Updated results from the Alta trial show that a single infusion with the highest dose of SB-525, an investigational gene therapy, yields dose-dependent and durable increases in clotting factor VIII (FVIII). The trial, in adults with severe hemophilia A , found no bleeding episodes up to 24 weeks following the infusion.<\/p>\n
That highest dose of SB-525 31013 vector genomes, vg\/kilogram, kg led patients to reach normal FVIII activity. Participants no longer needed replacement therapy following a short preventive course post-SB-525-administration.<\/p>\n
With these promising results, Pfizer has initiated a lead-in study (NCT03587116) to support SB-525 advancement to a Phase 3 registrational clinical trial. The six-month study will evaluate the current efficacy and safety of preventive replacement therapy in the usual care setting. It is currently recruiting participants worldwide.<\/p>\n
The Alta trials most recent findings will be shared at the upcoming 61st Annual Meeting of the American Society of Hematology (ASH), to be held Dec. 7-10 in Orlando, Fla.<\/p>\n
Data will be featured in a poster titled Updated Follow-up of the Alta Study, a Phase 1\/2, Open Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 Gene Therapy in Adult Patients with Severe Hemophilia A.<\/p>\n
SB-525 is a gene therapy candidate to treat hemophilia A thats being developed by Sangamo Therapeutics in collaboration with Pfizer. It consists of a DNA sequence coding for the production of a working FVIII the clotting factor missing in hemophilia A. That FVIII is carried and delivered to liver cells, where clotting factors are produced, using a harmless adeno-associated viral (AAV) vector.<\/p>\n
The goal of the therapy is for patients to regain the ability to continuously produce the coagulation factor, and reduce or eliminate the need for FVIII replacement therapy.<\/p>\n
The therapys safety and effectiveness for the treatment of adults with severe hemophilia A are currently being evaluated in the open-label Phase 1\/2 Alta trial (NCT03061201).<\/p>\n
The study is testing a single infusion into the vein (intravenous) of one of four ascending doses of SB-525: 91011 vg\/kg; 21012 vg\/kg; 11013 vg\/kg; and 31013 vg\/kg. Two people have been dosed per group, except for the highest dose group, which was expanded to five patients.<\/p>\n
Updated trial data now released show the results for the two patients dosed in the third group those given 11013 vg\/kg and the five individuals receiving the highest dose of 31013 vg\/kg.<\/p>\n
In the third group, a single infusion of SB-525 resulted in stable and clinically relevant increases in FVIII activity.<\/p>\n
Stronger results were seen with SB-525s highest dose. Of the five patients treated, data were available for four. For these participants, a single infusion with the highest dose of SB-525 led to normal FVIII levels with no bleeding events reported up to 24 weeks post-administration. These individuals no longer needed replacement therapy after the initial prophylactic period of up to about three weeks after SB-525 dosing.<\/p>\n
In addition, preliminary tests from the high-dose group indicate similar activity of SB-525-derived FVIII and the clotting factor provided by Xyntha, Pfizers recombinant therapy for hemophilia A.<\/p>\n
As to safety, one patient had treatment-related serious adverse events, namely low blood pressure and fever, occurring about six hours after infusion. These effects resolved with treatment within 24 hours, with no loss of FVIII expression.<\/p>\n
Some patients also showed elevated blood levels of liver enzymes(ALT, alanine aminotransferase). However, these were reported to be mild and temporary increases, which were treated in a timely manner with corticosteroids.<\/p>\n
Dosing in the fourth group is ongoing. At the upcoming meeting, Sangamo will disclose additional analyses of the trial data, including a follow-up of approximately 4 to 11 months after treatment.<\/p>\n
The rapid kinetics of Factor VIII expression, durability of response, and the relatively low intra-cohort variability in the context of a complete cessation of bleeding events and elimination of exogenous Factor VIII usage continues to suggest SB-525 is a differentiated hemophilia A gene therapy, Bettina Cockroft, MD, MBA, chief medical officer of Sangamo said in a press release.<\/p>\n
We are pleased with the progress of the program toward a registrational Phase 3 study led by Pfizer, who announced it has enrolled its first patient in the 6-month Phase 3 lead-in study. We have recently completed the manufacturing technology transfer to Pfizer and initiated the transfer of the IND [investigational new drug].<\/p>\n
Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.<\/p>\n
Total Posts: 121<\/p>\n
Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.<\/p>\n
<\/p>\n
View original post here: <\/p>\n
Updated Alta Trial Results Support SB-525 Gene Therapy for Hemophilia A - Hemophilia News Today<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" Updated results from the Alta trial show that a single infusion with the highest dose of SB-525, an investigational gene therapy, yields dose-dependent and durable increases in clotting factor VIII (FVIII). The trial, in adults with severe hemophilia A , found no bleeding episodes up to 24 weeks following the infusion Continue reading