NextCell increases market potential and reduces the risk of ProTrans' clinical trial program in type 1 diabetes

NextCell Pharma AB ("NextCell" or the "Company") updates the strategy for the clinical trial program in type 1 diabetes with ProTrans. The aim is to increase potential while reducing the risk. This can be achieved by waiting for preliminary safetydata from the approved ProTrans-Young pediatric trial before submitting the application for ProTrans-3, scheduled for H2 2022.

Advantages of the updated strategy for future application for marketing approval are:

• Lower age limit for treatment is desirable and could potentially double the intitial market
• Increased long term data from patients treated with one and two doses of ProTrans

While type 1 diabetes is usually diagnosed at 10-14 years of age, completed trials with ProTrans have only included adult patients. Patients diagnosed at young age experience more complications later in life and have reduced life expectancy.

ProTrans-Young is a recently approved sponsor-initiated trial to evaluate the possibility of treating children with ProTrans. The principal investigators are Professor Per-Ola Carlsson, Uppsala University Hospital and Uppsala University, together with co-investigators Professor Helena Elding Larsson, Skåne University Hospital and Professor Johnny Ludvigsson, Linköping University Hospital. The full titel of the study is "A Double-blinded, Randomized, Parallel, Placebo-controlled trial of Wharton's Jelly-derived Allogeneic Mesenchymal Stromal Cells to treat Type I Diabetes in Children and Adolescents" (EudraCT 2020-004520-42).

A total of 66 children and adolescents with type 1 diabetes will be treated with ProTrans or placebo. The study is a phase I/II study divided into two parts; in the first part, three adolescents (12-18 years) and then three children (7-11 years) are treated to evaluate safety. These preliminary safetydata are planned to be included in a phase III application. The second part of ProTrans-Young is randomized and placebo-controlled, for evaluation of treatment efficacy, 30 adolescents (12-22 years) and then 30 children (7-11 years) are randomized 1:1.

Preliminary safety data for the treatment of children and adolescents from ProTrans-Young may allow younger patients to be included in ProTrans-3. With this background in mind the strategy is updated in order to include safetydata from the phase I-part of ProTrans-Young in Pro-Trans-3. The pivotal phase III study is intended to form the basis for future market approval. The application is expected to be submitted as early as the second half of 2022.

NextCell has during the last year had a constructive dialogue with the European Medicines Agency (EMA) on the path to commercialization. Although the company believes that an application for phase III study can be granted based on available data, it is strategically advantageous to wait for safety data from a pediatric population to enable a significant expansion of market potential and the number of individuals with Type-1 diabetes who may be in need of treatment.

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 26-10-2021 08:15 CET.

För further information, please contact:

Mathias Svahn, CEO
Patrik Fagerholm, CFO

Tel: +46 8 735 5595
E-mail: info@nextcellpharma.com

Home pages:

NextCell Pharma AB: www.nextcellpharma.com
Cellaviva Sverige: www.cellaviva.se
Cellaviva Danmark: www.cellaviva.dk

About NextCell Pharma AB

NextCell is a phase II cell therapy company with the drug candidate ProTrans for the treatment of type 1 diabetes. The focus is to take ProTrans to market approval via a Phase III study. ProTrans is in addition to diabetes, used in two clinical trials for Covid-19, in Örebro and Montreal (Canada). The company is in the processes of establishing its own GMP facility for production of ProTrans. The GMP facility is expected to be ready for production of smaller quantities of ProTrans in 2023. NextCell furthermore owns 10% in FamicordTX, a CAR-T start-up in oncology and 100 % of Cellaviva, Scandinavia's largest stem cell bank for family saving stem cells from umbilical cord blood and umbilical cord tissue with permission from the Swedish Health and Social Care Inspectorate (IVO).

FNCA Sweden AB is assigned as Certified Adviser, 08-528 00 399, info@fnca.se.