Travere Therapeutics Showcases HCU Studies at SSIEM Symposium
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Travere Therapeutics to Present at SSIEM Symposium
Travere Therapeutics, Inc. (NASDAQ: TVTX) has announced participation in an upcoming symposium where the company will showcase groundbreaking research in the field of classical homocystinuria (HCU). This event, occurring in Porto, Portugal, from September 3 to 6, will feature two key poster presentations related to their investigational enzyme replacement therapy, pegtibatinase.
Poster Presentations Overview
At the Society for the Study of Inborn Errors of Metabolism (SSIEM) symposium, Travere Therapeutics is set to unveil the trial designs for the pivotal Phase 3 HARMONY Study, along with the ENSEMBLE long-term extension study that focuses on pegtibatinase. This innovative therapy aims to address the root causes of classical HCU by potentially restoring deficient cystathionine beta synthase (CBS) enzyme activity.
Details of the Poster Presentations
The first presentation will highlight the design of the HARMONY and ENSEMBLE Phase 3 studies for pegtibatinase, scheduled for September 4, 2024. The poster, categorized under clinical studies and patient-reported outcome measures, will delve into the study methods and patient engagement strategies.
The second presentation will focus on the COMPOSE Phase 1/2 study, specifically examining the safety of pegtibatinase in children aged 5 to less than 12 years with classical HCU. This e-poster will also represent significant findings related to clinical outcomes and safety profiles.
Understanding Classical Homocystinuria
Classical homocystinuria is a rare genetic metabolic disorder that occurs due to a deficient CBS enzyme. The CBS enzyme plays a critical role in managing methionine and cysteine amino acids, and its deficiency leads to harmful levels of homocysteine in the body. This condition can trigger life-threatening events, including strokes and various complications affecting vision, bones, and cognitive development. Current treatment options primarily consist of dietary restrictions and supplementation with vitamin B6 and betaine, which do not adequately address the underlying enzymatic deficiency.
The Role of Pegtibatinase
Pegtibatinase represents a significant advancement as an investigational PEGylated, recombinant enzyme replacement therapy. Early studies have indicated its potential effectiveness in reducing total homocysteine levels, thereby improving clinical outcomes for patients suffering from classical HCU. In recent developments, the company initiated the pivotal Phase 3 HARMONY Study, designed to evaluate the efficacy and safety of pegtibatinase comprehensively.
Recent Clinical Trials and Data
Encouraging preliminary data from the Phase 1/2 COMPOSE Study revealed a notable 67.1% mean relative reduction in total homocysteine levels among patients receiving the highest dose of pegtibatinase after 12 weeks. These findings highlight the potential for pegtibatinase to be a transformative treatment option for individuals with classical HCU. The FDA has recognized the therapy's promise by granting Breakthrough Therapy designation, Rare Pediatric Disease designation, and Fast Track designation, illustrating the urgency and significance of this treatment in the marketplace.
About Travere Therapeutics
Travere Therapeutics is dedicated to improving the lives of patients with rare diseases. With a commitment to innovation and patient-centered research, the company continually collaborates with healthcare professionals and patients to develop therapies that target rare metabolic disorders. By prioritizing safety and efficacy, Travere Therapeutics aims to provide hope and tangible solutions for individuals and families coping with the challenges of rare diseases.
Contact Information
For inquiries, Travere Therapeutics can be reached at the following:
Media Contact:
Phone: 888-969-7879
Email: mediarelations@travere.com
Investor Contact:
Phone: 888-969-7879
Email: IR@travere.com
Frequently Asked Questions
What is classical homocystinuria?
Classical homocystinuria is a rare genetic disorder caused by a deficiency in the CBS enzyme, leading to elevated homocysteine levels.
What is the role of pegtibatinase?
Pegtibatinase is an investigational therapy designed to replace the deficient CBS enzyme, aiming to reduce homocysteine levels in patients with classical HCU.
When will the findings of the trials be available?
The findings from the presentations at the SSIEM symposium will be shared during the event, with further details likely published in scientific journals thereafter.
What approvals has pegtibatinase received?
Pegtibatinase has been granted Breakthrough Therapy designation, Rare Pediatric Disease designation, and Fast Track designation by the FDA.
How can I learn more about Travere Therapeutics?
For more information about the company and their therapies, visit travere.com.
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