Dive Brief:

• Novo Nordisk on Sunday disclosed detailed clinical trial results for an experimental hemophilia treatment dubbed Mim8, showing once-weekly and once-monthly doses of the antibody drug controlled bleeding in people with the more common “A” form of the disorder.
• The data, which were presented at the International Society on Thrombosis and Haemostasis Annual Congress in Thailand, fill in a positive picture for Mim8’s effectiveness and safety. Novo had said in May that the Phase 3 trial, called Frontier-2, succeeded and shared topline findings.
• Among people who had not previously been on preventive treatment, researchers reported zero bleeds in 86% of study participants who received once-weekly Mim8, and 95% of those given the once-monthly dose. Those figures were 66% and 65%, respectively, among people in the trial who had prior preventive treatment.

Dive Insight:

The success of Ozempic and Wegovy, Novo’s drugs for diabetes and weight loss, have transformed the Danish drugmaker into the pharmaceutical industry’s second largest. Not surprisingly, analysts and Wall Street investors are now laser focused on that portion of the company’s business.

But Novo continues to develop drugs for rare blood disorders, and has placed emphasis on the potential of Mim8. It’s also invested in sickle cell disease, acquiring Forma Therapeutics two years ago and advancing another drug it licensed in 2018.

The recent results for Mim8 are the first from a Phase 3 trial program that includes three other studies beyond Frontier-2.

Novo enrolled 254 people with hemophilia A into Frontier-2, including those with and without “inhibitors,” which can prevent standard drugs from effectively clotting blood. The one-year study compared Mim8 to either no prophylaxis among those not on preventive treatment, or to prior coagulation factor prophylaxis among those who were.

In the former group, a once-weekly dose of Mim8 reduced the average annualized bleeding rate by 97%, while the once-monthly dose led to a 99% reduction. That translated to an average of 0.45 treated bleeds per patient year among the once-weekly group and 0.20 among the once-monthly patients. In the control arm of people who did not receive preventive treatment, the average rate was 15.75 treated bleeds per patient year.

Patients in the study who previously were on preventive treatment went through a “run-in” phase of the study, and then were given one of the two Mim8 doses. The average rate of treated bleeds was 2.51 per patient year for those on once-weekly dosing, versus 4.83 on their prior prophylaxis, and 1.78 for those on the once-monthly, versus 3.10 on their prior treatment.

There were no thromboembolic events or related serious side effects reported in the trial, Novo said.

“With Mim8, we have the potential of offering a substantial proportion of patients the prospect of zero bleeds and convenient dosing flexibility to fit their lifestyles and needs,” said Martin Holst Lange, head of development for Novo, in the company’s Sunday statement.

Novo plans to submit an approval application for Mim8 toward the end of the year, and will disclose more trial data for the drug at upcoming medical meetings.

A “bispecific” antibody, Mim8 is designed to bridge Factors IXa and X to replace the Factor VIII that’s missing in people with hemophilia A.

If approved, Mim8 would likely compete with Roche’s Hemlibra, which is also a bispecific anitbody and holds approval in the U.S. as a routine preventive treatment for people with hemophilia A. Dosing is flexible to either once-weekly, bi-weekly or once-monthly. Roche reported sales of 4.1 billion Swiss francs last year, or about $4.6 billion, using an average U.S. dollar exchange rate for 2023.