PolTREG initiates preclinical CAR-Treg development for neuroinflammatory autoimmune diseases

GDANSK, Poland I September 26, 2024 I PolTREG S.A. (Warsaw Stock Exchange: PTG), a biotechnology company in the clinical phase of developing cellular therapies for autoimmune diseases, has initiated a study involving CAR-Treg cells in a humanized mouse model of neuroinflammatory disease. The study aims to evaluate the safety and efficacy of CAR-Treg cell therapy, administered both systemically and intrathecally. Results from this study are expected by March 30, 2025, and will provide essential data for future clinical evaluation.

PolTREG’s CAR-Treg cells are derived using the same technology platform as PTG-007, their lead product, a polyclonal Treg cell therapy. PTG-007 has amassed over a decade of safety and efficacy data, primarily in type-1 diabetes (T1D) patients. According to Prof. Piotr Trzonkowski, CEO of PolTREG, this preclinical study is a significant advancement for the company's CAR-Treg cell therapy program targeting neurodegenerative diseases. The extensive experience gained from developing and treating patients with PTG-007 positions PolTREG advantageously in the field of next-generation engineered Tregs.

PolTREG's PTG-007 Treg cells are an advanced autologous polyclonal product candidate with 12 years of patient data in T1D, manufactured in their proprietary GMP facility in Poland. PolTREG claims to have unmatched expertise in this domain, potentially providing technical and clinical advantages in autoimmune therapy.

An exploratory Phase 1/2a safety study in relapsing-remitting multiple sclerosis (MS) demonstrated promising results. Patients who received intrathecal administration of PTG-007 showed no significant increase in existing brain lesions and almost no new lesions, outperforming those who received the therapy intravenously. This positive outcome is notable given the small sample size of 14 patients.

Upon successful preclinical testing of the CAR-Treg, PolTREG plans to begin the first-in-human trial of the engineered candidate for MS and amyotrophic lateral sclerosis (ALS) as early as next year. The company is also working on two additional engineered Treg cell therapies: Antigen-specific Tregs and T-cell receptor (TCR) Tregs for autoimmune diseases, currently in early preclinical proof-of-concept stages.

PolTREG recently announced the acquisition of three new patents. The United States Patent and Trademark Office (USPTO) granted a patent for CAR-Treg therapy in autoimmune neurodegenerative diseases to AZTherapies, a clinical biopharmaceutical company from Boston, which has exclusively licensed the therapy to PolTREG for all indications. Additionally, the Japanese Patent Office issued a patent for PolTREG’s method of producing antigen-specific Treg cells, under the PTG-020 product name. Earlier in September, the Chinese Patent Office granted a patent for PolTREG’s method of intrathecal administration of Treg cell therapy in MS.

PolTREG is a leader in developing T-regulatory cell (Treg)-based autoimmune therapies. Its primary product, PTG-007, aims to treat early-onset Type-1 Diabetes (T1D) and is ready for Phase 2/3 clinical testing, pending a partnership. The company plans to launch Phase 2 trials for PTG-007 in MS treatment in late 2024, targeting relapsing-remitting MS (RRMS) and primary progressive MS (PPMS). PolTREG’s pipeline includes various engineered Tregs, such as CAR-Tregs, antigen-specific Tregs, and TCR-Tregs, all in preclinical stages. The company has completed four clinical trials, treating over 100 patients with Tregs.