An Open-Label Efficacy and Safety Study of Pozelimab in Patients with CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)

This was an open-label, single-arm, historical-controlled, 144‑week treatment study in participants aged 1 year and older with active clinical signs and symptoms of protein-losing enteropathy (PLE) with biallelic CD55 loss-of-function mutation (CHAPLE disease). Participants served as their own historical control. Pozelimab was given as a single loading dose of 30 mg/kg intravenously on day 1 followed by weekly subcutaneous maintenance doses based on body weight.

Regeneron Pharmaceuticals, Inc.

Sponsor’s responsible officers are listed in Section 13.2

27 Jan 2020 (first participant first visit)

24 May 2022 (last participant last visit for the primary endpoint: proportion of participants who achieved normalization of serum albumin and improvement or no worsening in clinical outcomes [frequency of abdominal pain, bowel movement frequency, facial edema severity, and peripheral edema severity] at week 24)

02 May 2024 (last participant last visit for week 144)

The analyses presented in this report are based on the week 144 database lock date of 05 Jun 2024.