human recombinant apoA-I/sphingomyelin/dipalmitoylphosphatidylglycerol (CER-001) / Abionyx Pharma - LARVOL DELTA

Brain-Kidney Interplay during Sepsis-Associated AKI: Results from the RACERS Study (KIDNEY WEEK 2024) - "CER-001 treatment downregulated IDO1 and reduce neuroactive metabolites and waste accumulation."

Cognitive Disorders • Infectious Disease • Septic Shock • ICAM1 • IDO1 • VCAM1

Beneficial effects of recombinant CER-001 high-density lipoprotein infusion in sepsis: results from a bench to bedside translational research project. (PubMed, BMC Med) - P2 | "CER-001 shows promise as a therapeutic strategy for sepsis management, improving outcomes and mitigating inflammation and organ damage."

Journal • Acute Kidney Injury • Immunology • Infectious Disease • Inflammation • Nephrology • Renal Disease • Septic Shock • APOA1

CER-001, an Engineered High-Density Lipoprotein, Shows Beneficial Pleiotropic Effects in Patients with Sepsis in RACERS: A Phase 2A Randomized Control Trial (KIDNEY WEEK 2023) - P2 | "CER-001 replicates the beneficial pleiotropic effects of natural HDL by scavenging LPS, reducing inflammation, and protecting the endothelium. Therefore, CER-001 represents a promising therapeutic strategy for sepsis management, improving outcomes and mitigating the cytokine storm and associated organ damage often observed in our patients."

Clinical • Late-breaking abstract • P2a data • Acute Kidney Injury • Immunology • Infectious Disease • Inflammation • Nephrology • Renal Disease • Septic Shock • CXCL8 • IL6 • TNFA

CER-001, a Recombinant HDL, Modulates Ischemia/Reperfusion Injury (IRI) in Hypothermic (HMP) and Normothermic Perfusion (NMP) Solutions Through pAKT/eNOS Activation (KIDNEY WEEK 2023) - "The use of CER-001 in the perfusion liquid counteracted vasocostriction and inflammation and improved renal resistance during kidney perfusion, thereby increasing pool of organ for transplant."

Cardiovascular • Inflammation • Renal Disease • Reperfusion Injury • Transplantation • CD31 • EDN1 • IL6 • NOS3 • PECAM1 • TNFA

Pre-beta HDL discoidal mimetic, CER-001, and novel Apolipoprotein A-I (ApoA-I) multimers, Cargomer, as new targeted delivery vehicles for therapeutic cancer medicines (EORTC-NCI-AACR 2018) - "CER-001-Pacli is at least as efficacious as Abraxane nanoparticles to deliver and treat tumor-bearing mice. The Cargomer is a flexible targeting nanoparticle platform with promising delivery activities."

Breast Cancer • Esophageal Cancer • Gastrointestinal Cancer • Oncology • Pancreatic Cancer • Solid Tumor

A Recombinant BIO-HDL (CER-001) Can Prevent SARS-COV2-Induced Renal Dysfunction by Restoring SR-BI Signalling (ERA-EDTA 2022) - "Consistent with in vitro results, analysis of renal biopsies from COVID-19 patients with proteinuria showed increased SR-BI and ACE-2 cytoplasmic signals and reduced expression at the apical domain of injured tubules. CONCLUSION Our data confirmed the key role of lipid profile in SARS-COV2 infection, evaluating the molecular signalling involved in HDL metabolism and inflammatory processes, and could offer new therapeutic strategies for COVID-19 patients."

Infectious Disease • Nephrology • Novel Coronavirus Disease • Renal Disease • Respiratory Diseases • APOA1 • NOS3

Clinical trial in which people are allocated by chance to receive one of several clinical treatments, different doses, to prevent sepsis-induced acute kidney injury Studio clinico in cui le diverse opzioni di trattamento vengono assegnate in modo casuale, per identificare l’intervallo di dosi efficaci del farmaco CER-001 nel prevenire il danno renale acuto secondario a sepsi. (clinicaltrialsregister.eu) - P2; N=20; Ongoing; Sponsor: CERENIS THERAPEUTICS

Clinical • New P2 trial • Acute Kidney Injury • Dermatology • Immunology • Infectious Disease • Inflammation • Nephrology • Renal Disease • Septic Shock • Systemic Inflammatory Response Syndrome • CSF2 • CXCL8 • IL6 • TNFA

CER-001 ameliorates lipid profile and kidney disease in a mouse model of Familial LCAT Deficiency. (PubMed, Metabolism) - "Treatment with CER-001 ameliorates the dyslipidemia typically associated with LCAT deficiency and more importantly limits renal damage in a mouse model of renal disease in LCAT deficiency. The present results provide a rationale for using CER-001 in FLD patients."

