First results of the Phase II TITAN trial: anti-von Willebrand ... - Ablynx

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Treatment outcome • Disease duration is variable • The clinical response is usually achieved after 9-16 days of plasma therapy • Mortality reduced to 10-20% with PE, but still high • Mortality is highest in the first days from disease onset • Exacerbation: new clinical signs and symptoms within 30 days after normalisation of platelet count (25-50%) • Relapse: new clinical sign and symptoms more than 30 days after normalisation of platelet count (20-30%) (Rose M et al Am J Med 1987, Rock GA et al NEJM 1991, Bell WR et al NEJM 1991, Onundarson PT et al Ann Intern Med 1992, Thompson CE et al Blood 1992, Hayward CP et al Arch Intern Med 1994, Sarode R et al Am J Hematol 1997, Lara PN Jr et al Am J Hematol 1999, Burns ER et al Am J Hematol 2004, Zheng Xl et al Blood 2004, Scully M et al BJH 2006, Sadler JE Blood 2008) 10
Perspectives • Recombinant ADAMTS13 (Baxter) • Anti VWF-GPIb agents: ─ ARC-1779 aptamer (Archemix - Baxter) ─ ALX-0081 Nanobody (Ablynx) ─ rGPG 290 (Aarvon Biosciences) • Novel anti-CD20 products (LFB, Roche etc) 11
Page 1 and 2: First results of the Phase II TITAN
Page 3 and 4: Clinical symptoms at the first thre
Page 5 and 6: ADAMTS13 • VWF cleaving protease
Page 7 and 8: Treatment of acute TTP • Survival
Page 9: Immunosuppressive agents • Cortic
Page 13 and 14: ALX-0081: mode of action in TTP •
Page 15 and 16: ALX-0081 potently neutralises ULVWF
Page 17 and 18: The TITAN trial: study design Inclu
Page 19 and 20: Nanobodies ® - Inspired by nature
Page 21 and 22: Demographic and baseline characteri
Page 23 and 24: PE details • PE treatment - varia
Page 25 and 26: Preliminary observations: RiCo (n =
Page 27 and 28: Preliminary observations: FVIII aci
Page 29 and 30: Preliminary observations: anti-ADAM
Page 31 and 32: Serious adverse events • 2 SAEs r
Page 33: Acknowledgements • Hemophilia and

First results of the Phase II TITAN trial: anti-von Willebrand ... - Ablynx

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