Travere Therapeutics to present abstracts at the annual symposium of the Society for the Study of Genetic Errors in Metabolism

Travere Therapeutics, Inc.

Travere Therapeutics, Inc.

SAN DIEGO, August 25, 2022 (GLOBE NEWSWIRE) — Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the company and its collaborators will provide a genetic assessment and analysis of cognitive function from the company’s ongoing longitudinal natural history. Study of people with classic homocystinuria (HCU), at the Annual Symposium of the Society for the Study of Inherited Errors in Metabolism in Freiburg, Germany, August 30 – September 2, 2022. Data that explores population-based genomic estimates of the incidence of HCU are also presented. . The company is currently developing a new investigational enzyme replacement therapy, pegtibatinase, for the treatment of HCU.

The authors will be present at the poster rally at Messe Freiburg on August 31, 2022, from 18:45 – 20:15 CET. Posters will also be available electronically and will be on display in the Poster Gallery Hall from Tuesday, August 30 through Friday, September 2.

Population-based incidence estimates for classic homocystinuria using the genome assembly database (gnomAD)
Label No.: PO10-2316

A longitudinal study of cognitive function in classic homocystinuria illustrating distinct deficiencies in inhibitory control
Label No.: PO10-2388

Insights from the first genetic evaluation of a longitudinal natural history study in classical homocystinuria (HCU)
Label No.: PO10-2629

About classic homocystinuria

Classical homocystinuria (HCU) is a rare genetic metabolic disorder caused by a deficiency of the enzyme beta cystathionine synthase (CBS). CBS is a pivotal enzyme necessary for the management of methionine and cysteine ​​in the body. Classic HCU leads to toxic levels of homocysteine ​​that can lead to life-threatening thrombotic events such as stroke, heart attack, ophthalmic and skeletal complications, as well as delayed growth. Current treatment options are limited to a protein-restricted diet and supplemental use of vitamin B6 and betaine.

About Pegtibatinase

Pegtibatinase is a recombinant, PEGylated enzyme replacement therapy designed to address the underlying cause of classic HCU homocystinuria. In preclinical studies, pegtibatinase has demonstrated the ability to reduce total homocysteine ​​levels and improve clinical parameters. Pegtibatinase is currently progressing in an ongoing phase 1/2 compound study to evaluate its safety, tolerability, pharmacodynamics, pharmacodynamics, and clinical effects in patients with classic HCU. Pegtibatinase has been granted breakthrough therapy, rare pediatric diseases, and fast-track designations by the U.S. Food and Drug Administration, as well as orphan drug designation in the United States and Europe.

About Traverse Therapeutics

At Travere Therapeutics, we are lifelong rares. We are a biopharmaceutical company that meets every day to help patients, families, and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know that the need for treatment options is urgent – which is why our global team works with the rare disease community to identify, develop, and deliver life-changing treatments. In pursuit of this mission, we continually strive to understand the diverse perspectives of rare patients and courageously create new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit

forward-looking statements

This press release contains “forward-looking statements” as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, such statements are often identified by the words “may,” “may,” “believe,” “think,” “expect,” “plan,” “expect,” “intend,” or similar expressions. In addition, expressions of our strategies, intentions or plans are also forward-looking statements. These forward-looking statements include, but are not limited to, references to pegtibatinase that are designed to address the underlying cause of classic homocystinuria and references to a phase 1/2 structure study. These forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, distort or change any of them, and could cause actual results and outcomes to differ materially from current expectations. No forward-looking statement can be guaranteed. Factors that could cause actual results to differ materially from those indicated in the forward-looking statements include risks and uncertainties associated with the regulatory review and approval process, risks associated with enrollment in clinical trials for rare diseases and ongoing or planned clinical risks. Trials may not be successful or may be delayed for safety, regulatory, or other reasons. The Company faces a risk that it will not be able to raise additional financing that may be required to complete development of any or all of the Candidate Products; Risks related to the company’s reliance on contractors for clinical drug supply and commercial manufacturing; Uncertainties related to patent protection, periods of exclusivity and intellectual property rights of third parties; risks associated with regulatory interactions; and risks and uncertainties related to competitive products, including current and potential future general competition with some of the company’s products, and technological changes that may limit demand for the company’s products. The Company faces additional risks associated with the potential impacts of the COVID-19 pandemic on its business, including but not limited to (i) the Company’s ability to continue its ongoing development activities and clinical trials, (ii) the timing of such clinical trials and the release of data from those trials, (iii) ) the ability of the company and its suppliers to successfully manufacture its commercial products and candidate products, and (iv) the market and sales of its commercial products. You are cautioned not to place undue reliance on these forward-looking statements because there are significant factors that could cause actual results to differ materially from those in the forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Investors are referred to the full discussion of risks and uncertainties as contained in the company’s most recent Form 10-Q, Form 10-K, and other filings with the Securities and Exchange Commission.

Chris Klein, Chartered Financial Analyst
Senior Vice President, Investor Relations and Corporate Communications

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