Travere Therapeutics to Present Abstracts at the Society for the Study of Inborn Errors of Metabolism Annual Symposium
August 25 2022 - 4:30PM
Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the
Company and its collaborators will present a genetic evaluation and
an analysis of cognitive function from the Company’s ongoing
longitudinal natural history study of people living with classical
homocystinuria (HCU), at the Society for the Study of Inborn Errors
of Metabolism Annual Symposium in Freiburg, Germany, August 30 –
September 2, 2022. Data exploring genomic population-based
estimates of the incidence of HCU will also be presented. The
Company is currently advancing a novel investigational enzyme
replacement therapy, pegtibatinase, for the treatment of HCU.
Authors will be present at the poster walk in
Messe Freiburg on August 31, 2022, from 18:45 – 20:15 CEST. Posters
will also be available electronically and displayed in the poster
exhibition hall from Tuesday, August 30 until Friday, September
2.
Population-based Incidence Estimates of
Classical Homocystinuria Using the Genome Aggregation Database
(gnomAD) Poster #: PO10-2316
A Longitudinal Study of Cognitive
Function in Classical Homocystinuria Demonstrates Distinct Deficits
in Inhibitory Control Poster #: PO10-2388
Insights from the First Genetic
Evaluation of a Longitudinal Natural History Study in Classical
Homocystinuria (HCU)Poster #: PO10-2629
About Classical
Homocystinuria
Classical homocystinuria (HCU) is a rare genetic
metabolic disorder caused by a deficiency in the enzyme
cystathionine beta synthase (CBS). CBS is a pivotal enzyme that is
essential for the management of methionine and cysteine in the
body. Classical HCU leads to toxic levels of homocysteine that can
result in life-threatening thrombotic events such as stroke and
heart attacks, ophthalmologic and skeletal complications, as well
as developmental delay. Current treatment options are limited to
protein-restricted diet and supplemental use of vitamin B6 and
betaine.
About Pegtibatinase
Pegtibatinase is an investigational PEGylated,
recombinant enzyme replacement therapy designed to address the
underlying cause of classical homocystinuria HCU. In preclinical
studies, pegtibatinase has demonstrated an ability to reduce total
homocysteine levels and improve clinical parameters. Pegtibatinase
is currently advancing in the ongoing Phase 1/2 COMPOSE Study to
assess its safety, tolerability, pharmacokinetics, pharmacodynamics
and clinical effects in patients with classical HCU. Pegtibatinase
has been granted Breakthrough Therapy, Rare Pediatric Disease and
Fast Track designations by the FDA, as well as Orphan Drug
designation in the US and Europe.
About Travere Therapeutics
At Travere Therapeutics, we are in rare for
life. We are a biopharmaceutical company that comes together every
day to help patients, families and caregivers of all backgrounds as
they navigate life with a rare disease. On this path, we know the
need for treatment options is urgent – that is why our global team
works with the rare disease community to identify, develop and
deliver life-changing therapies. In pursuit of this mission, we
continuously seek to understand the diverse perspectives of rare
patients and to courageously forge new paths to make a difference
in their lives and provide hope – today and tomorrow. For more
information, visit travere.com
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,
these statements are often identified by the words "may", "might",
"believes", "thinks", "anticipates", "plans", "expects", "intends"
or similar expressions. In addition, expressions of our strategies,
intentions or plans are also forward-looking statements. Such
forward-looking statements include, but are not limited to,
references to pegtibatinase being designed to address the
underlying cause of classical homocystinuria and references to the
Phase 1/2 COMPOSE Study. Such forward-looking statements are based
on current expectations and involve inherent risks and
uncertainties, including factors that could delay, divert or change
any of them, and could cause actual outcomes and results to differ
materially from current expectations. No forward-looking statement
can be guaranteed. Among the factors that could cause actual
results to differ materially from those indicated in the
forward-looking statements are risks and uncertainties associated
with the regulatory review and approval process, risk associated
with enrollment of clinical trials for rare diseases and risk that
ongoing or planned clinical trials may not succeed or may be
delayed for safety, regulatory or other reasons. The Company faces
risk that it will be unable to raise additional funding that may be
required to complete development of any or all of its product
candidates; risk relating to the Company's dependence on
contractors for clinical drug supply and commercial manufacturing;
uncertainties relating to patent protection and exclusivity periods
and intellectual property rights of third parties; risks associated
with regulatory interactions; and risks and uncertainties relating
to competitive products, including current and potential future
generic competition with certain 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 the COVID-19 pandemic may have 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 Company’s and its suppliers’ ability to
successfully manufacture its commercial products and product
candidates, and (iv) the market for and sales of its commercial
products. You are cautioned not to place undue reliance on these
forward-looking statements as there are important factors that
could cause actual results to differ materially from those in
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 included in the Company's
most recent Form 10-Q, Form 10-K and other filings with the
Securities and Exchange Commission.
Contact:Chris Cline, CFASenior Vice President,
Investor Relations & Corporate
Communications888-969-7879IR@travere.com
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