TiumBio confirms safety of TU7710 for hemophilia

TiumBio has confirmed the safety of its hemophilia drug candidate TU7710 in phase 1a clinical trial.

In a public disclosure on Monday, TiumBio announced the top-line results of the phase 1a trial of TU7710.

The phase 1a study was conducted in 40 healthy adult males at Seoul National University Hospital for about one year, from July 2023 to August 2024. After pre-treatment with anticoagulant warfarin, the study was divided into 30 TU7710-treated and 10 placebo-treated patients, with no dropouts.

Based on the primary endpoint of safety, there were no serious adverse events (SAEs), and all adverse events were mild or moderate (Grade 1 or 2). All patients who experienced an adverse event were confirmed to have recovered.

In phase 1a, the Cmax of TU7710 increased proportionally with the dose, from 8.62 IU/ml at the lowest dose (0.1 mg/kg) to 190.10 IU/ml at the highest dose (1.6 mg/kg).

The dose-specific treatment-emergent adverse event (TEAE) rate was highest at 83.3 percent (five patients) at the 0.8 mg/kg dose and decreased to 33.3 percent (two patients) at the highest dose of 1.6 mg/kg. The incidence of TEAEs in the placebo group was 50 percent.

In the pharmacokinetic (PK) evaluation, a secondary endpoint, the half-life of TU7710 ranged from 8.66 to 30.64 hours across dose groups. This is longer than the half-lives of the marketed products NovoSeven (eptacog alfa) and SevenFact (coagulation factor Vlla-jncw), which are 2.3 and 1.6 hours, respectively.

Notably, in the 0.4 mg/kg dose group, some patients had half-lives of more than 30 hours.

“(Blood coagulation) Factor VII is an endogenous substance produced by the body, and due to the nature of endogenous substances, there is a large inter-individual variation, and pharmacokinetic analysis can be difficult, which may explain the longer half-life observed in the 0.4 mg/kg dose group compared to other dose groups,” TiumBio explained in its disclosure.

The company is conducting a phase 1b study on hemophilia patients in Italy, Spain, and other European countries.