Phase 3, Multicenter, Randomized, 48 Week, Double Blind, Parallel Group, Placebo Controlled Study to Evaluate Efficacy and Safety of CER-001 on Vessel Wall Area in Patients With Genetically Defined Familial Primary Hypoalphalipoproteinemia

The purpose of this study is to assess the impact of 29 intravenous infusions of CER-001 vs. placebo, given at weekly (9 infusions) and biweekly (20 infusions) intervals on carotid vessel wall area as measured by 3TMRI, when administered to patients with familial primary hypoalphalipoproteinemia with proven CVD and appropriate background lipid-lowering therapy.

Start Date01 Dec 2015

Sponsor / Collaborator

Cerenis Therapeutics, Inc.

NCT02484378

/ CompletedPhase 2

CER-001 Atherosclerosis Regression ACS Trial; A Phase II Multi-Center, Double-Blind, Placebo-Controlled, Dose-Focusing Trial Of Cer-001 In Subjects With Acute Coronary Syndrome

The purpose of this study is to assess the impact of ten intravenous infusions of 3 mg/kg CER 001 vs. placebo, given at weekly intervals for ten weeks, on atherosclerotic plaque volume as measured by coronary IVUS, when administered to subjects presenting with Acute Coronary Syndrome (ACS) with significant plaque volume.

Start Date01 Aug 2015

Sponsor / Collaborator

Cerenis Therapeutics, Inc.

[+1]

100 Clinical Results associated with CER-001

100 Translational Medicine associated with CER-001

100 Patents (Medical) associated with CER-001

Literatures (Medical) associated with CER-001

01 Dec 2022·Journal of nuclear medicine : official publication, Society of Nuclear Medicine

⁸⁹Zr-Labeled High-Density Lipoprotein Nanoparticle PET Imaging Reveals Tumor Uptake in Patients with Esophageal Cancer

Article

Author: Kroon, Jeffrey ; Stroes, Erik S G ; Gisbertz, Suzanne S ; Gurney-Champion, Oliver ; Verberne, Hein J ; van Dongen, Guus A M S ; Vugts, Danielle J ; Zheng, Kang H ; van Laarhoven, Hanneke W M ; Hulshof, Maarten C C M ; Coolen, Bram F ; Schoormans, Jasper ; Meijer, Sybren L ; Nederveen, Aart J

Nanomedicine holds promise for the delivery of therapeutic and imaging agents to improve cancer treatment outcomes. Preclinical studies have demonstrated that high-density lipoprotein (HDL) nanoparticles accumulate in tumor tissue on intravenous administration. Whether this HDL-based nanomedicine concept is feasible in patients is unexplored. Using a multimodal imaging approach, we aimed to assess tumor uptake of exogenously administered HDL nanoparticles in patients with esophageal cancer. Methods: The HDL mimetic CER-001 was radiolabeled using ⁸⁹Zr to allow for PET/CT imaging. Patients with primary esophageal cancer staged T2 and above were recruited for serial ⁸⁹Zr-HDL PET/CT imaging before starting chemoradiation therapy. In addition, patients underwent routine ¹⁸F-FDG PET/CT and 3-T MRI scanning (diffusion-weighted imaging/intravoxel incoherent motion imaging and dynamic contrast-enhanced MRI) to assess tumor glucose metabolism, tumor cellularity and microcirculation perfusion, and tumor vascular permeability. Tumor biopsies were analyzed for the expression of HDL scavenger receptor class B1 and macrophage marker CD68 using immunofluorescence staining. Results: Nine patients with adenocarcinoma or squamous cell carcinoma underwent all study procedures. After injection of ⁸⁹Zr-HDL (39.2 ± 1.2 [mean ± SD] MBq), blood-pool SUV_mean decreased over time (11.0 ± 1.7, 6.5 ± 0.6, and 3.3 ± 0.5 at 1, 24, and 72 h, respectively), whereas liver and spleen SUV_mean remained relatively constant (4.1 ± 0.6, 4.0 ± 0.8, and 4.3 ± 0.8 at 1, 24, and 72 h, respectively, for the liver; 4.1 ± 0.3, 3.4 ± 0.3, and 3.1 ± 0.4 at 1, 24, and 72 h, respectively, for the spleen) and kidney SUV_mean markedly increased over time (4.1 ± 0.9, 9.3 ± 1.4, and 9.6 ± 2.0 at 1, 24, and 72 h, respectively). Tumor uptake (SUV_peak) increased over time (3.5 ± 1.1 and 5.5 ± 2.1 at 1 and 24 h, respectively [P = 0.016]; 5.7 ± 1.4 at 72 h [P = 0.001]). The effective dose of ⁸⁹Zr-HDL was 0.523 ± 0.040 mSv/MBq. No adverse events were observed after the administration of ⁸⁹Zr-HDL. PET/CT and 3-T MRI measures of tumor glucose metabolism, tumor cellularity and microcirculation perfusion, and tumor vascular permeability did not correlate with tumor uptake of ⁸⁹Zr-HDL, suggesting that a specific mechanism mediated the accumulation of ⁸⁹Zr-HDL. Immunofluorescence staining of clinical biopsies demonstrated scavenger receptor class B1 and CD68 positivity in tumor tissue, establishing a potential cellular mechanism of action. Conclusion: To our knowledge, this was the first ⁸⁹Zr-HDL study in human oncology. ⁸⁹Zr-HDL PET/CT imaging demonstrated that intravenously administered HDL nanoparticles accumulated in tumors of patients with esophageal cancer. The administration of ⁸⁹Zr-HDL was safe. These findings may support the development of HDL nanoparticles as a clinical delivery platform for drug agents. ⁸⁹Zr-HDL imaging may guide drug development and serve as a biomarker for individualized therapy.

