393
Views
1
CrossRef citations to date
0
Altmetric
Review

Investigational drugs in phase I and phase II clinical trials for hereditary angioedema

, &
Pages 87-103 | Received 15 Oct 2017, Accepted 06 Dec 2017, Published online: 15 Dec 2017

References

  • Osler W. Hereditary angio-neurotic Œdema. Am J Med Sci. 1888;95(4):362–367.
  • Donaldson VH, Evans RR. A biochemical abnormality in herediatry angioneurotic edema: absence of serum inhibitor of C’ 1-esterase. PubMed PMID: 14046003. Am J Med. 1963 Jul;35:37–44.
  • Cicardi M, Aberer W, Banerji A, et al. Classification, diagnosis, and approach to treatment for angioedema: consensus report from the Hereditary Angioedema International Working Group. Allergy. 2014 May;69(5):602–616. PubMed PMID: 24673465.
  • Farkas H, Veszeli N, Kajdacsi E, et al. “Nuts and Bolts” of laboratory evaluation of angioedema. Clin Rev Allergy Immunol. 2016 Oct;51(2):140–151. PubMed PMID: 27142368.
  • Rosen FS, Pensky J, Donaldson V, et al. Hereditary angioneurotic edema: two genetic variants. Science. 1965 May 14;148(3672):957–958. PubMed PMID: 14277836.
  • Tosi M, Stoppa-Lyonnet D, Carter P, et al. Molecular defects of the C1-inhibitor gene in hereditary angioedema. Behring Inst Mitt. 1989 Jul;(84):173–179. PubMed PMID: 2572212.
  • Dewald G, Bork K. Missense mutations in the coagulation factor XII (Hageman factor) gene in hereditary angioedema with normal C1 inhibitor. Biochem Biophys Res Commun. 2006 May 19;343(4):1286–1289. PubMed PMID: 16638441. DOI:10.1016/j.bbrc.2006.03.092
  • Bafunno V, Firinu D, D’Apolito M, et al. Mutation of the angiopoietin-1 gene (ANGPT1) associates with a new type of hereditary angioedema. J Allergy Clin Immunol. PubMed PMID: 28601681 Jun 08 2017. 10.1016/j.jaci.2017.05.020.
  • Bork K, Wulff K, Steinmuller-Magin L, et al. Hereditary angioedema with a mutation in the plasminogen gene. Allergy. PubMed PMID: 28795768 Aug 10 2017. 10.1111/all.13270.
  • Csuka D, Veszeli N, Varga L, et al. The role of the complement system in hereditary angioedema. Mol Immunol. 2017 Sep;89:59–68. PubMed PMID: 28595743. DOI:10.1016/j.molimm.2017.05.020
  • Kaplan AP, Joseph K. Complement, kinins, and hereditary angioedema: mechanisms of plasma instability when C1 inhibitor is absent. Clin Rev Allergy Immunol. 2016 Oct;51(2):207–215. PubMed PMID: 27273087. DOI:10.1007/s12016-016-8555-6
  • Nussberger J, Cugno M, Cicardi M, et al. Local bradykinin generation in hereditary angioedema. J Allergy Clin Immunol. 1999 Dec;104(6):1321–1322. PubMed PMID: 10589018.
  • Han ED, MacFarlane RC, Mulligan AN, et al. Increased vascular permeability in C1 inhibitor-deficient mice mediated by the bradykinin type 2 receptor. J Clin Invest. 2002 Apr;109(8):1057–1063. PubMed PMID: 11956243; PubMed Central PMCID: PMC150945. DOI:10.1172/JCI14211
  • Brackertz D, Kueppers F. Hereditary angioneurotic oedema. Lancet. 1973 Sep 22;2(7830):680. PubMed PMID: 4125654.
