PolTREG S.A., a biotechnology company specializing in cellular therapies for autoimmune diseases, has commenced a Phase 2 clinical trial evaluating PTG-007, a Treg cell therapy, in children with presymptomatic type 1 diabetes (T1D). The trial, which received approval from the European Medicines Agency, aims to assess the safety and efficacy of early intervention in individuals at high risk of developing T1D.
The study will enroll 150 patients between 6 and 16 years of age who have a high genetic predisposition for T1D but have not yet manifested any clinical symptoms. Clinical sites are being established, with patient recruitment and randomization scheduled to begin later this year.
Rationale for Early Intervention
Professor Piotr Trzonkowski, CEO of PolTREG, emphasized the importance of early intervention, stating, "When patients are diagnosed with diabetes, they typically have already lost the vast majority of pancreatic islets producing insulin… When administering PTG-007 at an earlier stage of the disease, the number of viable islets is still sufficient to prevent symptoms from occurring." This approach aims to prevent the onset of symptomatic diabetes and maintain clinical health.
PTG-007: A Polyclonal Autologous Treg Cellular Therapy
PTG-007 is an Advanced Therapy Medicinal Product (ATMP) consisting of polyclonal autologous Treg cells. PolTREG has accumulated 12 years' worth of proprietary safety and efficacy data for PTG-007 in patients with early-onset T1D. Previous data indicated that a subset of patients remained insulin-independent for 18 to 24 months following treatment, while another group experienced clinical remission, defined as a reduced need for external insulin, for 7 to 12 years after treatment.
Funding and Future Plans
The study is supported by a grant of 31.7 million zloty (€7.3 million) from Poland’s Medical Research Agency. PolTREG is also planning to initiate a pivotal Phase 2/3 trial with PTG-007 in early-onset T1D and is currently seeking external funding for this endeavor.
PolTREG's Broader Pipeline
PolTREG is developing various Treg modalities, including CAR-Treg and other engineered Treg cells. The company has completed five clinical trials across T1D, multiple sclerosis (MS), and graft-versus-host disease. The extensive safety and efficacy data collected over a decade with PTG-007 positions PolTREG favorably in the development of new therapies for autoimmune diseases.
