Investor Presentation First Nine Months of 2023
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Investor presentation
First nine months of 2023
Main part of the Explorer 8 trial with concizumab in people with
HA or HB without inhibitors has been completed
Explorer 8 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)
•
Key inclusion criteria:
Aged ≥12 years with
haemophilia A or
haemophilia B, patients
mainly from phase 2
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
•
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; HwI: Haemophilia with inhibitors; Prophylaxis: PPX; OnD: On-demand, QD: Once-dailyView entire presentation