Ad hoc announcement pursuant to Art. 53 LR
Acer Therapeutics and Relief Therapeutics Announce FDA Acceptance for Filing of New Drug
Application for ACER-001 to Treat Urea Cycle Disorders
FDA sets PDUFA target action date of June 5, 2022
NEWTON, MA and GENEVA, SWITZERLAND October 6, 2021 Acer Therapeutics Inc. (Nasdaq: ACER)
(Acer) and its collaboration partner, RELIEF THERAPEUTICS Holding SA (SIX: RLF, OTCQB: RLFTF)
(Relief), today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing
the New Drug Application (NDA) for ACER-001 (sodium phenylbutyrate) for the treatment of patients
with Urea Cycle Disorders (UCDs). The FDA has assigned a Prescription Drug User Fee Act (PDUFA)
target action date of June 5, 2022.
Acers 505(b)(2) NDA is supported by results from two previously announced bioequivalence (BE) trials
in which ACER-001 showed similar relative bioavailability for both phenylbutyrate (PBA) and
phenylacetate (PAA), the active metabolite of sodium phenylbutyrate, compared to the reference
listed drug, BUPHENYL® (sodium phenylbutyrate).
"With FDA commencing a substantive review of our NDA, ACER-001 is one step closer to potentially
providing an alternative treatment option for patients with UCDs," said Chris Schelling, Chief Executive
Officer and Founder of Acer. "We look forward to working with the FDA during their review of our
application. In addition, we continue to collaborate with our partners to ensure we are well positioned
to support a successful commercial launch of ACER-001, subject to FDA approval.
Jack Weinstein, Chief Financial Officer and Treasurer of Relief, added, Our collaboration with Acer is
thriving and we are pleased with the progress they have made in advancing ACER-001. In parallel with
Acers activities, we continue to execute on our global commercial strategy for ACER-001 which
includes our intended submission of a Marketing Authorization Application (MAA) for the treatment of
patients with UCDs in Europe in Q2/Q3 2022.
Parties interested in the ACER-001 program for UCDs may sign up for updates at:
www.acertx.com/rare-disease-research/...cycle-disorders-ucds/
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 is no guarantee that this product
candidate will receive regulatory authority approval in any territory or become commercially available
for the indications under investigation.
Ad hoc announcement pursuant to Art. 53 LR
About UCDs
UCDs are a group of disorders caused by genetic mutations that result in a deficiency in one of the six
enzymes that catalyze the urea cycle, which can lead to an excess accumulation of ammonia in the
bloodstream, a condition known as hyperammonemia. Acute hyperammonemia can cause lethargy,
somnolence, coma, and multi-organ failure, while chronic hyperammonemia can lead to headaches,
confusion, lethargy, failure to thrive, behavioral changes, and learning and cognitive deficits. Common
symptoms of both acute and chronic hyperammonemia also include seizures and psychiatric
symptoms.1,2 The current treatment of patients with UCDs consists of dietary management to limit
ammonia production in conjunction with medications that provide alternative pathways for the
removal of ammonia from the bloodstream. Some patients may also require individual branched-chain
amino acid supplementation.
Current medical treatments for patients with UCDs include nitrogen scavengers, RAVICTI® and
BUPHENYL®, in which the active pharmaceutical ingredients are glycerol phenylbutyrate and sodium
phenylbutyrate, respectively. According to a 2016 study by Shchelochkov et al., published in Molecular
Genetics and Metabolism Reports, while nitrogen scavenging medications have been shown to be
effective in helping to manage ammonia levels in some patients with UCDs, non-compliance with
treatment is common. Reasons referenced for non-compliance associated with some available
medications include unpleasant taste, frequency with which medication must be taken, required
number of pills, and the high cost of the medication.3
About ACER-001
ACER-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). The formulation is a multi-particulate dosage formulation for oral
administration consisting of a core center, a layer of active drug, and a taste-masked coating designed
to avoid the bitter taste of sodium phenylbutyrate in the mouth while quickly dissolving in the low pH
of the stomach. Acer is also being developed for MSUD and has been granted orphan drug designation
by the FDA for this indication. ACER-001 is an investigational product candidate which has not been
approved by FDA, the European Medicines Agency (EMA), or any other regulatory authority.
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. Acers
pipeline includes four 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 infectious
diseases, including COVID-19. Each of Acers product candidates is believed to present a comparatively
de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data,
mechanistic differentiation and/or accelerated paths for development through specific programs and
procedures established by the FDA. In March 2021, Acer entered into a Collaboration and License
Ad hoc announcement pursuant to Art. 53 LR
Agreement with Relief for development and commercialization of ACER-001. For more information,
visit www.acertx.com.
