Hunt­ing a cure, Spark re­ports steady progress on he­mo­phil­ia B gene ther­a­py as Pfiz­er preps for piv­otal hand­off

Af­ter some fine tun­ing on the man­u­fac­tur­ing side, Spark Ther­a­peu­tics $ONCE says it’s racked up a new round of promis­ing re­sults for its Phase I/II study of a ground­break­ing new gene ther­a­py that aims to cure he­mo­phil­ia B. And the biotech is prep­ping a hand­off of SPK-9001 to its Big Phar­ma al­lies at Pfiz­er $PFE this sum­mer, who are plan­ning to start a Phase III piv­otal study.

Re­searchers are now re­port­ing on 15 pa­tients in the ear­ly-stage study get­ting a re­place­ment fac­tor IX gene to cor­rect the mu­ta­tion that re­quires con­stant care and fac­tor re­place­ment ther­a­py to pre­vent bleeds. The an­nu­al­ized bleed rate for all of them was cut by 97% and re­searchers were care­ful to high­light three pa­tients who are do­ing fine — with fac­tor IX lev­els rang­ing from 38.1% to 54.5% be­yond the 12-week mark— af­ter be­ing treat­ed with an en­hanced ther­a­py pro­duced by a new man­u­fac­tur­ing ap­proach.

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