Novo Nordisk said Monday that its investigational treatment met both primary endpoints in an open-label Phase 3a study in patients with hemophilia A, meaning patients had fewer bleeding episodes.
Following the topline FRONTIER 2 data disclosure via a press release, the Danish drugmaker’s shares $NVO were up about 3.5%.
Novo revealed only a few headline data points on the 26-week, 254-patient trial, but said it will release more data at “upcoming congresses and in publications” this year and in 2025. By the end of this year, Novo said it plans to submit the treatment known as Mim8 for its first regulatory approval.
For patients who had no prior prophylaxis treatment, the once-weekly and once-monthly Mim8 showed nearly 100% reductions in treated bleeds, at 97% and 99%, respectively. Most patients experienced zero bleeds that needed treatment. In the once-weekly group, 86% had no treated bleeds. For the once-monthly cohort, 95% had zero treated bleeds.
In patients with prior coagulation factor prophylaxis, Mim8 showed superior reductions of treated bleeds by 48% and 43% in the once-weekly and once-monthly groups, respectively, as compared to their prior prophylaxis. About two-thirds of patients on both dosing schedules experienced zero treated bleeds, Novo said.
“Headline mim8 efficacy results near our ‘best’ case,” Jefferies analyst Peter Welford wrote in a note shortly after the data release, “with 86% (weekly) and 95% (monthly) of patients not on prior prophylaxis experiencing zero treated bleeds, compared to the high of 80% reported by Roche’s HAVEN-6 at its interim readout, or 56%-60% in HAVEN-3, albeit caveating for cross trial comparisons.”
Analysts await a fuller dataset to see how competitive Mim8 could be with Roche’s Hemlibra and Sanofi’s Altuviiio. BioMarin also markets a treatment for the condition, with its gene therapy Roctavian approved last summer, but few patients have received it so far, and executives have floated the idea of a potential divestment.
Martin Holst Lange“These data demonstrate the ability of Mim8 to prevent bleeding episodes effectively and safely in people with haemophilia A, regardless of their dosing frequency,” Martin Holst Lange, Novo’s executive vice president for development, said in a press release. “Given the differing needs of people living with haemophilia A, a convenient once-weekly or once-monthly dosing provides optionality and flexibility for people living with haemophilia A with or without inhibitors.”
Other treatments could be on the way for the rare inherited bleeding disorder, in which patients have missing or defective clotting factor VIII. Pfizer’s antibody marstacimab is set for a fourth-quarter approval decision at the FDA.
Novo is also developing another asset, concizumab, for hemophilia A and B. The FDA rejected it last May. It’s approved in Canada, where it’s marketed as Alhemo.