- Company intends to file NDA for EDSIVO™ in
first half of 2018 -
Acer Therapeutics Inc., (Nasdaq:ACER), a pharmaceutical company
focused on the acquisition, development and commercialization of
therapies for serious rare and ultra-rare diseases with critical
unmet medical need, today announced positive results from the
pivotal clinical trial of EDSIVO™ (celiprolol) for the treatment of
vascular Ehlers-Danlos Syndrome (vEDS). Acer’s retrospective source
verified analysis of the trial data, including the primary and
secondary endpoints, confirmed the data from a previously published
randomized controlled clinical study of celiprolol(1). Acer will
use this pivotal clinical data to support a New Drug Application
(NDA) regulatory filing in the U.S. in the first half of 2018.
Ehlers-Danlos Syndrome (EDS) is a group of hereditary disorders
of connective tissue. vEDS is the most severe subtype where
patients suffer from life threatening arterial dissections and
ruptures, as well as intestinal and uterine ruptures. There
are currently no FDA approved therapies for vEDS(2).
“We have studied celiprolol for nearly two decades in vEDS
patients and this is the only drug to ever demonstrate a clinical
benefit in this difficult to treat patient population in a
randomized, controlled clinical study,” said Pierre Boutouyrie
M.D., Ph.D., co-director of the clinical pharmacology service at
the Georges-Pompidou European Hospital, Greater Paris University
Hospitals (AP-HP) and Principal Investigator for the published
celiprolol study. “Having established celiprolol as the standard of
care in France for vEDS patients, we are excited to collaborate
with Acer to help bring celiprolol to U.S. patients who are
suffering from this devastating, life-threatening disease.”
The previously completed European study, published on October
30, 2010, in The Lancet, was stopped early having achieved
statistical significance in its primary endpoints, with arterial
dissection or rupture affecting 5 (20%) celiprolol patients and 14
(50%) subjects in the non-treated control group (hazard ratio [HR]
0.36; p-value 0.04). The combined primary and secondary endpoints
of intestinal or uterine rupture affected 6 (24%) celiprolol
patients and 17 (61%) subjects in the non-treated control group (HR
0.31; p-value 0.01). The study was conducted in 53 patients, who
were randomly assigned either a twice daily treatment of celiprolol
or no treatment. Mean duration of follow-up was 47 months
prior to trial halt.
“We are committed to bringing EDSIVO™ to vEDS patients who
currently do not have access to this treatment,” said Robert D.
Steiner, M.D., Chief Medical Officer of Acer. “Our confirmation of
the published celiprolol clinical data with an Acer-sponsored
retrospective source verified analysis of the trial
data represents a critical element of the clinical module in
our NDA, which we are diligently building, along with current
manufacturing, non-clinical and other components of the regulatory
package.”
“We continue to successfully rapidly advance our lead product
candidate, EDSIVO™, a potential life-saving therapy for patients
with vEDS, towards an NDA filing, which we expect to accomplish in
the first half of 2018,” said Chris Schelling, CEO and Founder of
Acer. “In addition to source verifying a definitive Event-Free
Survival endpoint from a previously completed robust clinical
study, modernizing manufacturing and assembling other components of
the regulatory package, we are executing on a number of key medical
affairs focused initiatives for vEDS patients. Specifically,
we are setting up Centers of Excellence to optimize patient care,
and intend to develop a prospective vEDS Patient Registry and
provide integrated care support programs.”
About EDSIVO™ and vEDS
Ehlers-Danlos Syndrome (EDS) is a group of hereditary disorders
of connective tissue. vEDS is the most severe subtype where
patients suffer from life threatening arterial dissections and
ruptures, as well as intestinal and uterine ruptures. The average
mortality is 51 years of age. An Acer commissioned patient-finder
study identified 2,200 vEDS patients in the U.S. from an analysis
of commercially available patient claims data. However, experts
estimate as many as 5,000 patients may be affected. There are
currently no FDA-approved therapies for vEDS(2).
Acer is advancing EDSIVO™ (celiprolol), a new chemical entity
(NCE), for the treatment of vEDS and plans to file a NDA based on a
randomized controlled clinical study of celiprolol.(1) In 2015, the
U.S. Food and Drug Administration (FDA) granted EDSIVO™ orphan drug
designation for the potential treatment of vEDS.
About Acer Therapeutics
Acer, headquartered in Cambridge, MA, is a pharmaceutical
company that acquires, develops and intends to commercialize
therapies for patients with serious rare and ultra-rare diseases
with critical unmet medical need. Acer’s late-stage clinical
pipeline includes two candidates for severe genetic disorders for
which there are few or no FDA-approved treatments: EDSIVO™
(celiprolol) for vEDS, and ACER-001 (a fully taste-masked,
immediate release formulation of sodium phenylbutyrate) for urea
cycle disorders (UCD) and Maple Syrup Urine Disease (MSUD). There
are no FDA-approved drugs for vEDS and MSUD and limited options for
UCD, which collectively impact more than 4,000 patients in the
United States. Acer’s products have clinical proof-of-concept and
mechanistic differentiation, and Acer intends to seek approval for
them in the U.S. by using the regulatory pathway established under
section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act, or
FFDCA, that allows an applicant to rely for approval at least in
part on third-party data, which is expected to expedite the
preparation, submission, and approval of a marketing
application.
For more information, visit www.acertx.com.
References
(1) 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: 1476–84.
(2) Pepin MG, et al. Survival is affected by mutation type and
molecular mechanism in vascular Ehlers–Danlos syndrome (EDS type
IV) Genet Med. 16: 881-888.
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, future financial position, future
revenue, projected expenses, prospects, plans and objectives of
management are forward-looking statements. Examples of such
statements include, but are not limited to, statements relating to
Acer’s listing on the Nasdaq Capital Market; expectations regarding
the capitalization, resources and ownership structure of the
company; the potential for EDSIVO™ (celiprolol) and ACER-001 to
safely and effectively target diseases; the adequacy of the
company’s capital to support its future operations and its ability
to successfully initiate and complete clinical trials; the nature,
strategy and focus of the company; the development and commercial
potential of any product candidates of the company; and the
executive and board structure of the company. Acer 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 of the company to meet its 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 Acer’s
intellectual property, risks related to the drug development and
the regulatory approval process and the impact of competitive
products and technological changes. Acer disclaims 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.
Investor Contact:Hans VitzthumLifeSci
AdvisorsPh: 212-915-2568hans@lifescieadvisors.com
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