Mim8 seen as better than standard hem A therapies at bleed control

Phase 3 trial finds fewer bleeds over 26 weeks of investigative treatment's use

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Weekly and monthly treatment with Mim8, an investigational antibody therapy, was seen to be better than on-demand or standard preventive treatments at reducing bleeding episodes in patients with hemophilia A, regardless of inhibitor status, who took part in a clinical trial.

Top-line results from the global Phase 3 FRONTIER 2 (NCT05053139) study were announced by Novo Nordisk, the therapy’s developer. The company plans to submit a first regulatory application seeking Mim8’s approval by year’s end.

“We are very pleased with the positive results from the FRONTIER 2 clinical trial. 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, executive vice president of development at Novo Nordisk, said in a company press release.

“Given the differing needs of people living with haemophilia A, a convenient once-weekly or once-monthly dosing provides optionality and flexibility,” Lange added.

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Hemophilia A is marked by the lack or dysfunction of the blood clotting protein factor VIII (FVIII), leading to disease symptoms due to excessive and uncontrolled bleeding. Replacement therapies, providing the missing or faulty FVIII from an external source, are standard prophylactic (preventive) or on-demand therapies.

Certain patients, however, develop inhibitors, oneutralizing antibodies against the delivered FVIII, which can limit its effectiveness.

Mim8 is a next-generation prophylactic treatment designed to bypass the need for conventional FVIII by mimicking its activity. The antibody-based therapy works to bring together two other clotting factors — factor IXa and factor X — that mimic the natural function of FVIII in the body.

Administered as an under-the-skin injection, Mim8 is designed to stimulate the production of thrombin, an enzyme that helps blood to clot.

Early positive results from the FRONTIER 1 trial (NCT04204408), which evaluated the safety and pharmacological properties of Mim8 in healthy volunteers and hemophilia A patients, supported the launch of several Phase 3 trials, including FRONTIER 2.

In this Phase 3 trial, 254 hemophilia A patients with or without inhibitors, age 12 and older, randomly were assigned to either once weekly or monthly injections of Mim8, or to continue with routine preventive or on-demand FVIII replacement treatment for 26 weeks, or about six months.

Significantly fewer bleeds reported in patients given Mim8 weekly or monthly

A significantly lower number of bleeding episodes were found in patients treated with Mim8 compared with those on standard treatment, Novo Nordisk reported in its release.

In patients not using preventive therapies, once-weekly dosing with Mim8 led to 97% reduction in the number of treated bleeds, while once-monthly dosing lowered that number by 99%. In the Mim8 weekly dosing group, 86% of patients had no bleeds requiring treatment during the main trial’s 26 weeks, as did 95% of people in the monthly dosing group.

Patients using preventive FVIII therapies prior to enrollment continued to do so for at least 26 weeks in a run-in period before starting with Mim8 for another 26 weeks. Compared with treated bleeds during the run-in, the number of treated bleeds among patients given Mim8 weekly fell by 48%, and by 43% for those on monthly treatment.

Among this prior prophylactic group, 66% of patients on a weekly regimen and 65% of those on a monthly regimen had no bleeds requiring treatment while being given Mim8.

Mim8’s safety profile was in agreement with that seen in previous trials, with the treatment deemed safe and well tolerated, the company stated. No deaths or blood clotting events were reported.

Patients who complete Phase 2 and 3 studies into Mim8 are being invited to continue treatment in a long-term safety and efficacy extension study, FRONTIER4 (NCT05685238).

Findings in the FRONTIER clinical program, which also includes a Phase 3 trial (NCT05306418) of Mim8 in children with hemophilia A, 1 to 11 years old, will be published in scientific journals and presented at scientific meetings later this year and across 2025, the company reported.