Applied Therapeutics Inc. (Nasdaq:APLT), a clinical-stage
biopharmaceutical company developing a pipeline of novel drug
candidates against validated molecular targets in indications of
high unmet medical need, today announced the initiation of a Phase
1/2 study of AT-007 in Galactosemia. The study, termed
ACTION-Galactosemia, will investigate safety and pharmacokinetics
(PK) of AT-007, a central nervous system (CNS) penetrant Aldose
Reductase inhibitor in healthy volunteers, and will then explore
safety, PK and biomarker effects in adult subjects with
Galactosemia.
The multicenter study will primarily enroll
patients with Classic Galactosemia, the most common form of
Galactosemia in the US, but will also seek to enroll a single
cohort of GALK deficient (GALKD) patients, which is more common in
Europe.
“Dietary restriction alone does not prevent long
term complications of disease because the body makes galactose
endogenously,” said Riccardo Perfetti, MD, PhD, Chief Medical
Officer of Applied Therapeutics. “Galactosemia patients are in
desperate need of treatment options, and we are committed to
advancing AT-007 through clinical development as quickly as
possible due to the urgent unmet medical need.”
Applied Therapeutics plans to leverage recent
FDA guidance permitting biomarker-based development in low
prevalence, slowly progressing rare metabolic diseases, such as
Galactosemia.
Aldose Reductase (AR) is responsible for
formation of a toxic metabolite of galactose, called galactitol,
which has been shown to be responsible for many of the long-term
complications in Galactosemia. Patients will be treated
initially with a single dose of AT-007, followed by 27 days of
consecutive dosing. The study endpoints will be safety,
tolerability, PK and biomarker-based analyses. The trial will
determine the ability of AT-007 to suppress toxic accumulation of
galactitol in patients and will explore feasibility of measuring
galactitol levels in the brain via MRI.
“The initiation of the first therapeutic
clinical trial in Galactosemia marks an important milestone for the
Galactosemia community,” said Kellie Wilcox, President of the
Galactosemia Foundation. “Decades of work by the Galactosemia
community have culminated in this day - advocacy for newborn
screening, academic research, and participation in natural history
studies by families. We look forward to working closely with
Applied Therapeutics and the Galactosemia community in the next
phase of this journey.”
About GalactosemiaGalactosemia
is a rare metabolic disease that affects how the body processes a
simple sugar called galactose, and for which there is no known cure
or approved treatment available. Galactose is found in foods, but
the human body also naturally produces galactose on its own, so
dietary restriction can’t prevent complications of disease.
It is estimated that the U.S. Galactosemia population is
approximately 2,800 patients, based on newborn screening data
identifying 2,500 infants through 2014, and the estimated birth
rate of 80 patients per year. High levels of galactose circulating
in the blood and tissues of Galactosemia patients enables Aldose
Reductase to convert galactose to a toxic metabolite, called
galactitol, which causes long-term complications ranging from CNS
dysfunction to cataracts.
About AT-007AT-007 is a central
nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in
Phase 1/2 development for treatment of Galactosemia. AT-007 has
been studied in in an animal model of Galactosemia, which
demonstrated that AT-007 reduces toxic galactitol levels and
prevents disease complications. Applied Therapeutics is planning a
biomarker based development program in patients with Galactosemia,
based on the recently released draft industry guidance on drug
development for low prevalence, slowly progressing rare metabolic
diseases. The company received Orphan Designation for AT-007 for
Galactosemia in May 2019.
About Applied
TherapeuticsApplied Therapeutics is a clinical-stage
biopharmaceutical company developing a pipeline of novel drug
candidates against validated molecular targets in indications of
high unmet medical need. The company’s lead drug candidate, AT-001,
is a novel aldose reductase inhibitor (ARI) that is being developed
for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal
fibrosis of the heart. The company plans to initiate a Phase 2/3
pivotal study in DbCM in 2019. Applied Therapeutics is also
developing AT-007, a central nervous system penetrant ARI, for the
treatment of Galactosemia, a rare pediatric metabolic disease, and
initiated a Phase 1/2 clinical trial in June 2019. The preclinical
pipeline also includes AT-003, an ARI designed to cross through the
back of the eye when dosed orally, for the treatment of diabetic
retinopathy, expected to advance into a Phase 1 study in 2020.
Forward-looking StatementsThis
press release contains “forward-looking statements” that involve
substantial risks and uncertainties for purposes of the safe harbor
provided by the Private Securities Litigation Reform Act of 1995.
Any statements, other than statements of historical fact, included
in this press release regarding strategy, future operations,
prospects, plans and objectives of management, including words such
as "may," "will," "expect," "anticipate," "plan," "intend," and
similar expressions (as well as other words or expressions
referencing future events, conditions or circumstances) are
forward-looking statements. These include, without limitation,
statements regarding (i) the design, timing, scope and results of
our clinical trials, (ii) anticipated timing of disclosure of
results of our clinical trials, (iii) the potential benefits of the
formulated product candidate, (iv) the likelihood data will support
future development and (iv) the likelihood of obtaining regulatory
approval of our product candidates. Forward-looking statements in
this release involve substantial risks and uncertainties that could
cause actual results to differ materially from those expressed or
implied by the forward-looking statements, and we, therefore cannot
assure you that our plans, intentions, expectations or strategies
will be attained or achieved. Such risks and uncertainties include,
without limitation, the uncertainties inherent in the initiation,
execution and completion of clinical trials, in the timing of
availability of trial data, in the results of the clinical trials,
in the actions of regulatory agencies, in the commercialization and
acceptance of new therapies. Other factors that may cause actual
results to differ from those expressed or implied in the
forward-looking statements in this press release are discussed in
our filings with the U.S. Securities and Exchange Commission,
including the section titled “Risk Factors” contained therein.
Except as otherwise required by law, we disclaim any intention or
obligation to update or revise any forward-looking statements,
which speak only as of the date they were made, whether as a result
of new information, future events or circumstances or
otherwise.
ContactsInvestors:Maeve Conneighton(212)
600-1902 orappliedtherapeutics@argotpartners.com
Media:media@appliedtherapeutics.com
Galactosemia Patients/ Families:If you are a
patient or family member interested in receiving information
regarding participation in the Phase 1/2 clinical trial, please
email: galactosemia@appliedtherapeutics.com.
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