TREG – innovative cell therapy targeting Diabetes Type 1

Información del proyecto

TREG

Identificador del acuerdo de subvención: 830559

DOI

10.3030/830559

Proyecto cerrado

Fecha de la firma de la CE 20 Noviembre 2018

Fecha de inicio 1 Noviembre 2018

Fecha de finalización 31 Octubre 2023

Financiado con arreglo a

• INDUSTRIAL LEADERSHIP - Innovation In SMEs

Coste total

€ 3 571 427,50

Aportación de la UE

€ 2 499 999,00

Coordinado por

POLTREG SPOLKA AKCYJNA
Poland

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

Período documentado: 2022-11-01 hasta 2023-10-31

Resumen del contexto y de los objetivos generales del proyecto

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.

Trabajo realizado desde el comienzo del proyecto hasta el final del período abarcado por el informe y los principales resultados hasta la fecha

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.

Avances que van más allá del estado de la técnica e impacto potencial esperado (incluida la repercusión socioeconómica y las implicaciones sociales más amplias del proyecto hasta la fecha)

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