Program badawczy leku Hemlibra i wkład polskich ośrodków w jego rozwój

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Program badawczy leku Hemlibra i wkład polskich ośrodków w jego rozwój Maria Podolak-Dawidziak Warszawa 22 23.

1 Program badawczy leku Hemlibra i wkład polskich ośrodków w jego rozwój Maria Podolak-Dawidziak Warszawa Katedra i Klinika Hematologii, Nowotworów Krwi i Transplantacji Szpiku Uniwersytet Medyczny we Wrocławiu

2 Emicizumab budowa, czynność, zastosowanie u chorych na hemofilię A 2

3 Nowe podejście do leczenia hemofilii z inhibitorami Ragni MV: Novel alternate hemostatic agents for patients with inhibitors: beyond bypass therapy Hematology ASH 2017:

4 Cz. VIIIa vs emicizumab charakterystyka i wpływ na generację cz. Xa Lenting PJ, Denis CV, Christophe OD: Emicizumab, a biospecific antibody recognizing coagulation factors IX and X: how does it actually compare to factor VIII? Blood 2017; 130:

5 Emicizumab: Clinical Development Plan Non-interventional study (BH29768) Inhibitor, non-inhibitor, pediatrics 221 pts enrolled HAVEN 1 (BH29884): Phase III inhibitor adults/adolescents ( 12 years old) Weekly dosing 113 pts enrolled HAVEN 2 (BH29992): Phase III inhibitor children (0 11 years old) Weekly, every 2 and every 4 weeks dosing 88 pts enrolled HAVEN 3 (BH30071): Phase III non-inhibitor adults/adolescents ( 12 years old) Weekly and every other week dosing 152 pts enrolled HAVEN 4 (BO39182): Phase III non-inhibitor/inhibitor adults/adolescents Every 4 weeks dosing 48 pts enrolled 5

6 Emicizumab clinical trials HAVEN 1 (NCT ) HAVEN 2 (NCT ) HAVEN 3 (NCT ) HAVEN 4 (NCT ) Population PwHA 12 years with FVIII inhibitors PwHA <12 years with FVIII inhibitors PwHA 12 years without FVIII inhibitors PwHA 12 years with or without FVIII inhibitors Key outcome Statistically significant reduction in treated bleeds with QW (87%) emicizumab prophylaxis vs no prophylaxis 1 63% of patients on QW emicizumab prophylaxis vs 6% on no prophylaxis had zero treated bleeds 79% reduction in bleeding rate with QW emicizumab vs previous BPA prophylaxis 87% of children who received QW emicizumab prophylaxis experienced zero treated bleeds 2 99% reduction in bleeding rate with QW emicizumab vs previous BPA prophylaxis Statistically significant reduction in treated bleeds with QW (96%) and Q2W (97%) emicizumab prophylaxis vs no prophylaxis 3,4 56% patients on QW and 60% patients on Q2W emicizumab prophylaxis vs 0% on no prophylaxis had zero treated bleeds 68% reduction in bleeding rate with QW emicizumab vs previous FVIII prophylaxis Primary analyses evaluating emicizumab prophylaxis on bleed rate, safety, PK 1. Oldenburg J, et al. N Engl J Med 2017;377(9): Young G, et al. Blood 2017;130:Abstract Genentech Press Release Nov 19, WFH 2018 HAVEN 3 citation TBC FVIII, factor VIII; PK, pharmacokinetics; PwHA, persons with haemophilia A; Q2W, every 2 weeks; Q4W, every 4 weeks; QW, once weekly. 6

7 Program badań klinicznych z emicizumabem w Polsce 7

8 Emicizumab badania kliniczne w Polsce Hemofilia A z inhibitorem 10 (HAVEN 1) + 1 (HAVEN 3) + 3 ( STACEY), n =14 Hemofilia A bez inhibitora 19-1 (nabył inhibitor), n = 18 Dane HAVEN 1 (BH29884) n = 10 STACEY (M039129) n = 3 HAVEN 3 (BH30071) n = 13 ( 1 z inhibitorem) czyli n = 12 HAVEN 4 (bez & z inhibitorem) (B039182) n = 6 Główni badacze / Ośrodki Prof. J. Windyga, Warszawa 4 Prof. M. Hus, Lublin 4 Prof. A. Hellmann, Gdańsk 1 Dr T. Woźny, Poznań 1 Prof. J. Windyga, Warszawa 3 Prof. J. Windyga, Warszawa 4 Prof. M. Hus, Lublin 4 Prof. A. Hellmann, Gdańsk 2 (u 1 inhibitor, nadal w badaniu) Dr T. Woźny, Poznań 3 Prof. M. Podolak- Dawidziak, Wrocław 5 Prof. J. Windyga, Warszawa 1 Data aktywacji pierwszego ośrodka Data włączenia pierwszego pacjenta Data włączenia ostatniego pacjenta

