Investor Presentation Q1 2023
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Investor presentation
First three months of 2023
Main part of the explorer8 trial with concizumab in people with
HA or HB without inhibitors has been completed
explorer8 trial design
Key trial highlights
1) Maintained OnD treatment
Previously OnD treatment
R
1:2
2 Concizumab PPX, QD
Prophylaxis treatment
(continued from phase 2)
3 Concizumab PPX, QD
Prophylaxis treatment
4 Concizumab PPX, QD
(additional patients)
Novo NordiskⓇ
Extension with
concizumab
prophylaxis
Efficacy
• The trial met its primary endpoint, confirming superiority
of concizumab prophylaxis compared to no PPX (OnD
treatment)
The secondary confirmatory endpoint, confirming non-
inferiority of concizumab PPX to previous PPX factor
treatment was not met
•
Key inclusion criteria:
Aged ≥12 years with
haemophilia A or
haemophilia B, patients
mainly from phase 2
Objective:
•
Main part
24 weeks
Assess the efficacy of
Concizumab PPX vs no PPX
(OnD treatment) in
reducing number of
bleeding episodes
Extension part
Up to 143 weeks
Endpoints:
Number of treated
bleeding episodes
(spontaneous/traumatic)
Safety
•
Concizumab appeared to have a safe and well-tolerated
profile with no thromboembolic events reported after the
treatment restart¹
Next steps
•
Initial commercial launch for concizumab is expected to be
focused on HwI followed by Haemophilia B
Further assessment of development opportunities and
submissions based on the results from the explorer8 trial
1 Restart refers to the start of treatment with the new concizumab dosing regimen, which was implemented after the treatment pause
HA: Haemophilia A; HB: Haemophilia B; Prophylaxis: PPX; OnD: On-demand, QD: Once-dailyView entire presentation