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 poster presentations at
the 2022 43rd Annual Meeting of the Society for Inherited Metabolic
Disorders, April 10 - 13, 2022 in Orlando, Florida.
“Baseline data from the ACTION-Galactosemia Kids
study illustrates the high burden of disease associated with
Galactosemia, including neurological complications, due to high
galactitol levels,” said Riccardo Perfetti, MD, PhD, Chief Medical
Officer of Applied Therapeutics. “This data underscores the urgent
need to bring a treatment to people living with Galactosemia.”
Presentation Details
POSTER #68: Baseline
Disease Characteristics of 47 Pediatric Classic Galactosemia
Patients in the Action-Galactosemia Kids AT-007 Interventional
Study Demonstrate the Multi-System Burden of DiseaseR
Perfetti, A Ahmad, H Phan, J Thomas, F Lawson, E Bailey, L
Saltonstall, and S Shendelman
- In the
ACTION-Galactosemia Kids study, children presented with deficits in
speech, cognition, motor skills, and adaptive behavior.
POSTER #69: Galactitol, But Not Gal-1p,
Level is a Predictor of Disease Severity in Children with Classic
Galactosemia on Galactose Restricted DietR Perfetti, E
Bailey, F Lawson, S Wang, L Saltonstall, M Krumnow, and S
Shendelman
-
Higher galactitol levels, but not higher Gal-1p levels, were
associated with greater disease severity overall and on each of the
four quadrants of CNS function.
About Galactosemia
Galactosemia is a rare genetic metabolic disease
resulting in an inability to metabolize the simple sugar galactose.
Galactose is found in foods but is also produced endogenously by
the body. When not metabolized properly, galactose is converted to
the toxic metabolite, galactitol, which causes neurological
complications, including deficiencies in speech, cognition,
behavior, and motor skills, and also results in juvenile cataracts
and ovarian insufficiency (in women). There are approximately 3,000
patients with Galactosemia in the US and 80 new births per
year.
About AT-007
AT-007 is a central nervous system (CNS)
penetrant Aldose Reductase inhibitor (ARI) in development for the
treatment of several rare neurological diseases, including
Galactosemia, SORD Deficiency and PMM2-CDG. In an animal model of
Galactosemia, AT-007 reduced toxic galactitol levels and prevented
disease complications. In clinical trials, AT-007 significantly
reduced plasma galactitol levels vs. placebo in adults and children
with Galactosemia. AT-007 is currently being studied in a Phase 3
clinical outcomes trial (ACTION-Galactosemia Kids) in children ages
2-17 with Galactosemia, as well as a long-term open label study in
adults with Galactosemia. AT-007 has received both Orphan Drug and
Pediatric Rare Disease designations from the U.S. Food and Drug
Administration (FDA) for the treatment of Galactosemia and
PMM2-CDG, and Fast Track designation for 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.
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, 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 “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.
Contacts
Investors:
Maghan Meyers(212) 600-1902
appliedtherapeutics@argotpartners.com
Media:
media@appliedtherapeutics.com
Applied Therapeutics, Inc.
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