- Positive results on primary and secondary endpoints,
identifying dose for future development
- Direct and significant effect of CER-001 on endotoxin
removal and consequent reduction in the inflammatory cascade or
“cytokine storm”
- Significant protective effect of CER-001 on endothelial
functionality
- Trends towards fewer ICU days for patients treated, lower
requirement for organ support and improved 30-day survival were
also seen
- Trial reinforces the well-established safety profile of
CER-001
- Efficacy results consistent with those observed in
COVID-19
Regulatory News:
ABIONYX Pharma (FR0012616852 – ABNX – PEA PME
eligible), a new generation biotech company dedicated to the
discovery and development of innovative therapies for patients,
today reported that the pilot Phase 2a clinical trial evaluating
CER-001, the only natural recombinant apoA-I, as a treatment for
septic patients at high risk of developing Acute Kidney Injury
(AKI) met its primary objective. There are no approved treatments
for septic patients in the world.
Loreto Gesualdo, full Professor, Head of the Nephrology,
Dialysis and Transplantation Unit, University of Bari Aldo Moro,
Italy, and lead investigator of the RACERS study stated: “We are
incredibly excited to share results from this pilot Phase 2a trial
evaluating CER-001 in septic patients at high risk of developing
acute kidney injury. There is a complete lack of treatment options
for septic patients at high risk of developing acute kidney injury,
a disease that in 2019 had an estimated 13.7 million related deaths
globally (Lancet 2022; 400: 2221–48). The trial shows promising
positive results across a variety of primary and secondary
endpoints. CER-001 was significantly able to scavenge endotoxins,
modulate the cytokine storm, and provide endothelial protection.
The trend observed in reducing renal damage, the need for organ
support and ICU-day stay underscores the potential clinical
significance of these results.”
The RACERS study included 20 patients with gram-negative sepsis
who were at high risk for acute kidney injury due to high levels of
endotoxin activity and decline in function of one or more organ
systems. Patients received either standard of care treatment alone,
or in combination with one of three dosage regimens of CER-001
(five patients per group).
The main objective of this pilot study was to investigate
whether the use of CER-001 at different doses, in combination with
standard of care (SOC) treatment, is safe and effective, providing
a potential new strategy to treat septic patients, reducing the
inflammatory response to endotoxin and preventing the progression
to AKI according to KDIGO (Kidney Disease: Improving Global
Outcomes) criteria, as well as safety and tolerability of the
dosage regimens in order to select the optimal dose of CER-001.
One of the metabolic characteristics of bacterial (like sepsis)
or virus infections (like sars-Cov-2) is the strong decrease of
circulating lipoprotein and particularly the High-Density
Lipoprotein (HDL) with its main containing protein apolipoprotein
A-I (apoA-I). As an example, apoA-I level was recently described as
the biomarker predictive of long-term mortality after surgical
sepsis1. The rational of Abionyx was to restore, using CER-001, the
apoA-I levels to reestablish all the functionality of this
individualized biomarker leading to potential benefit in sepsis
pathology.
RACERS pilot study has shown for the first time in a human pilot
trial that the recovery of a normal apoA-I level in patient stop
the cytokine storm and improve the clinical outcomes. CER-001
demonstrated rapid and sustained reduction in endotoxin levels and
consequent reduction in the inflammatory cascade or “cytokine
storm” relative to SOC alone. Endothelial biomarkers demonstrated a
significant protective effect of CER-001. Trends towards fewer ICU
days, lower requirement for organ support and improved 30-day
survival. Evaluation of safety data, taken together with the
pharmacokinetic and pharmacodynamic data, has identified the dose
that will be used in subsequent studies.
Connie Peyrottes, Senior VP clinical development at ABIONYX
Pharma, added: “In this pilot study, CER-001 was shown to directly
decrease endotoxin and inhibit inflammation, limiting the
associated downward spiral that septic patients often experience.
The broad pleiotropic effect of our apoA-I bioproduct can target
multiple facets of septic disease, rather than focusing on a single
step in the inflammatory process. Primary and secondary endpoints
showed benefits of CER-001 therapy when added to standard
treatments. The positive results from this Phase 2a trial show
CER-001 has the potential to be a gamechanger for critical
illnesses marked by inflammation and organ failure across different
high mortality clinical indications which continue to have high
unmet medical needs.”
