Many of the nonconfidential advances in our premier research and development pipeline are shared through an extensive list of peer-reviewed publications and posters presented at international conferences.Medical and Scientific Publications
Narsoplimab (OMS721) Publications:
Narsoplimab is an investigational product not approved by any regulatory agency.
2024
Successful use of narsoplimab to treat allogeneic transplant-associated thrombotic microangiopathy while maintaining sirolimus
Alhomoud M, Scordo M, Perales MA. Bone Marrow Transplant. Published online March 11, 2024.
2023
IgA nephropathy: the lectin pathway and implications for targeted therapy
Barratt J, Lafayette RA, Zhang H, et al. Kidney International. 2023;104(2):254-264.
Association of urine protein excretion levels, as a measure of disease activity, and quality of life in IgA nephropathy patients
Barratt J, Clayton S, Lowe M, Jackson J, Roughley A, Tsong WH. Nephrology Dialysis Transplant. 2023;38(suppl 1):i1143-i1144.
MASP2 inhibition by narsoplimab suppresses endotheliopathies characteristic of transplant-associated thrombotic microangiopathy: in vitro and ex vivo evidence
Elhadad S, Redmond D, Huang J, Tan A, Laurence J. Clin Exp Immunol. 2023;213(2):252-264.
Inhibition of the lectin pathway of complement activation reduces LPS-induced acute respiratory distress syndrome in mice
Ali YM, Lynch NJ, Shaaban AA, et al. Frontiers in Immunology. 2023;14:1192767.
Compassionate use of narsoplimab in a child with high-risk neuroblastoma and severe HSCT-TMA
Garcia ML, Garvin J, Satwani P, et al. Pediatric Blood & Cancer. 2023;70(suppl 3):e30390.
Narsoplimab for severe transplant-associated thrombotic microangiopathy
Pandrowala A, Ganatra P, Krishnan VP, et al. Thrombosis Journal. 2023;21(1):26.
Hematopoietic stem cell transplantation-associated thrombotic microangiopathy and the role of advanced practice providers and pharmacists
Mahmoudjafari Z, Alencar MC, Alexander M, Johnson DJ, Yeh J, Evans MD. Bone Marrow Transplantation. 2023;58(6):625-634.
Systematic review of signs and symptoms associated with hematopoietic stem cell transplantation-associated thrombotic microangiopathy
Dandoy CE, Tsong WH, Sarikonda K, McGarvey N, Perales M-A. Transplantation and Cellular Therapy. 2023;29(4):282.e1-282.e9.
2022
Trial in progress: An open-label, multi-center phase 2 study evaluating efficacy and safety of the MASP-2 inhibitor narsoplimab in pediatric patients with high-risk hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA)
Pullman W, Brough M, Soto C, Meng T-C. Blood. 2022;140(suppl 1):8493-8494.
Narsoplimab treatment for recurrent IgA nephropathy stabilized eGFR and proteinuria
Storrar J, Rainone F, Middleton R, Barratt J. J Am Soc Nephrol. 2022;33:195.
Use of narsoplimab in a paediatric patient with IgA nephropathy
Oni L, McCormack V, Jones CA, Barratt J. J Am Soc Nephrol. 2022;33:959.
C5b-9 and MASP2 deposition in skin and bone marrow microvasculature characterize hematopoietic stem cell transplant-associated thrombotic microangiopathy
Elhadad S, Chadburn A, Magro C, et al. Bone Marrow Transplant. 2022;57(9):1445-1447.
Premortem skin biopsy assessing microthrombi, interferon type I antiviral and regulatory proteins, and complement deposition correlates with coronavirus disease 2019 clinical stage
Laurence J, Nuovo G, Racine-Brzostek SE, et al. Am J Pathol. 2022;192(9)1282-1294.
Inhibition of the lectin pathway of complement ameliorates hypocomplementemia and restores serum bactericidal activity in patients with severe COVID-19
Lynch NJ, Chan ACY, Ali YM, et al. Clin Transl Med. 2022;12(7):e980.
Narsoplimab, a mannan-binding lectin-associated serine protease-2 inhibitor, for the treatment of adult hematopoietic stem-cell transplantation–associated thrombotic microangiopathy
Khaled SK, Claes K, Goh YT, et al. J Clin Oncol. 2022;40(22):2447-2457.
Secondary complement deficiency impairs anti-microbial immunity to Klebsiella pneumoniae and Staphylococcus aureus during severe acute COVID-19
Ali YM, Lynch NJ, Khatri P, et al. Front Immunol. 2022;13:841759
Compassionate use narsoplimab to treat transplant associated thrombotic microangiopathy in a pediatric patient with multi-organ failure
Schoettler M, Bryson E, Deeb L, et al. Bone Marrow Transplant. 2022;57(suppl 1):350.
