Table of Contents

• Trial overview
• Conditions studied
• Who can participate
• Trial phases and designs
• Main endpoints and what they mean
• What these trials aim to show

Trial overview

The clinical research program for Spesolimab includes studies in several skin conditions and uses different trial designs.^[1] These trials are looking at both short-term treatment response and longer-term safety and benefit.^[1]

The available studies include authorised, completed, and ongoing or authorised trials, with enrollment sizes ranging from 36 to 465 participants.^[1]

Conditions studied

One trial is studying Generalized Pustular Psoriasis (GPP) in people who need treatment for repeated flares, which means repeated worsening episodes of the disease.^[1] Another GPP study is looking at long-term treatment in people who already took part in earlier Spesolimab trials.^[1]

Two studies focus on hidradenitis suppurativa (HS), a long-term skin disease, and one of them includes people with moderate to severe disease who took part in earlier study parts.^[1] Another trial is studying pyoderma gangrenosum, a condition that can cause painful skin ulcers.^[1] One study also includes Netherton syndrome (NS), a rare skin disease.^[1]

Who can participate

Participation depends on the study. Some trials are for people who already completed previous Spesolimab treatment in an earlier study, especially the long-term follow-up studies in GPP and HS.^[1]

Other studies include people with active disease, such as recurrent GPP flares, moderate to severe HS, or pyoderma gangrenosum with a target ulcer to measure.^[1] The Netherton syndrome trial included people with that specific diagnosis and measured treatment response in the group studied.^[1]

Trial phases and designs

The studies are in different research stages: Phase 2, Phase 3, Phase 4, and one low-intervention trial.^[1] Phase 2 studies usually help researchers learn more about dose and early benefit, while Phase 3 studies test whether the treatment works better than placebo in a larger group.^[1]

Phase 4 studies are used after earlier research and often focus on longer-term safety and real-world use.^[1] The low-intervention GPP study is also designed to look at response to repeated flare treatment after an earlier flare was treated with Spesolimab.^[1]

Several trials compare Spesolimab with placebo, which is a look-alike treatment with no active study drug.^[1] This helps researchers see whether any improvement is likely due to the study treatment itself.^[1]

Main endpoints and what they mean

In the GPP flare study, the main endpoint is reaching a GPPGA pustulation subscore of 0 at Week 1, which means no visible pustules.^[1] This is a fast measure of whether the skin flare improves quickly.^[1]

In the pyoderma gangrenosum study, the main endpoint is complete closure of the target ulcer, also called PGAR-100, meaning 100% area reduction, confirmed at a later visit.^[1] This tells researchers whether the ulcer fully heals and stays closed for a short follow-up period.^[1]

In the HS study, the main endpoint is the percent change from baseline in dT count, which measures how much the number of draining tunnels changes from the start of the study.^[1] In the Netherton syndrome study, the main endpoint is IASI response, defined as at least a 50% improvement in the score from baseline.^[1]

Safety is also important in several trials, especially the long-term studies, where the main endpoint is the occurrence of treatment-emergent adverse events (TEAEs).^[1] TEAEs are health problems that appear or get worse after treatment starts.^[1]

What these trials aim to show

Across the study program, the main goal is to learn whether Spesolimab can help people with specific skin diseases improve their symptoms and maintain benefit over time.^[1] The trials also aim to show whether the treatment can be used safely in longer studies and in people who already had earlier Spesolimab exposure.^[1]

Because the studies use different conditions, phases, and endpoints, they together give a broader picture of how Spesolimab is being tested in clinical research.^[1]