  • Gelfand JA, Sherins RJ, Alling DW, et al. Treatment of hereditary angioedema with danazol. Reversal of clinical and biochemical abnormalities. N Engl J Med. 1976 Dec 23;295(26):1444–1448. PubMed PMID: 792688. DOI:10.1056/NEJM197612232952602
  • Cicardi M, Agostoni A. Hereditary angioedema. N Engl J Med. 1996 Jun 20;334(25):1666–1667. PubMed PMID: 8628365. DOI:10.1056/NEJM199606203342510
  • Caballero T, Aygoren-Pursun E, Bygum A, et al. The humanistic burden of hereditary angioedema: results from the burden of illness study in Europe. Allergy Asthma Proc. 2014 Jan-Feb;35(1):47–53. PubMed PMID: 24268449. DOI:10.2500/aap.2013.34.3685
  • Pappalardo E, Zingale LC, Cicardi M. Increased expression of C1-inhibitor mRNA in patients with hereditary angioedema treated with danazol. Immunol Lett. 2003 May 01;86(3):271–276. PubMed PMID: 12706530.
  • Drouet C, Desormeaux A, Robillard J, et al. Metallopeptidase activities in hereditary angioedema: effect of androgen prophylaxis on plasma aminopeptidase P. J Allergy Clin Immunol. 2008 Feb;121(2):429–433. PubMed PMID: 18158172; PubMed Central PMCID: PMC4126900. DOI:10.1016/j.jaci.2007.10.048
  • Farkas H, Zotter Z, Csuka D, et al. Short-term prophylaxis in hereditary angioedema due to deficiency of the C1-inhibitor–a long-term survey. Allergy. 2012 Dec;67(12):1586–1593. PubMed PMID: 23025435. DOI:10.1111/all.12032
  • Cicardi M, Bergamaschini L, Cugno M, et al. Long-term treatment of hereditary angioedema with attenuated androgens: a survey of a 13-year experience. J Allergy Clin Immunol. 1991 Apr;87(4):768–773. PubMed PMID: 2013670.
  • Bork K, Bygum A, Hardt J. Benefits and risks of danazol in hereditary angioedema: a long-term survey of 118 patients. Ann Allergy Asthma Immunol. 2008 Feb;100(2):153–161. PubMed PMID: 18320917. DOI:10.1016/S1081-1206(10)60424-3
  • Fust G, Farkas H, Csuka D, et al. Long-term efficacy of danazol treatment in hereditary angioedema. Eur J Clin Invest. 2011 Mar;41(3):256–262. PubMed PMID: 20955212. DOI:10.1111/j.1365-2362.2010.02402.x
  • Farkas H, Martinez-Saguer I, Bork K, et al. International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency. Allergy. 2017 Feb;72(2):300–313. PubMed PMID: 27503784; PubMed Central PMCID: PMC5248622.
  • Caballero T, Farkas H, Bouillet L, et al. International consensus and practical guidelines on the gynecologic and obstetric management of female patients with hereditary angioedema caused by C1 inhibitor deficiency. J Allergy Clin Immunol. 2012 Feb;129(2):308–320. PubMed PMID: 22197274.
  • Frank MM, Sergent JS, Kane MA, et al. Epsilon aminocaproic acid therapy of hereditary angioneurotic edema. A double-blind study. N Engl J Med. 1972 Apr 13;286(15):808–812. PubMed PMID: 4551861. DOI:10.1056/NEJM197204132861503
  • Kleniewski J, Blankenship DT, Cardin AD, et al. Mechanism of enhanced kinin release from high molecular weight kininogen by plasma kallikrein after its exposure to plasmin. J Lab Clin Med. 1992 Jul;120(1):129–139. PubMed PMID: 1535355.
