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 extended the review period for the
New Drug Application (NDA) for govorestat (AT-007) for the
treatment of Classic Galactosemia by three months. The FDA has set
a new Prescription Drug User Fee Act (PDUFA) target action date of
November 28, 2024.
The FDA notified Applied Therapeutics that it
required additional time to review supplemental analyses of
previously submitted data that had been provided by Applied in
response to the FDA’s routine information requests and determined
that the additional information constitutes a Major Amendment to
the NDA. In February 2024, the Company announced that the FDA
accepted and granted Priority Review to the NDA. Govorestat was
previously granted Pediatric Rare Disease designation, and will
qualify for a Priority Review Voucher (PRV) upon approval.
“While the PDUFA action date extension
represents a delay, we remain confident in the potential for
govorestat approval for Galactosemia and we will continue to work
closely with the FDA throughout the review process,”
said Shoshana Shendelman, PhD, Founder and CEO of Applied
Therapeutics. “During this time, we are committed to maintaining
the expanded access program for govorestat to ensure patients with
Galactosemia have the opportunity to receive this important
treatment.”
Govorestat is an investigational, novel Aldose
Reductase Inhibitor (ARI) being developed for the treatment of
several rare diseases. The NDA filing of govorestat is supported by
rapid and sustained reduction in galactitol, which resulted in a
meaningful benefit on clinical outcomes across pediatric patients,
alongside a favorable safety profile. The submission package
included clinical outcomes data from the Phase 3 registrational
ACTION-Galactosemia Kids study in children aged 2-17 with
Galactosemia, the Phase 1/2 ACTION-Galactosemia study in adult
patients with Galactosemia, and preclinical data.
If approved, govorestat would be the first
medication indicated for the treatment of Galactosemia and would be
Applied Therapeutics’ first commercial product. The Company has
also submitted a Marketing Authorization Application (MAA) for
govorestat for the treatment of Classic Galactosemia to the EMA,
which was validated in December 2023 and is under review by the
EMA’s Committee for Medicinal Products for Human Use (CHMP). The
Company expects a decision by the EMA in the fourth quarter of
2024.
About Galactosemia
Galactosemia 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. Newborn screening for Galactosemia is
mandatory in the US and most EU countries, leading to rapid
identification of affected patients.
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 govorestat 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 12 months,
govorestat achieved statistical significance on the prespecified
primary endpoint of correlation of sorbitol with the clinical
outcome composite and demonstrated sustained, statistically
significant reduction in sorbitol level vs. placebo. Govorestat
also demonstrated highly statistically significant effects on the
CMT Health Index (CMT-HI) patient reported outcome measure, with
benefit of govorestat on categories of lower limb function,
mobility, fatigue, pain, sensory function, and upper limb
function.
Govorestat has received Orphan Medicinal Product
Designation from the European Medicines Agency (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 likelihood that the
Company’s ongoing NDA and MMA submissions will be approved and the
timing of any approval decision. 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, (xviii)
our ability to achieve the anticipated benefits from the agreements
entered into in connection with our partnership with Advanz Pharma
and (xix) 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@apliedtherapeutics.com
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