Journal • Preclinical • Dyslipidemia • Metabolic Disorders • Nephrology • Renal Disease

ABIONYX Announces the Publication of Pre-clinical Data in the Journal Metabolism Demonstrating that CER-001 Improves Lipid Profile and Kidney Function for an Ultra-rare Kidney Disease (Businesswire) - "ABIONYX Pharma...today announced the publication of a set of pre-clinical data...In a genetically modified mouse model of LCAT deficiency, CER-001 had beneficial effects not only on lipid profile but also on renal function, reducing urinary albumin to creatinine ratio and restoring nestin and nephrin content in the glomerulus....The company will continue to work on strengthening the understanding the mechanism of action of CER-001 in kidney disease....Treatment with CER-001 ameliorates renal function in a mouse model with ultra-rare renal disease, reducing urinary albuminuria and restoring podocyte functionality; The data set the basis for the potential use of CER-001 in other renal diseases."

Preclinical • Chronic Kidney Disease • Renal Disease

No benefit of HDL mimetic CER-001 on carotid atherosclerosis in patients with genetically determined very low HDL levels. (PubMed, Atherosclerosis) - "In patients with genetically determined very low HDL-cholesterol, 24 weeks of treatment with HDL mimetic CER-001 did not reduce carotid vessel wall dimensions or arterial wall inflammation, compared with placebo."

Clinical • Journal • Atherosclerosis • Cardiovascular • Dyslipidemia • Familial Primary HypoAlphalipoproteinemia • Immunology

No benefit of HDL mimetic CER-001 on carotid atherosclerosis in patients with genetically determined very low HDL levels (ScienceDirect) - P3, N=30; TANGO (NCT02697136); Sponsor: Cerenis Therapeutics, SA; "In patients with genetically determined very low HDL-cholesterol levels based on FHA, we did not observe a reduction of atherosclerotic plaque size, nor arterial wall inflammation after 24 weeks of treatment with CER-001 infusions compared with placebo, despite an increase in cholesterol efflux capacity. These results are in line with contemporary randomized clinical trials, which failed to demonstrate benefit of HDL infusion on imaging endpoints in atherosclerosis."

P3 data

Cerenis Therapeutics: Cash position and revenue for Q3 2016 (Cerenis Press Release) - P2, N=302; NCT02484378; Sponsor: Cerenis; "Enrolment in the CARAT study has completed on schedule and results are anticipated no later than the first quarter of 2017. Subject to the positive outcome of CARAT, a phase III pivotal study (CALMS) will then be launched...No safety or tolerability issues have been identified in CARAT to prevent the study being completed as planned – periodic safety reviews have been performed during the on-treatment period by a data safety monitoring board (DSMB)...Preclinical data showing favorable safety and tolerability profile of CER-001 were presented at the 2016 European Society of Cardiology Congress (ESC)."

Anticipated new P3 trial • Anticipated P2 data • Conference • DSMB • Enrollment closed • Preclinical • Dyslipidemia

Cerenis Therapeutics announces the results of the LOCATION study (Cerenis Press Release) - P2, N=8; NCT02484378; "Cerenis Therapeutics...today announced the results of the LOCATION clinical study, which evaluated the selectivity of CER-001...by collecting serial blood samples, the investigators showed that one hour after CER-001 infusion plasmamediated cholesterol efflux increased by 13.8% and mean plasma apoA-I levels increased by 9.9 mg/dL. Both apoA-I levels and cholesterol efflux capacity returned to baseline values after 24 hours"

P2 data • Dyslipidemia

Effects of the high-density lipoprotein mimetic agent CER-001 on coronary atherosclerosis in patients with acute coronary syndromes: A randomized trial (Eur Heart J) - P2, N=507; NCT01201837; Sponsor: Cerenis Therapeutics; "The nominal change in the total atheroma volume (adjusted means) was -2.71, -3.13, -1.50, and -3.05 mm3 with placebo, CER-001 3 mg/kg, 6 mg/kg, and 12 mg/kg, respectively (primary analysis of 12 mg/kg vs. placebo: P = 0.81). There was also no difference among groups for the nominal change in per cent atheroma volume (0.02, -0.02, 0.01, and 0.19%; nominal P = 0.53 for 12 mg/kg vs. placebo)....CER-001 infusions did not reduce coronary atherosclerosis on IVUS and QCA when compared with placebo"

P2 data • Acute Coronary Syndrome • Atherosclerosis • Dyslipidemia

Cerenis: Corporate Presentation (Cerenis) - "Increase in HDL cholesterol: +700% for 45 mg/kg dose"

P1 data • Dyslipidemia

Cerenis Therapeutics announces last patient dosed in CARAT phase II study with CER-001 in post-acute coronary syndrome patients (Cerenis Press Release) - P2, N=301; NCT02484378; Sponsor: Cerenis; "Cerenis Therapeutics...announces the dosing of the last patient in the global Phase II CARAT study...Enrollment in CARAT was completed in August 2016 and the last patient has now received the final infusion of CER-001 or placebo...the final follow-up safety visit is expected to occur at the end of the month...Data analysis will commence thereafter and the Company expects to report topline results no later than the first quarter of 2017."