  • Van Geffen M, Cugno M, Lap P, et al. Alterations of coagulation and fibrinolysis in patients with angioedema due to C1-inhibitor deficiency. Clin Exp Immunol. 2012 Mar;167(3):472–478. PubMed PMID: 22288590; PubMed Central PMCID: PMC3374279. DOI:10.1111/j.1365-2249.2011.04541.x
  • Zanichelli A, Vacchini R, Badini M, et al. Standard care impact on angioedema because of hereditary C1 inhibitor deficiency: a 21-month prospective study in a cohort of 103 patients. Allergy. 2011 Feb;66(2):192–196. PubMed PMID: 21039598. DOI:10.1111/j.1398-9995.2010.02433.x
  • Saule C, Boccon-Gibod I, Fain O, et al. Benefits of progestin contraception in non-allergic angioedema. Clin Exp Allergy. 2013 Apr;43(4):475–482. PubMed PMID: 23517043. DOI:10.1111/cea.12055
  • Farkas H, Jakab L, Temesszentandrasi G, et al. Hereditary angioedema: a decade of human C1-inhibitor concentrate therapy. J Allergy Clin Immunol. 2007 Oct;120(4):941–947. PubMed PMID: 17761272. DOI:10.1016/j.jaci.2007.06.026
  • Berinert® [C1 Esterase Inhibitor (Human)]. For intravenous use. Freeze-dried powder for reconstitution. Initial U.S. Approval: 2009. FULL PRESCRIBING INFORMATION, 2016 cited 2017 Nov 16. Available from: http://labeling.cslbehring.com/PI/US/Berinert/EN/Berinert-Prescribing-Information.pdf.
  • Craig T, Shapiro R, Vegh A, et al. Efficacy and safety of an intravenous C1-inhibitor concentrate for long-term prophylaxis in hereditary angioedema. Allergy Rhinol (Providence). 2017 Mar 01;8(1):13–19. PubMed PMID: 28381322; PubMed Central PMCID: PMC5380447. DOI:10.2500/ar.2017.8.0192
  • Longhurst H, Cicardi M, Craig T, et al. Prevention of hereditary angioedema attacks with a subcutaneous C1 inhibitor. N Engl J Med. 2017 Mar 23;376(12):1131–1140. PubMed PMID: 28328347. DOI:10.1056/NEJMoa1613627
  • HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human]) For Subcutaneous Injection, Freeze-Dried Powder for Reconstitution. Initial U.S. Approval: 2017. FULL PRESCRIBING INFORMATION, 2017 cited 2017 Nov 16. Available from: https://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM564335.pdf.
  • CINRYZE® (C1 Esterase Inhibitor [Human]) for intravenous use, freeze -dried powder for reconstitution. Initial U.S. Approval: 2008. FULL PRECSRIBING INFORMATION, 2016 cited 2017 Nov 16. Available from: http://pi.shirecontent.com/PI/PDFs/Cinryze_USA_ENG.pdf.
  • Moldovan D, Bernstein JA, Cicardi M. Recombinant replacement therapy for hereditary angioedema due to C1 inhibitor deficiency. PubMed PMID: 26250409 Immunotherapy. 2015;77:739–752. DOI:10.2217/imt.15.44
  • RUCONEST® (C1 esterase inhibitor [recombinant]) for intravenous use, lyophilized powder for reconstitution. FULL PRESCRIBING INFORMATION; cited 2017 Oct 15. Available from: https://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM405634.pdf.
  • Prematta M, Gibbs JG, Pratt EL, et al. Fresh frozen plasma for the treatment of hereditary angioedema. Ann Allergy Asthma Immunol. 2007 Apr;98(4):383–388. PubMed PMID: 17458436. DOI:10.1016/S1081-1206(10)60886-1
  • Bowen T, Cicardi M, Farkas H, et al. 2010 International consensus algorithm for the diagnosis, therapy and management of hereditary angioedema. Allergy Asthma Clin Immunol. 2010 Jul 28;6(1):24. PubMed PMID: 20667127; PubMed Central PMCID: PMC2921362. DOI:10.1186/1710-1492-6-24
  • Cicardi M, Bork K, Caballero T, et al. Evidence-based recommendations for the therapeutic management of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an international working group. Allergy. 2012 Feb;67(2):147–157. PubMed PMID: 22126399.
