Acer Therapeutics Announces Presentation of HCP Urea Cycle Disorder Treatment Preference Data at the Society for Inherited Metabolic Disorders Annual Meeting and Provides OLPRUVA™ Program Update
March 15 2023 - 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 data will be
presented from a survey designed to quantify preferences of
healthcare providers for Urea Cycle Disorders (UCDs) at the
upcoming 44th Annual Meeting of the Society for Inherited Metabolic
Disorders (SIMD), March 18th-21st in Salt Lake City, Utah. Results
from the discrete choice experiment will be available on Acer’s
website following the poster presentation at SIMD on March 19 by
Dr. Robert Steiner (University of Wisconsin School of Medicine and
Public Health) at
https://www.acertx.com/publications-and-presentations/.
Poster #97: Quantifying Preferences for Urea Cycle Disorder
Treatments Using a Discrete-Choice ExperimentThis poster summarizes
results from a web-based, quantitative survey study using a
discrete choice experiment (DCE) methodology. This study sought to
capture the perspectives of healthcare providers (HCPs) with
experience treating UCDs to quantify the attributes of
nitrogen-binding medications (such as sodium phenylbutyrate and
glycerol phenylbutyrate) for UCDs that may influence overall
prescription and patient adherence.
“OLPRUVA™1 leverages the well-established efficacy of sodium
phenylbutyrate in an innovative dual-coating formulation designed
for palatability,” said Adrian Quartel, MD, FFPM, CMO of Acer. “We
look forward to the presentation of the survey results at SIMD as
we continue to prepare for the launch of OLPRUVA™, and to the
delivery of an important alternative treatment option to those
facing the unmet needs of existing therapies.”
ACER-001 (sodium phenylbutyrate) Palatability Data
Presented at SIMD and GMDI 2022Taste-Masked Coating of
Sodium Phenylbutyrate (ACER-001) Improves the Palatability of
Sodium Phenylbutyrate for Treatment of Urea Cycle
Disorders2,3Results from two Phase 1, open-label, repeated
measures, taste assessment studies of ACER-001 (sodium
phenylbutyrate) suspension and sodium phenylbutyrate (BUPHENYL®)
powder were presented at the SIMD Annual Meeting and Genetic
Metabolic Dieticians International (GMDI) Conference in April 2022
and May 2022, respectively. ACER-001 for the treatment of UCDs is
now marketed in the U.S. as OLPRUVA™ (sodium phenylbutyrate)
following FDA approval in December 2022. Results from both studies
concluded that ACER-001 (sodium phenylbutyrate) suspension was
shown to have overall lower flavor intensity scores than sodium
phenylbutyrate (BUPHENYL®) powder when administered within five
minutes of preparation.
OLPRUVA™ Program UpdateAcer also provided an
update on its commercial launch activities for OLPRUVA™ in advance
of the SIMD Annual Meeting, where it will be exhibiting in booths
312 and 213. To support the launch of OLPRUVA™ in Q2 2023, the
company is actively adding resources to establish its commercial
and medical affairs presence in the U.S. As a part of its OLPRUVA™
commercialization strategy, Acer has recently introduced its
patient support service, OLPRUVA™ Navigator by Acer Therapeutics,
that is designed to assist UCD patients with support, access,
education, and patient adherence to treatment. Representatives will
begin accepting prescriptions in late Q2 2023. Acer is also
actively engaged in negotiations regarding access for OLPRUVA™ with
the major commercial payers and state Medicaid organizations.
In connection with the company’s ongoing support for the rare
disease patient community, Acer has established a pricing strategy
that reflects its commitment to deliver innovative treatments that
are responsibly priced and accessible to those in need. As a
result, Acer intends to price OLPRUVA™ competitively, at a
significant discount to the currently available commercial product
RAVICTI®, while implementing predictable pricing that will not
increase beyond the rate of inflation. The company also plans to
invest a portion of OLPRUVA™ revenue back into additional solutions
aimed at improving outcomes for UCD patients. More information will
be available at OLPRUVA.com.
