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 magnetic resonance
spectroscopy (MRS) data on reduction of galactitol levels in the
brain of Galactosemia patients treated with AT-007 in the
ACTION-Galactodsemia adult study. Overall, plasma reduction in
galactitol correlated with brain reduction in galactitol. There
were two exceptions, which may have resulted from incomplete peak
separation on the MRS scans. At the two doses which demonstrated
statistically significant reduction in plasma galactitol, 20 and
40mg/kg, 3 out of 4 patients displayed substantial galactitol
reduction ranging from 61.94% to 69.80% reduction from baseline.
“We are pleased to share our MRS data from
ACTION-Galactosemia and believe this represents an important
scientific advancement in the field of Galactosemia research,”
said Riccardo Perfetti, MD, PhD, Chief Medical Officer of
Applied Therapeutics. “We have demonstrated that galactitol can be
quantitated in the brain of Galactosemia patients on a restricted
diet, and that levels of galactitol in the brain can be reduced
through treatment with a AT-007, a CNS penetrant aldose reductase
inhibitor.”
Additional data on galactitol reduction in
ACTION-Galactosemia can be found below. Applied Therapeutics will
hold a conference call to discuss the data in more detail at 8:30
a.m. ET, and slides can be downloaded prior to the meeting at
https://ir.appliedtherapeutics.com/.
Graphic 1 accompanying this announcement is
available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/a3c76cbc-4979-4ad2-8937-24b1c5cf1706
Conference Call at 8:30 a.m.
Eastern
TimeApplied Therapeutics will
hold a conference call to discuss MRS data from the
ACTION-Galactosemia study today, December 9, 2020 at 8:30 a.m. ET.
The live event will be available on the investor page of the
Applied Therapeutics website at
https://ir.appliedtherapeutics.com/ or by calling (800)
369-8554 (toll-free domestic) or (409) 937-8917 (international)
five minutes prior to the start time and entering passcode 9978322.
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.
About GalactosemiaGalactosemia
is a rare, slowly progressing metabolic disease caused by a genetic
inability to break down the sugar galactose. Aldose Reductase (AR),
an enzyme known to play a role in many diseases including
Galactosemia, converts galactose into galactitol, a toxic
metabolite that builds up in tissues and organs and can cause
long-term disease complications. There are approximately 3,000
individuals with Galactosemia in the U.S. and about 3,500
individuals in the E.U. Most patients with Galactosemia are under
the age 40, as newborn screening was not widely adopted until the
1980s. Galactosemia is now included as part of routine newborn
screening in all 50 U.S. states, and in many countries in
Europe.
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 pivotal Phase 1/2
clinical trial in June 2019, read out positive top-line
biomarker data in adult Galactosemia patients in January
2020 and announced full data from the trial in April
2020. A pediatric Galactosemia study commenced in June 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, as well as novel dual PI3k inhibitors in
preclinical development for orphan oncology indications. To learn
more, please visit www.appliedtherapeutics.com and
follow the company on Twitter @Applied_Tx.
Investors:Maeve
Conneighton(212) 600-1902
orappliedtherapeutics@argotpartners.com
Media:Gleb
Sagitovmedia@appliedtherapeutics.com
Forward-Looking Statements
This press release may contain “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) the timing of
our NDA submission for potential approval of AT-007, which will
include data from the ACTION-Galactosemia Kids trial and the 90-day
safety data in adults with Galactosemia, (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.
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