Subcutaneous MG1113 in severe hemophilia A and B: phase 1b study for safety, pharmacokinetics, and pharmacodynamics.

What was studied

This phase 1b trial tested MG1113 under the skin in severe hemophilia A or B. MG1113 is an antibody that targets TFPI. 15 men took part, with 5 per dose group. 14 had hemophilia A and 1 had hemophilia B. None had inhibitors. Weekly doses were 2.0, 3.0 or 3.3 mg/kg. Dosing ran for 8 weeks.

What they found

MG1113 was well tolerated. Side events occurred in 6 of 15 people. No serious side events, early withdrawals or dose breaks occurred. Peak blood levels came at 33 to 48 hours after a dose. Free TFPI fell and clotting markers rose. Bleeding rates fell in an exploratory look, more at the higher doses. Some people had no bleeding at each dose.

What it means for patients

The authors say the drug had acceptable safety. They say its blood levels fit weekly dosing. They say the marker changes fit TFPI blocking. They link this to fewer bleeds.

Why it is not certain yet

Only 15 men took part. The trial ran 8 weeks. The bleeding results were exploratory. The abstract describes no comparison group.

Source

Subcutaneous MG1113 in severe hemophilia A and B: phase 1b study for safety, pharmacokinetics, and pharmacodynamics.

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