Subgroup Analysis: NextCell Presents Preliminary One-Year Results from the Older Age Group in ProTrans-Young

Summary of Results
In the open-label part of the study, the three treated patients showed an average increase in insulin production at 12 months compared to pre-treatment levels. This is in line with previous observations in our studies involving adult patients.

In the randomised, placebo-controlled part of the study, no substantial difference was observed between ProTrans and placebo. The spread in results was wide, resulting in large margins of error and warranting caution in interpreting the data at this early stage. Notably, three patients in the placebo group showed increased insulin production after one year, which may be attributed to the so-called honeymoon phase – a temporary period following diagnosis during which the body’s natural insulin production partially recovers. This phase can last from several months to a year and varies significantly between individuals.

The substantial individual variation in disease progression, along with unusually high retained insulin production in parts of the placebo group, makes it currently difficult to determine whether adolescents respond differently to treatment compared with adults. This variation is especially pronounced early after diagnosis and tends to decrease over time – particularly during adolescence, when differences are initially greatest due to puberty-related factors.

Our previous studies have treated adult patients later in the disease course, up to 24 months after diagnosis. A statistically significant treatment effect was observed as early as 12 months, and this effect persisted for at least five years – an outcome that may well also be seen in the adolescent group over time.

Sex-specific differences
Girls often experience a slower decline in insulin production during the early phase after diagnosis, which may be due to a combination of hormonal influences, more regulated immune responses, and genetic factors. It is therefore relevant to analyse treatment responses by sex across all patients. Girls treated with ProTrans retained 81% of their insulin production after one year, compared with 75% in the placebo group. For boys, the corresponding figures were 78% and 72%.

Factors that may have influenced the analysis
Several factors may have influenced the outcome of the analysis. On average, patients in the ProTrans group had a higher body weight and BMI at baseline, factors previously associated with a faster disease progression. In addition, four patients in the ProTrans group presented with elevated fasting plasma glucose levels at baseline – a condition also linked to a less favourable disease course.

Comment from the CEO
“We remain confident in the potential of ProTrans and see clear signs of a positive treatment effect. As the patients were recently diagnosed, many are undergoing puberty, and there are baseline differences between the groups, it may take longer to demonstrate a clear treatment advantage. Our hypothesis remains: ProTrans has a long-lasting effect, and differences are expected to become more evident over time,” says Mathias Svahn, CEO of NextCell.

Next Steps
Patients in the trial are being followed for a total of five years, with insulin production measured every six months. This interim analysis includes only baseline and one-year data. As the adolescents progress through puberty and hormonal fluctuations stabilise, the study groups are expected to become more homogeneous – which in turn may make it easier to draw more robust conclusions on treatment efficacy.

Recruitment of the younger age group (7–11 years) is ongoing and expected to be completed before summer 2025. One-year efficacy results for this group are anticipated in the second half of 2026. Insulin production will be measured in all patients every six months over the five-year follow-up period.

This disclosure contains information that NextCell Pharma AB is obliged to make public pursuant to the EU Market Abuse Regulation (EU nr 596/2014). The information was submitted for publication, through the agency of the contact person, on 07-04-2025 18:05 CET.