01 Sep 2018·JAMA cardiologyQ1 · MEDICINE

Effect of Serial Infusions of CER-001, a Pre-β High-Density Lipoprotein Mimetic, on Coronary Atherosclerosis in Patients Following Acute Coronary Syndromes in the CER-001 Atherosclerosis Regression Acute Coronary Syndrome Trial

Q1 · MEDICINE

Article

Author: Scherer, Daniel J. ; Schwartz, Gregory G. ; Ray, Kausik K. ; Psaltis, Peter J. ; Di Giovanni, Giuseppe ; Nicholls, Stephen J. ; Worthley, Stephen G. ; Dasseux, Jean-Louis ; Keyserling, Connie ; Andrews, Jordan ; Butters, Julie ; Kastelein, John J. P. ; Janssan, Alex ; Merkely, Bela ; Pisaniello, Anthony D. ; Nissen, Steven E. ; Kim, Susan W. ; Griffith, Liddy

Importance:

CER-001 is a negatively charged, engineered pre-β high-density lipoprotein (HDL) mimetic containing apolipoprotein A-I and sphingomyelin. Preliminary studies demonstrated favorable effects of CER-001 on cholesterol efflux and vascular inflammation. A post hoc reanalysis of a previously completed study of intravenous infusion of CER-001, 3 mg/k, showed that the intravenous infusion in patients with a high coronary plaque burden promoted regression as assessed by intravascular ultrasonography.

Objective:

To determine the effect of infusing CER-001 on coronary atherosclerosis progression in statin-treated patients.

Design, Setting, and Participants:

A double-blind, randomized, multicenter trial evaluating the effect of 10 weekly intravenous infusions of CER-001, 3 mg/kg, (n = 135) or placebo (n = 137) in patients with an acute coronary syndrome (ACS) and baseline percent atheroma volume (PAV) greater than 30% in the proximal segment of an epicardial artery by intravascular ultrasonography. The study included 34 academic and community hospitals in Australia, Hungary, the Netherlands, and the United States in patients with ACS presenting for coronary angiography. Patients were enrolled from August 15, 2015, to November 19, 2016.

Interventions:

Participants were randomized to receive weekly CER-001, 3 mg/kg, or placebo for 10 weeks in addition to statins.

Main Outcomes and Measures:

The primary efficacy measure was the nominal change in PAV from baseline to day 78 measured by serial intravascular ultrasonography imaging. The secondary efficacy measures were nominal change in normalized total atheroma volume and percentage of patients demonstrating plaque regression. Safety and tolerability were also evaluated.

