Acer Therapeutics Announces Resubmission of New Drug Application for ACER-001 for Treatment of UCDs
July 18 2022 - 8:30AM
Acer Therapeutics Inc. (Nasdaq: ACER), a pharmaceutical company
focused on the acquisition, development and commercialization of
therapies for serious rare and life-threatening diseases with
significant unmet medical needs, today announced the resubmission
of Acer’s New Drug Application (NDA) to the U.S. Food and Drug
Administration (FDA) for ACER-001 (sodium phenylbutyrate) for oral
suspension for the treatment of patients with urea cycle disorders
(UCDs). Acer believes the resubmission addresses in full the items
raised by the FDA in the Complete Response Letter (CRL).
In June 2022, as previously announced, the FDA issued Acer a CRL
stating that satisfactory inspection of its third-party contract
packaging manufacturer is required before the ACER-001 NDA may be
approved. Acer notified the FDA in the resubmission that the
third-party contract packaging manufacturer is ready for
inspection. FDA did not cite any other approvability issues in the
CRL pertaining to the NDA, nor request any additional clinical or
pharmacokinetic studies be conducted prior to FDA action.
Additional existing nonclinical information as requested by the FDA
in the CRL but identified as “not an approvability issue”, as well
as labeling and other routine updates to the original NDA, were
provided in the resubmission of the NDA.
“Our team did an outstanding job resubmitting the NDA one month
after we received the CRL,” said Chris Schelling, Founder &
Chief Executive Officer of Acer. “Our third-party contract
manufacturing partner has been incredibly responsive and has
confirmed that it is ready for inspection. Our manufacturing
partner is regarded as a global leader in clinical supply chain and
commercial packaging services with more than 70 years of
experience. Along with the other requested updates we provided to
the FDA in our resubmitted NDA, assuming it is accepted for review,
we now look forward to assisting the FDA in the completion of its
review of our NDA as soon as possible.”
Acer expects to be notified by the Agency of its decision to
accept or reject the resubmission for review within 14 calendar
days of receipt of the NDA resubmission.1 If the resubmission is
deemed complete by the FDA, a resubmission classification (Class 1
or 2) will be assigned and a new Prescription Drug User Fee Act
(PDUFA) target action date will be established of either two or six
months from the resubmission date depending on the classification
and an inspection of the facility will be requested. However, there
can be no assurance the resubmission will be accepted for review,
classified for action, or the target action deadline will be
established or met.
About ACER-001ACER-001 (sodium phenylbutyrate)
is being developed for the treatment of various inborn errors of
metabolism, including UCDs and Maple Syrup Urine Disease (MSUD).
ACER-001 is a nitrogen-binding agent in development for use as
adjunctive therapy in the chronic management of patients with UCDs
involving deficiencies of carbamylphosphate synthetase (CPS),
ornithine transcarbamylase (OTC), or argininosuccinic acid
synthetase (AS). ACER-001 is a polymer coated formulation that,
when taken within 5 minutes, helps prevent the coating from
dissolving. ACER-001 has been granted orphan drug designation by
the FDA for MSUD. ACER-001 is an investigational product candidate
which has not been approved by FDA, the European Medicines Agency
(EMA), or any other regulatory authority. There can be no assurance
that the FDA inspection of the third-party contract packaging
manufacturer facility will be satisfactory, that such inspection is
the only impediment to FDA approval of a resubmitted NDA, that a
resubmitted NDA will otherwise be approved by the FDA, or that
ACER-001 will be approved for any indication.
About Acer Therapeutics Inc.Acer is a
pharmaceutical company focused on the acquisition, development and
commercialization of therapies for serious rare and
life-threatening diseases with significant unmet medical needs.
Acer’s pipeline includes four investigational programs: ACER-001
(sodium phenylbutyrate) for treatment of various inborn errors of
metabolism, including urea cycle disorders (UCDs) and Maple Syrup
Urine Disease (MSUD); ACER-801 (osanetant) for treatment of induced
Vasomotor Symptoms (iVMS); EDSIVO™ (celiprolol) for treatment of
vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed
type III collagen (COL3A1) mutation; and ACER-2820 (emetine), a
host-directed therapy against a variety of viruses, including
cytomegalovirus, Zika, dengue, Ebola and COVID-19. For more
information, visit www.acertx.com.
References
- FDA SOPP 8405.1: Procedures for
Resubmissions to an Application or Supplement Version: 7 Effective
Date: November 1, 2021
https://www.fda.gov/media/84417/download
Acer 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.
All statements, other than statements of historical facts, included
in this press release are forward-looking statements. Examples of
such statements include, but are not limited to, statements about
the Company’s belief that the resubmission addresses in full the
items raised by the FDA in the CRL, the Company’s timing
expectations with respect to notification by the Agency of its
decision to accept or reject the resubmission for review, the
assignment of a resubmission classification and the establishment
of a new PDUFA target action date, and the timing thereof, and a
potential request from the FDA for an inspection of the facility of
our third-party contract manufacturing partner. Our pipeline
products are under investigation and their safety and efficacy have
not been established and there is no guarantee that any of our
investigational products in development will receive health
authority approval or become commercially available for the uses
being investigated. We may not actually achieve the plans, carry
out the intentions or meet the expectations or projections
disclosed in the forward-looking statements and you should not
place undue reliance on these forward-looking statements. Such
statements are based on management’s current expectations and
involve risks and uncertainties. Actual results and performance
could differ materially from those projected in the forward-looking
statements as a result of many factors, including, without
limitation, risks related to the drug development and the
regulatory approval process, including the timing and requirements
of regulatory actions. We disclaim any intent or obligation to
update these forward-looking statements to reflect events or
circumstances that exist after the date on which they were made.
You should review additional disclosures we make in our filings
with the Securities and Exchange Commission, including our Annual
Report on Form 10-K and Quarterly Reports on Form 10-Q. You may
access these documents for no charge
at http://www.sec.gov.
CORPORATE CONTACTSJim DeNikeAcer Therapeutics
Inc.jdenike@acertx.com+1-844-902-6100
INVESTOR RELATIONS CONTACTSNick
ColangeloGilmartin Groupnick@gilmartinIR.com+1-339-225-1047
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