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 accepted the filing of the New Drug
Application (NDA) for govorestat (AT-007) for the treatment of
Classic Galactosemia. The NDA was granted Priority Review status,
and the FDA assigned a Prescription Drug User Free Act (PDUFA)
target action date of August 28, 2024. The FDA also noted that it
is planning to hold an advisory committee meeting to discuss the
application. Govorestat was previously granted Pediatric Rare
Disease designation, and will qualify for a Priority Review Voucher
(PRV) upon approval.
“The FDA’s acceptance of the NDA for govorestat
for the treatment of Galactosemia represents a critical milestone
for Applied Therapeutics and more importantly, for patients with
Galactosemia and their families. The Agency’s decision to grant
Priority Review for this NDA underscores the urgent unmet medical
need as there are currently no treatment options for this
devastating disease,” said Shoshana Shendelman, PhD, Founder and
CEO of Applied Therapeutics. “We want to thank the patients,
families, collaborators and physicians involved in reaching this
achievement. We look forward to continuing to work with the FDA
throughout the review process, as we hope to bring govorestat to
patients as quickly as possible.”
“We are excited to see that the FDA has accepted
the govorestat NDA for priority review,” said Nicole Casale,
President of the Galactosemia Foundation. “The Galactosemia
community is in urgent need of a safe and effective treatment, and
the potential approval of govorestat provides hope for many
families. Acceptance of the govorestat NDA brings us one step
closer to availability of a first-ever treatment.”
Govorestat is an investigational, novel Aldose
Reductase Inhibitor (ARI) being developed for the treatment of
several rare diseases. It is a potent and selective compound, which
crosses the blood brain barrier into the Central Nervous System
(CNS). 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 FDA grants
Priority Review to applications for potential medicines that, if
approved, may offer significant improvements in the treatment,
prevention or diagnosis of a serious condition. Govorestat for the
treatment of Galactosemia has received Orphan Medicinal Product
Designation from the European Medicines Agency (EMA) and Orphan
Drug Designation, Pediatric Rare Disease Designation and Fast Track
Designation from the FDA. 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 NDA submission will be
approved. 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
(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
Applied Therapeutics (NASDAQ:APLT)
Historical Stock Chart
From Mar 2024 to Apr 2024
Applied Therapeutics (NASDAQ:APLT)
Historical Stock Chart
From Apr 2023 to Apr 2024