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 it has reached
agreement with the U.S. Food and Drug Administration (FDA) under a
Special Protocol Assessment (SPA) for its pivotal Phase 3 DiSCOVER
(
Decentral
ized
Study of
Celiprolol
on
vEDS-related
Event
Reduction) clinical trial
of EDSIVO™ (celiprolol) for the treatment of patients with
COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS). In April
2022, FDA granted celiprolol Breakthrough Therapy designation in
the U.S.
The SPA agreement is a process through which sponsors may seek
agreement with FDA on the design and size of certain clinical
trials, clinical studies, or animal studies. Agreement on Acer’s
Phase 3 trial design indicates concurrence by FDA with the adequacy
and acceptability of specific critical elements of the overall
protocol design that could support a future regulatory submission
and marketing application if the trial results meet the agreed-upon
criteria. For more information on Special Protocol Assessments,
please visit:
https://www.fda.gov/regulatory-information/search-fda-guidance-documents/special-protocol-assessment-guidance-industry.
“Receiving SPA agreement from FDA is a significant milestone in
our ongoing development of EDSIVO™ for patients with
COL3A1-positive vEDS, who urgently need an approved treatment for
this devastating disease,” said Adrian Quartel, MD, CMO of Acer.
“The SPA underscores our alignment with FDA on important
regulatory, clinical and scientific requirements for our planned
Phase 3 trial and reflects our ongoing commitment to the vEDS
patient community. We are planning for trial initiation by the end
of Q2 2022.”
About the EDSIVO™ DiSCOVER TrialThe DiSCOVER
trial is a prospective, Phase 3, randomized, double-blind,
placebo-controlled efficacy trial designed to evaluate EDSIVO™ in
patients with genetically confirmed COL3A1-positive vEDS using a
decentralized clinical trial design and independent adjudication
committee. The primary objective of the trial is to determine
whether EDSIVO™ reduces the occurrence of vEDS-related clinical
events requiring medical attention (fatal and non-fatal cardiac or
arterial events, uterine rupture, intestinal rupture, and/or
unexplained sudden death) relative to placebo as measured by time
to event. One interim analysis (based on number of primary events)
is planned at approximately 24 months after full enrollment. Acer
plans to enroll approximately 150 COL3A1-positive vEDS patients,
all in the U.S., randomized 2:1 to receive either EDSIVO™ or
placebo, respectively.
The duration of the DiSCOVER trial is currently estimated to be
approximately 3.5 years to completion (based on statistical power
calculations and number of primary events), once fully enrolled,
which will require additional capital beyond mid-2022. In April
2022, FDA granted celiprolol Breakthrough Therapy designation in
the U.S. for the treatment of patients with COL3A1-positive vEDS.
EDSIVO™ (celiprolol) is an investigational product candidate which
has not been approved by FDA. There is no guarantee that this
product candidate will receive regulatory authority approval or
become commercially available for any indications in the U.S.
More information on the planned EDSIVO™ pivotal clinical trial
can be found at www.discoverceliprolol.com.
About vEDS Ehlers-Danlos syndrome (EDS) is an
inherited disorder caused by mutations in the genes responsible for
the structure, production, or processing of collagen, an important
component of the connective tissues in the human body, or proteins
that interact with collagen. EDS is a spectrum disorder where
patients present with various forms, the most serious of which is
vascular Ehlers-Danlos syndrome (vEDS), also known as vEDS type IV,
which is generally caused by a mutation in the COL3A1 gene
resulting in reduced collagen levels. vEDS causes abnormal
fragility in blood vessels, which can give rise to aneurysms,
abnormal connections between blood vessels known as arteriovenous
fistulas, arterial dissections, and spontaneous vascular ruptures,
all of which can be potentially life-threatening. Gastrointestinal
and uterine fragility or rupture also commonly occur in vEDS
patients. Spontaneous arterial rupture has a peak incidence in the
third or fourth decade of life in vEDS patients but may occur
earlier and is the most common cause of sudden death in vEDS
patients. Arterial rupture or dissection events occur in about 25%
of patients before the age of 20 but increase to roughly 90% of
patients by the age of 40. The median survival age of vEDS patients
in the U.S. is 51 years, with arterial rupture being the most
common cause of sudden death.1
About EDSIVO™ (celiprolol)Acer is developing
EDSIVO™ (celiprolol), a new chemical entity (NCE), for the
treatment of COL3A1-positive vEDS patients. Celiprolol received FDA
Orphan Drug Designation for the treatment of vEDS in 2015. The
EDSIVO™ NDA was originally submitted based on data obtained from
the BBEST trial2 and accepted for filing in October 2018 with
priority review. Following FDA review, Acer received a Complete
Response Letter (CRL) in June 2019 stating that it will be
necessary to conduct an adequate and well-controlled trial to
determine whether EDSIVO™ reduces the risk of clinical events in
patients with vEDS. In April 2022, FDA granted celiprolol
Breakthrough Therapy designation in the U.S. for the treatment of
patients with COL3A1-positive vEDS. In May 2022, Acer reached
agreement with FDA under a SPA for its pivotal Phase 3 clinical
trial of EDSIVO™ (celiprolol) for the treatment of patients with
COL3A1-positive vEDS.
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 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. Each of Acer’s
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. For more information, visit
www.acertx.com.
References
- Pepin, et al. Survival is affected
by mutation type and molecular mechanism in vascular Ehlers–Danlos
syndrome (EDS type IV). Genet Med. 2014 Dec;16(12):881-8.
- Ong KT, et al. Effect of celiprolol
on prevention of cardiovascular events in vascular Ehlers-Danlos
syndrome: a prospective randomised, open, blinded-endpoints trial.
Lancet. 2010;376(9751):1476-1484
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 regarding strategy, future operations,
timelines for clinical study enrollment or regulatory actions, or
otherwise, 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 for ACER-001,
ACER-801, EDSIVO™ or our other product candidates; 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
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
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 Annual
Report on Form 10-K. You may access these documents for no charge
at http://www.sec.gov.
Corporate and IR Contact:Jim DeNikeAcer
Therapeutics Inc.Ph: 844-902-6100jdenike@acertx.com
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