The Paediatric Committee (PDCO) of the European Medicines Agency (EMA) has issued a positive opinion on PolTREG's Pediatric Investigation Plan (PIP) for its investigational therapy PTG-007, a significant advancement in the company's efforts to develop treatments for pre-symptomatic type 1 diabetes in children.
The favorable opinion represents a crucial regulatory milestone for PolTREG S.A. (Warsaw Stock Exchange: PTG), potentially accelerating the path toward marketing authorization across the European Union and European Economic Area. Notably, the PDCO has recommended expanding the eligible patient population from the original 6-16 years to include children as young as 3 and adolescents up to 18 years of age.
"Securing a positive opinion from the PDCO brings PolTREG one step closer to the potential approval of PTG-007 for pediatric use across the European Union and the European Economic Area," said Prof. Piotr Trzonkowski, CEO of PolTREG. "Pre-symptomatic type 1 diabetes (Stage 1) represents a significant unmet medical need, making this therapeutic area highly attractive."
Innovative Cell Therapy Approach
PTG-007 utilizes polyclonal T-regulatory (Treg) lymphocytes to target the autoimmune mechanisms underlying type 1 diabetes. The therapy aims to intervene at Stage 1 of the disease, before clinical symptoms appear, potentially preventing progression to insulin dependence.
The PDCO's opinion builds upon PolTREG's original preTreg clinical trial protocol initiated in October 2024, which has been enrolling children with Stage 1 type 1 diabetes. This early-stage intervention strategy targets patients who have developed autoantibodies but have not yet experienced significant beta-cell destruction or hyperglycemia.
PolTREG stands out in the cellular therapy landscape with over 12 years of clinical data supporting the safety and efficacy of its polyclonal Treg therapies. The company claims to be the first in the world to administer Treg therapies to patients and the first to generate revenue from a Treg therapeutic for autoimmune disease under a hospital exemption valid in Poland.
Expanding Therapeutic Pipeline
Beyond type 1 diabetes, PolTREG is advancing its next-generation cellular therapy programs. The company recently reported encouraging preliminary safety and efficacy data from in vivo studies of CAR-engineered Treg lymphocytes in murine models, conducted in collaboration with AZTherapies.
"We are also delighted by the progress in developing next-generation Tregs. The in vivo murine results obtained a few days ago using CAR-TREG lymphocytes shows the preliminary safety and efficacy of our therapy," Prof. Trzonkowski noted. "In the coming months, we will intensively prepare to initiate a Phase 1 clinical trial in multiple sclerosis and amyotrophic lateral sclerosis."
The company's CAR-TREG program is being developed with plans to create an allogeneic version in cooperation with Swiss-based Antion Biosciences. This approach could potentially expand treatment access by eliminating the need to derive cells from each individual patient.
Manufacturing Capabilities
A key advantage in PolTREG's development strategy is its GMP-certified manufacturing facility, described as one of Europe's largest and most advanced for cellular therapies. The facility encompasses over 2,100 square meters of laboratory space with 15 production lines, capable of shipping cellular therapy products across Europe within 24 hours.
The company has indicated that its facility can be substantially expanded to accommodate manufacturing of next-generation engineered therapies and other cell therapies, positioning PolTREG for potential commercial-scale production as its pipeline advances.
Future Directions
While the positive PIP opinion strengthens PolTREG's pediatric development program, the company has disclosed that its lead product, PTG-007 for early-onset Type 1 Diabetes, is ready for Phase 2/3 clinical testing. PolTREG is actively seeking partnership opportunities to advance this program.
The company's technology platform extends beyond polyclonal Tregs to include multiple cellular therapy approaches: CAR-TREGs, allogeneic TREGs, antigen-specific TREGs, and TCR-TREGs. This diversified portfolio positions PolTREG to potentially address multiple autoimmune conditions with tailored cellular therapy approaches.
As regulatory and clinical development continues, the positive PDCO opinion represents an important validation of PolTREG's approach to addressing pediatric autoimmune conditions, particularly the significant unmet need in early-stage type 1 diabetes intervention.
