PolTREG starts Phase 2 cell therapy trial for children with presymptomatic diabetes

On October 24, 2024, PolTREG S.A., a biotechnology company based in Warsaw, announced the initiation of a significant Phase 2 clinical trial focusing on PTG-007 Treg cell therapy for presymptomatic type-1 diabetes (T1D) patients. This decision followed the approval from the European Medicines Agency. The placebo-controlled study will investigate the safety and efficacy of PTG-007 in 150 patients, specifically targeting children aged 6 to 16 who are at a high genetic risk for developing T1D but have not yet exhibited any symptoms. The clinical sites have already begun operations, with patient recruitment and randomization expected to proceed later in the year.

According to Prof. Piotr Trzonkowski, the Chief Executive Officer of PolTREG, early diagnosis of diabetes typically coincides with the significant loss of pancreatic islets responsible for insulin production. Administering PTG-007 at an earlier stage aims to preserve the remaining viable islets, thereby preventing symptoms and maintaining clinical health. This early intervention approach underpins PolTREG's belief that diabetes can potentially be prevented, allowing patients to avoid symptom manifestation altogether.

The study has received a substantial financial boost in the form of a 31.7 million zloty (€7.3 million) grant from Poland’s Medical Research Agency. PolTREG holds an exclusive position with 12 years' worth of proprietary safety and efficacy data for PTG-007 in early-onset T1D patients. This data indicates that some patients have maintained insulin independence for 18 to 24 months post-treatment, while another group has remained in clinical remission, characterized by a minimal need for external insulin, for 7 to 12 years after therapy.

Looking ahead, the company plans to initiate a pivotal Phase 2/3 trial for PTG-007 in early-onset T1D and is actively seeking external funding to support this endeavor. PTG-007, classified as an Advanced Therapy Medicinal Product (ATMP), represents a versatile platform for developing treatments across a spectrum of autoimmune diseases. PolTREG has completed five clinical trials encompassing T1D, multiple sclerosis (MS), and graft versus host disease, showcasing its comprehensive expertise in Treg cell therapy development.

PolTREG distinguishes itself by developing all available Treg modalities in-house, including CAR-Treg and other engineered Treg cells. Its extensive safety and efficacy history with PTG-007, spanning more than a decade, positions the company ahead of its competitors. The bioequivalence of new products in PolTREG’s pipeline with PTG-007 suggests that the company can expedite the development and market introduction of these therapies.

PolTREG is recognized as a global leader in autoimmune therapy development, specifically utilizing T-regulatory cells (Tregs). Their lead product, PTG-007, is an autologous Treg treatment for early-onset Type-1 diabetes, poised for Phase 2/3 clinical testing. The company seeks partnerships to advance this phase. Additionally, PolTREG plans to initiate Phase 2 trials for PTG-007 in treating multiple sclerosis (MS), targeting both RRMS and PPMS, in the latter half of 2024. The company's pipeline also includes engineered Tregs, such as CAR-Tregs, antigen-specific Tregs, and TCR-Tregs, currently in preclinical stages. PolTREG has successfully completed four clinical trials, treating over 100 patients with Tregs.

This strategic progression underscores PolTREG’s commitment to pioneering advancements in the treatment of autoimmune diseases through innovative cellular therapy solutions. The company's comprehensive data and robust research infrastructure ensure it remains at the forefront of Treg cell therapy development.