Results:

Among 293 patients (mean [SD] age, 59.8 [9.4] years; 217 men [79.8%] and 261 white race/ethnicity [96.0%]), 86 (29%) had statin prior use prior to the index ACS and 272 (92.8%) had evaluable imaging at follow-up. The placebo and CER-001 groups had similar posttreatment median levels of low-density lipoprotein cholesterol (74 mg/dL vs 79 mg/dL; P = .15) and high-density lipoprotein cholesterol (43 mg/dL vs 44 mg/dL; P = .66). The primary efficacy measure, PAV, decreased 0.41% with placebo (P = .005 compared with baseline), but not with CER-001 (-0.09%; P = .67 compared with baseline; between group differences, 0.32%; P = .15). Similar percentages of patients in the placebo and CER-001 groups demonstrated regression of PAV (57.7% vs 53.3%; P = .49). Infusions were well tolerated, with no differences in clinical and laboratory adverse events observed between treatment groups.

Conclusions and Relevance:

Infusion of CER-001 did not promote regression of coronary atherosclerosis in statin-treated patients with ACS and high plaque burden.

Trial Registration:

ClinicalTrials.gov Identifier: NCT2484378.

01 Jun 2017·Cardiovascular diagnosis and therapyQ4 · MEDICINE

Regression of coronary atherosclerosis with infusions of the high-density lipoprotein mimetic CER-001 in patients with more extensive plaque burden

Q4 · MEDICINE

Article

Author: Stephen J. Nicholls ; John F. Paolini ; Yu Kataoka ; Jordan Andrews ; Julie Butters ; Rishi Puri ; Constance Keyserling ; MyNgan Duong ; Jean-Louis Dasseux ; Jessica Fendler ; Nisha Schwarz ; Tracy Nguyen

BACKGROUND:

CER-001 is an engineered pre-beta high-density lipoprotein (HDL) mimetic, which rapidly mobilizes cholesterol. Infusion of CER-001 3 mg/kg exhibited a potentially favorable effect on plaque burden in the CHI-SQUARE (Can HDL Infusions Significantly Quicken Atherosclerosis Regression) study. Since baseline atheroma burden has been shown as a determinant for the efficacy of HDL infusions, the degree of baseline atheroma burden might influence the effect of CER-001.

METHODS:

CHI-SQUARE compared the effect of 6 weekly infusions of CER-001 (3, 6 and 12 mg/kg) vs. placebo on coronary atherosclerosis in 369 patients with acute coronary syndrome (ACS) using serial intravascular ultrasound (IVUS). Baseline percent atheroma volume (B-PAV) cutoff associated with atheroma regression following CER-001 infusions was determined by receiver-operating characteristics curve analysis. 369 subjects were stratified according to the cutoff. The effect of CER-001 at different doses was compared to placebo in each group.

RESULTS:

A B-PAV ≥30% was the optimal cutoff associated with PAV regression following CER-001 infusions. CER-001 induced PAV regression in patients with B-PAV ≥30% but not in those with B-PAV <30% (-0.45%±2.65% vs. +0.34%±1.69%, P=0.01). Compared to placebo, the greatest PAV regression was observed with CER-001 3mg/kg in patients with B-PAV ≥30% (-0.96%±0.34% vs. -0.25%±0.31%, P=0.01), whereas there were no differences between placebo (+0.09%±0.36%) versus CER-001 in patients with B-PAV <30% (3 mg/kg; +0.41%±0.32%, P=0.39; 6 mg/kg; +0.27%±0.36%, P=0.76; 12 mg/kg; +0.32%±0.37%, P=0.97).

CONCLUSIONS:

Infusions of CER-001 3 mg/kg induced the greatest atheroma regression in ACS patients with higher B-PAV. These findings identify ACS patients with more extensive disease as most likely to benefit from HDL mimetic therapy.

