The data shows that despite patients being enrolled into the Balance study that were already on Fabryzyme for an average of more than 4 years and with high Antibody activity against Fabryzyme (and hence continues decline in renal function), when these patients were switched to pegunigalidase, the Antibody activity was significantly reduced.
- IgG: from average 23,330 Fabryzyme to 9,418 pegunigalidase (note less IgG is better) - IgG, dropping more than half (60% drop). - serum neutralising Ab: from average 83.58% Fabryzyme enzyme inhibition, to 61.65% pegunigalidase enzyme inhibition, (again less inhibition is better) - nAB activity is a net 21.93% drop, meaning a 26% reduction in enzyme inhibition.
And most importantly. Residual active enzyme from 16.42% Fabryzyme to 38.35% active enzyme for pegunigalidase. A net 21.93% increase in enzyme activity, but a very nice 130% increase in enzyme activity of pegunigalsidase over Fabryzyme.
Thus after the timecourse of these tests, pegunigalidase had 130% greater maintained active enzyme than when these patients were on Fabryzyme.
This should mean:
1. That existing patients patients should be able to switch over from Fabryzyme to pegunigalidase and have a better efficacy of the treatment.
2. Patients that are naive and start on pegunigalidase (without having had anti-Fabryzme antibodies already built up in their system) could get the greatest benefit from pegunigalidase therapy.
Both of these substantiate the idea that pegunigalidase would be the better treatment for existing and naive patients. Of while course this needs to be finally proved in final clinical trial results, this does provide early data to support pegunigalidase in its claims.
Also importantly this definitely puts on the right path for at least meeting the endpoints from the EMA of Comparability against Fabryzyme, as well as a strong logic for Superiority for the FDA. Good news indeed.
What is also good to read from this PR, is that: "In addition, the results generated in a recent analysis of 60 patients screened for the BALANCE study to date, including the 37 patients analyzed for the poster presentation, have been substantially in-line with the above analysis implying that the remaining effective enzyme in blood samples was more than double for pegunigalsidase alfa compared to Fabrazyme."
This new data furthers corroborates findings in the Hughes and Warnock posters data from this past February 2018, and combined make things looks very attractive indeed.
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