Novo Nordisk Reveals Phase 3 Hemophilia A Data, Highlighting Roche Rivalry

Novo Nordisk recently provided a detailed update on the phase 3 results of its hemophilia A therapy candidate, Mim8, showcasing how it compares to Roche's Hemlibra in terms of efficacy and tolerability. The update comes after the initial topline results were shared in May, which suggested that Mim8’s once-monthly dosing could be as effective as Hemlibra’s once-weekly regimen.

In the initial report, analysts from Jefferies noted that the results appeared promising, with the once-monthly dosing of Mim8 showing similar efficacy to the once-weekly regimen. However, the initial results lacked specifics on annualized bleed rates (ABR) and primarily focused on percentage changes and injection site reactions.

Novo filled these gaps during a presentation at the International Society of Thrombosis and Haemostasis Annual Congress. The company reiterated the topline efficacy results and disclosed data on annualized bleeds and injection site reactions. In patients without prior prophylaxis treatment, the mean ABR was 15.75 in the control group. For those on once-weekly and once-monthly Mim8 prophylaxis, the mean ABRs were significantly lower at 0.45 and 0.20, respectively.

In patients with prior coagulation factor prophylaxis, the mean ABR for the once-weekly Mim8 group was 2.51, compared to 4.83 for those on prior clotting factor prophylaxis. The once-monthly Mim8 group had a mean ABR of 1.78, versus 3.10 for those on previous clotting factor prophylaxis.

Comparing these results to Roche’s Hemlibra proves challenging due to differences in study designs. Roche’s phase 3 trial for Hemlibra focused on patients with Factor VIII inhibitors and another group without inhibitors. In patients without inhibitors who were previously treated with on-demand bypassing agents, Roche reported an ABR of 2.9 in the Hemlibra group, compared to 23.3 in the control group. Another study for patients without inhibitors on on-demand treatment reported ABRs of 1.5 and 1.3 in the Hemlibra group, versus 38.2 in the control group.

Injection site reactions are a potential vulnerability for Hemlibra. Jefferies analysts noted in May that for a new competitor to clearly differentiate itself, the frequency of injection site reactions should be below 10%. Mim8 reported injection site reaction rates of 5.0%, 8.2%, 9.5%, and 12.2% across different patient cohorts.

Novo aims to file for regulatory approval of Mim8 by late 2024. Concurrently, Roche is bolstering Hemlibra's market position, which generated 4.1 billion Swiss francs ($4.6 billion) in revenue last year. Roche has introduced two new vial options and a new administration kit to enhance the convenience of Hemlibra dosing.

This development in hemophilia A treatment showcases the ongoing competition between Novo Nordisk and Roche, with both companies striving to optimize their offerings in terms of efficacy, dosing convenience, and minimization of side effects. As Novo prepares for its approval submission, the detailed phase 3 data on Mim8 will play a crucial role in its competitive positioning against the established Hemlibra.