Macugen (pegaptanib sodium), a novel ocular therapeutic that targets vascular endothelial growth factor (VEGF)

International Ophthalmology Clinics

Volumn 46, Issue 4, 2006, Pages 141-154

  Katz, Barrett ^a   Goldbaum, Mauro ^b  

^a Fovea Pharmaceuticals Inc   (United States)

^b OSI PHARMACEUTICALS INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BENZOPORPHYRIN DERIVATIVE; PEGAPTANIB; VASCULOTROPIN;

CLINICAL TRIAL; DIABETIC MACULAR EDEMA; DISEASE COURSE; DOSE RESPONSE; DRUG BLOOD LEVEL; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; ENDOPHTHALMITIS; EYE DISEASE; HUMAN; LASER COAGULATION; PHOTODYNAMIC THERAPY; PRIORITY JOURNAL; RETINA MACULA AGE RELATED DEGENERATION; RETINA MACULA EDEMA; RETINA NEOVASCULARIZATION; REVIEW; VISUAL ACUITY;

APTAMERS, NUCLEOTIDE; HUMANS; MACULAR DEGENERATION; RETINAL NEOVASCULARIZATION; TREATMENT OUTCOME; VASCULAR ENDOTHELIAL GROWTH FACTOR A;

EID: 33750364435     PISSN: 00208167     EISSN: None     Source Type: Journal    
DOI: 10.1097/01.iio.0000212130.91136.31     Document Type: Review

References (47)

