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 successful completion of a
recent pre-New Drug Application (“NDA”) meeting with the Food and
Drug Administration (FDA) regarding its govorestat (AT-007)
Galactosemia program. Based on discussions with the FDA, the
Company believes they are aligned with the FDA and plans to submit
an NDA for govorestat (AT-007) for the treatment of Galactosemia in
the fourth quarter of this year.
“In the pre-NDA
meeting, the FDA expressed their support for a potential NDA based
on the govorestat data generated to date in Galactosemia, and
provided constructive recommendations for successful NDA acceptance
and review,” said Shoshana Shendelman, PhD, Founder and CEO of
Applied Therapeutics. “If approved, govorestat will fill an
important unmet need for the Galactosemia community. We will work
to prepare our regulatory package as expeditiously as possible and
expect to submit the NDA to the FDA in the fourth quarter of this
year.”
“We thank the FDA for
their productive feedback and for their partnership in this
process,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of
Applied Therapeutics. “We believe that the clinical efficacy
demonstrated to date, combined with galactitol biomarker data and a
favorable safety profile, provides us with a solid filing package
to the FDA. We are excited to advance towards a regulatory
submission for the first potential treatment for Galactosemia.”
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
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 the strategy, future operations, prospects, plans
and objectives of management, including words such as “may,”
“will,” “expect,” “anticipate,” “plan,” “intend,” “predicts” 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 the (i) timing for the Company’s submission of
an NDA for govorestat (AT-007) for the treatment of Galactosemia
and (ii) strength of the submission package and likelihood that the
NDA submission will be accepted. 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, (i) our plans to develop, market and
commercialize our product candidates, (ii) the initiation, timing,
progress and results of our current and future preclinical studies
and clinical trials and our research and development programs,
(iii) our ability to take advantage of expedited regulatory
pathways for any of our product candidates, (iv) our estimates
regarding expenses, future revenue, capital requirements and needs
for additional financing, (v) our ability to successfully acquire
or license additional product candidates on reasonable terms and
advance product candidates into, and successfully complete,
clinical studies, (vi) our ability to maintain and establish
collaborations or obtain additional funding, (vii) our ability to
obtain and timing of regulatory approval of our current and future
product candidates, (viii) the anticipated indications for our
product candidates, if approved, (ix) our expectations regarding
the potential market size and the rate and degree of market
acceptance of such product candidates, (x) our ability to fund our
working capital requirements and expectations regarding the
sufficiency of our capital resources, (xi) the implementation of
our business model and strategic plans for our business and product
candidates, (xii) our intellectual property position and the
duration of our patent rights, (xiii) developments or disputes
concerning our intellectual property or other proprietary rights,
(xiv) our expectations regarding government and third-party payor
coverage and reimbursement, (xv) our ability to compete in the
markets we serve, (xvi) the impact of government laws and
regulations and liabilities thereunder, (xvii) developments
relating to our competitors and our industry, (xvii) our ability to
achieve the anticipated benefits from the agreements entered into
in connection with our partnership with Advanz Pharma and (xiv)
other factors that may impact our financial results. In light of
the significant uncertainties in these forward-looking statements,
you should not rely upon forward-looking statements as predictions
of future events. Although we believe that we have a reasonable
basis for each forward-looking statement contained in this press
release, we cannot guarantee that the future results, levels of
activity, performance or events and circumstances reflected in the
forward-looking statements will be achieved or occur at all.
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:Maeve
Conneighton (212) 600-1902
orappliedtherapeutics@argotpartners.com
Media:media@appliedtherapeutics.com
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