Applied Therapeutics Announces Positive Data Trend in AT-007 ACTION-Galactosemia Kids Pediatric Trial; Trial Will Continue to 18 Months in Blinded Format
October 06 2022 - 7:00AM
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 that the
ACTION-Galactosemia Kids Phase 3 trial has demonstrated a trend in
clinical benefit favoring AT-007 vs. placebo. The study is designed
to evaluate the impact of AT-007 vs. placebo on clinical outcomes
in children with Classic Galactosemia, with a review of safety and
efficacy every 6 months by a firewalled Data Monitoring Committee
(DMC) until the study reaches statistical significance. Review of
the data at 12 months of treatment by the DMC indicated that while
the study primary endpoint has not yet reached statistical
significance, a trend exists favoring AT-007 vs. placebo. The
clinical benefit at this early time point was most pronounced in
patients with significant deficits in clinical performance at
baseline. Safety data demonstrated that AT-007 continues to be safe
and well tolerated. The study will proceed in blinded format to the
next review at 18 months of treatment. In the meantime, the Company
will meet with the EMA to discuss potential submission of an MAA
based on existing data for conditional approval.
“Galactosemia is a debilitating disease that
greatly impacts patients and families, and there are currently no
approved treatments available,” said Riccardo Perfetti, MD, PhD,
Chief Medical Officer of Applied Therapeutics. “The data to date in
the ACTION-Galactosemia Kids study provides hope for patients and
families living with this disease. We know that entering a child in
a long-term placebo-controlled study is a difficult decision, and
we are truly thankful to the families who are participating in the
ACTION-Galactosemia clinical program.”
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 in adults with SORD Deficiency, AT-007 significantly reduced
blood sorbitol levels. 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. The drug has been generally safe and well
tolerated in all clinical studies to date. 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.
To learn more, please visit
www.appliedtherapeutics.com and follow the company on Twitter
@Applied_Tx.
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Contacts
Investors: Maeve
Conneighton(212) 600-1902
orappliedtherapeutics@argotpartners.com
Media:
media@appliedtherapeutics.com
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