Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that the
Company will present 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 will also
present 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 will
present 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.
Oral Presentation
Pegtibatinase, an Investigational Enzyme Replacement
Therapy for the Treatment of Classical Homocystinuria (HCU): Latest
Findings from the COMPOSE Phase 1/2 TrialParallel Session:
Clinical Studies and Outcomes IAugust 30, 9:45 – 10:00 a.m. IDT,
Oren 2
Poster PresentationsPoster presentations will
take place in the Exhibitions Hall (ICC) on August 29, 3:30 – 7:30
p.m. IDT; August 30, 7:30 a.m. – 8:35 p.m. IDT; August 31, 7:45
a.m. – 3:30 p.m. IDT; and September 1, 8:15 a.m. – 12:30 p.m.
IDT.
Clinical Characterization of Classical Homocystinuria
Due to Cystathionine-beta Synthase Deficiency: Results from the
ACAPPELLA StudyPoster B18Poster Section: Clinical Studies,
Patient Reported Outcome Measures
Clinical Burden of Classical Homocystinuria in the
United States: A Retrospective Analysis of Optum Market
ClarityPoster B36Poster Section: Clinical Studies, Patient
Reported Outcome MeasuresAugust 30, 7:11 p.m. IDT, Exhibitions Hall
(ICC) – Poster Walk & Highest Ranked Posters
Validation of a Patient Identification Algorithm to
Estimate the Prevalence of ClassicalHomocystinuria
(HCU) in the United States (US)Poster B24Poster Section:
Clinical Studies, Patient Reported Outcome Measures
Understanding the Burden of Classical Homocystinuria
(HCU) from the Patient’s Perspective: A Qualitative
StudyPoster B102Poster Section: Inborn Errors of
Metabolism in Adults
RESTORE, a Phase 3 Study to Evaluate the Effects of
Chenodeoxycholic Acid in Adults and Pediatric Patients with
Cerebrotendinous Xanthomatosis Poster B13Poster Section:
Clinical Studies, Patient Reported Outcome Measures
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,
pulmonary embolism and deep vein thrombosis, 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. To date, the
pegtibatinase program has been granted Breakthrough Therapy
designation, 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, including as a result of macroeconomic conditions;
risks 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 also faces additional risks associated with
its other products and products in development, global and
macroeconomic conditions, including health epidemics and pandemics,
including risks related to potential disruptions to clinical
trials, commercialization activity, supply chain, and manufacturing
operations. 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, including under the heading
“Risk Factors”, as included in the Company's most recent Form 10-K,
Form 10-Q and other filings with the Securities and Exchange
Commission.
Media:Nivi NehraVice President, Corporate
Communications888-969-7879mediarelations@travere.com |
Investors:Naomi EichenbaumVice President, Investor
Relations888-969-7879IR@travere.com |
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