NextCell Pharma highlights the positive trends seen in the preliminary ProTrans data in adolescents, while underscoring the need to wait until the end of the trial before drawing statistical conclusions. However, the market has a strong aversion to uncertainty and reacted hard to the one-year results revealed in mid-April. BioStock spoke with CEO Mathias Svahn to gain a deeper understanding of the data.

NextCell Pharma is a Swedish biotech company developing stromal cell therapies primarily for the treatment of autoimmune and inflammatory diseases. Its most advanced programme is ProTrans for the treatment of type 1 diabetes, where existing treatment options target symptom management and fail to modify the progression of the disease.

Evaluating ProTrans in children

Treatment with ProTrans, a cell therapy derived from umbilical cord mesenchymal stromal cells, has previously shown a positive therapeutic effect in adult patients, where patients who underwent treatment retained a higher proportion of their own insulin production. Now, ProTrans is evaluated in children in the phase II study called ProTrans-Young. It includes a total of 60 patients divided into an older age group (12–21 years) and a younger age group (7–11 years), and the patients are followed for a total of five years, with follow-up every six months.

The company recently presented an administrative analysis of preliminary one-year results, where ProTrans demonstrates a trend of insulin preservation in females and males when stratified according to sex in the older age group. The company states it is too early to draw conclusions as to the statistical significance of this effect until conclusion of the trial in the latter part of 2026.

Early analysis reveals a need to consider sex and age when comparing treatment impact

An analysis based on data from 30 patients in the older age cohort ranging from 12-21 years old – enrolled within six months of diagnosis – reveals variability in disease development and response to therapy at this early stage. This is likely to impact the interpretation of outcomes in the ProTrans-Young study when analysis is placed on the complete group with no stratification or sub-group analysis.

Notably, in the placebo group, three patients experienced an increase in insulin production due to the so-called “honeymoon phase” – a transitional period following diagnosis during which insulin production and blood sugar control temporarily improve. The degree of individual variability is highest shortly after diagnosis and diminishes over time. This variation is particularly pronounced among adolescents, largely due to hormonal fluctuations associated with puberty and differences between the sexes.

As these hormonal changes stabilise as puberty progresses, and honeymoon related effects are lost with disease development, the patient groups are expected to become more homogeneous – allowing for more robust and reliable conclusions regarding treatment effects with longer-term follow-up data.

ProTrans effect expected to become more evident over time

The company’s previous studies have focused on adult patients treated later in the course of the disease – up to 24 months after diagnosis. Already after 12 months of follow-up, a statistically significant treatment effect was demonstrated – an effect that persisted for at least five years. It is therefore hypothesised that a similar long-term effect will be observed in adolescents with longer follow-up.

According to the company, the goal is to initiate ProTrans treatment as early as possible after diagnosis, in order to preserve the remaining insulin production. It is anticipated that patients in the placebo group will lose their insulin production over time, whereas ProTrans treatment is intended to keep patients in the so-called honeymoon phase, or even promote disease remission.

CEO clarifies the results

BioStock reached out to NextCell Pharma’s CEO, Mathias Svahn, to learn more about the patients in the study and the implications of the preliminary results.

Starting with ProTrans, developed to treat type 1 diabetes – what does current treatment look like for these patients and what do you hope to improve with your therapy?

– Today, there is no treatment that alters the course of type 1 diabetes. Patients, regardless of age, require insulin therapy to survive – but this only treats the symptoms, not the underlying autoimmune cause.

– That is where ProTrans comes in. Our treatment is based on mesenchymal stromal cells, which can modulate the immune system and suppress the harmful autoimmune attack on insulin-producing cells in the pancreas.

– The goal is to preserve the body’s own insulin production, which can lead to more stable blood glucose levels, reduced insulin doses, and, over time, a decreased risk of serious complications like kidney damage, vision issues, and cardiovascular disease.

»The goal is to preserve the body’s own insulin production, which can lead to more stable blood glucose levels, reduced insulin doses, and, over time, a decreased risk of serious complications like kidney damage, vision issues, and cardiovascular disease.«

– We have already seen positive and long-lasting effects in adult patients, where a single infusion of ProTrans was able to slow disease progression for at least five years. Now we want to show the same for children and adolescents – potentially even better results, as their immune systems are more adaptable.

What does a typical disease progression look like after diagnosis?

– Shortly after diagnosis, there is often some residual insulin production since not all beta cells are yet destroyed. This means that the need for insulin may initially be lower as the glucose is easier to control – a phase known as the “honeymoon period,” which is temporary.

– Over time, the autoimmune attack continues to destroy insulin-producing cells. Endogenous insulin production gradually decreases, requiring higher insulin doses and making glucose control more challenging.

– After one to a few years, the body typically loses all endogenous insulin production, making the patient fully dependent on exogenous insulin. The longer someone has type 1 diabetes, the greater the risk for chronic complications, especially if glucose levels are not well-controlled.

