Applied Therapeutics Announces FDA Orphan Drug Designation for AT-007 in Galactosemia
28 Maggio 2019 - 1:30PM
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 U.S. Food and
Drug Administration (FDA) has granted Orphan Drug Designation to
AT-007, a central nervous system (CNS) penetrant Aldose Reductase
inhibitor (ARI) in the late stages of preclinical development for
the treatment of Galactosemia.
“We are pleased to obtain Orphan Drug
Designation for AT-007 in Galactosemia, a devastating rare
metabolic disease for which there are no approved treatments,” said
Shoshana Shendelman, PhD, Founder, Chief Executive Officer and
Chair of the Board of Applied Therapeutics. “Galactosemia patients
are in desperate need of treatment options, and we are committed to
advancing AT-007 into clinical development as quickly as possible
due to the urgent unmet medical need. We look forward to
taking advantage of the opportunities that Orphan Designation
provides in order to bring this potential new treatment option to
patients and families as rapidly as possible.” Applied
Therapeutics plans to initiate a Phase 1/2 biomarker based clinical
study with AT-007 in Galactosemia patients later this year.
The FDA Office of Orphan Products Development
grants Orphan Drug Designation to drugs and biologics that are
defined as those intended for the safe and effective treatment,
diagnosis or prevention of rare diseases/disorders that affect
fewer than 200,000 people in the United States. The designation
allows Applied Therapeutics to qualify for a number of incentives,
including: seven years of market exclusivity upon regulatory
approval, if received; exemption from FDA application fees for
Galactosemia; and tax credits for qualified clinical trials.
About GalactosemiaGalactosemia
is a devastating rare pediatric metabolic disease that affects how
the body processes a simple sugar called galactose, and for which
there is no known cure or approved treatment available. Galactose
is found in foods, but the human body also naturally produces
galactose on its own, so dietary restriction can’t prevent
complications of disease. It is estimated that the U.S.
Galactosemia population is approximately 2,800 patients, based on
newborn screening data identifying 2,500 infants through 2014, and
the estimated birth rate of 80 patients per year. High levels of
galactose circulating in the blood and tissues of Galactosemia
patients enables Aldose Reductase to convert galactose to a toxic
metabolite, called galactitol, which causes long-term complications
ranging from CNS dysfunction to cataracts.
About AT-007AT-007 is a central
nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in
late-stage preclinical 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. Applied Therapeutics is planning a
biomarker-based development program in patients with Galactosemia,
based on the recently released draft industry guidance on drug
development for low prevalence, slowly progressing rare metabolic
diseases. The company intends to advance AT-007 into a
Phase 1/2 clinical trial in 2019.
About Applied
TherapeuticsApplied 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-001,
is a novel Aldose Reductase inhibitor (ARI) that is being developed
for the treatment of Diabetic Cardiomyopathy (DbCM), a fatal
fibrosis of the heart. The company plans to initiate a Phase 2/3
pivotal study in DbCM in 2019. Applied Therapeutics is also
developing AT-007, a central nervous system (CNS) penetrant ARI,
for the treatment of Galactosemia, a rare pediatric metabolic
disease, which is expected to advance into a Phase 1 clinical trial
in 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, expected to advance into
a Phase 1 study in 2020.
Forward-looking StatementsThis
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. These include, without limitation,
statements regarding our ability to rapidly advance the clinical
development of our product candidates, including AT-007, and obtain
approval of such product candidates, the likelihood data will
support future development and the expected timing of initiation of
our clinical trials. 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, the uncertainties inherent in the initiation, execution
and completion of clinical trials, in the timing of availability of
trial data, in the results of the clinical trials, in the actions
of regulatory agencies, in the commercialization and acceptance of
new therapies. Other 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 section
titled “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.
ContactsInvestors:Maeve Conneighton(212)
600-1902 orappliedtherapeutics@argotpartners.com
Media:media@appliedtherapeutics.com
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