Travere Therapeutics, Inc., (Nasdaq: TVTX) today 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.
“Classical HCU is an isolating and devastating
rare disorder with very limited treatment options, including
adherence to highly restrictive diets, leaving patients and their
caregivers with immense challenges,” said Jula Inrig, M.D., chief
medical officer of Travere Therapeutics. “We look forward to
participating at both of these prestigious metabolic medical
meetings, and we are excited about our work pioneering the first
potential disease-modifying treatment for the thousands of people
affected by classical HCU.”
SIMD Poster Presentations
Latest Results From the
COMPOSE Phase 1/2 Trial For the
Treatment of Classical Homocystinuria (HCU) Using Pegtibatinase, a
Novel Investigational Enzyme Replacement TherapyPoster: 139Abstract
Category: Innovative TherapiesSymphony Ballrooms 4-7; April 15,
2024, 8-9 p.m. ET
Pegtibatinase, an Investigational Enzyme
Replacement Therapy, For The Treatment of Classical Homocystinuria
(HCU): Design of the HARMONY Phase 3 Study Poster:
147Abstract Category: Innovative Therapies Symphony Ballrooms 4-7;
April 15, 2024, 8-9 p.m. ET
Economic Burden of Classical
Homocystinuria in the United StatesPoster: 48Abstract
Category: Clinical Care/ResearchSymphony Ballrooms 4-7; April 15,
2024, 7-8 p.m. ET
Clinical Burden of Classical
Homocystinuria in the United States: A Retrospective Claims
Analysis Poster: 64Abstract Category: Clinical
Care/ResearchSymphony Ballrooms 4-7; April 15, 2024, 7-8 p.m.
ET
Association Between Homocysteine and
Clinical Outcomes in Patients with Classical Homocystinuria: A
Systematic Literature ReviewPoster: 68Abstract Category:
Clinical Care/ResearchSymphony Ballrooms 4-7; April 15, 2024, 7-8
p.m. ET
GMDI Poster Presentations
Latest Results from the
COMPOSE Phase 1/2 Trial of Pegtibatinase, a
Novel Investigational Enzyme Replacement Therapy for Classical
Homocystinuria (HCU) Poster: 10April 18, 2024, 4:30-5:15
p.m. ET
Novel Tool for Dietary Management and
Monitoring in Clinical Trials of Pegtibatinase, an Investigational
Enzyme Replacement Therapy for Classical Homocystinuria
Poster: 16April 18, 2024, 4:30-5:15 p.m. ET
Novel Dietary Management Strategies for
Classical Homocystinuria (HCU) in HARMONY/ENSEMBLE Phase 3 Studies
of Pegtibatinase, an Investigational Enzyme Replacement
Therapy Poster: 33April 18, 2024, 5:15-6 p.m. ET
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 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. In May
2023, the Company announced that data from four patients treated
with the highest dose of pegtibatinase showed a clinically
meaningful 67.1% mean relative reduction in total homocysteine from
baseline and was generally well-tolerated after 12 weeks of
treatment. 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 U.S. 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 “anticipate,”
“believe,” “expect,” “intend,” “may,” “might,” “objective,” “plan,”
“will” 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, statements regarding pegtibatinase as the first
potential disease-modifying treatment for classical homocystinuria,
references to the HARMONY study and the other studies described
herein, including statements regarding expectations related
thereto, and statements regarding the development of an innovative
tool used for dietary management and monitoring. 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
related to the timing and outcome of the HARMONY study and the
other studies described herein; risks and uncertainties associated
with the regulatory review and approval process, risks associated
with enrollment of clinical trials for rare diseases, and risks
that ongoing or planned clinical trials may not succeed or may be
delayed for safety, regulatory or other reasons. The Company faces
risks related to its business and finances in general; risks
related to the commercial launch of a new product; risks associated
with market acceptance of FILSPARI and other current and future
products, including efficacy, safety, price, reimbursement and
benefit over competing therapies; and the risk that the Company’s
clinical candidates will not be found to be safe or effective and
that current or anticipated future clinical trials will not proceed
as planned. Specifically, the Company faces risks related to the
timing and potential outcome of the FDA’s potential acceptance for
filing and review of the sNDA submission for full approval of
FILSPARI in IgAN, and the timing and potential outcome of the
European Commission’s decision regarding conditional marketing
authorization of sparsentan for IgAN. There is no guarantee that
the FDA will accept the sNDA submission for filing, that the
European Commission will grant conditional marketing authorization
of sparsentan for IgAN, or that regulators will grant full approval
of sparsentan for IgAN or FSGS. The Company also faces the 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
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.
Contact Info
Media:888-969-7879 mediarelations@travere.com |
Investors:888-969-7879 IR@travere.com |
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