INmune Bio (INMB) Study update summary

Study background and rationale

• Mission EB is a phase III clinical trial evaluating CORDStrom, a pooled umbilical cord-derived mesenchymal stromal cell therapy, for recessive dystrophic epidermolysis bullosa (RDEB).

• RDEB is a progressive, multisystemic disease with limited effective treatments and high unmet need for systemic anti-inflammatory therapies.

• Existing therapies (Filsuvez, Vyjuvek) are topical, expensive, and lack systemic efficacy, highlighting the need for disease-modifying options.

Study design and execution

• Mission EB was a double-blind, placebo-controlled trial with 37 children enrolled, 30 completing, and 124 infusions administered.

• Patients received two infusions of CORDStrom or placebo, followed by a nine-month washout and crossover.

• The trial included both intermediate and severe RDEB patients, with a focus on safety, efficacy, and patient/parent acceptability.

Key efficacy and safety results

• CORDStrom demonstrated a 21% itch reduction at three months and 26% at six months, with the largest improvements in the most severe patients.

• Pain scores improved by 22% (average) and 28% (worst) in intermediate/severe patients; skin healing scores improved notably at six months.

• No serious adverse events were observed, and the therapy was well tolerated across all age groups.