About RELIEF THERAPEUTICS Holding SA
Relief focuses primarily on clinical-stage programs based on molecules with a history of clinical testing
and use in human patients or a strong scientific rationale. Reliefs lead drug candidate RLF-100
(aviptadil), a synthetic form of Vasoactive Intestinal Peptide (VIP), is in late-stage clinical testing in the
U.S. for the treatment of respiratory deficiency due to COVID-19. As part of its pipeline diversification
strategy, in March 2021, Relief entered into a Collaboration and License Agreement with Acer
Therapeutics for development and commercialization of ACER-001. ACER-001 is a taste-masked and
immediate release proprietary powder formulation of sodium phenylbutyrate (NaPB) for the treatment
of Urea Cycle Disorders and Maple Syrup Urine Disease. In addition, Reliefs recently completed
acquisitions of APR Applied Pharma Research SA and AdVita Lifescience GmbH, bring to Relief a diverse
pipeline of marketed and development-stage programs, a commercial infrastructure in selected
European countries and an internal, R&D capability, which Relief hopes to leverage for both internal
pipeline products as well as for third party product development on a fee for service basis.
RELIEF THERAPEUTICS Holding SA is listed on the SIX Swiss Exchange under the symbol RLF and quoted
in the U.S. on OTCQB under the symbol RLFTF. For more information, visit
www.relieftherapeutics.com. Follow Relief on LinkedIn.
References
1. Ah Mew N, et al. Urea cycle disorders overview. Gene Reviews. Seattle, Washington: University
of Washington, Seattle; 1993.
2. Häberle J, et al. Suggested guidelines for the diagnosis and management of urea cycle
disorders. Orphanet Journal of Rare Diseases. 2012;7(32).
3. Shchelochkov OA, et al. Barriers to drug adherence in the treatment of urea cycle disorders:
Assessment of patient, caregiver and provider perspectives. Mol Genet Metab. 2016;8:43-47.
Acer 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. All statements, other than statements of historical facts, included in this press release regarding
strategy, future operations, timelines, future financial position, future revenues, projected expenses,
regulatory submissions, actions or approvals, cash position, liquidity, prospects, plans and objectives of
management are forward-looking statements. Examples of such statements include, but are not
limited to, statements relating to the potential for our product candidates to safely and effectively
treat diseases and to be approved for marketing; the commercial or market opportunity of any of our
product candidates in any target indication and any territory; our ability to secure the additional capital
necessary to fund our various product candidate development programs; the adequacy of our capital
to support our future operations and our ability to successfully fund, initiate and complete clinical trials
and regulatory submissions; the ability to protect our intellectual property rights; our strategy and
business focus; and the development, expected timeline and commercial potential of any of our
product candidates. 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 managements
Ad hoc announcement pursuant to Art. 53 LR
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 and uncertainties associated with the ability to project future cash
utilization and reserves needed for contingent future liabilities and business operations, the availability
of sufficient resources to fund our various product candidate development programs and to meet our
business objectives and operational requirements, the fact that the results of earlier studies and trials
may not be predictive of future clinical trial results, the protection and market exclusivity provided by
our intellectual property, risks related to the drug development and the regulatory approval process,
including the timing and requirements of regulatory actions, and the impact of competitive products
and technological changes. 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 Quarterly Reports on Form 10-Q and our Annual Report on Form 10-K. You
may access these documents for no charge at www.sec.gov.
Relief Forward-Looking Statements
This communication expressly or implicitly contains certain forward-looking statements concerning
RELIEF THERAPEUTICS Holding SA and its businesses. Such statements involve certain known and
unknown risks, uncertainties and other factors, including (i) whether the FDA will approve Acers NDA
for ACER-001, (ii) whether Relief will be able to submit an application for approval of ACER-001 in
Europe in Q2/Q3 2022 (or at all), (iii) whether any such application submitted to European authorities
seeking marketing authorization for ACER-001 for the treatment of patient in Europe with UCDs will be
approved, (iv) whether RLF-100 (aviptadil) will receive emergency use authorization in the United
States, (v) whether RLF-100 (aviptadil) will ever be submitted for authorization in Europe, (vi) whether
Reliefs ongoing disputes with its U.S. collaboration partner for RLF-100 (aviptadil) can be resolved
amicably, and (vii) those other risks, uncertainties and factors described in Reliefs annual and periodic
filings with the SIX Stock Exchange, all of which could cause the actual results, financial condition,
performance or achievements of RELIEF THERAPEUTICS Holding SA to be materially different from any
future results, performance or achievements expressed or implied by such forward-looking
statements. RELIEF THERAPEUTICS Holding SA is providing this communication as of this date and does
not undertake to update any forward-looking statements contained herein as a result of new
information, future events or otherwise.
CORPORATE CONTACTS
Acer Therapeutics:
Jim DeNike
Acer Therapeutics Inc.
jdenike@acertx.com
+1 844-902-6100
RELIEF THERAPEUTICS Holding SA:
Jack Weinstein
Chief Financial Officer and Treasurer
contact@relieftherapeutics.com
INVESTOR RELATIONS CONTACTS
Acer Therapeutics:
Hans Vitzthum
RELIEF THERAPEUTICS Holding SA:
Michael Miller
Ad hoc announcement pursuant to Art. 53 LR
LifeSci Advisors
hans@lifesciadvisors.com
+1 617-430-7578
Rx Communications Group
mmiller@rxir.com
+1-917-633-6086
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Acer Therapeutics zieht an nur Relief nicht :-(