  • Craig T, Aygoren-Pursun E, Bork K, et al. WAO guideline for the management of hereditary angioedema. World Allergy Organ J. 2012 Dec;5(12):182–199. PubMed PMID: 23282420; PubMed Central PMCID: PMC3651186.
  • FIRAZYR® (icatibant) injection, for subcutaneous use. Initial U.S. Approval: 2011. FULL PRESCRIBING INFORMATION; 2015 Dec cited 2017 Oct 15. Available from: http://pi.shirecontent.com/PI/PDFs/Firazyr_USA_ENG.pdf.
  • KALBITOR® (ecallantide) injection, for subcutaneous use. Initial U.S. Approval: 2009. FULL PRESCRIBING INFORMATION, 2015 cited 2017 Nov 16. Available from: http://www.shirecontent.com/PI/PDFs/Kalbitor_USA_ENG.pdf.
  • Duffey H, Firszt R. Management of acute attacks of hereditary angioedema: role of ecallantide. J Blood Med. 2015;6:115–123. PubMed PMID: 25931832; PubMed Central PMCID: PMC4404974. DOI:10.2147/JBM.S66825
  • Lead programs of adverum biotechnologies, 2017 cited 2017 Oct 15. Available from: http://adverum.com/science/.
  • Overview of KalVista’s drug discovery; 2017 cited 2017 Oct 15. Available from: http://www.kalvista.com/discovery-overview.html.
  • Interim results of the drug development program of Verseon Corporation; 2017 Sep 25 cited 2017 Oct 2017 15. Available from: http://www.verseon.com/media-1/news/154-interim-results-2017.
  • Farkas H, Reshef A, Aberer W, et al. Treatment effect and safety of icatibant in pediatric patients with hereditary angioedema. J Allergy Clin Immunol Pract. PubMed PMID: 28601641 Jun 07 2017. 10.1016/j.jaip.2017.04.010.
  • van Veen HA, Koiter J, Vogelezang CJ, et al. Characterization of recombinant human C1 inhibitor secreted in milk of transgenic rabbits. J Biotechnol. 2012 Dec 31;162(2–3):319–326. PubMed PMID: 22995741. DOI:10.1016/j.jbiotec.2012.09.005
  • Varga L, Farkas H. rhC1INH: a new drug for the treatment of attacks in hereditary angioedema caused by C1-inhibitor deficiency. Expert Rev Clin Immunol. 2011 Mar;7(2):143–153. PubMed PMID: 21426252. DOI:10.1586/eci.11.5
  • van Doorn MB, Burggraaf J, van Dam T, et al. A phase I study of recombinant human C1 inhibitor in asymptomatic patients with hereditary angioedema. J Allergy Clin Immunol. 2005 Oct;116(4):876–883. PubMed PMID: 16210064. DOI:10.1016/j.jaci.2005.05.019
  • Baker JW, Reshef A, Moldovan D, et al. Recombinant human C1-esterase inhibitor to treat acute hereditary angioedema attacks in adolescents. J Allergy Clin Immunol Pract. 2017 Jul - Aug;5(4):1091–1097. PubMed PMID: 28202404. DOI:10.1016/j.jaip.2016.11.005
  • Farrell C, Hayes S, Relan A, et al. Population pharmacokinetics of recombinant human C1 inhibitor in patients with hereditary angioedema. Br J Clin Pharmacol. 2013 Dec;76(6):897–907. PubMed PMID: 23594263; PubMed Central PMCID: PMC3845313. DOI:10.1111/bcp.12132
  • Reshef A, Grivcheva-Panovska V, Kivity S, et al. Safety and efficacy of rhC1-INH for the treatment of HAE attacks in pediatric patients. Annual Scientific Meeting of American College of Allergy, Asthma & Immunology; 2015 Nov 5-9; San Antonio, TX: Ann Allergy Asthma Immunol; 2015.
