HARMONY and COMPOSE Cohort 7 are clinical studies sponsored by Travere Therapeutics that are evaluating pegtibatinase, an investigational enzyme replacement therapy for classical HCU. Pegtibatinase has not been approved by the U.S. Food and Drug Administration or any other regulatory authority.
HARMONY
- Phase 3 study of pegtibatinase for people ages 12 to 65 with classical HCU.
- Participants receive either pegtibatinase or placebo during the study treatment period.
- Participation lasts about nine months.
- Approximately 70 people expected to participate in locations in the United States and other countries.
- Additional eligibility requirements apply.
COMPOSE
- Study of pegtibatinase for children ages 5 to 11 with classical HCU who are currently receiving HCU treatment.
- Participation may last up to 42 weeks, or about 10 months.
- Up to 15 children expected to participate at sites in the United States, France and Qatar.
- Additional eligibility requirements apply.
After completion, eligible participants may have the option to join the ENSEMBLE long-term extension study. Option for at home participation available.
Want to learn more?
Visit HCUconnection.com or clinicaltrials.gov. You can also check out FAQs about the study.
Ready to join? Talk to your doctor today.
Recommendations to Questions to Ask Your Doctor
- Do I, or my child, meet the initial eligibility requirements?
- How would study participation affect current HCU medicines, supplements and dietary management?
- Are there possible benefits and risks, and what happens if the treatment does not help?
- For HARMONY, what does it mean to receive a placebo?
- Who should we contact to enroll?
September 03, 2025
New long-term data from Cohort 6 of the Phase 1/2 COMPOSE open-label extension (OLE) Study demonstrated that at the target dose of 2.5 mg/kg twice weekly, participants treated with pegtibatinase in the OLE maintained significant reductions in disease-related metabolite levels, including a 53.5% relative reduction in total homocysteine and a 67.1% relative reduction in methionine over 50 weeks of treatment. Importantly, within the observation period, homocysteine remained significantly below the clinical guidelines of 100 µM. These reductions are clinically meaningful, as the accumulation of these metabolites is associated with toxicity and disease progression. Pegtibatinase was generally well-tolerated, and no new safety signals were observed.
September 26, 2024
Travere Therapeutics, Inc., announced a voluntary pause of enrollment in the Phase 3 HARMONY Study evaluating pegtibatinase for the treatment of classical homocystinuria (HCU). The voluntary enrollment pause enables the Company to work to address necessary process improvements in manufacturing scale-up to support commercial-scale manufacturing as well as full enrollment in the HARMONY Study. Patients currently enrolled in pegtibatinase studies continue to receive study medication from small-scale batches which are unaffected by the scale-up process. Currently enrolled patients will be able to continue on study medication as scheduled for the duration of the trials they are participating in.
August 22, 2024
Travere Therapeutics, Inc. announced that the Company will present two posters in classical homocystinuria (HCU) at the Society for the Study of Inborn Errors of Metabolism (SSIEM) annual symposium in Porto, Portugal, September 3-6, 2024.
At SSIEM, the Company will present the trial designs of the pivotal Phase 3 HARMONY Study and ENSEMBLE long-term extension study of pegtibatinase, a novel investigational enzyme replacement therapy for the treatment of classical HCU. Additionally, the Company will share the trial design of cohort 7 in the Phase 1/2 COMPOSE Study of pegtibatinase in pediatric participants with classical HCU.
Posters:
- Poster 72: Pegtibatinase, an Investigational Enzyme Replacement Therapy for Classical Homocystinuria (HCU): Design of the HARMONY and ENSEMBLE Phase 3 Studies
- Mechanism of action and impact of thiol homeostasis on efficacy of enzyme replacement therapy for homocystinuria
- Compose Phase 1/2, Dose-Escalation Cohort 7 Design: Safety of Pegtibatinase in Children >5 to <12 With Classical Homocystinuria (HCU)
April 4, 2024
SAN DIEGO, April 04, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. announced that the Company will present eight posters in classical homocystinuria (HCU) at Society for Inherited Metabolic Disorders (SIMD) in Charlotte, North Carolina from April 14-17, 2024, and Genetic Metabolic Dieticians International (GMDI) in Charlotte, North Carolina from April 17-20, 2024.
At SIMD and GMDI, the Company will present the trial design of the pivotal Phase 3 HARMONY Study of pegtibatinase, a novel investigational enzyme replacement therapy for the treatment of classical HCU. Additionally, the Company will share insights on the development of an innovative tool used for dietary management and monitoring in the Phase 3 HARMONY Study and open-label extension ENSEMBLE Study, and the positive results from cohort 6 in the placebo-controlled Phase 1/2 COMPOSE Study of pegtibatinase in classical HCU.
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SIMDÂ Posters
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GMDIÂ Posters
December 14, 2023
Travere Therapeutics, Inc. announced the Company has opened enrollment in the HARMONY Study, a global, randomized pivotal Phase 3 clinical trial of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU). Classical HCU is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS). The study is designed to determine the safety and efficacy of pegtibatinase in reducing plasma total homocysteine (tHcy) levels, a key treatment goal in classical HCU, compared to placebo in participants who are receiving standard of care.
August 24, 2023
Travere Therapeutics presented clinical data from the Phase 1/2 COMPOSE Study of pegtibatinase, a novel investigational enzyme replacement therapy being evaluated for the treatment of classical homocystinuria (HCU), at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium in Jerusalem, Israel, August 29 – September 1, 2023.
The Company and its collaborators also presented analyses on its prospective HCU natural history study, the prevalence of HCU, and the burden of HCU from the patient perspective highlighting the devastating nature of this rare metabolic disease over patients’ lifetimes and underscoring the urgent need for new treatments. In addition, the Company presented data on the clinical burden of HCU and the relationship between total homocysteine (tHcy) and clinical outcomes, which has been recognized as one of the highest-ranked posters at SSIEM.
- Clinical Characterization of Classical Homocystinuria Due to Cystathionine-beta Synthase Deficiency: Results from the ACAPPELLA Study
- Clinical Burden of Classical Homocystinuria in the United States: A Retrospective Analysis of Optum Market Clarity
- Validation of a Patient Identification Algorithm to Estimate the Prevalence of Classical
Homocystinuria (HCU) in the United States (US) - Understanding the Burden of Classical Homocystinuria (HCU) from the Patient’s Perspective: A Qualitative Study
May 31, 2023
- Data from the four patients treated with the highest dose of pegtibatinase showed a clinically meaningful 67.1% mean relative reduction in total homocysteine from baseline
- Pegtibatinase has been generally well-tolerated to date
- The Company is engaging with regulators and expects to initiate a pivotal Phase 3 Study by year-end 2023Â
January 09, 2023
Travere Therapeutics Provides Corporate Update and 2023 Outlook
October 27, 2022
Travere Therapeutics Reports Third Quarter 2022 Financial Results and
Corporate Updates
Travere Therapeutics, Inc. Â today reported its third quarter 2022 financial results and provided a corporate update.
A Breakthrough Therapy Designation was granted to the pegtibatinase development program for classical homocystinuria (HCU).
August 25, 2022
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Population-based Incidence Estimates of Classical Homocystinuria Using the Genome Aggregation Database (gnomAD)
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A Longitudinal Study of Cognitive Function in Classical Homocystinuria Demonstrates Distinct Deficits in Inhibitory Control
- Insights from the First Genetic Evaluation of a Longitudinal Natural History Study in Classical Homocystinuria (HCU)
August 3, 2022
Travere Therapeutics Provides Regulatory Updates on its Development Programs
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Pegtibatinase granted Breakthrough Therapy Designation by FDA
