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 presentations at the 2022
Annual Meeting of the Peripheral Nerve Society, May 14 - 17 in
Miami, Florida.
“The data presented at PNS represents a
comprehensive understanding of the role of toxic sorbitol in
disease pathogenesis in SORD Deficiency, as well as ability to
reduce sorbitol levels and prevent disease progression with AT-007
treatment,” said Riccardo Perfetti MD, PhD, Chief Medical Officer
of Applied Therapeutics. “In a pilot study in patients with SORD
Deficiency, sorbitol level correlated with disease severity, and
AT-007 treatment substantially reduced sorbitol levels. In a
Drosophila model of disease, elevated sorbitol resulted in neuronal
damage and decline in mobility, and AT-007 treatment prevented the
disease phenotype by inhibiting sorbitol production. Taken
together, this data significantly advances our understanding of the
disease and potential for treatment with AT-007.”
Presentation Details
Oral Presentation: Aldose
Reductase Inhibitor AT-007 Prevents Mitochondrial Dysfunction and
Neurodegeneration in Sorbitol Dehydrogenase Deficiency-Induced
NeuropathyYi Zhu, MD, PhD, Amanda Lobato, Adriana Rebelo, PhD,
Tijana Canic, Sheyum Syed, PhD, MS, Riccardo Perfetti, MD, PhD,
Shoshana Shendelman, PhD, Stephan Züchner, MD, PhD, and Grace Zhai,
PhDOral Presentation Date and Time: Monday, May
16, 2022, 4:30 PM EDTPoster Date and Time: Sunday,
May 15, 2022, 12:00 – 2:00 PM EDTIn-Person Poster
Number: 152; E-Poster
Number: 1354
Poster: Circulating Sorbitol
Levels Correlate with Severity of Disease in Patients with Sorbitol
Dehydrogenase (SORD) DeficiencyRiccardo Perfetti, MD, PhD, and
Shoshana Shendelman, PhDDate and Time: Poster
Session II, Tuesday, May 17, 2022, 12:00 – 2:00 PM
EDTIn-Person Poster
Number: 162; E-Poster
Number: 1383
Poster: AT-007 Significantly
Lowers Blood Sorbitol Levels in Patients with Hereditary Neuropathy
Resulting from Sorbitol Dehydrogenase (SORD) DeficiencyRiccardo
Perfetti MD, PhD, Jahannaz Dastgir, DO, and Shoshana Shendelman,
PhDDate and Time: Poster Session II, Tuesday, May
17, 2022, 12:00 – 2:00 PM EDTIn-Person Poster
Number: 53; E-Poster
Number: 1203
About Sorbitol Dehydrogenase (SORD)
Deficiency
Sorbitol Dehydrogenase Deficiency (SORD
Deficiency) is a rare, progressive, debilitating hereditary
neuropathy that affects peripheral nerves and motor neurons. SORD
Deficiency is one of the most common forms of recessive hereditary
neuropathy and affects approximately 3,300 patients in the U.S. and
4,000 patients in Europe. The disease is caused by a lack of the
enzyme sorbitol dehydrogenase, responsible for the metabolism of
sorbitol, which causes sorbitol to accumulate at high levels and
become toxic to the body. Intracellular sorbitol accumulation
results in significant disability, loss of sensory function,
neuromuscular dysfunction, and decreased mobility.
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 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. In a pilot
study, AT-007 significantly reduced blood sorbitol levels in adults
with SORD Deficiency. AT-007 is currently being studied in a Phase
3 trial (INSPIRE) investigating biomarker efficacy, clinical
outcomes, and significantly reduced blood sorbitol levels in adults
with SORD Deficiency. 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.
Grafico Azioni Applied Therapeutics (NASDAQ:APLT)
Storico
Da Giu 2024 a Lug 2024
Grafico Azioni Applied Therapeutics (NASDAQ:APLT)
Storico
Da Lug 2023 a Lug 2024