From August 2024, children aged 7-11 years, newly diagnosed with type 1 diabetes will be able to participate in a study with ProTrans.
NextCellPharma has many active owners. Questions come in regularly. NextCell's CEO Mathias Svahn answers a number of these questions in this Q&A section.
The information below has previously been communicated by NextCell. This information has been compiled to make it easier for NextCell's stakeholders to obtain an overall picture. This English version is a translation of the Swedish version. The Swedish version is at all times to be deemed as the correct version.
– The paediatric study, or ProTrans-Young which is the formal name of the ongoing Phase 2 study, consists of two parts. The first part includes a group of 30 young people aged 12-21 years, and the second part includes a group of 30 children aged 7-11. Before recruitment of the 30 younger children begins, a statement from our safety committee regarding the 6 children who have recently developed diabetes and been treated with ProTrans is expected during the spring.
The study is progressing well and according to plan. For both groups, treatment regarding safety has been carried out and, as in other studies with ProTrans, no serious side effects were found.
The Phase 2 study has continued in the older group and hopefully the last patient in this group will be treated this year, which means that results may be available around the end of next year. As for the younger children, that part of the study will start in mid-2024 and will be evaluated 12 months after the last patient has been treated. Uppsala University is the sponsor of the study. The principal investigator is Professor Per-Ola Carlsson, and he decides when the data will be analysed. According to the study protocol, an analysis of the effect will be carried out when all patients have had a one-year follow-up.
Read an interview (in Swedish) with Mathias Svahn about the ProTrans-Young paediatric study
– After scientific consultation with EMA, we understand that the pending paediatric safety data and approved PIP (see below) allows us to design a pivotal Phase 3 study that includes both paediatric and adult patients. This limits the need for separate paediatric studies and offers the opportunity to apply for market approval to, in principle, all patients newly diagnosed with type 1 diabetes.
NextCell has submitted a Paediatric Investigational Plan (PIP), which is an EMA requirement for drug development. We have received a very good response and the main criticism, which is not really a criticism, was that EMA believes that all children should have access to treatment, regardless of age. In addition to demonstrated safety, the paediatric plan must be approved before a Phase 3 application can be submitted.
– First and foremost, we want to ensure that a Phase 3 study includes as wide an age range as possible to avoid having to conduct additional Phase 3 studies in other age groups. This is why the ongoing ProTrans-Young study is so important. This study will provide us with incredibly valuable data from a total of 66 patients, compared to the previous ProTrans 1 & 2 studies which together included 24 patients.
Thus, we will build up a significant database of clinical data that will be valuable when we submit the application for a Phase 3 study. The total number of patients who have undergone treatment is of great importance for both regulatory authorities and potential licensees. In addition, we continue to collect long-term data from, for example, the ProTrans-Repeat study (a follow-up study aimed at verifying whether a second treatment with ProTrans can extend or maintain any effect over a longer period of time). The results indicate that a single treatment changes the course of the disease and that two treatments are probably even better.
We estimate that the cost of the ProTrans-Young study amounts to 60-80 million SEK, funds that are sponsored by Uppsala University in exchange for us providing the drug. In addition, we obtain access to study data that can be used in the application for market authorisation. A win-win-win for our shareholders, the healthcare system, and all patients.
Before it was clear that Uppsala would actually carry out ProTrans-Young, the strategy was to go straight in with a Phase 3 application. When ProTrans-Young became a reality, the company chose to lower the risk of the operation and we have never regretted that decision.
– We believe that we create the best possible shareholder value if we can apply for a Phase 3 study together with a future licensee, who will cover the development costs. Alternatively, we await results from ProTrans-Young to optimise the study design and apply ourselves. It is possible that the process could be accelerated with the right financial partner.
– Tzield has set a new standard for diabetes treatment. The drug is administered intravenously, one injection a day during what is currently a 14-day hospital stay. Tzield also requires premedication to reduce side effects. The total cost of treatment with Tzield in the US has been estimated at 194,000 USD plus hospital costs per patient, while ProTrans is estimated to be priced significantly lower. In addition, ProTrans causes no known serious side effects and we expect comparable efficacy. In addition, Tzield is currently only approved to treat patients with Stage 2 type 1 diabetes, i.e. individuals who have two antibodies against insulin-producing beta cells, but where the disease has not yet progressed to Stage 3 insulin-dependent type 1 diabetes. I think the results indicate that an infusion of ProTrans is more attractive than 14 injections of Tzield when compared to previous studies.