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

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Clinical studies with ALX-0081 related to TTP indication Phase Population n Regimen/dose Status I Healthy volunteers I Healthy volunteers II Patients with acquired TTP • Clinical status 40 36 110 – Phase I trials successfully completed, up to 14d treatment well tolerated, no immunogenicity (limited exposure), mild bleeding and bruising, partial decrease of VWF:Ag and FVIII activity levels Single dose i.v. infusion 0.5-12 mg Single and multiple dose s.c. 2–16 mg and 10 mg (7-14 d) Multiple doses i.v. and s.c. 10-20 mg/d as adjunct to PE – Phase II (acquired TTP) primary endpoint: time-to-platelet recovery Completed – final study report Completed - final study report Ongoing 16
The TITAN trial: study design Inclusion criteria • men and women • 18 years or older • clinical diagnosis of TTP, necessitating PE Exclusion criteria • platelet count ≥ 100,000/µL • severe active infection indicated by sepsis • infection with E. coli 0157 or related organism • anti-phospholipid syndrome, DIC or congenital TTP • pregnancy or breast-feeding • active bleeding or high risk of bleeding • uncontrolled arterial hypertension • chronic anticoagulant treatment • bone marrow carcinosis • severe liver impairment Randomisation 1:1 n = 110 PE PE Primary endpoint time-to-response, Placebo anti-VWF Nanobody defined by recovery of platelets ≥ 150,000/µL and confirmation at 48 h by de novo measure of platelet count ≥ 150,000/µL and LDH ≤ 2 x ULN 30 days 30 days 1 year follow-up 1 year follow-up Secondary endpoints • PE frequency and volume • relapse • exacerbations • mortality • major clinical events • recovery from signs and symptoms 17
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 and 10: Immunosuppressive agents • Cortic
Page 11 and 12: Perspectives • Recombinant ADAMTS
Page 13 and 14: ALX-0081: mode of action in TTP •
Page 15: ALX-0081 potently neutralises ULVWF
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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