Tiumbio, a company focused on developing treatments for rare and incurable diseases, has announced positive results from its Phase 1a clinical trial of TU7710, a novel drug candidate for hemophilia treatment. The trial, conducted in 40 healthy male participants, evaluated the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of intravenously administered TU7710 following pretreatment with Warfarin.
Favorable Safety Profile
The Phase 1a trial revealed no serious adverse events among the participants. All drug-related side effects were mild, classified as Grade 1 or 2, indicating a high level of safety and tolerability for TU7710.
Extended Half-Life
A key finding from the trial was the extended half-life of TU7710 compared to existing treatments. Pharmacokinetic evaluation showed that all five dose groups (0.1 mg/kg, 0.2 mg/kg, 0.4 mg/kg, 0.8 mg/kg, and 1.6 mg/kg) exhibited half-lives approximately seven times longer than those of NovoSeven and SevenFact, which have half-lives of 2.3 hours and 1.6 hours, respectively. This suggests that TU7710 has the potential to be a long-acting therapy, potentially reducing the frequency of injections required for hemophilia patients.
Addressing Unmet Needs in Hemophilia Treatment
TU7710 is being developed as a Factor VIIa treatment candidate for hemophilia patients who have developed neutralizing antibodies to existing treatments for hemophilia A (caused by a deficiency of blood clotting factor VIII) or hemophilia B (caused by a deficiency of blood clotting factor IX). Current market leader NovoSeven, marketed by Novo Nordisk, requires frequent intravenous injections (every two hours) due to its short half-life, which presents a significant burden for patients. The global market for such treatments is estimated at KRW 2 trillion (approximately USD 1.3 billion).
Future Development Plans
"Building on the success of 'Afstyla,' a hemophilia treatment, we have achieved results that meet our expectations in the first clinical trial of TU7710, which represents a new challenge for us," said Kim Huntaek, CEO of Tiumbio. The company is now planning to initiate a Phase 1b clinical trial in Europe to further evaluate TU7710 in hemophilia patients.
