Coagulation factor VIIa Recombinant Human

Tiumbio completed Phase 1a trials for hemophilia drug TU7710, showing high safety and tolerability. TU7710's half-life is seven times longer than competitors, indicating potential as a long-acting treatment.

At ASH 2024, physicians discussed advancements in hemophilia care, including Roche's Hemlibra and curative AAV gene therapies like CSL Behring's Hemgenix, Biomarin's Roctavian, and Pfizer's Beqvez and Hympavzi. Despite progress, unmet needs remain, with concerns over long-term risks of gene therapies and limited access. Pfizer presented Phase III AFFINE trial data for giroctocogene fitelparvovec, showing comparable efficacy to Roctavian with better safety. Sanofi's fitusiran, a siRNA, demonstrated efficacy similar to clotting factor replacement therapy. Staidson's bemiltenase alfa, a fusion protein, showed favorable bleed clearance rates. Challenges persist, but advancements in curative, prophylaxis, and on-demand treatments offer more options for patients.

Hemophilia B market to grow significantly by 2034, driven by gene therapies like HEMGENIX and BEQVEZ, priced at $3.5 million each. Key companies include Centessa Pharmaceuticals, Sanofi, Pfizer, and Novo Nordisk. Market dynamics influenced by increasing prevalence, prophylactic treatment focus, and pipeline advancements.

TiumBio has initiated a Phase II trial of oral immuno-oncology drug TU2218, targeting advanced solid tumours, with the first HNSCC patient dosed. The trial aims to assess TU2218's safety and efficacy in combination with Keytruda for HNSCC, biliary tract, and colorectal cancers. TU2218, a dual inhibitor of TGFR1 and VEGFR2 pathways, seeks to enhance immune checkpoint inhibitor efficacy.