Pfizer B hemophilia gene therapy SPK-9001 I/II clinical harvest exciting data
May 26, 2016 Source: Bio Valley
Window._bd_share_config={ "common":{ "bdSnsKey":{ },"bdText":"","bdMini":"2","bdMiniList":false,"bdPic":"","bdStyle":" 0","bdSize":"16"},"share":{ }};with(document)0[(getElementsByTagName('head')[0]||body).appendChild(createElement('script')) .src='http://bdimg.share.baidu.com/static/api/js/share.js?v=89860593.js?cdnversion='+~(-new Date()/36e5)];Pfizer and partner Spark Therapeutics, the US pharmaceutical giant, recently announced that SPK-9001, a type B hemophilia gene therapy developed by the two companies, has obtained exciting data in a phase I/II clinical study. SPK-9001 is a lead compound in the gene therapy SPK-FIX project promoted by both parties and is currently being developed for the treatment of type B blood diseases. Pfizer said it will release detailed data at the 21st European Hematology Association (EHA) conference on June 11.
SPK-9001 is a novel bioengineered adeno-associated virus (AAV) capsid that expresses a codon-optimized, highly active human factor IX variant, a proprietary Spark company. Highly optimized gene therapy for technology platform selection, design, production and formulation.
In the Phase I/II clinical study, subjects received an intravenous infusion of SPK-9001. Data from the first three subjects showed that a single intravenous infusion of 5x10 (11th power) vg/kg of SPK-9001 successfully achieved long-lasting, therapeutic levels of clotting factor IX expression. The level exceeded the threshold IX level considered to be sufficient to reduce the risk of joint bleeding and prophylactic infusion of coagulation factors.
Among them, 2 subjects without a history of previous liver disease, the level of factor IX in the blood increased continuously during the first 4 weeks after a single infusion of SPK-9001. In the case of data submitted to EHA, 1 case was infused. The latter 8 weeks were stable at 28% of normal IX levels, and the other 1 was stable at 30% of normal IX levels 7 weeks after infusion. The third patient had a history of previous liver disease. After infusion of SPK-9001, blood factor IX levels also continued to increase, and remained stable at 16% of normal IX levels 3 weeks after infusion.
Data from a natural history of patients with hemophilia B indicate that when the level of factor IX activity in the patient's blood circulatory system is maintained at a certain threshold (≥12% of normal IX levels), it is considered sufficient to reduce the risk of joint bleeding and prevention The need for sexual infusion of clotting factors.
In the summary submitted to EHA, none of the three subjects received conventional factor IX infusion to prevent bleeding events in a total 28-week observation. Only one subject received a prophylactic infusion for suspected ankle hemorrhage within 2 days after infusion of SPK-9001. In the study, SPK-9001 was well tolerated and none of the subjects required or received immunosuppressive therapy.
Pfizer said the preliminary data is very encouraging and supports the hope that hemophilia gene therapy will achieve a one-time intravenous infusion to eliminate the need for regular infusion of factor IX control and prevention of bleeding events. The study predicts that treatment levels of expression repair can be achieved by a single intravenous infusion of low doses of highly optimized gene therapy while avoiding the need for immunosuppressive therapy.
Hemophilia B is a rare hereditary blood disease with approximately 4,000 patients in the United States and approximately 26,000 patients worldwide. Currently, the treatment of hemophilia B requires repeated intravenous infusion of plasma-derived or recombinant Factor IX to control and prevent bleeding episodes.
Pfizer and Spark reached a strategic partnership in 2014 to develop hemophilia gene therapy. Under the agreement, Spark will be responsible for all Phase I/II clinical studies of all candidate products in the SPK-FIX project, and Pfizer will be responsible for key clinical research, regulatory issues, and potential global commercialization activities.
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