Pfizer Inc. (NYSE:PFE) and Sangamo Therapeutics Inc. (NASDAQ:SGMO) have announced that their gene therapy was effective in maintaining Factor VIII inhibitors in patients with hemophilia A disorder where blood does not easily clot for 14 months.
Giroctogene fitelparvovec therapy shows encouraging results
The companies indicated that all the five subjects in the early stage study received a one-off giroctogene fitelparvovec treatment. The patients did not exhibit spontaneous bleeding events or need an infusion of lacking the blood-clotting protein. The therapy the companies are developing is among the several treatments currently under development for the treatment of hemophilia A. It will be an option to the current standard care that necessitates regular infusion of the clotting protein to replace Factor VIII.
The companies released the phase I/II Alta study data on giroctocogene Fitelparvovec ahead of the planned third phase trial. Results indicated that patients in the Alta study that were treated with 3e13vg/kg dose produced the Factor VIII inhibitors that prevent clotting. None of the patients required Factor VIII infusion or experienced bleeding episodes. The levels of Factor VIII show measurements of up to 61 weeks based on data from the group’s longest treated patient. Pfizer and Sangamo presented the updated data from the Alta study as an oral abstract during this year’s World Federation of Haemophilia Congress.
Late-stage study dosing to commence in the second half of 2020
Pfizer’s SVP and Rare Disease Research Unit Chief Scientific Officer Seng Cheng indicated that the company was delighted that the data validated previous results from the Phase1/2 study. Cheng added that they were encouraged by giroctocogene fitelparvovec’s potential to demonstrate durability in the long term, which is vital for hemophilia A patients.
The therapy is meant to help patients produce Factor VIII on their own as well as stop the painful joints and muscles bleeding. This is typical of hemophilia and can lead to severe joint damage with time. According to the companies, they expect to commence patient dosing in the late-stage study in the second half of 2020.