Investor Presentaiton slide image

Investor Presentaiton

Investor presentation Full year 2018 Slide 100 N8-GP administered every fourth day reduces median bleeding rate to 1.3 episode per year in phase 3 trial N8-GP phase 1 pharmacokinetics FVIII activity (IU/mL) 1.2 Dose 50 IU/kg (n=8) 1.0 One stage clot assay 0.8 0.6 0.4 0.2 0.0 0 FVIII - N8-GP • 24 48 72 Time (h) 96 120 144 168 Pathfinder 2 headline results (phase 3) PK documented single dose half-life of 18.4 hours and mean trough level before next dose of 3% • Patients on every fourth day prophylaxis (50 IU/kg) had a median ABR of 1.3 • • • 95% of mild to moderate bleeds managed with 1-2 doses N8-GP appeared to have a safe and well tolerated profile One patient developed inhibitors, as expected in a population of previously treated haemophilia A patients Pathfinder 2 extension trial results • 55 patients with ≤2 bleeds during 6 months in the main phase were randomised 2:1 to either once-weekly (75 IU/ kg) or every fourth day (50 IU/kg) treatment for 180 days¹ Patients in both treatment arms had a median ABR of 0 Next steps • Awaiting regulatory decisions in the USA, the EU and Japan Source: Tiede et al. J Thromb Haemot. 2013;11:670-675 changing diabetes® PK: Pharmacokinetic; ABR: Annualised bleeding rate; IU: International unit 1 Prophylaxis 75 IU/kg every 7 days (n=38) or prophylaxis 50 IU/kg every 4 days (n=17) novo nordisk
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