Topas Therapeutics Reports Positive Phase 2a Results for TPM502 in Celiac Disease

HAMBURG, Germany I October 15, 2024 I

Topas Therapeutics has released promising topline results from its Phase 2a trial of TPM502, which is being tested in patients with celiac disease. These results represent the first clinical proof of concept for Topas' nanoparticle platform, showcasing its potential to elicit targeted, antigen-specific tolerogenic effects.

The Phase 2a trial (NCT05660109) was a global, double-blind, randomized, placebo-controlled study started in 2023. It aimed to evaluate the safety, tolerability, and pharmacodynamics of TPM502 administered through two infusions in adult celiac patients. The study involved a gluten challenge to assess pharmacodynamic responses post-treatment with TPM502 or placebo. Initial findings demonstrated that tolerance induction, marked by specific antigenic responses, showed a clear dose-dependent pattern achieving statistical significance. These specific antigenic effects were sustained throughout the follow-up period. TPM502 was found to be safe at all dose levels tested. Topas plans to present the complete data at an upcoming scientific conference and seek publication in a peer-reviewed journal.

Hugo Fry, CEO of Topas Therapeutics, remarked, "This initial data from the TPM502 Phase 2a trial is a significant achievement for the Topas team. It supports our aim to prove that our platform induces antigen-specific tolerogenic effects. The study's findings highlight the potential of our novel platform across a wide array of autoimmune and immune-mediated diseases." He added, "We believe these results will serve as a strong foundation for the next development phase of Topas and improve our standing in the immune-tolerance field."

TPM502 is composed of nanoparticles carrying the main gluten epitopes for HLA-DQ2.5, common in most celiac disease patients. It aims to establish long-term immune tolerance, potentially offering significant therapeutic advantages for celiac patients, who currently have no treatment options. The Phase 2a study's initiation is based on encouraging preclinical data and the excellent safety results observed in the Phase 1 study of TPM203 in treating pemphigus vulgaris. The next steps for TPM502 will be determined based on the complete data analysis.

Topas Therapeutics is a clinical-stage biotechnology company focusing on creating immune tolerance for autoimmune and immune-mediated diseases. Utilizing a unique Topas Platform, which includes antigen-coupled nanoparticles, the company targets liver sinusoidal endothelial cells to drive T cells towards tolerance. The Phase 2a clinical trial results in celiac disease underscore the potential of this new drug modality, positioning Topas to significantly impact a variety of immune-mediated conditions and provide substantial therapeutic benefits to patients.