Anticipated P2 data • Anticipated trial completion date • Enrollment closed • Acute Coronary Syndrome • Dyslipidemia

First patient enters phase III TANGO clinical trial to evaluate efficacy to regress atherosclerosis and safety of CER-001 in patients with familial primary hypoalphalipoproteinemia (FPHA) (Cerenis Press Release) - P3, N=NA; "Cerenis Therapeutics...today announces that the first patient has been enrolled into the Phase III TANGO trial that is designed to assess both the efficacy of CER-001 to regress atherosclerosis and its safety in patients with FPHA...The TANGO trial, which began in the 4th quarter of 2015 as announced to investors at the time of the Company’s IPO, is on schedule and results are expected in Q3 2017."

Anticipated P3 data • Enrollment open • Dyslipidemia

Cerenis receives EMA Orphan Drug Designations for CER-001 for the treatment of ApoA-I and ABCA-1 deficiencies (Herald) - "Cerenis Therapeutics...announced today that it has received two separate Orphan Drug Designations from the European Medicines Agency (EMA) for the use of CER-001 in the treatment of patients with rare genetic defects in HDL synthesis/maturation pathways, specifically apoA-I deficiency and ABCA1 deficiency."

Orphan drug • Atherosclerosis • Dyslipidemia

Cerenis Therapeutics announces that CARAT phase 2 study dataon CER-001 have been presented at ACC annual meeting 2017 (Cerenis Press Release) - P2, N=NA; NCT02484378 (CARAT); Sponsor: Cerenis; "Cerenis Therapeutics...announces that CARAT Phase 2 Study data on CER-001 in post-acute coronary syndrome (ACS) patients have been presented at the Annual American College of Cardiology (ACC) Scientific Sessions in Washington DC, USA...The CARAT Phase 2 Study confirmed the safety profile of CER-001 but did not meet its primary efficacy endpoint of regression of atherosclerotic plaques in post-ACS patients."

P2 data • Acute Coronary Syndrome • Dyslipidemia

Cerenis: Corporate Presentation (Cerenis) - "Primary endpoint (reduction in plaque at 12 mg/kg dose vs. placebo) not achieved"; "Reduction in the total volume of atherosclerosis vs. baseline was statistically significant at 3 mg/kg"

P2 data • Dyslipidemia

Cerenis reports two positive phase II studies for HDL mimetic CER-001 at EAS (Cerenis Press Release) - "...announced that two of its Phase II studies, SAMBA and MODE (Modifying Orphan Disease Evaluation), with CER-001...
P2, N=7; 'CER-001 increased cholesterol mobilization and esterification in the HDL fraction, and one month of treatment with 9 doses of CER-001 resulted in a statistically significant reduction in carotid artery Mean Vessel Wall Area...'; P2,N=23;NCT01412034;'...a statistically significant reduction in carotid artery Mean Vessel Wall Area, as measured by Magnetic Resonance Imaging (3T-MRI), after 6-months of bi-weekly CER-001 treatment on top of optimized LDL-c-lowering therapy, including apheresis.'"

P2 data • Atherosclerosis • Dyslipidemia

Cash position and revenue for Q3 2015 (Cerenis Press Release) - "The first patient in TANGO trial will be enrolled during the last quarter of 2015."

Anticipated new P3 trial • Dyslipidemia

Cerenis reports top-line phase II results for its HDL mimetic CER-001 - P2b, N=507; CHI-SQUARE (NCT01201837); Sponsor: Cerenis; "...its Phase IIb CHI-SQUARE (Can HDL Infusions Significantly Quicken Atherosclerosis REgression?) study did not reach its primary endpoint in post-Acute Coronary Syndrome (ACS) patients."

P2b data • Acute Coronary Syndrome • Atherosclerosis • Dyslipidemia

Publication of the LOCATION clinical study results in the renowned scientific journal of the European Atherosclerosis Society (EAS) (Cerenis Press Release) - P2, N=8; EudraCT2014-001666-10 (Location); NCT02484378 (CARAT); "Cerenis Therapeutics...today announces the publication of the LOCATION clinical study results in the internationally peer-reviewed journal...they are particularly reassuring prior to the publication of the CARAT study results, planned for first quarter of 2017..."

Anticipated conference • Anticipated P2 data • Dyslipidemia

Big pharma has eyes on Cerenis' HDL drug (FierceBiotech) - Cerenis is testing in P2 trials of CER-001, whether infusions of the drug will prevent people who've already had a heart attack from suffering another; Mid-stage trials are expected to conclude next year; CER-001 could be approved by 2017 & top $8B in sales by 2021

Anticipated regulatory • Anticipated sales • Anticipated trial completion date • Dyslipidemia