Novo Nordisk plans to seek approval in the US and the EU this year for their drug Mim8 for use on pediatric patients with Hemophilia A after promising results from its FRONTIER3 trial.

As reported by FIERCE Biotech, Phase 1 of the trial consisted of 70 children between the ages of 1 and 11 who treated once a week with Mim8 for a total of 26 weeks.  Fourteen of the children enrolled have hemophilia A with inhibitors.  Inhibitors cause the body to reject standard factor treatments.

Phase 3 FRONTIER3 Trial Results

74.3% of the children involved in the trial experienced no treated bleeds during the trial period, including all 14 participants with hemophilia A with inhibitors. The average annualized rate of treated bleeds, or the ABR, landed at 0.53.

Concerning safety measures, none of the participants experienced any major reactions or events needing emergency intervention and no deaths occurred.  Results also showed that 98% of caregivers to the patients preferred Mim8 over previous options, 73% strongly preferred.  Physical functioning reportedly improved as well as quality of life.

The second phase of the study now begins.  45% of the children will shift to treating monthly with the remainder continuing to treat weekly.

About Mim8

Mim8 is categorized as a Factor VIIIa, or FVIIIa, mimetic bispecific antibody that is administered under the skin of patients living with Hemophilia A, with and without inhibitors.  Mim8 is designed to replace Factor VIII and restore the thrombin’s ability to generate, helping blood to clot.  It must be noted that this treatment is not approved by any regulatory authorities in the US or worldwide and is not available for customer use.

Source: FIERCE Biotech