1. Gragoudas ES, Adamis AP, Cunningham ET Jr, et al. Pegaptanib for neovascular age-related macular degeneration. N Engl J Med. 2004;351:2805-2816.
2. D'Amico DJ, Patel M, Adamis AP, et al. Pegaptanib sodium for neovascular age-related macular degeneration: two-year safety results of the two prospective, multicenter, controlled clinical trials. Ophthalmology. 2006;113:992-1001.
3. Cunningham ET Jr, Adamis AP, Altaweel M, et al. A phase II randomized double-masked trial of pegaptanib, an anti-vascular endothelial growth factor aptamer, for diabetic macular edema. Ophthalmology. 2005;112:1747-1757.
4. Adamis AP, Altaweel M, Bressler NM, et al. Changes in retinal neovascularization after pegaptanib (Macugen) therapy in diabetic individuals. Ophthalmology. 2006;113:23-28.
5. OSI (Eyetech) Pharmaceuticals. Preliminary topline efficacy results from phase II data show Macugen(R) (pegaptanib sodium injection) maintained or improved vision in over 90 percent of patients with macular edema associated with central retinal vein occlusion (CRVO). Available at: http://www.pfizer.com/ pfizer/are/news_releases/2006pr/mn_2006_0202.jsp 2006. February 2, 2006. Accessed June 15, 2006.
6. Comer GM, Ciulla TA, Criswell MH, et al. Current and future treatment options for nonexudative and exudative age-related macular degeneration. Drugs Aging. 2004;21:967-992.
7. Ferrara N. Vascular endothelial growth factor: basic science and clinical progress. Endocr Rev. 2004;25:581-611.
8. Robinson CJ, Stringer SE. The splice variants of vascular endothelial growth factor (VEGF) and their receptors. J Cell Sci. 2001;114:853-865.
9. Adamis AP, Shima DT, Yeo KT, et al. Synthesis and secretion of vascular permeability factor/vascular endothelial growth factor by human retinal pigment epithelial cells. Biochem Biophys Res Commun. 1993;193:631-638.
10. Aiello LP, Northrup JM, Keyt BA, et al. Hypoxic regulation of vascular endothelial growth factor in retinal cells. Arch Ophthalmol. 1995;113:1538-1544.
11. Famiglietti EV, Stopa EG, McGookin ED, et al. Immunocytochemical localization of vascular endothelial growth factor in neurons and glial cells of human retina. Brain Res. 2003;969:195-204.
12. Ishida S, Usui T, Yamashiro K, et al. VEGF164 is proinflammatory in the diabetic retina. Invest Ophthalmol Vis Sci. 2003;44:2155-2162.
13. Ishida S, Usui T, Yamashiro K, et al. VEGF164-mediated inflammation is required for pathological, but not physiological, ischemia-induced retinal neovascularization. J Exp Med. 2003;198:483-489.
14. Gardner TW, Antonetti DA, Barber AJ, et al. Diabetic retinopathy: more than meets the eye. Surv Ophthalmol. 2002;47(suppl 2):S253-S262.
15. Leung DW, Cachianes G, Kuang WJ, et al. Vascular endothelial growth factor is a secreted angiogenic mitogen. Science. 1989;246:1306-1309.
16. Senger DR, Connolly DT, Van de Water L, et al. Purification and NH2-terminal amino acid sequence of guinea pig tumor-secreted vascular permeability factor. Cancer Res. 1990;50:1774-1778.
17. Asahara T, Takahashi T, Masuda H, et al. VEGF contributes to postnatal neovascularization by mobilizing bone marrow-derived endothelial progenitor cells. Embo J. 1999;18:3964-3972.
18. Usui T, Ishida S, Yamashiro K, et al. VEGF164(165) as the pathological isoform: differential leukocyte and endothelial responses through VEGFR1 and VEGFR2. Invest Ophthalmol Vis Sci. 2004;45:368-374.
19. Kvanta A, Algvere PV, Berglin L, et al. Subfoveal fibrovascular membranes in age-related macular degeneration express vascular endothelial growth factor. Invest Ophthalmol Vis Sci. 1996;37:1929-1934.
20. Grossniklaus HE, Ling JX, Wallace TM, et al. Macrophage and retinal pigment epithelium expression of angiogenic cytokines in choroidal neovascularization. Mol Vis. 2002;8:119-126.
21. Otani A, Takagi H, Oh H, et al. Vascular endothelial growth factor family and receptor expression in human choroidal neovascular membranes. Microvasc Res. 2002;64:162-169.
22. Matsuoka M, Ogata N, Otsuji T, et al. Expression of pigment epithelium derived factor and vascular endothelial growth factor in choroidal neovascular membranes and polypoidal choroidal vasculopathy. Br J Ophthalmol. 2004;88:809-815.
23. Hera R, Keramidas M, Peoc'h M, et al. Expression of VEGF and angiopoietins in subfoveal membranes from patients with age-related macular degeneration. Am J Ophthalmol. 2005;139:589-596.
24. Kliffen M, Sharma HS, Mooy CM, et al. Increased expression of angiogenic growth factors in age-related maculopathy. Br J Ophthalmol. 1997;81:154-162.
25. Funatsu H, Yamashita H, Ikeda T, et al. Angiotensin II and vascular endothelial growth factor in the vitreous fluid of patients with diabetic macular edema and other retinal disorders. Am J Ophthalmol. 2002;133:537-543.
26. Funatsu H, Yamashita H, Ikeda T, et al. Vitreous levels of interleukin-6 and vascular endothelial growth factor are related to diabetic macular edema. Ophthalmology. 2003;110:1690-1696.
27. Funatsu H, Yamashita H, Sakata K, et al. Vitreous levels of vascular endothelial growth factor and intercellular adhesion molecule 1 are related to diabetic macular edema. Ophthalmology. 2005;112:806-816.
28. Aiello LP, Avery RL, Arrigg PG, et al. Vascular endothelial growth factor in ocular fluid of patients with diabetic retinopathy and other retinal disorders. N Engl J Med. 1994;331:1480-1487.
29. Tripathi RC, Li J, Tripathi BJ, et al. Increased level of vascular endothelial growth factor in aqueous humor of patients with neovascular glaucoma. Ophthalmology. 1998;105:232-237.
30. Lashkari K, Hirose T, Yazdany J, et al. Vascular endothelial growth factor and hepatocyte growth factor levels are differentially elevated in patients with advanced retinopathy of prematurity. Am J Pathol. 2000;156:1337-1344.
31. Qaum T, Xu Q, Joussen AM, et al. VEGF-initiated blood-retinal barrier breakdown in early diabetes. Invest Ophthalmol Vis Sci. 2001;42:2408-2413.
32. Ferrara N, Chen H, Davis-Smyth T, et al. Vascular endothelial growth factor is essential for corpus luteum angiogenesis. Nat Med. 1998;4:336-340.
33. Nissen NN, Polverini PJ, Koch AE, et al. Vascular endothelial growth factor mediates angiogenic activity during the proliferative phase of wound healing. Am J Pathol. 1998;152:1445-1452.
34. Gerber HP, Vu TH, Ryan AM, et al. VEGF couples hypertrophic cartilage remodeling, ossification and angiogenesis during endochondral bone formation. Nat Med. 1999;5:623-628.
35. Storkebaum E, Carmeliet P. VEGF: a critical player in neurodegeneration. J Clin Invest. 2004;113:14-18.
36. Shima DT, Nishijima K, Jo N, et al. VEGF-mediated neuroprotection in ischemic retina. Invest Ophthalmol Vis Sci. 2004;45. E-abstract 3270.
37. Avastin (bevacizumab). United States Food and Drug Administration web site. February 13, 2004. Available at http://www.fda.gov/cder/foi/label 2004/125085lbl.pdf. Accessed June 15, 2006.
38. Tuerk C, Gold L. Systematic evolution of ligands by exponential enrichment: RNA ligands to bacteriophage T4 DNA polymerase. Science. 1990;249:505-510.
39. Ruckman J, Green LS, Beeson J, et al. 20-Fluoropyrimidine RNA-based aptamers to the 165-amino acid form of vascular endothelial growth factor (VEGF165). Inhibition of receptor binding and VEGF-induced vascular permeability through interactions requiring the exon 7-encoded domain. J Biol Chem. 1998;273:20556-20567.
40. Bell C, Lynam E, Landfair DJ, et al. Oligonucleotide NX1838 inhibits VEGF165-mediated cellular responses in vitro. In Vitro Cell Dev Biol Anim. 1999;35:533-542.
41. Eyetech Study Group. Preclinical and phase 1A clinical evaluation of an anti-VEGF pegylated aptamer (EYE001) for the treatment of exudative age-related macular degeneration. Retina. 2002;22:143-152.
42. Eyetech Study Group. Anti-vascular endothelial growth factor therapy for subfoveal choroidal neovascularization secondary to age-related macular degeneration: phase II study results. Ophthalmology. 2003;110:979-986.
43. Ng EW, Adamis AP. Targeting angiogenesis, the underlying disorder in neovascular age-related macular degeneration. Can J Ophthalmol. 2005;40:352-368.
44. Chakravarthy U, Adamis AP, Cunningham ET Jr, et al. Year 2 efficacy results of two randomized, controlled clinical trials of pegaptanib, an anti-VEGF aptamer, for neovascular age-related macular degeneration. Ophthalmology. 2006;113:1508-1521.
45. Gonzales CR. Enhanced efficacy associated with early treatment of neovascular age-related macular degeneration with pegaptanib sodium: an exploratory analysis. Retina. 2005;25:815-827.
46. Jager RD, Aiello LP, Patel SC, et al. Risks of intravitreous injection: a comprehensive review. Retina. 2004;24:676-698.
47. Miyamoto K, Hiroshiba N, Tsujikawa A, et al. In vivo demonstration of increased leukocyte entrapment in retinal microcirculation of diabetic rats. Invest Ophthalmol Vis Sci. 1998;39:2190-2194.