Novo Highlights Positive Outlook for Preventive Hemophilia Drug

Novo Nordisk recently unveiled comprehensive clinical trial results for Mim8, an experimental treatment for hemophilia A. This new antibody drug, administered either weekly or monthly, has demonstrated significant effectiveness in controlling bleeding among patients with this condition. The data were shared at the International Society on Thrombosis and Haemostasis Annual Congress held in Thailand, painting a favorable picture of Mim8's safety and efficacy.

The extensive results come from the Phase 3 trial known as Frontier-2. Previous reports in May had already highlighted the trial's success, but these new details offer a more comprehensive look. Among those who had never received preventive treatment before, 86% of participants on a weekly dose of Mim8 reported zero bleeds, while this figure rose to 95% for those on a monthly dose. For participants who had prior preventive treatments, the numbers were slightly lower, showing zero bleeds in 66% of weekly dosed individuals and 65% of those on a monthly regimen.

Novo Nordisk, predominantly known for its diabetes and weight loss medications like Ozempic and Wegovy, is also making strides in rare blood disorders. The pharmaceutical giant continues to invest in treatments for conditions like sickle cell disease, demonstrated by its acquisition of Forma Therapeutics two years ago and the advancement of another drug it licensed in 2018. The promising results from the Mim8 trial further indicate Novo's commitment to these areas.

The Frontier-2 trial enrolled 254 hemophilia A patients, both with and without inhibitors that can hinder the efficacy of standard blood-clotting drugs. The year-long study aimed to compare Mim8's effect against no prophylaxis for untreated patients and against prior coagulation factor prophylaxis for those previously on preventive treatment. In patients without prior prevention, a weekly dose of Mim8 reduced the annualized bleeding rate by 97%, while the monthly dose achieved a 99% reduction. This resulted in an average of 0.45 treated bleeds per patient annually for the weekly group and 0.20 for the monthly group, compared to 15.75 bleeds in the control group.

For patients who had been on preventive treatment before joining the trial, the run-in phase was followed by either a weekly or monthly Mim8 dose. Their average annualized bleeding rates were 2.51 for the weekly dose, down from 4.83 on their previous treatment, and 1.78 for the monthly dose, compared to 3.10 previously. Importantly, no thromboembolic events or related serious side effects were reported during the trial.

Martin Holst Lange, Novo's head of development, highlighted the potential of Mim8 to offer patients zero bleeds and flexible dosing to suit various lifestyles. The company intends to submit an approval application for Mim8 by the end of the year and will release more trial data at upcoming medical conferences.

Mim8 is a bispecific antibody designed to bridge Factors IXa and X, effectively replacing the missing Factor VIII in hemophilia A patients. If approved, Mim8 will likely compete with Roche's Hemlibra, another bispecific antibody currently approved in the U.S. for routine preventive treatment of hemophilia A. Hemlibra offers flexible dosing options, including weekly, bi-weekly, and monthly regimens.

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