Efficacy Profile

Jivi efficacy in adults and adolescents^1*

Efficacy in ABR with routine prophylaxis (Part A)*

Median ABR was 2.09 for all prophylaxis groups combined during the 26-week treatment period¹
42 subjects (38.2%) in the prophylaxis arms had no bleeds during the 26-week period¹

Bleeding tendency	All bleeds Median ABR (Q1; Q3)	Spontaneous bleeds Median ABR (Q1; Q3)	Joint bleeds Median ABR (Q1; Q3)	Subjects with zero bleeding episodes (secondary endpoint) % (n)
TWICE WEEKLY _{30-40 IU/kg} ≥2 SPONTANEOUS BLEEDS^† _(n=13)	4.1 _{(2.0; 10.6)}	3.9 _{(0.0; 4.1)}	4.0 _{(2.0; 8.0)}	15.4% ₍₂₎
≤1 SPONTANEOUS BLEED^‡ _(n=11)	1.9 _{(0.0; 5.2)}	0 _{(0.0; 1.9)}	1.9 _{(0.0; 5.2)}	45.5% ₍₅₎

EVERY 5 DAYS _{40-60 IU/kg} ≤1 SPONTANEOUS BLEED^‡ _(n=43)	1.9 _{(0.0; 4.2)}	0 _{(0.0; 4.0)}	1.9 _{(0.0; 4.0)}	44.2% ₍₁₉₎

Adapted from Jivi Product Monograph.

Median ABR in the every-7-days treatment arm was 3.85 for the ITT population. Twenty six percent of subjects in this arm experienced bleeding and moved to more frequent dosing during Part A of the PROTECT VIII study.¹

Efficacy in control of bleeding episodes (Part A)*

386 bleeding episodes were treated with Jivi during Week 0 to 36 in the on-demand arm and 316 in all prophylaxis groups combined
Patients rated the adequacy of hemostasis with Jivi as “good” or “excellent” in 73.3% of treatment for bleeds, with 23.4% rated as “moderate” and 3.3% as “poor” (2° endpoint)

Perioperative management (Part B)*

Treatment with Jivi provided “good” or “excellent” hemostatic control in all 20 major surgeries^§

ITT=intent-to-treat.

* PROTECT VIII study: A Phase 2/3, multi-national, open-label, uncontrolled, partially randomized study examining the pharmacokinetics, safety and efficacy of Jivi in previously-treated patients (≥150 exposure days [EDs]), adolescents and adults (12 to 65 years of age), with severe hemophilia A (<1%). The main study duration (Part A; n=134) was 36 weeks and evaluated the pharmacokinetics (single dose of 60 IU/kg), safety and efficacy of Jivi for on-demand treatment and routine prophylaxis with three regimens. Safety and efficacy of Jivi in hemostasis during major surgical procedures was evaluated in Part B (n=16). An optional extension study included patients (n=121) completing Part A to accumulate at least 100 EDs. The primary efficacy variable was annualized bleed rate (ABR).

† Patients with ≥2 spontaneous bleeds (defined as joint or muscle bleeds and no identified trauma) during the first 10 weeks of the trial.

‡ Patients with ≤1 spontaneous bleed (defined as joint or muscle bleeds and no identified trauma) during the first 10 weeks of the trial.

§ Fourteen major surgeries were orthopedic joint surgeries, including 3 arthroplasties, 6 joint replacements and 3 synovectomies, and 2 other joint procedures.