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Topas Therapeutics GmbH

Falkenried 88, Haus A
20251 Hamburg GERMANY

+49 40 302089010
info@topas-therapeutics.com

Pipeline

Topas is utilizing its highly differentiated and versatile nanoparticle technology platform to develop a broad pipeline of novel therapies to treat autoimmune and antigen-driven diseases. With our Phase 2a study, we have established clinical proof of concept for the Topas Tolerance-Inducing Platform, supporting the potential application of our platform in celiac disease and in a range of other autoimmune and immune-mediated diseases.

Clinical Programs

Celiac Disease

Celiac disease (CeD) is a serious autoimmune disorder where the ingestion of gluten—a protein found in wheat, barley, and rye—triggers an immune response in the small intestine. This reaction can cause damage to the lining of the small intestine, leading to malabsorption of nutrients and a range of severe health complications. Globally CeD affects approximately 1.4% of the population and an estimated 3.3 million people in the USA alone, as indicated by seroprevalence studies. About 130,000 patients in the USA suffer from severe and refractory celiac disease, leading to additional complications such as malabsorption, ulcerative jejunitis, and enteropathy-associated T-cell lymphoma. Additionally, CeD patients miss about 50% more workdays compared to those without the disease, and they often face substantial psychosocial challenges. Celiac disease is strongly associated with specific HLA class II gene variants, particularly HLA-DQ2 and HLA-DQ8. Furthermore, CeD patients are 3-12 times more likely to develop non-Hodgkin’s lymphoma, a life-threatening complication. Management of the disease currently relies solely on a strict gluten-free diet, yet approximately 30% of patients continue to experience severe symptoms.

The Phase 2a study in celiac disease delivered initial clinical proof of concept for Topas Tolerance-Inducing Platform. TPM502, consisting of nanoparticles coupled with CeD-relevant antigens, demonstrated a good safety and tolerability profile in celiac disease patients. Our lead candidate achieved significant and dose-dependent reduction in IL-2 and IFN-γ release by gluten-specific T cells with a treatment effect that persisted throughout the study follow-up period. TPM502’s immunomodulatory effect was demonstrated by post-treatment phenotypic changes in gluten-specific CD4+ T cells consistent with T cell anergy or exhaustion and a significant increase in gluten-specific regulatory CD4+ T cells. In addition, the assessment of patient-reported outcomes (PROs), indicated a dose-dependent reduction in score intensity. Access the overview of the Phase 2a study outcomes here.

Topas is currently preparing for the initiation of its global, double-blind, randomized, placebo-controlled Phase 2b clinical study.  

Pemphigus Vulgaris

Pemphigus vulgaris (PV) is a rare and serious autoimmune skin disease that can lead to high morbidity and mortality. It is characterized by flaccid blisters and erosions on the skin and mucous membranes. These painful lesions significantly impact a patient’s quality of life and overall health. The incidence of PV ranges from approximately 0.5 to 3.2 cases per 100,000 people. Despite its rarity, PV can be life-threatening, with a mortality rate of 5% to 15%. Patients often experience multiple comorbidities, such as opportunistic infections, hypertension, and type 2 diabetes, which complicate their condition and treatment. The financial burden on PV patients is substantial, averaging about $30,000 per year. Most of these costs are related to outpatient care, but those requiring hospitalization typically stay for an average of twelve days. Current treatments for PV typically involve corticosteroids and immunosuppressive drugs, which can help manage symptoms but often lead to significant side effects and do not address the underlying cause of the disease, leaving patients vulnerable to relapses and other health complications. 

In the Phase 1 study, TPM203 demonstrated a good safety and tolerability profile, with pharmacokinetics data confirming rapid clearance from circulation. The encouraging results supported the Phase 2a study initiation for TPM502 in celiac disease patients.