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 new data and scientific
presentations from the pivotal Phase 2 ACTION-Galactosemia trial.
The full study data, originally planned to be presented at the
Society for Inherited Metabolic Disorders conference this week,
will be available on the company’s website.
- Slides from the presentation
“Development of AT-007 for the treatment of
Galactosemia”, presented by Riccardo Perfetti, MD, PhD,
Chief Medical Officer of Applied Therapeutics, will be made
available today, April 21, at 8:00 a.m. EDT on the Events page
under the Investor Relations section of the Applied Therapeutics
website at www.appliedtherapeutics.com. Key data from the
presentation are summarized below.
- A Galactosemia Educational
Symposium for healthcare providers hosted by Applied
Therapeutics will be webcast with a live Q&A moderated by Eva
Morava, MD, PhD, Professor of Clinical Genomics, Mayo Clinic, on
April 27 at 1:00 p.m. EDT. The live event will be available on the
Presentations & Publications section of the Applied
Therapeutics website at www.appliedtherapeutics.com. A replay of
the call will be available on the Applied Therapeutics website
approximately two hours after the completion of the call and will
be archived for 30 days.
The double-blind placebo-controlled
ACTION-Galactosemia trial evaluated safety and pharmacokinetics of
AT-007, a Central Nervous System (CNS) penetrant Aldose Reductase
Inhibitor (ARI) in healthy volunteers, as well as safety,
pharmacokinetics, and efficacy biomarkers in adult Galactosemia
patients. The key biomarker outcome of the study was reduction in
galactitol, an aberrant toxic metabolite of galactose, formed by
Aldose Reductase in Galactosemia patients. Accumulation of
galactitol causes long-term complications ranging from CNS
dysfunction to cataracts.
“We are pleased to share the full results of
ACTION-Galactosemia,” said Riccardo Perfetti, MD, PhD, Chief
Medical Officer of Applied Therapeutics. “Patients with this
devastating disease experience life-long accumulation of galactitol
in the CNS and in other tissues, and our data demonstrates rapid
and sustained reduction in this toxic metabolite.”
“We remain steadfast in our commitment to bring
this treatment to patients as quickly as possible, and understand
the urgency to potentially prevent worsening of disease in adults
and onset of disease-related complications in children,” said
Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics.
“We look forward to initiating a pediatric study in Q2 and remain
on track to submit an NDA later this year.”
Galactitol ReductionAs
previously announced, once-daily 20mg/kg AT-007 reduced galactitol
levels by approximately 50% in Galactosemia patients. Reduction in
galactitol levels was rapid (within 6 days of consecutive AT-007
treatment) and sustained throughout the treatment period of 27
days. Reduction in galactitol from baseline was statistically
significant at the 20mg/kg vs placebo (p<0.01). The lower dose
tested, 5mg/kg, demonstrated a similar trend in reducing galactitol
levels approximately 20% from baseline (p=NS from placebo).
Other Galactose
MetabolitesReduction in galactitol was not accompanied by
any increase in galactose. This data confirms that reduction of the
toxic metabolite galactitol through Aldose Reductase inhibition
does not result in derangement of other metabolites in the
galactose pathway as previously demonstrated in animal models of
Galactosemia.
MRI/MRS DataPatients treated
with AT-007 (once-daily over 27 days) demonstrated a positive trend
in MRI outcomes, specifically on measures of edema and overall
neuronal health. AT-007 treated patients demonstrated improvement
in ventricular volume, a measure of edema, which has been shown to
occur in the brain of Galactosemia patients due to osmotic
dysregulation caused by galactitol. Patients treated with AT-007
also demonstrated improvements in N-acetyl-aspartate, a marker of
neuronal health. Galactitol was visible on MRI in the brain of all
patients at baseline, and a positive trend toward decreased brain
galactitol levels in AT-007 treated patients was observed by
quantitative MR Spectroscopy (MRS).
40mg/kg DataAs no drug-related
adverse events were seen at the once-daily 20mg/kg dose, a
once-daily 40mg/kg dose was subsequently studied in healthy
volunteers and is ongoing in Galactosemia patients. The 40mg/kg
dose was safe and well tolerated. Evaluation of once-daily 40mg/kg
AT-007 in Galactosemia patients remains ongoing and this data will
be shared when available.
SafetyNo drug-related adverse
events were reported at any dose of AT-007 in ACTION-Galactosemia.
This robust safety data includes all 80 healthy volunteers and 8
adult Galactosemia patients who received active drug during the
core study.
Future DevelopmentA 90-day
safety extension study for ACTION-Galactosemia is ongoing. The
extension study is open to patients from the core study and to new
adult Galactosemia patients. A pediatric study with a safety and
biomarker design similar to ACTION-Galactosemia is planned to
commence in Q2 of this year. Both the extension study and the
pediatric study are designed to incorporate primarily home health
visits in order to limit travel and risk of exposure to COVID-19.
More information on the pediatric study will be shared in coming
weeks.
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-007,
is a novel central nervous system penetrant aldose reductase
inhibitor (ARI) for the treatment of Galactosemia, a rare pediatric
metabolic disease. The Company initiated a Phase 1/2 clinical trial
in June 2019 and read out positive top-line biomarker data in adult
Galactosemia patients in January of 2020. The Company is also
developing AT-001, a novel potent ARI that is being developed for
the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis
of the heart. The Company initiated a Phase 3 registrational study
in DbCM in September 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, as well as novel dual PI3k
inhibitors in preclinical development for orphan oncology
indications.
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 (i) our plan to move quickly towards
regulatory filing following our pivotal Phase 2 ACTION-Galactosemia
study, while preparing for Galactosemia commercial launch and
growing our organization, (ii) the design, scope and results of our
clinical trials, (iii) the timing of the initiation and completion
of our clinical trials, (iv) the likelihood that data from our
clinical trials will support future development of our product
candidates, and (v) the likelihood of obtaining regulatory approval
of our product candidates and qualifying for any special
designations, such as orphan drug designation. 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 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, (vi) our ability to maintain and establish
collaborations or obtain additional funding, (vii) our ability to
obtain regulatory approval of our current and future product
candidates, (viii) our expectations regarding the potential market
size and the rate and degree of market acceptance of such product
candidates, (ix) our ability to fund our working capital
requirements and expectations regarding the sufficiency of our
capital resources, (x) the implementation of our business model and
strategic plans for our business and product candidates, (xi) our
intellectual property position and the duration of our patent
rights, (xii) developments or disputes concerning our intellectual
property or other proprietary rights, (xiii) our expectations
regarding government and third-party payor coverage and
reimbursement, (xiv) our ability to compete in the markets we
serve, (xv) the impact of government laws and regulations and
liabilities thereunder, (xvi) developments relating to our
competitors and our industry, (xvii) the impact of the COVID-19
pandemic on the timing and progress of our ongoing clinical trials
and our business in general and (xviii) 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:Trammy
Lai917.297.5956media@appliedtherapeutics.com
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