Sangamo Therapeutics, Inc. · NASDAQ:SGMO
Sangamo Therapeutics — World Federation of Hemophilia (WFH) Virtual Summit 2020
15 slides from Sangamo Therapeutics's conference presentation, Jun 18, 2020
Slide 1: Updated Follow-up of the High-Dose Cohort in the Alta Study
Updated Follow-up of the High-Dose Cohort in the Alta Study, a Phase 1/2 Study of giroctocogene fitelparvovec (SB-525) Gene Therapy in Adults With Severe Hemophilia A Thomas J. Harrington, MD1; Barbara A. Konkle, MD2; Kimo Stine, MD3; Nathan Visweshwar, MD4; Andrew D. Leavitt, MD5; Adam Giermasz, MD
Slide 2: Disclosures for Thomas J. Harrington, MD
Disclosures for: Thomas J. Harrington, MD Conflict Disclosure Research Support Sangamo/Pfizer Inc. Director, Officer, Employee none Shareholder none Honoraria none Advisory Committee none Consultant none
Slide 3: Disclaimer
Data in this presentation are presented “as-is” and potentially subject to change. Disclaimer
Slide 4: Hemophilia A
Characterized by increased bleeding caused by low levels of factor VIII (FVIII) activity resulting from mutations in the F8 gene Treatment is currently based on replacement therapy with exogenous FVIII, along with emerging mimetic-based therapy Current treatments require frequent dosing to be effect
Slide 5: Giroctocogene Fitelparvovec (SB-525) Gene Therapy for Hemophilia A
Giroctocogene fitelparvovec (SB-525) Gene Therapy for Hemophilia A Alta is a phase 1/2 dose-ranging, single-dose, multicenter study to assess the safety and tolerability of giroctocogene fitelparvovec (SB-525) in adult subjects (aged ≥18 years) with severe hemophilia A Giroctocogene fitelparvovec (S
Slide 6: Study End Points
Study End Points Primary end points Safety and tolerability of SB-525, as assessed by the incidence of adverse events (AEs) and serious adverse events (SAEs) and by changes in clinical laboratory assessments, vital signs and electrocardiogram, and liver imaging Changes in circulating FVIII activity
Slide 7: Study Status
Study Status 4 dose cohorts of 2 subjects each and a high-dose cohort expansion of 3 subjects (total of 11 subjects dosed); no prophylactic steroid use Steroid treatment is initiated for alanine aminotransferase (ALT) elevation that exceeds 1.5x baseline value The safety and efficacy data of each co
Slide 8: Patient Demographics
Min-max, minimum-maximum. Data cut: March 2020 Patient Demographics Characteristic Cohort 1 9e11 vg/kg Cohort 2 2e12 vg/kg Cohort 3 1e13 vg/kg Cohort 4 3e13 vg/kg All Subjects Age, years n 2 2 2 5 11 Mean (SD) 30.5 (9.19) 35.5 (16.26) 32.0 (1.41) 26.8 (6.30) 30.0 (7.94) Median 30.5 35.5 32.0 29.0 30
Slide 9: Safety Summary
Safety Summary: Cohort 4 (3x1013 vg/kg) 1 subject had a treatment-related serious adverse event (SAE) of grade 3 hypotension and grade 2 fever, with symptoms of headache and tachycardia occurring ≈6 hours after completion of the vector infusion, with resolution ≈12 hours postinfusion No additional t
Slide 10: Safety Summary: Treatment-Related Adverse Events
Safety Summary: Treatment-Related Adverse Events Cohort 4 (3x1013 vg/kg) MedDRA Preferred Term Cohort 4 3e13 vg/kg (N=5) Subjects, n (%) No. of Events Any treatment-related event 5 (100.0) 42 Alanine aminotransferase increased* 3 (60.0) 9 Pyrexia 4 (80.0) 4 Aspartate aminotransferase increased 1 (20
Slide 11: ALT Elevations
ALT Elevations: Cohort 4 (3x1013 vg/kg) Subject ID Number Time of First ALT Elevation (Week) Maximum ALT Value, U/L (Grade) Steroids, >60mg (Weeks) Steroids, Taper (Weeks) FVIII levels (Chromo, IU/dL) at Start of Steroids FVIII Levels (Chromo, IU/dl) at End of Taper Time of Second ALT Elevation (Wee
Slide 12: Efficacy: Latest Available FVIII Values from March 2020 Data Cut
Efficacy: Cohort 4 (3x1013 vg/kg) Latest available FVIII values from March 2020 data cut FVIII Activity as measured at Central Laboratory with Chromogenic Assay
Slide 13: Efficacy: Factor VIII Activity Over Study Weeks
Efficacy: Cohort 4 (3x1013 vg/kg) N=5 Patients (Up to Wk 28) N=4 (Up to Wk 36) 50% Factor VIII activity (Lower bound of Normal) Study Week Mean Factor VIII activity value from Week 9 to Week 36 (based on group mean) * Mean (Box-Whisker plot) Data cut: March 2020
Slide 14: Efficacy: Steady-State FVIII Activity
Efficacy: Cohort 4 (3x1013 vg/kg) Steady-state FVIII activity achieved by week 9 post infusion Subjects have been followed for 33-65 weeks, FVIII activity values available up to week 30 and up to week 61 Median steady-state (of geometric means since week 9) FVIII activity level 64.2% via central lab
Slide 15: Conclusions
Conclusions Cohort 4 (3x1013 vg/kg): With follow-up ranging 33 to 65 weeks, data continues to show that giroctocogene fitelparvovec (SB-525) is generally well tolerated Sustained FVIII activity levels in the mild to normal range No use of exogenous FVIII beyond week 3 post infusion No bleeding event
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