Michael Davidson, M.D., Chairman of the Scientific Advisory
Board of ABIONYX concluded: “The impressive results of this Phase
2a study confirm that CER-001 may potentially treat sepsis and
other severe, acute inflammatory diseases. These results are
consistent with the previously published results in COVID-19 and
opens a new chapter in the development of CER-001 in the field of
short-term therapy for acute conditions.”
The observed safety and efficacy in RACERS were generally
consistent with historical data including clinical results for
CER-001 in COVID-19 that were published recently in the scientific
journal "Frontiers in Medicine", a specialty medicine journal, in
September 2022.
The potential use of CER-001 in septic patients is currently
under clinical development. These data will be discussed with
regulatory authorities, starting with Europe but also the U.S.
later this year in order to design an appropriate clinical and
regulatory development strategy for this disease state that
currently has no available treatment options.
About RACERS
RACERS is a clinical trial named RACERS (a RAndomized study
comparing short-term CER-001 infusions at different doses to
prevent Sepsis-induced acute kidney injury) with CER-001 in septic
patients at high risk of developing acute kidney injury.
Following the positive signals observed in the Temporary
Authorization for Named Use (ATUn) in an ultra-rare kidney disease,
the study assessed the role of CER-001, a novel, in preventing
Acute Kidney Injury (AKI) in septic patients. The core component of
the program is the launch of a 30-day Phase 2a clinical
dose-finding trial with the Company's lead product candidate,
CER-001, in the prevention of AKI in septic patients. Researchers
have demonstrated that in humans, reconstituted HDLs have a
scavenger role in reducing circulating endotoxin, as well as major
anti-inflammatory and endothelial activity. These important effects
were also demonstrated with CER-001 in a rigorous preclinical model
of sepsis-induced AKI developed in collaboration with an Italian
Veterinarian Hospital (Surgical Section, Chief: Prof. Antonio
Crovace). Several other AKI/sepsis models showed that HDL is a
critical factor in modifying the disease.
This clinical study, designed in concert with expert Italian
nephrologists (Nephrology, Dialysis and Transplantation Unit,
Chief: Prof. Loreto Gesualdo) and intensivists (Anesthesiology and
Resuscitation Unit, Chief: Prof. Salvatore Grasso), was a
randomized, open labelled, placebo-controlled, parallel-group study
evaluating the safety and efficacy of intravenously administered
CER-001 in patients with sepsis at high risk for AKI based on their
endotoxin levels and Sequential Organ Failure Assessment (SOFA
score). A total of 20 patients were randomized to receive 8 doses
of CER-001 over 6 days on top of standard of care, or standard of
care alone. The primary endpoint of the study was the onset and
severity of AKI according to KDIGO criteria as well as safety and
tolerability of the dosage regimens in order to select the optimal
dose of CER-001.
The clinical study was partnered with the University of
Bari.
About CER-001 in COVID
The complete data are available in the article:
“Apolipoprotein-A-I for severe COVID-19-induced hyperinflammatory
states: A prospective case study”
https://www.frontiersin.org/articles/10.3389/fphar.2022.936659/full
About ABIONYX Pharma
ABIONYX Pharma is a new generation biotech company that aims to
contribute to health through innovative therapies in indications
where there is no effective or existing treatment, even the rarest
ones. Thanks to its partners in research, medicine,
biopharmaceuticals and shareholding, the company innovates on a
daily basis to propose drugs for the treatment of renal and
ophthalmological diseases, or new HDL vectors used for targeted
drug delivery.
1 Guirgis, Faheem W.,et al. Annals of Intensive Care 11, n 1
(décembre 2021): 82.
https://doi.org/10.1186/s13613-021-00865-x.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230116005256/en/
NewCap Investor relations Nicolas Fossiez Louis-Victor
Delouvrier abionyx@newcap.eu +33 (0)1 44 71 98 53
NewCap Media relations Arthur Rouillé abionyx@newcap.eu
+33 (0)1 44 71 00 15
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