Applied Therapeutics Announces Restart of Pediatric Galactosemia Study
01 Febbraio 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 announced that the FDA has lifted
the hold and the AT-007 ACTION-Galactosemia Kids pediatric clinical
study will resume effective immediately. Applied Therapeutics has
worked closely with FDA to modify the trial, with the shared goal
of ensuring that all patients have the opportunity to receive
clinical benefit.
Previously, the ACTION-Galactosemia Kids program
was split into two separate clinical studies – a dose escalation
and biomarker study followed by a separate long-term clinical
outcomes study. The two studies have now been combined into a
single two-part study to ensure that all patients who complete the
dose escalation and biomarker portion of the study will seamlessly
continue into the long-term outcomes study without treatment
interruption. Additionally, the dose-escalation portion of the
study has been operationally modified to ensure continuous drug
treatment and participation throughout the study. All patients who
were already randomized and participating in the
ACTION-Galactosemia Kids study will be eligible to return to the
study, and the Company anticipates being fully enrolled within a
matter of weeks. The Company remains on target to submit an NDA no
later than Q3 2021.
“We thank the Galactosemia community for their
patience and support, and we are grateful to the FDA for their
partnership,” said Shoshana Shendelman, PhD, Founder and CEO of
Applied Therapeutics. “We believe that the program is in a stronger
position for overall success and potential approval due to our
close collaboration with FDA. AT-007 represents an important
advancement for Galactosemia patients, and offering a therapeutic
option to children is a priority for Applied Therapeutics.”
About AT-007
AT-007 is a central nervous system (CNS)
penetrant Aldose Reductase inhibitor (ARI) in clinical development
for treatment of Galactosemia. AT-007 has been studied in an animal
model of Galactosemia, which demonstrated that AT-007 reduces toxic
galactitol levels and prevents disease complications. AT-007
was also studied in an adult biomarker-based clinical study and
demonstrated significant reduction in plasma galactitol levels vs.
placebo; the long-term extension in adults remains ongoing. The
company received Orphan Designation for AT-007 for Galactosemia
in May 2019 and Pediatric Rare Disease Voucher (PRV)
designation in 2020.
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 Galactosemia, a rare pediatric
metabolic disease. The Company initiated a pivotal Phase 1/2
clinical trial in June 2019, read out positive top-line
biomarker data in adult Galactosemia patients in January
2020 and announced full data from the trial in April
2020. A pediatric Galactosemia study commenced in June 2020.
The Company is also developing AT-001, a novel potent ARI that is
being developed for the treatment of Diabetic Cardiomyopathy, or
DbCM, a fatal fibrosis of the heart. The Company initiated a Phase
3 registrational study in DbCM in September 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, as well as novel dual PI3k inhibitors in
preclinical development for orphan oncology indications.
Investors:Maeve
Conneighton(212) 600-1902
orappliedtherapeutics@argotpartners.com
Media:Gleb
Sagitovmedia@appliedtherapeutics.com
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