– This is precisely the progression we aim to slow or change with ProTrans – by halting the autoimmune attack early and preserving insulin production as long as possible.

You recently presented preliminary 1-year results from the ProTrans-Young study in patients aged 12–21, showing limited effect compared to placebo. Can you elaborate on what was observed?

– Yes, we recently shared preliminary 1-year results from the older age group (12–21) in the ongoing ProTrans-Young study. These are from an administrative subgroup analysis of the first 30 randomised patients – so it is still early in follow-up and not possible for us to perform a statisitical analysis without impacting the integrity of the trial.

– We saw that insulin production was well preserved in both groups – ProTrans and placebo – making it hard to detect differences without sub-group stratification after just one year.

– We are not disappointed; this is a natural part of tracking early disease. We have a five-year follow-up and believe differences will become clearer over time, as we have seen in adult studies.

– We observed a clear positive trend when analysing girls and boys separately. Both sexes had higher preserved insulin levels, but differing disease progression between sexes blurred this effect in the full group.

– The placebo group performed unusually well, likely due to the “honeymoon phase,” where the body’s insulin production temporarily recovers after diagnosis. This is typical of adolescent trials where adolescents are highly susceptible to the placebo effect early after the intervention. There was also great individual variation, again expected in this age group during puberty – a time of major hormonal and immune changes. This is why sub-group analysis and consideration of how the cell therapy works in individuals will be so important when we perform the full data analysis on conclusion of the trial in 2026.

Knowing the honeymoon phase may impact results, why conduct an analysis so early? What did you hope to learn?

– It is important to clarify that this was not an interim analysis but an administrative subgroup analysis. The main goal was to prepare and optimise future data reads, in terms of study design and statistical methodology. It also helps with regulatory discussions and potential licensing partners.

– We wanted to understand the high variability in this age group, characterised by puberty and early disease. This helps set realistic expectations for future analyses and refine our strategy.

– Even though a clear treatment effect was not seen after one year looking at the group as a whole, the analysis provided valuable insights – for example, how strong the honeymoon effect is in the placebo group and early signs of treatment effect in some subgroups such as sex separation.

»Even though a clear treatment effect was not seen after one year looking at the group as a whole, the analysis provided valuable insights – for example, how strong the honeymoon effect is in the placebo group and early signs of treatment effect in some subgroups such as sex separation.«

You mention factors that may have influenced results – such as gender distribution. How might these have affected outcomes?

– As mentioned, disease progression differs between sexes – slower in girls. The placebo group had more girls, skewing the group result. When we analyse boys and girls separately, we see a clear positive trend.

Could you be more explicit on what you observed in the previous adult trials? And what parallels can be drawn to the pediatric study?

– Yes, our previous adult studies showed strong results. Differences were statistically significant after 12 months, and most importantly, the effect lasted up to five years – suggesting ProTrans may truly modify the disease.

– There are several parallels to the pediatric study, but also key differences. The disease mechanism is the same – autoimmune attack on insulin-producing cells – so we expect ProTrans to work in younger patients too. However, in the pediatric trial, we treat at an earlier stage. Their immune systems are more plastic, possibly leading to even better results in the long-term. But this also means the honeymoon phase affects early results more, unlike in adults treated at later stages of the disease.

– So, for a similar trend to be seen in adult and pediatric trials we would need to evaluate over a longer time period.

In ProTrans-Young, you have also included a younger group (7–11 years). What is the timeline for that part of the study and your expectations?

– The study for the 7–11 age group is progressing very well. Families are highly engaged, and many actively seek out the study teams in Uppsala, Linköping, and Lund.

»The study for the 7–11 age group is progressing very well. Families are highly engaged, and many actively seek out the study teams in Uppsala, Linköping, and Lund.«

– Professor Per-Ola Carlsson and his team at Uppsala University are doing a fantastic job – both in care and execution – which is vital in studies like this. Word about ProTrans has spread in patient communities, which is boosting interest. What makes the study especially attractive for families is that treatment involves a single infusion, with no serious side effects so far – a major reassurance for parents facing lifelong treatment for their child.

– We expect all patients in the younger group to be treated before summer. This means we can expect 1-year results in the second half of 2026.

Do the subgroup results impact the study going forward?

– The administrative subgroup analysis does not change the study design, unlike an interim analysis. Based on the results, we and Uppsala University will assess optimal timings for future analyses.

The market responded heavily to the results. How do you interpret the results?

– Of course, we would have liked to show a clear treatment effect after one year. But we are happy with the results and they are as anticipated. It is important to remember that;

• Placebo effects also tend to be stronger in children and adolescents.
• It is a balance between showing results quickly and understanding when the disease allows us to truly see the effect. We simply need more time.
• Our assessment is that differences between the treatment groups will become clearer the longer we follow patients, as we saw in adults. It is a matter of patience – because the difference will come.

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