New Drugs in the Pipeline: From Concept to Clinic

Rebecca Kruse-Jarres, Jean St. Louis, Anne Greist, Amy D. Shapiro, Hedy Smith, Pratima Chowdary, Anja B Drebes, Edward D. Gomperts, Miranda Chapman, Min Mo, Aaron Novack, Heinrich D. Farin, Treatment Of Serious Bleeds With a B-Domain Deleted Recombinant Porcine Sequence Factor VIII (OBI-1) In Patients With Acquired Hemophilia A: A Prospective Clinical Trial Blood. ,vol. 122, pp. 206- 206 ,(2013) , 10.1182/BLOOD.V122.21.206.206

Erich V De Paula, Kaan Kavakli, Johnny Mahlangu, Yasmin Ayob, Steven R Lentz, Massimo Morfini, Laszlo Nemes, Silva Z Šalek, Midori Shima, Jerzy Windyga, Silke Ehrenforth, Ampaiwan Chuansumrit, 1804 (ADEPTTM1) INVESTIGATORS, None, Recombinant factor VIIa analog (vatreptacog alfa [activated]) for treatment of joint bleeds in hemophilia patients with inhibitors: a randomized controlled trial. Journal of Thrombosis and Haemostasis. ,vol. 10, pp. 81- 89 ,(2012) , 10.1111/J.1538-7836.2011.04549.X

L. A. VALENTINO, V. MAMONOV, A. HELLMANN, D. V. QUON, A. CHYBICKA, P. SCHROTH, L. PATRONE, W.‐Y. WONG, , A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management Journal of Thrombosis and Haemostasis. ,vol. 10, pp. 359- 367 ,(2012) , 10.1111/J.1538-7836.2011.04611.X

J. N. MAHLANGU, M. J. COETZEE, M. LAFFAN, J. WINDYGA, T. T. YEE, J. SCHROEDER, J. HAANING, J. E. SIEGEL, G. LEMM, Phase I, randomized, double-blind, placebo-controlled, single-dose escalation study of the recombinant factor VIIa variant BAY 86-6150 in hemophilia. Journal of Thrombosis and Haemostasis. ,vol. 10, pp. 773- 780 ,(2012) , 10.1111/J.1538-7836.2012.04667.X

J. SPIRA, O. PLYUSHCH, N. ZOZULYA, R. YATUV, I. DAYAN, A. BLEICHER, M. ROBINSON, M. BARU, Safety, pharmacokinetics and efficacy of factor VIIa formulated with PEGylated liposomes in haemophilia A patients with inhibitors to factor VIII – an open label, exploratory, cross‐over, phase I/II study Haemophilia. ,vol. 16, pp. 910- 918 ,(2010) , 10.1111/J.1365-2516.2010.02273.X

Moshe Baru, The use of PEGylated liposomes in the development of drug delivery applications for the treatment of hemophilia International Journal of Nanomedicine. ,vol. 5, pp. 581- 591 ,(2010) , 10.2147/IJN.S8603

Donna Dimichele, None, Inhibitors: resolving diagnostic and therapeutic dilemmas. Haemophilia. ,vol. 8, pp. 280- 287 ,(2002) , 10.1046/J.1365-2516.2002.00626.X

Jerry S. Powell, Neil C. Josephson, Doris Quon, Margaret V. Ragni, Gregory Cheng, Ella Li, Haiyan Jiang, Lian Li, Jennifer A. Dumont, Jaya Goyal, Xin Zhang, Jurg Sommer, Justin McCue, Margaret Barbetti, Alvin Luk, Glenn F. Pierce, Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients Blood. ,vol. 119, pp. 3031- 3037 ,(2012) , 10.1182/BLOOD-2011-09-382846

Uri Martinowitz, Aaron Lubetsky, Phase I/II, open-label, multicenter, safety, efficacy and PK study of a recombinant coagulation factor IX albumin fusion protein (rIX-FP) in subjects with hemophilia B Thrombosis Research. ,vol. 131, ,(2013) , 10.1016/S0049-3848(13)70152-X

Alessandro Gringeri, Lorenzo G. Mantovani, Luciana Scalone, Pier Mannuccio Mannucci, , Cost of care and quality of life for patients with hemophilia complicated by inhibitors: The COCIS study group Blood. ,vol. 102, pp. 2358- 2363 ,(2003) , 10.1182/BLOOD-2003-03-0941