  • Bernstein JA, Relan A, Harper JR, et al. Sustained response of recombinant human C1 esterase inhibitor for acute treatment of hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2017 Apr;118(4):452–455. PubMed PMID: 28284978. DOI:10.1016/j.anai.2017.01.029
  • Pharming announces positive data from paediatric clinical trial with RUCONEST® - Press release; 2017 Oct 2 cited 2017 Oct 15. Available from: https://www.pharming.com/pharming-announces-positive-data-from-paediatric-clinical-trial-with-ruconest/.
  • Riedl MA, Grivcheva-Panovska V, Moldovan D, et al. Recombinant human C1 esterase inhibitor for prophylaxis of hereditary angio-oedema: a phase 2, multicentre, randomised, double-blind, placebo-controlled crossover trial. Lancet. 2017 Sep 30;390(10102):1595–1602. PubMed PMID: 28754491. DOI:10.1016/S0140-6736(17)31963-3
  • Hack CE, Relan A, Van Amersfoort ES, et al. Target levels of functional C1-inhibitor in hereditary angioedema. Allergy. 2012;67(1):123–130.
  • CSL™ - R&D briefing presentation; 2014 Dec 3 cited 2017 Oct 15. Available from: http://www.csl.com.au/docs/805/134/Investor%20Briefing%20Presentation%20FINAL%202014.pdf.
  • Cao H, Biondo M, Rayzman V, et al. Development and characterization of an anti-FXIIa monoclonal antibody for the treatment of hereditary angioedema. American Academy of Allergy, Asthma, and Immunology, annual meeting; 2015 Feb 20-24; Houston, TX: J Allergy Clin Immunol; 2015. J Allergy Clin Immunol. 2015;135:AB194.
  • Roberts A, Panousis C, Green T, et al. FXIIa-mediated kallikrein activity discriminates between HAE and normal plasma samples and is a pharmacodynamic marker for CSL312. 10th C1-inhibitor deficiency workshop; 2017 May 18-21; Budapest, Hungary: Allergy, Asthma & Clinical Immunology; 2017.
  • Liu J, Qin J, Cooley B, et al. Reduction of hepatic factor XII expression in mice by ALN-F12 inhibits thrombosis without increasing bleeding risk. XXVI ISTH Congress and 63rd Annual SSC Meeting; 2017 Jul 8- 13;Berlin, Germany: Research and Practice in Thrombosis and Haemostasis.;2017
  • Liu J, Qin J, Cooley B, et al. Reduction of hepatic factor XII expression in mice by ALN-F12 inhibits thrombosis without increasing bleeding risk - presentation held at ISTH 2017; cited 2017 Oct 15. Available from: http://www.alnylam.com/wp-content/uploads/2017/07/ALN-F12-ISTH2017-Presentation-Final.pdf.
  • Akinc A, Liu J, Qin J, et al. An investigational RNAi therapeutic targeting factor XII (ALN-F12) for the treatment of hereditary angioedema. American Academy of Allergy, Asthma, and Immunology Annual Meeting; 2016 Mar 4-7; Los Angeles, CA: J Allergy Clin Immunol; 2016.
  • Bhattacharjee G, Revenko AS, Crosby JR, et al. Inhibition of vascular permeability by antisense-mediated inhibition of plasma kallikrein and coagulation factor 12. Nucleic Acid Ther. 2013 Jun;23(3):175–187. PubMed PMID: 23582057.
  • Pipeline of preclinical and clinical studies by ionis pharmaceuticals; 2017 cited 2017 Oct 15. Available from: http://www.ionispharma.com/pipeline/.
  • Lehmann A. Ecallantide (DX-88), a plasma kallikrein inhibitor for the treatment of hereditary angioedema and the prevention of blood loss in on-pump cardiothoracic surgery. Expert Opin Biol Ther. 2008 Aug 8;8:1187–1199. DOI:10.1517/14712598.8.8.1187
  • Farkas H, Varga L. Ecallantide is a novel treatment for attacks of hereditary angioedema due to C1 inhibitor deficiency. Clin Cosmet Investig Dermatol. 2011;4:61–68. PubMed PMID: 21760740; PubMed Central PMCID: PMC3133501. DOI:10.2147/CCID.S10322
  • Advisory committee briefing document, Kalbitor® (ecallantide) for acute attacks of hereditary angioedema (BLA 125277) Jan 02, 2009 cited 2017 Nov 16. Available from: https://www.fda.gov/ohrms/dockets/ac/09/briefing/2009-4413b1-03-Dyax.pdf.
