Concizumab Phase 2 Data Demonstrate Its Potential as a Safe and Efficacious Subcutaneous Prophylaxis Treatment for All Patients with Haemophilia

July 09, 2019/Bio ValleyBIOON/--Danish pharmaceutical giant Novo Nordisk recently announced positive data from two clinical studies of its innovative hemophilia therapy, concizumab, at the 27th Annual Congress of the International Society on Thrombosis and Haemostasis (ISTH 2019) held in Melbourne, Australia. The results confirmed the favorable safety profile of concizumab and provided proof-of-concept for its efficacy in preventing bleeding episodes in patients with hemophilia A (Explorer 5 study) and in patients with hemophilia A or B with inhibitors (Explorer 4 study).

In this study, concizumab was well tolerated, with no discontinuations due to adverse events and no thromboembolic events. Phase II clinical trials showed that although many patients were undertreated due to conservative dosing rules, the annualized bleeding rate was comparable to that of factor prophylaxis. Importantly, no safety concerns were observed regarding the treatment of breakthrough bleeding, and all patients opted to continue into the extension phase of the study.

Amy Shapiro, investigator of the Explorer 4 study, stated, “These results are particularly important for patients with hemophilia B who have inhibitors, as treatment options for these patients are limited. Previously,”FDAConcizumab has been granted Breakthrough Therapy Designation (BTD) for the treatment of patients with hemophilia B who have inhibitors.

Ludovic Helfgot, Executive Vice President of Novo Nordisk Biopharm Operations, stated: “We are pleased that the results of the Phase II clinical study of concizumab support its use as a safe and well-tolerated subcutaneous prophylactic therapy in all patients with hemophilia, regardless of inhibitor status. We are delighted to continue advancing into Phase III clinical development, with the global Phase III program scheduled to launch later this year.”

Concizumab is a first-in-class, high-affinity, humanized recombinant monoclonal antibody that inhibits tissue factor pathway inhibitor (TFPI) by binding to its Kunitz-2 domain, thereby enabling the FVIIa:tissue factor complex to generate sufficient activated factor X to restore hemostatic potential in patients with hemophilia. Due to its unique mechanism of action, concizumab is expected to be equally effective in both hemophilia A and B, regardless of inhibitor status. Furthermore, subcutaneous administration may improve adherence, leading to better treatment outcomes.

Explorer 5 was a Phase II study that enrolled 36 patients with hemophilia A. Explorer 4 was a randomized controlled Phase II study that enrolled a total of 26 patients with hemophilia A or B who had inhibitors. In this study, patients were randomized in a 2:1 ratio to receive either prophylactic treatment with concizumab or on-demand treatment with the recombinant activated factor VII (rFVIIa) product eptacog alfa (activated). Based on insights gathered from the Phase II studies, including an individualized dose-escalation regimen guided by conservative efficacy criteria, Novo Nordisk has developed an optimized dosing strategy and trial design for its pivotal Phase III clinical studies. (Bioon.com)

Original Source:Concizumab phase 2 data demonstrate its potential as a safe and efficacious subcutaneous prophylaxis treatment for all patients with haemophilia