The 31st Annual Meeting of the International Society on Thrombosis and Haemostasis (ISTH) 2023 was held recently in Montreal, Quebec, Canada. Prof. Yang Renchi from the Hospital of Hematology, Chinese Academy of Medical Sciences, as the first Chinese clinical PI to make a presentation on China's self-developed products at ISTH, announced the results of a clinical study on an ultra-long-acting recombinant coagulation factor product, i.e., the recombinant human coagulation factor VIII-Fc fusion protein for injection developed by Gensciences, in the form of an oral presentation, which is expected to bring more better options for more than a million patients around the globe to get rid of the sufferings and return to the normal life.

Meet clinical needs and improve patients’medication compliance

Hemophilia is a group of hereditary bleeding disorders where a deficiency of coagulation factors leads to coagulation dysfunction, and is also a birth defect that poses a serious health risk. It is often clinically manifested as spontaneous bleeding or massive bleeding caused by mild trauma, tending to result in disability or life-threatening risks in serious cases, and therefore hemophiliacs are also called "fragile man". There are more than one million hemophiliacs worldwide, including about 140,000 in China, all requiring lifelong injections of coagulation factors.

The first generation of therapeutic drugs for hemophilia were blood-derived coagulation factor products, mainly derived from plasma extraction, with shortcomings such as blood-borne infections and insufficient supply. The second generation of recombinant coagulation factor products, introduced in the 1990s, were effective in reducing the risk of blood-borne virus transmission and enhancing the patient safety. The emergence of third-generation long-acting coagulation factors will further improve the patients’ medication compliance.

The combination of two long-lasting efficacy technologies, PEG modification and Fc fusion protein, adopted in the recombinant human coagulation factor VIII-Fc fusion protein for injection (FRSW117) allows for "once-a-week" prophylaxis medication. As the world's second and China's first ultra-long-acting recombinant factor VIII product under development, it is expected to help those with hemophilia A to return to normal life. The product possesses independent intellectual property rights, and core patents have been granted or in substantive review phase in several countries and regions in the world.

FRSW117 allows for once-a-week treatment and it can greatly improve the patients' quality of life; no factor VIII inhibitors were detected in any study subject. Data from the Phase I clinical study showed that, in two groups of subjects at different doses (25 IU/kg and 50 IU/kg), the duration of factor VIII activity above 1% was prolonged by 2.59 and 2.49 times, respectively, when compared with the existing commercially available product (ADVATE®). And the half-life of factor VIII in both groups was 2.129 and 2.232 times that of the ADVATE®, respectively.