“We are proud that we can provide OLPRUVA™ — an innovative,
responsibly priced treatment option — to patients with UCDs.
And we are committed to working closely with the UCD community to
ensure that those affected by this terrible disease live their
lives with as few challenges and interruptions as possible,”
commented Chris Schelling, CEO and Founder of Acer. “While our
future plans are subject to the availability of working capital
moving forward, we feel strongly that Acer is well positioned to
access additional sources of capital to fund day-to-day operations
and OLPRUVA’s™ launch requirements.”
New FDA-Approved UCD Treatment Option:
OLPRUVA™ACER-001 (sodium phenylbutyrate) was
approved for the treatment of certain UCDs in December 2022 and is
marketed under the brand name, OLPRUVA™. OLPRUVA™ (sodium
phenylbutyrate) for oral suspension is a prescription medicine used
along with certain therapy, including changes in diet, for the
long-term management of adults and children weighing 44 pounds (20
kg) or greater and with a body surface area (BSA) of 1.2 m2 or
greater, with urea cycle disorders (UCDs), involving deficiencies
of carbamylphosphate synthetase (CPS), ornithine transcarbamylase
(OTC), or argininosuccinic acid synthetase (AS).1 Please see
Important Safety Information and full Prescribing Information,
including Patient Information.
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. In the U.S., OLPRUVA™ (sodium
phenylbutyrate) is approved for the treatment of urea cycle
disorders (UCDs) involving deficiencies of carbamylphosphate
synthetase (CPS), ornithine transcarbamylase (OTC), or
argininosuccinic acid synthetase (AS). Acer is also advancing a
pipeline of investigational product candidates for rare and
life-threatening diseases, including: OLPRUVA™ (sodium
phenylbutyrate) for treatment of various disorders, including Maple
Syrup Urine Disease (MSUD); ACER-801 (osanetant) for treatment of
Vasomotor Symptoms (VMS), Post-traumatic Stress Disorder (PTSD) and
prostate cancer; 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. In March 2021, Acer entered into
a Collaboration and License Agreement with RELIEF THERAPEUTICS
Holding SA for development and commercialization of OLPRUVA™
(ACER-001) in which Acer retains development and commercialization
rights in the U.S., Canada, Brazil, Turkey, and Japan. For more
information, visit www.acertx.com.
References
- OLPRUVA™ (sodium phenylbutyrate) for oral suspension.
Prescribing information. Newton, MA: Acer Therapeutics Inc.
- Steiner R, et al. The Pharmacokinetics of Taste-Masked Sodium
Phenylbutyrate (ACER-001) for the Treatment of Urea Cycle Disorders
Under Fasting and Fed Conditions in Healthy Volunteers. SIMD April
2022
- Cederbaum S, et al. Taste-Masked Coating of Sodium
Phenylbutyrate (ACER-001) Improves the Palatability of Sodium
Phenylbutyrate for Treatment of Urea Cycle Disorders. GMDI May
2022
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
survey data to be presented, including the timing, detail and
content thereof, and OLPRUVA™ launch activities, including the
addition of resources, the launch of patient support services,
discussions with payers and organizations, and the Company’s
pricing strategy and related plans and expectations. 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, the availability of financing to fund our pipeline
product development programs and general corporate operations as
well as risks related to 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 and IR ContactsJim DeNikeAcer
Therapeutics Inc.jdenike@acertx.com+1-844-902-6100
Nick ColangeloGilmartin
Groupnick@gilmartinIR.com+1-332-895-3226
Acer Therapeutics (NASDAQ:ACER)
Historical Stock Chart
From Jun 2024 to Jul 2024
Acer Therapeutics (NASDAQ:ACER)
Historical Stock Chart
From Jul 2023 to Jul 2024