NextCell Pharma (NXTCL) Study Result summary

Study design and background

• ProTrans-Young is a pediatric clinical trial for type 1 diabetes using umbilical cord-derived mesenchymal stromal cells, conducted in collaboration with Uppsala University.

• The trial evaluates ProTrans in newly diagnosed type 1 diabetes patients aged 12–21, with all patients enrolled within six months of diagnosis.

• The trial is divided into three steps, starting with a completed phase I safety trial, followed by a phase II double-blind, randomized trial.

• Subgroup analysis covers the first 30 randomized patients, a period with higher residual insulin production but greater variability in disease progression.

• The younger age group (7–11 years) is still recruiting, with full primary endpoint data expected by the end of 2026.

Safety and tolerability results

• No severe adverse events were observed in children as young as seven, indicating strong safety and tolerability.

• Both placebo and ProTrans treatments were safely tolerated, with no need for immunosuppressive therapy.

• The product is administered as a single intravenous infusion and is well tolerated by both children and adults.

• No trauma, pre-medication, or continuous examinations are required for patients.

• The product can be stored long-term at clinics, enabling fast access and scalability.

Efficacy and clinical trends

• Early data show a reduction in insulin loss with ProTrans compared to placebo: females lost 19% vs. 25%, males 22% vs. 28% over one year.

• Girls treated with ProTrans retained 81% of insulin production after one year versus 75% for placebo; boys retained 78% versus 72%.

• In the randomized, placebo-controlled arm, no substantial difference was observed between ProTrans and placebo, with wide variability and large margins of error.

• Some placebo patients experienced increased insulin production, likely due to the honeymoon phase, complicating interpretation.

• Adult trials showed over 50% insulin retention over five years with ProTrans, compared to rapid loss with placebo.