Protalix BioTherapeutics, Inc. (de) · AMEX:PLX

Protalix BioTherapeutics, Inc. (de) — Technical Presentation (2014)

20 slides from Protalix BioTherapeutics, Inc. (de)'s technical presentation, Aug 1, 2014

  1. Slide 1: Taliglucerase Alfa 36-Month Clinical Safety and Efficacy in Treatment-Naïve Patients

    Taliglucerase Alfa 36-Month Clinical Safety and Efficacy in Treatment-Naïve Patients Ari Zimran Share Zadeck Medical Center and Hadassah Medical School - Hebrew University, Jerusalem, Israel Ari Zimran1; Gloria Durán2; Atul Mehta3; Pilar Giraldo4; Hanna Rosenbaum5; Fiorina Giona6; Dominick J. Amato7

  2. Slide 2: Acknowledgement of fellow investigators

    The authors would like to acknowledge fellow investigators Drs. Rene Heitner and Paul Fernhoff who have passed away. 2

  3. Slide 3: Disclosures

    Disclosures AZ is a consultant for Protalix BioTherapeutics and is a member of the Speakers’ Bureau for Protalix BioTherapeutics, Shire, and Genzyme/Sanofi; is a stockholder in Protalix BioTherapeutics, and receives honoraria from Shire, Genzyme/Sanofi, and Pfizer. In addition, the Gaucher Clinic re

  4. Slide 4: Introduction

    Introduction • Taliglucerase alfa is an enzyme replacement therapy (ERT) approved for treatment of adults with Type 1 Gaucher disease (GD) and is the first approved plant cell–expressed recombinant therapeutic protein.1,2 • The safety and efficacy of taliglucerase alfa 30 U/kg and 60 U/kg were demon

  5. Slide 5: Study Design

    Study Design • Phase 3, multicenter, double-blind, randomized trial (PB-06-003; NCT00705939) of taliglucerase alfa at 2 dose levels • Dosing: 30-U/kg or 60-U/kg intravenous (IV) infusion every other week • Efficacy assessments – Spleen volume, liver volume, platelet count, hemoglobin concentration,

  6. Slide 6: Study Design (cont'd)

    Study Design (cont’d) • Main inclusion criteria: PB-06-001 – Treatment-naïve, adult patients (>18 years of age) with a diagnosis of GD – Leukocyte glucocerebrosidase activity ≤30% of the mean activity of the reference range – Splenomegaly ≥8 times the expected normal volume as determined by magnetic

  7. Slide 7: Patient Disposition

    Patient Disposition Treatment Number of duration Patients 0 Discontinuations (n=3) PB-06-001  2: AEs, both hypersensitivity 9 Months  1: pregnancy 9 months Not treated in PB-06-003 (n=3)  1: pregnancy  1: patient planned to become pregnant  1: patient not interested in treatment PB-06-003 Exten

  8. Slide 8: Patient Demographics

    Patient Demographics Taliglucerase Taliglucerase Alfa Alfa 30 U/kg 60 U/kg Parameter (n=12) (n=14) Age, mean ± SD, y 38.9 ± 12.1 35.6 ± 12 (Range) (24–74) (19–58) Male, n (%) 7 (58.3) 8 (57.1) Female, n (%) 5 (41.7) 6 (42.9) Ashkenazi Jewish, n (%) 4 (33.3) 2 (14.3) Non-Jewish, n (%) 8 (66.7) 12 (85

  9. Slide 9: Baseline Disease Characteristics

    Baseline Disease Characteristics Taliglucerase Alfa Taliglucerase Alfa Parameter 30 U/kg (n=12) 60 U/kg (n=14) Absolute spleen volume, mL Mean ± SD 2324.0 ± 1209.0 2120.0 ± 1426.5 Range 1025.6-4901.1 913.7-5417.8 Spleen volume, MN Mean ± SD 16.4 ± 8.3 16.8 ± 14.2 Range 7.7-34.5 7.8-54.2 Absolute liv

  10. Slide 10: Spleen Volume Response to Taliglucerase Alfa

    Spleen Volume Response to Taliglucerase Alfa * Values are mean  standard error *n=11 at 36 months 10

  11. Slide 11: Liver Volume Response to Taliglucerase Alfa

    Liver Volume Response to Taliglucerase Alfa * Values are mean  standard error *n=11 at 36 months 11

  12. Slide 12: Hemoglobin Response to Taliglucerase Alfa

    Hemoglobin Response to Taliglucerase Alfa * † Values are mean  standard error *n=11 at 24 and 36 months; †n=11 at 36 months 12

  13. Slide 13: Platelet Response to Taliglucerase Alfa

    Platelet Response to Taliglucerase Alfa 147,727 94,683 69,042 64,900 * * Values are mean  standard error *n=11 at 36 months 13

  14. Slide 14: Chitotriosidase Response to Taliglucerase Alfa

    Chitotriosidase Response to Taliglucerase Alfa * Values are mean  standard error *n=10 at 36 months 14

  15. Slide 15: Safety: Adverse Events

    Safety: Adverse Events • Most common AEs No. of AEs • Nasopharyngitis • 30 U/kg, n=2; 60 U/kg, n=3 (No. of % of • Arthralgia Patients) Total • 30 U/kg, n=4; 60 U/kg, n=4 Parameter [N=26] AEs • Upper respiratory tract infection • 30 U/kg, n=3; 60 U/kg, n=3 Total AEs 280 (24) 100 • Headache • 30 U/kg,

  16. Slide 16: Safety: Infusion-related Adverse Events

    Safety: Infusion-related Adverse Events Infusion-related AEs • 4 patients in the Possibly, probably, or definitely related to 30-U/kg group experienced 18 treatment* infusion-related AEs Taliglucerase alfa Taliglucerase alfa • 6 patients in the 30 U/kg 60 U/kg 60-U/kg group experienced 18 • Fixed dr

  17. Slide 17: Summary and Conclusions

    Summary and Conclusions These 36-month results of taliglucerase alfa 30 U/kg and 60 U/kg in treatment-naïve adult patients with GD: • Represent the longest multiple-dose study of taliglucerase alfa in patients with GD • Demonstrate continuous improvement of the disease parameters in GD patients afte

  18. Slide 18: Disclosure Information

    Disclosure Information WORLD Symposium, San Diego 2012 ARI ZIMRAN, MD  Disclosure of Relevant Financial Relationships  I have the following financial relationships to disclose:  Consultant for: Protalix  Speaker’s Bureau for: Shire HGT, Pfizer, Actelion  Grant/Research support from: Genzyme (fo

  19. Slide 19: Acknowledgments

    Acknowledgments Thank You • The authors thank the patients, their families, and the clinical research personnel. 19

  20. Slide 20: Thank You

    THANK YOU 20

Protalix BioTherapeutics — Technical Presentation (2014) on 6ix