CER-001 explained

Drug Name:CER-001
Routes Of Administration:Infusion
Synonyms:CER-001, CER 001
Cas Number:1383435-67-3
Drugbank:DB18538

CER-001 is a recombinant high-density lipoprotein (HDL) mimetic that has orphan drug status.[1] It is in early-stage clinical trials for the potential treatment of hypoalphalipoproteinaemia,[2] acute coronary syndrome (ACS),[3] acute kidney injury (AKI),[4] [5] [6] atherosclerosis[7] and lecithin cholesterol acyltransferase (LCAT) deficiency.[8] CER-001 is also under investigation as possible agent for treating hyperinflammatory states based on lipid profile alterations due to COVID-19.[9] [10]

Chemistry

CER-001 is an artificially synthesized mimetic of recombinant human apolipoprotein AI (ApoA1), the main structural protein of natural HDL, together with two phospholipids, which are: sphingomyelin and dipalmitoyl-phosphatidylglycerol.[11]

Mechanism of action

CER-001 is designed to mimic the natural structure and function of nascent HDL, also known as pre-beta HDL.[12] This mimicry stimulates cholesterol efflux from macrophages, captures cholesterol and eliminates it via reverse lipid transport (RLT) pathway, also known as reverse cholesterol transport (RCT) pathway.[13]

Clinical trials

CER-001 was given in clinical human studies by intravenous infusion. It did not appear to affect clinical chemistry, hematology or coagulation parameters and is considered safe.[14]

Notes and References

  1. Web site: EU/3/21/2490 - orphan designation for treatment of lecithin-cholesterol acyltransferase deficiency . https://web.archive.org/web/20220316142619/https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2490 . 2022-03-16 . 2024-03-11 . European Medicines Agency.
  2. Web site: May 2018 . CER-001 for Familial Primary Hypoalphalipoproteinemia . https://web.archive.org/web/20240320142405/https://www.io.nihr.ac.uk/techbriefings/cer-001-for-familial-primary-hypoalphalipoproteinemia/ . 2024-03-20 . 2024-03-20 . NIHR Innovation Observatory Evidence Briefing . en-US.
  3. Tardif JC, Ballantyne CM, Barter P, Dasseux JL, Fayad ZA, Guertin MC, Kastelein JJ, Keyserling C, Klepp H, Koenig W, L'Allier PL, Lespérance J, Lüscher TF, Paolini JF, Tawakol A, Waters DD . Effects of the high-density lipoprotein mimetic agent CER-001 on coronary atherosclerosis in patients with acute coronary syndromes: a randomized trial . European Heart Journal . 35 . 46 . 3277–3286 . December 2014 . 24780501 . 4258222 . 10.1093/eurheartj/ehu171 . 2024-04-29 . 1522-9645 .
  4. Web site: 2023-01-17 . CER-001 identified as potential treatment for septic patients . https://web.archive.org/web/20230117140046/https://www.europeanpharmaceuticalreview.com/news/178595/cer-001-potential-treatment-identified-for-septic-patients/ . 2023-01-17 . 2024-03-09 . European Pharmaceutical Review . en . Eckford C.
  5. Web site: 2023-01-16 . Abionyx Pharma announces success in fighting sepsis-triggered AKI . https://web.archive.org/web/20230124091847/https://european-biotechnology.com/up-to-date/latest-news/news/abionyx-pharma-announces-success-in-fighting-sepsis-triggered-aki.html . 2023-01-24 . 2024-03-20 . european-biotechnology.com . en . 2023-01-16.
  6. Stasi A, Fiorentino M, Franzin R, Staffieri F, Carparelli S, Losapio R, Crovace A, Lacitignola L, Cimmarusti MT, Murgolo F, Stufano M, Cafiero C, Castellano G, Sallustio F, Ferrari C, Ribezzi M, Brienza N, Schirinzi A, Di Serio F, Grasso S, Pontrelli P, Tupin C, Barbaras R, Keyserling-Peyrottes C, Crovace A, Gesualdo L . Beneficial effects of recombinant CER-001 high-density lipoprotein infusion in sepsis: results from a bench to bedside translational research project . BMC Medicine . 21 . 1 . 392 . November 2023 . 37915050 . 10621167 . 10.1186/s12916-023-03057-5 . 2023-11-02 . free .
  7. Tardy C, Goffinet M, Boubekeur N, Ackermann R, Sy G, Bluteau A, Cholez G, Keyserling C, Lalwani N, Paolini JF, Dasseux JL, Barbaras R, Baron R . CER-001, a HDL-mimetic, stimulates the reverse lipid transport and atherosclerosis regression in high cholesterol diet-fed LDL-receptor deficient mice . Atherosclerosis . 232 . 1 . 110–118 . January 2014 . 24401224 . 10.1016/j.atherosclerosis.2013.10.018 . 2013-11-08 . free .
  8. Pavanello C, Turri M, Strazzella A, Tulissi P, Pizzolitto S, De Maglio G, Nappi R, Calabresi L, Boscutti G . The HDL mimetic CER-001 remodels plasma lipoproteins and reduces kidney lipid deposits in inherited lecithin:cholesterol acyltransferase deficiency . Journal of Internal Medicine . 291 . 3 . 364–370 . March 2022 . 34761839 . 9299003 . 10.1111/joim.13404 . 2021-11-11 .
  9. Stasi A, Franzin R, Fiorentino M, Squiccimarro E, Castellano G, Gesualdo L . Multifaced Roles of HDL in Sepsis and SARS-CoV-2 Infection: Renal Implications . International Journal of Molecular Sciences . 22 . 11 . gfac067.086 . June 2021 . 9383927 . 10.1093/ndt/gfac067.086 .
  10. Faguer S, Del Bello A, Danet C, Renaudineau Y, Izopet J, Kamar N . Apolipoprotein-A-I for severe COVID-19-induced hyperinflammatory states: A prospective case study . Frontiers in Pharmacology . 13 . 936659 . 2022-09-26 . 36225555 . 9550000 . 10.3389/fphar.2022.936659 . free .
  11. Web site: CER-001 . https://web.archive.org/web/20200811025936/https://www.abionyx.com/en/our-assets/cer-001 . 2020-08-11 . 2024-04-10 . Abionyx Pharma SA, Balma, France.
  12. Web site: Cerenis Reports Top-Line Phase II Results for its HDL Mimetic CER-001 . https://web.archive.org/web/20201022183044/https://www.fiercebiotech.com/biotech/cerenis-reports-top-line-phase-ii-results-for-its-hdl-mimetic-cer-001 . 2020-10-22 . 2024-03-12 . Fierce Biotech . 2014-01-03 . Helfand C.
  13. Barbaras R . Non-clinical development of CER-001 . Frontiers in Pharmacology . 6 . 220 . 2015-10-06 . 26500552 . 4594020 . 10.3389/fphar.2015.00220 . free .
  14. Keyserling CH, Barbaras R, Benghozi R, Dasseux JL . Development of CER-001: Preclinical Dose Selection Through to Phase I Clinical Findings . Clinical Drug Investigation . 37 . 5 . 483–491 . May 2017 . 28213743 . 5394142 . 10.1007/s40261-017-0506-3 . 2017-02-17 .

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