TREG – innovative cell therapy targeting Diabetes Type 1

Project Information

TREG

Grant agreement ID: 830559

DOI

10.3030/830559

Project closed

EC signature date 20 November 2018

Start date 1 November 2018

End date 31 October 2023

Funded under

• INDUSTRIAL LEADERSHIP - Innovation In SMEs

Total cost

€ 3 571 427,50

EU contribution

€ 2 499 999,00

Coordinated by

POLTREG SPOLKA AKCYJNA
Poland

Periodic Reporting for period 5 - TREG (TREG – innovative cell therapy targeting Diabetes Type 1)

Reporting period: 2022-11-01 to 2023-10-31

Summary of the context and overall objectives of the project

The objective of the project is to develop R&D works on TREG – a ground breaking Type 1 Diabetes (T1D) somatic cell therapy based on T-regulatory cells obtained from the patient’s blood. T-regulatory lymphocytes (Treg) form a special population in the immune system and although accounting for less than 1% of the leukocytes in the peripheral blood, they play a significant role in a human body due to the fact that they regulate the immune response so that pathogens are swiftly eliminated while own tissues remain protected.

T1D is an example of autoimmune disease which frequency is contineously increasing in pediatric population in Europe. Yet, there is no commonly accepted treatment which could be administered to stop the progression of disease and all afected subjects develop full onset T1D. It is therefore important to go through formal path of registration and start offering the treatment which has some efficacy in this disease.

The Treg therapy will be the first cell therapy product with the proven ability to hamper T1D progression by suppling the missing cells to the patient. The technology is based on ex vivo expansion of patients’ naturally occurring regulatory T cells, without genetic manipulation, followed by autologous re-transfusion to induce immune tolerance - each vaccine will be produced individually for a particular patient on the basis of their blood sample. The uniqueness and innovativeness of the treatment lies in the reduction of the disease causes, and not only its clinical symptoms which improves the quality of life and clinical parameters of patients.

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

We have chosen Parexel International as subcontracted contract research organisation (CRO) after the public bid. This subcontractor has been doing preparation for phase III trial with our TREGS medicinal product.

We have completed phase II trial and started preparing report from it. Looking into the safety, no serious adverse effects were reported. In terms of efficacy, the most promising results were obtained with combined treatment covering TREGS and rituximab as measured with the level of fasting and stimulated c-peptide and HbA1C. Treg only group was worse but still better than control one.

In cooperation with CRO we are preparing the documentation in accordance with EMA procedure. Poltreg’s documentation was translated to English, archived electronically and stored at password-protected virtual room for exchange with CRO. Based on the documents, CRO performed gap analysis for each of the stages of the product development, i.e. preclinical, CMC, clinical.

As the first stage of submission for scientific advice procedure, we have submitted letter of intent for PIP (pediatric investigation Plan) on 7th November 2019. It is submitted to the European Medicines Agency's Paediatric Committee (PDCO).

Submitted PIP will allow us to proceed further. We plan to submit intent letter for scientific advice in Q1 2020 Having some feedback from PDCO and the results of phase II will enable us to prepare better the outline of phase III.

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

As a result of the proposed project, PolTREG will be prepared for the next phase of clinical trials (Phase III) on the safety and efficacy of the therapy in a broader paediatric population. If successful, the project will accelerate availability of a breakthrough therapy to the T1D patients and put PolTREG on a path of business development by a full-scale commercialisation of the innovation.

When T regulatory cells (TREGS) are missing, other immune cells stop distinguishing self from non-se

T regulatory cells (TREGS) constitute a gendarmerie in the immune system. In the presence of TREGS