Travere Therapeutics, Inc. is set to present their latest clinical data on an investigational enzyme replacement therapy for homocystinuria at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Jerusalem, Israel. The data will be presented at the event, which takes place from August 29 to September 1, 2023.
Homocystinuria is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS). This deficiency leads to toxic levels of homocysteine in the body, resulting in life-threatening thrombotic events, ophthalmologic and skeletal complications, and developmental delay. Current treatment options are limited to a protein-restricted diet and the use of vitamin B6 and betaine supplements.
Travere Therapeutics will present clinical data from the Phase 1/2 COMPOSE Study of pegtibatinase, a novel investigational enzyme replacement therapy for the treatment of classical homocystinuria. Pegtibatinase has shown promising results in reducing total homocysteine levels and improving clinical parameters in preclinical studies.
In addition to the clinical data presentation, Travere Therapeutics and its collaborators will also present analyses on the prospective HCU natural history study, the prevalence of HCU, and the burden of HCU from the patient perspective. These analyses highlight the devastating nature of this rare metabolic disease over patients’ lifetimes and underscore the urgent need for new treatments.
The Company will also present data on the clinical burden of HCU and the relationship between total homocysteine and clinical outcomes. This particular presentation has been recognized as one of the highest-ranked posters at the SSIEM Annual Symposium.
The schedule of presentations includes an oral presentation on pegtibatinase in the Clinical Studies and Outcomes session on August 30. Poster presentations will also take place throughout the event, covering topics such as the clinical characterization of classical homocystinuria, the clinical burden of HCU in the United States, the prevalence of HCU, and the patient perspective on the burden of HCU.
Travere Therapeutics is dedicated to developing life-changing therapies for rare diseases and working closely with the rare disease community to understand their perspectives and needs. The Company’s ongoing pegtibatinase program has received Breakthrough Therapy designation, Rare Pediatric Disease and Fast Track designations from the FDA, as well as Orphan Drug designation in the US and Europe.
With their participation in the SSIEM Annual Symposium, Travere Therapeutics aims to contribute to the advancement of knowledge and potential new treatments for classical homocystinuria.