  • Cicardi M, Levy RJ, McNeil DL, et al. Ecallantide for the treatment of acute attacks in hereditary angioedema. N Engl J Med. 2010 Aug 05;363(6):523–531. PubMed PMID: 20818887. DOI:10.1056/NEJMoa0905079
  • Levy RJ, Lumry WR, McNeil DL, et al. EDEMA4: a phase 3, double-blind study of subcutaneous ecallantide treatment for acute attacks of hereditary angioedema. Ann Allergy Asthma Immunol. 2010 Jun;104(6):523–529. PubMed PMID: 20568386. DOI:10.1016/j.anai.2010.04.012
  • Lumry WR, Bernstein JA, Li HH, et al. Efficacy and safety of ecallantide in treatment of recurrent attacks of hereditary angioedema: open-label continuation study. Allergy Asthma Proc. 2013 Mar-Apr;34(2):155–161. PubMed PMID: 23484891. DOI:10.2500/aap.2013.34.3653
  • MacGinnitie AJ, Davis-Lorton M, Stolz LE, et al. Use of ecallantide in pediatric hereditary angioedema. Pediatrics. 2013 Aug;132(2):e490–7. PubMed PMID: 23878046.
  • Bernstein JA, Qazi M. Ecallantide: its pharmacology, pharmacokinetics, clinical efficacy and tolerability. Expert Rev Clin Immunol. 2010 Jan;6(1):29–39. PubMed PMID: 20383888.
  • Kenniston JA, Faucette RR, Martik D, et al. Inhibition of plasma kallikrein by a highly specific active site blocking antibody. J Biol Chem. 2014 Aug 22;289(34):23596–23608. PubMed PMID: 24970892; PubMed Central PMCID: PMC4156074. DOI:10.1074/jbc.M114.569061
  • Chyung Y, Vince B, Iarrobino R, et al. A phase 1 study investigating DX-2930 in healthy subjects. Ann Allergy Asthma Immunol. 2014 Oct;113(4):460–6 e2. PubMed PMID: 24980392. DOI:10.1016/j.anai.2014.05.028
  • Banerji A, Busse P, Shennak M, et al. Inhibiting plasma kallikrein for hereditary angioedema prophylaxis. N Engl J Med. 2017 Feb 23;376(8):717–728. PubMed PMID: 28225674. DOI:10.1056/NEJMoa1605767
  • EU clinical trial register - HELP study™: a multicenter, randomized, double-blind, placebo-controlled efficacy and safety study to evaluate DX 2930 for long-term prophylaxis against acute attacks of hereditary angioedema (HAE), [ cited 2017 Nov 16]. Available from: https://www.clinicaltrialsregister.eu/ctr-search/search?query=2015-003943-20.
  • ClinicalTrials.gov - efficacy and safety study of DX-2930 to prevent acute angioedema attacks in patients with type I and type II HAE. 2017 cited 2017 Nov 16. Available from: https://clinicaltrials.gov/ct2/show/NCT02586805.
  • Riedl MA, Bernstein JA, Craig T, et al. An open-label study to evaluate the long-term safety and efficacy of lanadelumab for prevention of attacks in hereditary angioedema: design of the HELP study extension. Clin Transl Allergy. 2017;7:36. PubMed PMID: 29043014; PubMed Central PMCID: PMCPMC5629784. DOI:10.1186/s13601-017-0172-9
  • EU clinical trials register - HELP study extension™: an open-label study to evaluate the long-term safety and efficacy of DX-2930 for prevention against acute attacks of hereditary angioedema (HAE), cited 2017 Nov 16. Available from: https://www.clinicaltrialsregister.eu/ctr-search/search?query=2015-005255-27.
