Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the
Company will present two posters in classical homocystinuria (HCU)
at the Society for the Study of Inborn Errors of Metabolism (SSIEM)
annual symposium in Porto, Portugal, September 3-6, 2024.
At SSIEM, the Company will present the trial
designs of the pivotal Phase 3 HARMONY Study and ENSEMBLE long-term
extension study of pegtibatinase, a novel investigational enzyme
replacement therapy for the treatment of classical HCU.
Additionally, the Company will share the trial design of cohort 7
in the Phase 1/2 COMPOSE Study of pegtibatinase in pediatric
participants with classical HCU.
“We believe that pegtibatinase has the potential
to effectively replace the deficient CBS enzyme activity, and that
it could ultimately become the first disease-modifying therapy for
people living with classical HCU,” said Jula Inrig, M.D., chief
medical officer of Travere Therapeutics. “We look forward to
sharing the study designs of our innovative clinical programs in
this rare metabolic disorder at the SSIEM symposium this year.”
SSIEM Poster PresentationsRosa Mota Pavilion,
Porto, Portugal
Pegtibatinase, an Investigational Enzyme
Replacement Therapy for ClassicalHomocystinuria
(HCU): design of the HARMONY and ENSEMBLE Phase 3 studies
Poster: EP-013Abstract Category: Clinical studies, patient-reported
outcome measures Wednesday, September 4, 2024, 6:15 – 8:15 p.m.
GMT+1
COMPOSE Phase 1/2, Dose-Escalation
Cohort 7 Design: safety of pegtibatinase
inchildren aged ≥5–<12 years with classical
homocystinuria (HCU)e-Poster: PO-072Abstract Category:
Clinical studies, patient reported outcome measures Wednesday,
September 4, 2024, 6:15 – 8:15 p.m. GMT+1
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 HCU. In preclinical studies,
pegtibatinase has demonstrated an ability to reduce total
homocysteine levels and improve clinical parameters.
In December 2023 the Company initiated the pivotal Phase 3
HARMONY Study to support the potential approval of pegtibatinase
for the treatment of classical HCU. 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 total homocysteine
(tHcy) levels. In May 2023 the Company announced that
data from four patients treated with the highest dose of
pegtibatinase in the Phase 1/2 COMPOSE Study 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
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
effectively replace the deficient CBS enzyme activity, the
potential for pegtibatinase to ultimately become the first
disease-modifying therapy for people living with classical HCU, and
statements relating to clinical studies, including but not limited
to trial design, anticipated results and timing related thereto.
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 studies described herein
and uncertainties associated with the regulatory review and
approval process, as well as risks and uncertainties 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 also
faces risks related to its business and finances in general, the
success of its commercial products and risks and uncertainties
associated with its preclinical and clinical stage pipeline.
Specifically, the Company faces risks associated with the ongoing
commercial launch of FILSPARI, 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 the studies described herein, and the timing
and potential outcome of the FDA’s review of the Company’s sNDA
submission for full approval of FILSPARI in IgAN. There is no
guarantee 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:888-969-7879mediarelations@travere.com |
Investors:888-969-7879IR@travere.com |
Travere Therapeutics (NASDAQ:TVTX)
過去 株価チャート
から 9 2024 まで 10 2024
Travere Therapeutics (NASDAQ:TVTX)
過去 株価チャート
から 10 2023 まで 10 2024