Lectin pathway inhibitor for transplant-associated thrombotic microangiopathy
Ganatra P, Kakunje M, Mishra V, Pandrowala A, Hiwarkar P. Bone Marrow Transplant. 2022;57(suppl 1):349.
Trial design: Phase 3 randomized, double-blind, placebo-controlled study of narsoplimab safety and efficacy in IgA nephropathy (ARTEMIS-IGAN)
Lafayette R, Rovin B, Floege J, et al. Kidney Int Rep. 2022;7(2):S57.
2021
Role of the lectin pathway of complement in hematopoietic stem cell transplantation-associated endothelial injury and thrombotic microangiopathy
Gavriilaki E, Ho VT, Schwaeble W, et al. Exp Hematol Oncol. 2021;10:57.
Characterization of narsoplimab, a selective inhibitor of lectin pathway-mediated complement activation and thrombosis
Dudler T, Yaseen S, Freeman J, et al. Mol Immunol. 2021;141:145.
Real world use of narsoplimab in a case of severe HSCT-TMA
Long-term Phase 2 efficacy of the MASP-2 inhibitor narsoplimab for treatment of severe IgA nephropathy
Reduction of urinary levels of lectin pathway complement components in an IgA vasculitis patient after MASP-2 inhibition with narsoplimab
Lectin pathway mediates complement activation by SARS-CoV-2 proteins
Narsoplimab (OMS721) treatment contributes to improvements in organ function in adult patients with high-risk transplant-associated thrombotic microangiopathy
Perales M, Cairo M, Duarte R, et al. HemaSphere. 2021;5(suppl 2):79.
Narsoplimab (OMS721), a MASP-2 inhibitor, for the treatment of adult hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA): Subgroup analyses
Rambaldi A, Claes K, Goh YT, et al. Bone Marrow Transplant. 2021;56:147-149.
Clinical pharmacology and population pharmacokinetic/pharmacodynamic modeling of lectin pathway inhibition by narsoplimab (OMS721)
In acute HSCT/BMT-TMA the activation of the lectin pathway induces C5b-9 formation on endothelial cells and favors microvascular thrombosis
Narsoplimab (OMS721), a MASP-2 inhibitor, for the treatment of adult hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA)
MASP2 levels are elevated in thrombotic microangiopathies: association with microvascular endothelial cell injury and suppression by anti-MASP2 antibody narsoplimab
Elhadad S, Chapin J, Copertino D, Van Besien K, Ahamed J, Laurence J. Clin Exp Immunol. 2021;203(1):96-104.
2020
Development of pharmacodynamic assays to assess ex vivo MASP-2 inhibition and their use to characterize the pharmacodynamics of narsoplimab (OMS721) in humans and monkeys
Endothelial injury and thrombotic microangiopathy in COVID-19: Treatment with the lectin-pathway inhibitor narsoplimab
Safety, tolerability, and effect of narsoplimab (OMS721), a novel MASP-2 inhibitor for the treatment of IgA nephropathy
Inhibition of the lectin pathway of the complement system as a novel approach in the management of IgA vasculitis-associated nephritis
Narsoplimab (OMS721) for the treatment of adult hematopoietic stem cell transplant-associated thrombotic microangiopathy
Rambaldi A, Smith M, Whitaker S, Khaled S. HemaSphere. 2020;4(suppl 1):92.
MASP-2 inhibition as a potential strategy for the management of IgA nephropathy
2019
MASP-2 levels following allogeneic hematopoietic stem cell transplantation in adults: correlation with development of a thrombotic microangiopathy and implications for therapy with anti-complement agents
Elhadad S, Van Biesen K, Chapin J, Ahamed J, Laurence J. Blood. 2019;134(suppl 1):3305.
Absence of the lectin activation pathway of complement ameliorates proteinuria-induced renal injury
Interim results from an ongoing phase 2 study evaluating the use of a MASP-2 inhibitor for the treatment of IgA nephropathy
Barratt J, Leifke E, Whitaker S, DeTulleo L, Lafayette R. Nephrology Dialysis Transplant. 2019;34(suppl 1):111.
2018
Cardioprotection by an anti-MASP-2 antibody in a murine model of myocardial infarction
Clark JE, Dudler T, Marber MS, Schwaeble W. Open Heart. 2018;5(1):e000652.
Improved survival following OMS721 treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HCT-TMA)
Rambaldi A, Khaled S, Smith M, et al. HemaSphere. 2018;2(suppl 1):312-313.
2017
Effective treatment of GvHD-associated transplant-associated microangiopathy with a novel inhibitor of the lectin pathway of complement
Caprioli C, Grassi A, Micò C, Rambaldi A. Presented at: EBMT Transplant Complications Working Party – Crash Course on Diagnosis and Treatment of Noninfectious Complications after HCT; October 19-20, 2017; Granada, Spain.
