Travere Therapeutics, Inc. (Nasdaq: TVTX) today announced the
Company has opened enrollment in the HARMONY Study, a global,
randomized pivotal Phase 3 clinical trial of pegtibatinase, a novel
investigational enzyme replacement therapy being evaluated for the
treatment of classical homocystinuria (HCU). Classical HCU is a
rare genetic metabolic disorder caused by a deficiency in the
enzyme cystathionine beta synthase (CBS). The study is designed to
determine the safety and efficacy of pegtibatinase in reducing
plasma total homocysteine (tHcy) levels, a key treatment goal in
classical HCU, compared to placebo in participants who are
receiving standard of care.
“Classical HCU is a devastating rare disease
that often manifests in childhood and can lead to serious
complications due to toxic levels of homocysteine. These
complications include continuous risk of developing
life-threatening thrombotic events, such as heart attack and
stroke, skeletal abnormalities, cognitive developmental delays, and
intellectual disabilities. Patients and caregivers have limited
treatment options, including adherence to highly restrictive diets
that often are very challenging to follow and inadequate for
maintaining metabolic control of homocysteine levels,” said William
Rote, Ph.D., senior vice president of research and development at
Travere Therapeutics. “The initiation of the HARMONY Study is an
exciting step forward in addressing this significant unmet need and
advancing our goal to deliver pegtibatinase as the first
disease-modifying treatment option for the classical HCU
community.”
The HARMONY Study is a global, randomized,
multi-center, double-blind, placebo-controlled Phase 3 clinical
trial designed to evaluate the efficacy and safety of pegtibatinase
as a novel treatment to reduce tHcy levels. The trial is expected
to enroll approximately 70 patients (≥12 to ≤65 years of age at
screening) with a diagnosis of classical HCU who continue to have
tHcy levels ≥50 μM while maintaining their standard-of-care
treatment. Participants will be randomized 1:1 to receive 2.5 mg/kg
of pegtibatinase or placebo, administered subcutaneously, for a
24-week blinded treatment duration. The primary endpoint is
relative geometric mean change in plasma tHcy levels from baseline
compared to weeks 6 through 12, and durability of treatment
response through 24 weeks of treatment will be measured as a
secondary endpoint. The Company expects topline data from the
HARMONY Study to become available in 2026. The Company will also be
initiating the ENSEMBLE Study, a Phase 3b, open-label, long-term
extension, that will evaluate the ongoing efficacy and long-term
safety of pegtibatinase in participants with HCU following their
completion of the Phase 1/2 COMPOSE Study or the Phase 3 HARMONY
Study. The ENSEMBLE Study will include an optional protein
tolerance modification sub-study that will evaluate if eligible
patients can increase their natural dietary protein intake while
maintaining an acceptable level of metabolic control while
receiving pegtibatinase.
The initiation of the Phase 3 HARMONY Study is
based on the positive safety and efficacy data from the Phase 1/2
COMPOSE Study. In COMPOSE, pegtibatinase demonstrated
dose-dependent reductions in tHcy during 12 weeks of treatment. At
the 2.5 mg/kg dose, pegtibatinase provided rapid and sustained
reductions in tHcy, with a 67.1% mean relative reduction in tHcy
from baseline, as well as maintenance of mean tHcy below the
clinically meaningful threshold of 100 μM, over weeks 6 to 12. To
date in the COMPOSE Study, pegtibatinase has been generally
well-tolerated.
Pegtibatinase has been granted Breakthrough
Therapy, Rare Pediatric Disease and Fast Track designations by the
U.S. Food and Drug Administration (FDA), as well as Orphan Drug
designation in the U.S. and Europe.
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 “on-track”,
“positioned”, “look forward to”, “will,” “would,” “may”, “might”,
“believes”, “anticipates”, “plans”, “expects”, “intends,”
“potential” 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: the potential for pegtibatinase to
become first disease-modifying treatment for classical HCU;
expectations regarding the Phase 3 HARMONY Study and sub-studies
and the anticipated timing and outcome thereof; and expectations
regarding enrollment and data availability and the anticipated
timing thereof. 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, as well as risks and
uncertainties associated with the Company’s business and finances
in general and its recently announced strategic reorganization, the
success of its commercial products and risks and uncertainties
associated with the Company’s preclinical and clinical stage
pipeline. Specifically, the Company faces risks associated with
market acceptance of its commercial products including efficacy,
safety, price, reimbursement and benefit over competing therapies,
as well as risks associated with the successful development and
execution of commercial strategies for such products, including
FILSPARI. The risks and uncertainties the Company faces with
respect to its preclinical and clinical stage pipeline include 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 its Phase 3 HARMONY
Study, the timing and potential outcome of its planned sNDA
submission for full approval of sparsentan in IgAN, and the risk
that the results from the Phase 3 DUPLEX Study of sparsentan in
FSGS will not serve as a basis for a regulatory submission for
approval of sparsentan for FSGS. There is no guarantee that
regulators will grant full approval of sparsentan for IgAN or FSGS.
The Company also faces the risk that its cash runway might not last
as long as currently anticipated and 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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