI score at Week 48.^[4][14]

• NCT03217422: Phase 4 study in autoimmune hepatitis, completed and focused on ALT normalization and later confirmation of biochemical and histological remission.^[10]