Topas Therapeutics has hailed a win for its immune-tolerance platform, reporting positive phase 2a data on its celiac disease candidate without sharing numbers to validate its upbeat outlook.
Germany-based Topas spun out of Evotec in 2016 with a platform for inducing antigen-specific tolerance. The platform produces nanoparticles that carry disease-related epitopes to the liver. TPM502, Topas’ lead candidate, carries the major gluten epitopes for HLA-DQ2.5 to try to establish long-term tolerance in people with celiac disease.
The condition causes your immune system to attack your own tissues when you eat gluten, potentially damaging your gut.
Topas put TPM502 to the test in a phase 2a study that randomized 38 adults with celiac disease to receive two infusions of the drug candidate or placebo. The trial’s primary endpoint looked at adverse events. Topas said the candidate showed “a good safety profile in celiac disease patients.”
The biotech was similarly upbeat about the early signs of efficacy, hailing the phase 2a data as evidence its platform induces antigen-specific tolerogenic effects. That claim is underpinned by a gluten challenge test. Topas said its initial analysis showed that antigen-specific markers of tolerance induction exhibited a dose response that reached statistical significance and persisted throughout the follow-up period.
Like on the safety endpoint, Topas made the claims without sharing numbers to support its position. The biotech plans to submit the full data for presentation at a conference and publication in a journal. Topas will base the next steps for TPM502 on the full data analysis.
TPM502 is one of a number of assets in development for celiac disease. Takeda has a rival nanoparticle immune tolerance candidate, TAK-101, in phase 2 development. Kanyos Bio, a wholly owned subsidiary of Pfizer-backed Anokion, is running a phase 2 trial of another liver-targeted immune tolerance therapy. Others are trying to treat celiac disease by modifying gluten or reducing inflammation.
The impact of positive data on TPM502 could extend beyond celiac disease. As Topas’ lead candidate, TPM502 is serving as a proving ground for the platform. A phase 1 trial of another prospect, TPM203, is underway in pemphigus vulgaris and Topas sees opportunities to go after a range of autoimmune and immune-mediated diseases.