News (Medical) associated with CER-001

13 Jun 2024

·www.biospace.com

ABIONYX Pharma has completed a successful pre-IND meeting with the FDA for CER-001 Phase 2b/3 Clinical Trial for Patients with Sepsis

Based on encouraging Phase 2a data and a productive pre-IND Type B meeting with U.S. Food and Drug Administration (FDA), ABIONYX Pharma intends to Investigational New Drug application (IND) in the coming months which will include a Phase 2b/3 clinical trial for CER-001 in Sepsis Access here the full press release TOULOUSE, France & LAKELAND, Mich.--(BUSINESS WIRE)-- ABIONYX Pharma, (FR0012616852 - ABNX - eligible for PEA PME), a new generation biotech company dedicated to the discovery and development of innovative therapies based on the world’s only natural recombinant apoA-I, today announced that the company has completed a pre-IND (Investigational New Drug Application, IND) meeting with the US Food and Drug Administration and has received feedback to support an IND filing for its candidate drug. This is an important validation of the quality of the project and a significant step towards an application to include American study centers in future clinical trials. ABIONYX Pharma intends to IND application to the US authority in the coming months. About ABIONYX Pharma ABIONYX Pharma is a next-generation biotech company focused on developing innovative medicines for diseases where there is no effective or existing treatment, even the rarest ones. The company expedites the development of novel therapeutics through an extensive expertise in lipid science and a differentiated apoA-I-based technology platform. ABIONYX Pharma is committed to radically improving treatment outcomes in Sepsis and critical care. View source version on businesswire.com: Contacts Contacts: 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 Source: ABIONYX Pharma View this news release online at:

INDPhase 2

15 Feb 2024

·www.biospace.com

ABIONYX Pharma Acknowledges the Clinical Results of the Phase 3 AEGIS-II Study Evaluating the Efficacy and Safety of CSL Behring’s Human-plasma-derived apoA-I, CSL112

TOULOUSE, France & LAKELAND, Mich.--(BUSINESS WIRE)-- Access here the full press release ABIONYX Pharma, (FR0012616852 – ABNX – PEA PME eligible), a new generation biotech company dedicated to the discovery and development of innovative therapies based on the world’s only natural recombinant apoA-I, today acknowledges that the Phase 3 AEGIS-II study evaluating the efficacy and safety of CSL Behring’s human-plasma-derived apoA-I, CSL112, compared to placebo in reducing the risk of major adverse cardiovascular events (MACE) in patients following an acute myocardial infarction (AMI), did not meet its primary efficacy endpoint of MACE reduction at 90 days. About CER-001 CER-001 is a novel engineered recombinant human apoA-I that was designed to mimic the structural and functional biological properties of natural, nascent HDL, also known as pre-β HDL, and has been shown to perform all steps of the Reverse Lipid Transport pathway (RLT), the only natural pathway responsible for lipid elimination. Administered CER-001 particles increase transient apoA-I and the number of HDL particles and promote the elimination of trapped cholesterol and lipids in tissues in the absence of LCAT enzyme for example, but also the elimination of bacterial lipid endotoxin (LPS) in the case of sepsis. HDL particles are then recognized by the liver, leading to the elimination of these transported lipids via a process called Reverse Lipid Transport (RLT). About ABIONYX Pharma ABIONYX Pharma is a next-generation biotech company focused on developing innovative medicines in diseases where there is no effective or existing treatment, even the rarest ones. The company expedites the development of novel therapeutics through an extensive expertise in lipid science and a differentiated apoA-I -based technology platform. ABIONYX Pharma is committed to radically improving treatment outcomes in sepsis and critical care. View source version on businesswire.com: Contacts 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 Source: ABIONYX Pharma View this news release online at:

Phase 3Clinical Result

02 Nov 2023

·www.biospace.com

ABIONYX Pharma RACERS Study Data in Sepsis Presented at the American Society of Nephrology (ASN) 2023 Annual Meeting "Kidney Week"

