Applied Therapeutics Reports Initial Pediatric Biomarker Data from ACTION-Galactosemia Kids
09 Settembre 2021 - 1:00PM
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 reported initial biomarker data from
the pediatric ACTION-Galactosemia Kids study, as well as baseline
analysis of the 47 children enrolled in the study.
The results demonstrate a substantial reduction
in plasma galactitol of approximately 35% after 30 days of
treatment, which was statistically significant (p<0.001) vs.
placebo. AT-007 was safe and well tolerated in children of
all ages (2-17). A pharmacokinetic analysis of AT-007 plasma
drug levels at day 30 revealed that dosing could be further
optimized by adjusting dose based on weight rather than age.
As such, weight-based dosing brackets have been implemented for the
remainder of the study, and a subset of patients have been
dose-adjusted. The Company expects to present additional biomarker
data at the optimized drug levels pending analysis at day 30.
Due to the time required for these study changes, the Company now
plans to submit a New Drug Application (NDA) to the U.S. Food and
Drug Administration (FDA) in Q4 of this year, and has adjusted
projected timelines accordingly.
Additionally, analysis of the 47 children in the
ACTION-Galactosemia Kids study demonstrated a clear correlation
between baseline galactitol level and baseline clinical functional
outcomes. Children with higher plasma galactitol levels displayed
greater disease severity vs. children with lower plasma galactitol
levels at baseline. This data is the first demonstration of
correlation of a biochemical biomarker with severity of disease in
Galactosemia patients. Full data will be presented at a future
medical conference.
“Reduction in toxic galactitol is critically
important to patients with Galactosemia,” said Shoshana Shendelman,
PhD, CEO, Founder and Chair of the Board of Applied Therapeutics.
“AT-007 has previously demonstrated significant reduction in
galactitol in adults with Galactosemia. Now, we have demonstrated a
significant reduction in children with this devastating rare
disease. As the first interventional clinical study in pediatric
Galactosemia patients, ACTION-Galactosemia Kids has already
provided important insight into disease severity and progression.
This new data demonstrating correlation of galactitol level with
severity of disease underscores the importance of reducing this
toxic metabolite in patients with Galactosemia.”
About Applied Therapeutics
Applied 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-007,
is a novel central nervous system penetrant Aldose Reductase
Inhibitor (ARI) for the treatment of CNS rare metabolic diseases,
including Galactosemia, SORD Deficiency and PMM2-CDG. The Company
is also developing AT-001, a novel potent ARI, for the treatment of
Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart.
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, as well as novel dual PI3k
inhibitors in preclinical development for orphan oncology
indications.
To learn more, please visit
www.appliedtherapeutics.com and follow the company on Twitter
@Applied_Tx.
Forward-Looking Statements
This 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 timing of
our NDA submission for potential approval of AT-007, which will
include data from the ACTION-Galactosemia Kids trial and the 90-day
safety data in adults with Galactosemia, (ii) the timing of our
rare disease franchise expansion programs in SORD Deficiency, (iii)
the timing of the initiation and completion of our clinical trials,
including ARISE-HF, (iv) the likelihood that data from our clinical
trials will support future development of our product candidates
and (v) 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, (i) our plans to develop and commercialize our product
candidates, (ii) the initiation, timing, progress and results of
our current and future preclinical studies and clinical trials and
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advantage of expedited regulatory pathways for any of our product
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Contacts
Investors:Maghan Meyers (212) 600-1902
orappliedtherapeutics@argotpartners.com
Media:media@appliedtherapeutics.com
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