12 slides from Protalix Biotherapeutics, Inc. (de)'s technical presentation
Slide 1: Gout and Treatment with Uricases: Approved and Under Development
Gout and Treatment with Uricases Approved and Under Development Made More Crystal Clear Naomi Schlesinger, MD Harold J, Ardella T, and Helen T Stevenson Presidential Endowed Chair of Rheumatology Professor and Chief Division of Rheumatology Spencer Fox Eccles School of Medicine University of Utah Sa
Slide 2: PRX-115 Phase 1 Top Line Results
PRX-115 Phase 1 Top Line Results Interim Analysis (Cohorts 1-7) 2
Slide 3: PB115-SAD-101; First in Human Phase 1 Trial
PB115-SAD-101; First in Human Phase 1 Trial Study StudyTitle: Title: A double-blind, placebo-controlled, single ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics properties of PRX-115 in adult volunteers with elevated uric acid levels Primaryobjective:
Slide 4: Study schedule per patient
We are here Study Scheme Cohort 8 In each cohort: Cohort 7 • Single IV doses • N = 6 active + 2 placebo Cohort 6 Optional cohort • Safety Cohort 5 • PK Dose escalation meeting by • Uric Acid Cohort 4 a blinded Safety Monitoring Committee (SMC) • ADA Cohort 3 Dose administration Cohort 2 For subjects
Slide 5: Study Population
Study Population • Males or females 18 to 65 years of age, inclusive • Serum uric acid 6.0 mg/dL reduced to ≥ 4.7* mg/dL, at Screening • Body mass index within the range 18.5 to 40 kg/m2, inclusive, at the Screening visit • Had no gout flare in the last year prior to either Screening or Day -1 • H
Slide 6: Summary of Demographics and Baseline Characteristics
Summary of Demographics and Baseline Characteristics PRX-115 Pooled Parameter Statistic Overall Placebo Overall n 42 14 56 Age (years) Mean 36.8 34.3 36.2 SD 12.5 11.2 12.2 Sex n(%) Female 13 (31.0%) 3 (21.4%) 16 (28.6%) Male 29 (69.0%) 11 (78.6%) 40 (71.4%) Race n (%) American Indian or Alaska Nati
Slide 7: Overall Summary of Adverse Events: PRX-115 was Well-Tolerated
Overall Summary of Adverse Events: PRX-115 was Well-Tolerated Number of Participants Reporting at Least 1 AE PRX-115 PRX-115 Pooled Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 Cohort 6 Cohort 7 Overall Placebo n(%) n(%) n(%) n(%) n(%) n(%) n(%) n(%) n(%) N 6 6 6 6 6 6 6 42 14 Adverse Event 5(83.3)
Slide 8: Immunogenicity Evaluation: Preliminary Results
Immunogenicity Evaluation: Preliminary Results • Preliminary antidrug IgG antibody assessment revealed that many subjects developed a positive, low-titer IgG response following a single administration of PRX-115. • PRX-115 immunogenicity is still under evaluation including correlation to PK, PD (uri
Slide 9: Mean PRX-115 Plasma Concentrations
Mean PRX-115 Plasma Concentrations (µg/mL) 10 9 Cohort 1 • Exposure of PRX-115 increased Mean Plasma PRX-115 (µg/mL) 8 Cohort 2 with the increase of dose 7 Cohort 3 6 Cohort 4 • Detectable exposures beyond 5 Cohort 5 day 56 was observed in plasma 4 Cohort 6 of treated patients in cohorts 6 3 Cohort
Slide 10: Mean Uric Acid Concentrations
Mean Uric Acid Concentrations (mg/dL) 10.00 9.00 Pooled Placebo 8.00 Cohort 1 • Administration of PRX-115 Mean Plasma Uric Acid (mg/dL) 7.00 leads to a rapid reduction of Cohort 2 6.00 plasma uric acid Cohort 3 5.00 Cohort 4 • Effect of PRX-115 on plasma 4.00 Cohort 5 uric acid levels and duration o
Slide 11: Summary
Summary SAFETY SAFETY – All randomized patients completed the study – PRX-115 was found to be well tolerated in the study – Only 11/42 (26%) patients reported study drug related AE, most mild to moderate in severity, with the exception of one patient that experienced a severe AE of an anaphylactic r
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