Medical and Scientific Publications

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.

Narsoplimab (OMS721) Publications:

Narsoplimab is an investigational product not approved by any regulatory agency.


Systematic review of signs and symptoms associated with hematopoietic stem cell transplant-associated thrombotic microangiopathy

Dandoy CE, Tsong WH, Sarikonda K, McGarvey N, Perales M-A. Transplantation and Cellular Therapy. 2022; in press.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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

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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.

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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.

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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.

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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.

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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.

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Real world use of narsoplimab in a case of severe HSCT-TMA

Pantin JM, Billy A. Blood. 2021;138(suppl 1):4862.
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Long-term Phase 2 efficacy of the MASP-2 inhibitor narsoplimab for treatment of severe IgA nephropathy

Lafayette R, Carroll K, Barratt J. J Am Soc Nephrol. 2021;32:B10.
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Reduction of urinary levels of lectin pathway complement components in an IgA vasculitis patient after MASP-2 inhibition with narsoplimab

Pérez-Alós L, Scionti K, Molyneux K, Barratt J, Garred P. J Am Soc Nephrol. 2021;32:498.
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Lectin pathway mediates complement activation by SARS-CoV-2 proteins

Ali YM, Ferrari M, Lynch NJ, et al. Front Immunol. 2021;12:714511.
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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.

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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.

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Clinical pharmacology and population pharmacokinetic/pharmacodynamic modeling of lectin pathway inhibition by narsoplimab (OMS721)

Pullman W, Facius A, Lahu G, Smyth B. Bone Marrow Transplant. 2021;56:300-301.
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In acute HSCT/BMT-TMA the activation of the lectin pathway induces C5b-9 formation on endothelial cells and favors microvascular thrombosis

Galbusera M, Gastoldi S, Noris M, Bresin E, Benigni A, Remuzzi G. Bone Marrow Transplant. 2021;56:295-297
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Narsoplimab (OMS721), a MASP-2 inhibitor, for the treatment of adult hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA)

Khaled SK, Boelens JJ, Cairo MS, et al. Transplantation and Cellular Therapy. 2021;27(3):S24-S26.
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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.

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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

Freeman J, Cummings J, Chuidian M, Dudler T. Blood. 2020;136(suppl 1):26-27.
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Endothelial injury and thrombotic microangiopathy in COVID-19: Treatment with the lectin-pathway inhibitor narsoplimab

Rambaldi A, Gritti G, Micò MC, et al. Immunobiology. 2020;225(6):152001.
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Safety, tolerability, and effect of narsoplimab (OMS721), a novel MASP-2 inhibitor for the treatment of IgA nephropathy

Lafayette R, Rovin B, Floege J, et al. Kidney Int Rep. 2020;5(11):2032-2041.
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Inhibition of the lectin pathway of the complement system as a novel approach in the management of IgA vasculitis-associated nephritis

Selvaskandan H, Cheung CK, Dormer J, et al. Nephron. 2020;144(9):453-458.
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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.

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MASP-2 inhibition as a potential strategy for the management of IgA nephropathy

Barratt J, Lafayette R. Drugs of the Future. 2020;45(6):389-396.
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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.

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Absence of the lectin activation pathway of complement ameliorates proteinuria-induced renal injury

Alghadban S, Kenawy HI, Dudler T, Schwaeble WJ, Bunskill NJ. Front Immunol. 2019;10:2238.
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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.

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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.

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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.

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The effect of OMS721 on proteinuria in patients with IgA nephropathy

Block G, Whitaker S. Nephrology Dialysis Transplant. 2017;32(suppl 3):143.

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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.

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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.

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.

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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. doi:10.1096/fj.201601306R.

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Resolution of acute kidney injury secondary to TA-TMA by the anti-MASP-2 monoclonal antibody OMS721 in a pediatric HSCT recipient

Zecca M, Comoli P, Mina T, et al. Bone Marrow Transplantation. 2017;52:S124-S516.
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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. doi:10.1186/s12974-016-0684-6.

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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

Asgari E, Farrar CA, Lynch N, et al. FASEB J. 2014;28(9):3996-4003.
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The lectin pathway of complement activation is a critical component of the innate immune response to pneumococcal infection

Ali YM, Lynch NJ, Haleem KS, et al. PLoS Pathog. 2012;8:e1002793.
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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. doi:10.1073/pnas.1101748108.
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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. doi:10.1111/j.1365-2567.2009.03200.x.
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OMS906 Publications:

OMS906 is an investigational product not approved by any regulatory agency.

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.

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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.

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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.

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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. doi:10.1523/JNEUROSCI.3180-20.2021.
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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. doi:10.1038/npp.2014.268.

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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. doi:10.1007/978-1-60761-974-1_17.

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An inducible and reversible mouse genetic rescue system

Zeng H, Horie K, Madisen L, et al. PLoS Genet. 2008;4(5):e1000069. doi:10.1371/journal.pgen.1000069.

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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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