9 Program badań klinicznych z emicizumabem w Polsce Study Sites Investigators BH29768 (non-interventional) HAVEN1 (BH29884; inhibitor) HAVEN3 (BH30071; non-inhibitor) Warsaw Institute of Hematology and Tranfusion Medicine Gdańsk Department of Hematology and Transplantology Lublin Department of Hematology and Transplantology Poznań Department of Hematology J. Windyga, A. Buczma, E. Stefańska-Windyga A. Hellman, A. Mital, E. Zarzycka, M. Dudka M. Hus, J. Muzyka-Kasiedczuk, J. Kozińska T. Wożny, J. Malendowicz-Portala HAVEN4 (BO39182; q4w) STACEY (MO39129) Warsaw Institute of Hematology and Tranfusion Medicine Wrocław Department of Hematology and Transplantology Warsaw Institute of Hematology and Tranfusion Medicine J. Windyga, A. Buczma, E. Stefańska-Windyga M. Podolak-Dawidziak, K. Kuliczkowski, D. Urbaniak-Kujda E. Stefańska-Windyga, A. Buczma, A. Baran, E. Odnoczko 9

10 Emicizumab w profilaktyce u chorych na hemofilię A (New J Engl J Med. IF ) 10

258) Emicizumab subcutaneous dosing every 4 weeks is safe and

haemophilia A with and without inhibitors.

11 Emicizumab w profilaktyce u chorych na hemofilię A (New J Engl J Med. IF ) Emicizumab subcutaneous dosing every 4 weeks is safe and efficacious in the control of bleeding in persons with haemophilia A with and without inhibitors. Results from the phase 3 HAVEN 4 study Steven Pipe, MD University of Michigan, C.S. Mott Children's Hospital, Ann Arbor, MI, USA 11

12 Co-authors Victor Jiménez-Yuste: Hospital Universitario La Paz, Autónoma University, Madrid, Spain Amy Shapiro: Indiana Hemophilia and Thrombosis Center, Indianapolis, IN, USA Nigel Key: University of North Carolina, Chapel Hill, NC, USA Maria Podolak-Dawidziak: Medical University Department of Hematology, Wroclaw, Poland Cedric Hermans: Cliniques Universitaires St-Luc, Brussels, Belgium Kathelijne Peerlinck: UZ Leuven Gasthuisberg, Leuven, Belgium Michaela Lehle, Sammy Chebon, Agnes Portron and Nives Selak Bienz: F. Hoffmann-La Roche Ltd., Basel, Switzerland Gallia G. Levy: Genentech Inc., South San Francisco, CA, USA Midori Shima: Nara Medical University, Department of Pediatrics, Kashihara, Japan 12

13 Badania z emicizumabem HAVEN 4 Prof. Maria Podolak-Dawidziak - Wrocław Praca oryginalna z tego badania została zaakceptowana w marcu 2019 r. do druku w Lancet Haematology (IF=10,6). 13

14 Hemlibra ex-change Conference Prague Expert speaker: Prof. Jerzy Windyga, Warsaw, Poland Dr Jan Blatný Czech Republic Dr Ester Zápotocká Czech Republic Prof. Sophie Susen France Dr Carmen Escuriola- Ettingshausen Germany Dr Robert Klamroth Germany Prof. Johannes Oldenburg Germany Prof. Gili Kenet Israel Prof. Giancarlo Castaman Italy Dr Elena Santagostino Italy Prof. Jerzy Windyga Poland Prof. Johnny Mahlangu South Africa Dr Maria Teresa Alvarez Spain Dr Santiago Bonanad Spain Dr Victor Jimenez- Yuste Spain Dr Eva Mingot Spain 14 Dr Gerry Dolan UK Dr Michael Callaghan USA Dr Robert Sidonio USA Dr Guy Young USA

15 HEMLIBRA s story pivotal studies FPI NIS US approval for haemophilia A with inhibitors EU approval for haemophilia A with inhibitors US approval for haemophilia A without inhibitors HAVEN 1 HAVEN 2 HAVEN 3 EU approval for haemophilia A without inhibitors HAVEN 4 STASEY IIS Real world data IIS, investigator-initiated study 15

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