  • Cornpropst M, Dobo S, Collier J, et al. BCX7353, a potent inhibitor of plasma kallikrein, shows sustained maximal enzyme inhibition when dosed orally once daily: results from a phase I trial in healthy subjects. American Academy of Allergy, Asthma, and Immunology, Annual Meeting; 2016 Mar 4-7; Los Angeles, CA: J Allergy Clin Immunol; 2016.
  • Cornpropst M, Dobo S, Collier J BCX7353, a potent inhibitor of plasma kallikrein, shows sustained maximal enzyme inhibition when dosed orally once daily: results from a phase I trial in healthy subjects - full version of poster presented at AAAAI 2016; cited 2017 Oct 15. Available from: http://www.biocryst.com/application/files/3014/7709/2261/AAAAI2016_BCX7353-101Poster.pdf.
  • Avoralstat – oPuS-2 Results; 2016 Feb 8 cited 2017 Nov 16. Available from: http://files.shareholder.com/downloads/BCRX/5336066312x0x873686/8A78C027-983C-4219-A5F8-2B413B0D9C16/Avoralstat_OPuS-2_results_slides_8Feb16_FINAL.pdf.
  • Chen X, Kotian P, Wilson R, et al. Preclinical characterization of BCX7353, an oral plasma kallikrein inhibitor, for the treatment of hereditary angioedema (HAE). American Academy of Allergy, Asthma, and Immunology, Annual Meeting; 2017 Mar 3-6; Atlanta, GA: J Allergy Clin Immunol; 2017.
  • Chen X, Kotian P, Wilson R, et al. Preclinical Characterization of BCX7353, an Oral Plasma Kallikrein Inhibitor, for the Treatment of Hereditary Angioedema - Full version of a poster presentation held at the Annual Meeting of American Academy of Allergy, Asthma, and Immunology, 2017 Mar 3-6 cited 2017 Oct 15. Available from: http://www.biocryst.com/application/files/4014/8882/4138/BCX7353-AAAAI-2017_final-v2.pdf.
  • BCX7353 – aPeX-1, final analysis results, 2017 Sep 5 cited 2017 Oct 15. Available from: http://files.shareholder.com/downloads/BCRX/5336066312x0x955610/9932121A-876F-4F1E-A4C6-C4173A296D63/APeX-1_Final_Analysis_Results.pdf.
  • Kalfus I, McDonald A, Qian S Safety study of ATN-249, a new oral kallikrein inhibitor for hereditary angioedema. 10th C1-inhibitor deficiency workshop; 2017 May 18-21; Budapest, Hungary: Allergy, Asthma & Clinical Immunology; 2017.
  • Kalfus I, McDonald A, Qian S Safety Study of ATN-249, A New Oral Kallikrein Inhibitor for Hereditary Angioedema - Full version of a presentation, held at the 10th C1-inhibitor deficiency workshop; 2017 May 18-21 cited 2017 Oct 15. Available from: http://attunepharma.com/assets/C1INH_Deficiency_Workshop_Oral_Presentation_20170521.pdf.
  • Kalfus I, McDonald A, Qian S Potency, selectivity, and exposure evaluation of ATN-249, a new oral kallikrein inhibitor for hereditary angioedema. American Academy of Allergy, Asthma, and Immunology, Annual Meeting; 2017 Mar 3-6; Atlanta, GA: J Allergy Clin Immunol; 2017.
  • Longhurst HJ, Farkas H, Craig T, et al. HAE international home therapy consensus document. Allergy Asthma Clin Immunol. 2010 Jul 28;6(1):22. PubMed PMID: 20667125; PubMed Central PMCID: PMC2922091. DOI:10.1186/1710-1492-6-22
  • Schmaier AH, Cicardi M, Reshef A, et al. Abstracts from the 10th C1-inhibitor deficiency workshop. Allergy, Asthma Clin Immunol. 2017;13(S2). DOI:10.1186/s13223-017-0198-5

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.