The effect of OMS721 on proteinuria in patients with IgA nephropathy
Block G, Whitaker S. Nephrology Dialysis Transplant. 2017;32(suppl 3):143.
Maintenance of remission following completion of OMS721 treatment in Patients with IgA nephropathy (IgAN)
Block G, Whitaker S. J Am Soc Nephrol. 2017;28:749-750.
Early results of a phase 2 study using OMS721 in patients with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HCT-TMA)
Khaled SK, Kwong YL, Smith M, Metjian A, Whitaker S. Biol Blood Marrow Transplant. 2017;23:S282-S283.
Dose-finding clinical trial of OMS721 for the treatment of atypical hemolytic uremic syndrome (aHUS) – stage 1 results
Nowicki M, Wiecek A, Massart A, Weekers L, Whitaker S, Miglinas M. Presented at: World Congress of Nephrology (WCN); April 21-25, 2017; Mexico City, Mexico.
Lectin pathway effector enzyme mannan-binding lectin-associated serine protease-2 can activate native complement C3 in absence of C4 and/or C2
Yaseen S, Demopulos G, Dudler T, et al. FASEB J. 2017;31(5):2210-2219.
Resolution of acute kidney injury secondary to TA-TMA by the anti-MASP-2 monoclonal antibody OMS721 in a pediatric HSCT recipient
2016
Mannan binding lectin-associated serine protease-2 (MASP-2) critically contributes to post-ischemic brain injury independent of MASP-1
Orsini F, Chrysanthou E, Dudler T, et al. J Neuroinflammation. 2016;13(1):213.
2014
Mannan-binding lectin-associated serine protease 2 is critical for the development of renal ischemia reperfusion injury and mediates tissue injury in the absence of complement C4
2012
The lectin pathway of complement activation is a critical component of the innate immune response to pneumococcal infection
2011
Targeting of mannan-binding lectin-associated serine protease-2 confers protection from myocardial and gastrointestinal ischemia/reperfusion injury
Schwaeble WJ1, Lynch NJ, Clark JE,et al. Proc Natl Acad Sci USA. 2011;108(18):7523-7528.
2010
Activation of mannan-binding lectin-associated serine proteases leads to generation of a fibrin clot
Gulla KC1, Gupta K, Krarup A, et al. Immunology. 2010;129(4):482-495.
OMS906 Publications:
OMS906 is an investigational product not approved by any regulatory agency.
2023
OMS906, a mannan-binding lectin-associated serine protease-3 (MASP-3) inhibitor, normalizes hemoglobin levels in treatment-naïve PNH patients: Interim data from a proof-of-concept clinical trial
Karnabeda O, Gavriilaki E, Nangia N, Whitaker S, Panse J. HemaSphere. 2023;7(S3):LBA2714.
Alternative pathway MASP-3 inhibitor OMS906: Results from a first-in-man phase 1 study in healthy subjects and study design of two ongoing clinical trials in patients with PNH
Griffin M, Humphreys J, Philpot E, Cummings WJ, Pullman W. HemaSphere. 2023;7(suppl 3):1459-1460.
2022
Safety, tolerability, pharmacokinetics, and pharmacodynamics of the alternative pathway MASP-3 inhibitor OMS906 in a phase 1 study of healthy subjects
Pullman W, Humphreys J, Philpot E, Cummings WJ. Blood. 2022;140(suppl 1):5801-5802.
In vivo inhibition of alternative pathway activity via MASP-3 inhibitor OMS906
Cummings WJ, Li Y, Shaffer K, Freeman J, Yabuki M, Dudler T. Molecular Immunology. 2022;150:145.
MASP-2 and MASP-3 inhibitors block complement activation, inflammation, and microvascular stasis in a murine model of vaso-occlusion in sickle cell disease
Belcher JD, Nguyen J, Chen C, et al. Transl Res. 2022;249:1-12.
Other Publications:
Selective inhibition of phosphodiesterase 7 enzymes reduces motivation for nicotine use through modulation of mesolimbic dopaminergic transmission
Ciccocioppo R, de Guglielmo G, Li H, et al. J Neurosci. 2021 Jun 1;41(28):6128-6143.
PPARγ activation attenuates opioid consumption and modulates mesolimbic dopamine transmission
de Guglielmo G, Melis M, De Luca MA, et al. Neuropsychopharmacology. 2015;40(4):927-937.
Selection of targeted mutants from a library of randomly mutagenized ES cells
Horie K, Gaitanaris G, Gragerov A. Methods Mol Biol. 2011;693:283-294.
An inducible and reversible mouse genetic rescue system
Zeng H, Horie K, Madisen L, et al. PLoS Genet. 2008;4(5):e1000069.
Neuromedin U receptor 2-deficient mice display differential responses in sensory perception, stress, and feeding
Zeng H, Gragerov A, Hohmann JG, et al. Mol Cell Biol. 2006;26(24):9352-9363.
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