CER-001 showed robust efficacy in sepsis demonstrating statistically significant sustained reduction in endotoxins (LPS) and consequent decreases in inflammatory cytokines and markers of endothelial dysfunction Data showed a reduced severity of AKI, a trend for decreased mortality and shorter ICU stay CER-001 shows promise as a therapeutic strategy for sepsis management, improving outcomes and mitigating inflammation and organ damage with the potential to save lives Simultaneous publication of data from translational sepsis research project, including the RACERS study, exclusively in BMC Medicine (Nature Springer) TOULOUSE, France & LAKELAND, Mich.--(BUSINESS WIRE)-- ABIONYX Pharma, (FR0012616852 - ABNX - PEA PME eligible), a new generation biotech company dedicated to the discovery and development of innovative therapies based on the world's only recombinant human apoA-1, today announced the full results of the RACERS Phase 2 clinical trial of CER-001, an apoA-1-based therapy for the treatment of sepsis, in a late-breaking clinical trial poster presentation at the American Society of Nephrology (ASN) Kidney Week 2023. Key Points from the RACERS Data: CER-001 rapidly and significantly eliminated endotoxins, and the result was maintained (p<0.05 on days 3, 6 and 9), whereas even by day 9, patients on standard care alone showed no decreases in endotoxin levels relative to baseline. Mortality for all patients at 30 days was 6.7% for the CER-001 group and 20.0% for patients treated with standard care alone. This represents a Relative Risk Reduction (RRR) of 65%. Among critical care patients, mortality rates were 14.7% compared to 50.0% for patients on standard care (RRR=71%). ICU patients treated with CER-001 were discharged earlier than patients receiving standard care, with an average ICU stay 5 days shorter than that of patients on standard care. Access here the full press release 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 apoA-I vectors used for targeted drug delivery.

Clinical ResultPhase 2

100 Deals associated with CER-001

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Familial HDL Deficiency	Phase 3	United States	Cerenis Therapeutics, Inc.	01 Dec 2015
Familial HDL Deficiency	Phase 3	Belgium	Cerenis Therapeutics, Inc.	01 Dec 2015
Familial HDL Deficiency	Phase 3	Canada	Cerenis Therapeutics, Inc.	01 Dec 2015
Familial HDL Deficiency	Phase 3	France	Cerenis Therapeutics, Inc.	01 Dec 2015
Familial HDL Deficiency	Phase 3	Israel	Cerenis Therapeutics, Inc.	01 Dec 2015
Familial HDL Deficiency	Phase 3	Italy	Cerenis Therapeutics, Inc.	01 Dec 2015
Familial HDL Deficiency	Phase 3	Netherlands	Cerenis Therapeutics, Inc.	01 Dec 2015
Sepsis	Phase 2	Italy	ABIONYX Pharma SA	07 Apr 2022
Acute kidney injury due to sepsis	Phase 2	Italy	-	22 Dec 2020
Bacterial urinary infection	Phase 2	Italy	-	22 Dec 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02697136 (TANGO, CTgov)	Phase 3	Familial HDL Deficiency	30	CER-001 (CER-001)	vpoipvzbzv(kafljhpkhn) = uxyehwljnc wismhzgrvw (azdkhjjbkn, coidobqclv - dnhfzopuzz) View more	-	22 Jul 2025
				Placebo (Placebo)	vpoipvzbzv(kafljhpkhn) = ajazmyshtw wismhzgrvw (azdkhjjbkn, idvlfqazqw - zfhzrdglpd) View more
EUCTR2020-004202-60-IT (RACERS, ASN2023) Manual	Phase 2	Sepsis	20	CER-001	huoyoszabg(lkfvtgwgup) = no serious adverse events were attributed to CER-001 use mxeizadgzs (hgjfqqhqje )	Positive	02 Nov 2023
				standard of care
NCT02484378 (CARAT, Pubmed \| JAMA Cardiol) Manual	Phase 2	Acute Coronary Syndrome \| Coronary Artery Disease \| Atherosclerosis	293	CER-001	ajdptecjhj(mqpaddsqrd) = ciuatpaomx fmknlklvim (rggycftjth ) View more	Negative	01 Sep 2018
				Placebo	ajdptecjhj(mqpaddsqrd) = eldezwqlxq fmknlklvim (rggycftjth ) View more
NCT01412034 (MODE, Pubmed \| Am Heart J) Manual	Phase 2	Homozygous familial hypercholesterolemia	23	CER-001	xvryifyryh(wddmbacwbo) = enfegwixck rmfkaoytgk (lgxdlcweyi )	Positive	01 May 2015
NCT01201837 (Pubmed \| Eur Heart J)	Phase 2	Acute Coronary Syndrome	507	Placebo	idxnaxlisx(mqnqdyocto) = hoavgydqpq agajuxoevb (xaufsilldh ) View more	-	07 Dec 2014
				CER-001 3 mg/kg	idxnaxlisx(mqnqdyocto) = bhhtdroccw agajuxoevb (xaufsilldh ) View more

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