Travere Therapeutics to Present at 2024 Metabolism Symposium

Travere Therapeutics, Inc. will present two significant posters related to classical homocystinuria (HCU) at the Society for the Study of Inborn Errors of Metabolism (SSIEM) annual symposium in Porto, Portugal, taking place from September 3-6, 2024. The focus will be on the clinical study designs for pegtibatinase, which is being investigated as a potential disease-modifying treatment for this rare metabolic disorder.

At the SSIEM symposium, Travere Therapeutics will unveil the trial designs for both the pivotal Phase 3 HARMONY Study and the ENSEMBLE long-term extension study. These studies assess the efficacy and safety of pegtibatinase, a novel enzyme replacement therapy intended for the treatment of classical HCU. The company will also present the design of cohort 7 from the Phase 1/2 COMPOSE Study, focusing on pediatric participants with classical HCU.

Dr. Jula Inrig, the Chief Medical Officer of Travere Therapeutics, highlighted the potential of pegtibatinase to effectively replace the deficient CBS enzyme activity in HCU patients. She emphasized that this enzyme replacement could become the first disease-modifying therapy for individuals suffering from this disorder. The company is eager to share its innovative clinical program designs at the upcoming SSIEM symposium.

The presentations are scheduled as follows:

1. Pegtibatinase, an Investigational Enzyme Replacement Therapy for Classical Homocystinuria (HCU): Design of the HARMONY and ENSEMBLE Phase 3 Studies
- Poster: EP-013
- Abstract Category: Clinical studies, patient-reported outcome measures
- Date and Time: Wednesday, September 4, 2024, from 6:15 to 8:15 p.m. GMT+1

2. COMPOSE Phase 1/2, Dose-Escalation Cohort 7 Design: Safety of Pegtibatinase in Children Aged ≥5–<12 Years with Classical Homocystinuria (HCU)
- e-Poster: PO-072
- Abstract Category: Clinical studies, patient-reported outcome measures
- Date and Time: Wednesday, September 4, 2024, from 6:15 to 8:15 p.m. GMT+1

Classical homocystinuria (HCU) is a rare genetic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS). This enzyme plays a crucial role in managing methionine and cysteine in the body. A deficiency leads to toxic levels of homocysteine, which can result in severe health issues such as thrombotic events (including stroke and pulmonary embolism), ophthalmologic and skeletal complications, and developmental delays. Current treatments are limited to dietary restrictions and the use of vitamin B6 and betaine.

Pegtibatinase is an investigational PEGylated recombinant enzyme replacement therapy designed to tackle the root cause of classical HCU. Preclinical studies have shown that pegtibatinase can lower total homocysteine levels and improve clinical outcomes. In December 2023, Travere Therapeutics initiated the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase for treating classical HCU. The HARMONY Study is a global, randomized, multi-center, double-blind, placebo-controlled clinical trial aimed at evaluating the efficacy and safety of pegtibatinase in reducing total homocysteine levels.

In May 2023, data from the Phase 1/2 COMPOSE Study indicated that four patients who received the highest dose of pegtibatinase experienced a significant 67.1% reduction in total homocysteine levels from baseline after 12 weeks of treatment. The treatment was generally well-tolerated. Pegtibatinase has received several designations from the FDA, including Breakthrough Therapy, Rare Pediatric Disease, and Fast Track, as well as Orphan Drug designation in both the U.S. and Europe.

Travere Therapeutics is dedicated to developing and delivering innovative therapies for rare diseases. The company collaborates closely with the rare disease community to meet urgent treatment needs and provide hope for patients and their families.

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