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 provided a regulatory update on the
Galactosemia program, including updates on the regulatory paths for
govorestat (AT-007) in both the U.S. and Europe.
The United States Food
and Drug Administration (US FDA) has granted a Pre-New Drug
Application (Pre-NDA) meeting to be held this summer to discuss a
potential NDA submission for govorestat (AT-007) for the treatment
of Galactosemia. The Company believes that the clinical efficacy
demonstrated to date, combined with galactitol biomarker data and a
favorable safety profile, may support an NDA submission, and is
seeking feedback from the FDA and alignment on the details of the
submission. If the FDA is in agreement on the potential path
forward to approval, the Company will plan to submit an NDA in the
fall. The Company expects to provide a further update following the
meeting.
Regarding regulatory
submission plans in Europe, Applied Therapeutics and its European
commercial partner, Advanz Pharma, met with the European Medicines
Agency (EMA) rapporteurs earlier this summer, and plan to proceed
with an EMA Marketing Authorization Application (MAA) submission as
expeditiously as possible. The EMA submission is anticipated in the
fall in order to provide sufficient time for approval of the
Pediatric Investigational Plan (PIP), as well as incorporation of
rapporteur comments and suggestions from the meeting.
“We look forward to
advancing our regulatory submissions in both the U.S. and Europe,”
said Shoshana Shendelman, PhD, Founder and CEO of Applied
Therapeutics. “We believe that govorestat offers compelling
efficacy alongside a favorable safety profile and represents a
transformative treatment option for patients with Galactosemia.
Galactosemia is a devastating disease that progressively worsens
with age, despite adherence to a galactose-restricted diet, due to
endogenous production of galactose by the body and subsequent
conversion to the toxic metabolite, galactitol. There are currently
no approved treatments for Galactosemia, and we hope to bring
govorestat to patients as the first treatment for Galactosemia as
soon as possible.”
About
GalactosemiaGalactosemia 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, and approximately 4,000 patients with Galactosemia
in the EU and 120 new births per year.
About
Govorestat (AT-007)Govorestat 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 a study in children
with Galactosemia aged 2-17, treatment with AT-007 demonstrated
clinical benefit on activities of daily living, behavioral
symptoms, cognition, fine motor skills and tremor. Govorestat also
significantly reduced plasma galactitol levels in both adults and
children with Galactosemia. Galactitol is a toxic metabolite
responsible for tissue damage and long-term complications in
Galactosemia.
Govorestat is also
being studied in the ongoing Phase 3 INSPIRE trial, which is
evaluating the effect of AT-007 vs. placebo in patients with SORD
Deficiency on sorbitol reduction as well as clinical outcomes in
approximately 50 patients aged 16-55 in the U.S. and Europe. In an
interim analysis, AT-007 reduced sorbitol by a mean of 52%, or
approximately 16,000 ng/ml, over a 90-day period, which was highly
statistically significant vs. placebo (p<0.001).
Govorestat has
received Orphan Medicinal Product Designation from the European
Medicines Agency (EMA)EMA for both Galactosemia and SORD
Deficiency. Govorestat has also received Orphan Drug Designation
from the U.S. Food and Drug Administration (FDA) for the treatment
of Galactosemia, PMM2-CDG, and SORD Deficiency; Pediatric Rare
Disease designation for 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, govorestat, 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.
Forward-Looking
Statements
This press release contains
“forward-looking statements” that involve substantial risks and
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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, including those about
our upcoming pre-NDA meeting, the potential NDA submission,
including the timing thereof and the timing for our MAA submission,
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
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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:Maeve
Conneighton (212) 600-1902
orappliedtherapeutics@argotpartners.com
Media:media@appliedtherapeutics.com
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