Pro­QR shares spike on pos­i­tive PoC study for lead CF drug

Shares of the Dutch biotech Pro­QR Ther­a­peu­tics $PRQR shot up this morn­ing af­ter it re­leased up­beat re­sults from an ear­ly proof-of-con­cept study for its cys­tic fi­bro­sis drug QR-010.

“Pa­tients with CF feel and do bet­ter when the CFTR pro­tein chan­nel works more nor­mal­ly,” said Noreen R. Henig, chief de­vel­op­ment of­fi­cer of Pro­QR, in a state­ment. “Our im­por­tant first step in help­ing pa­tients with CF was to demon­strate that QR-010 could re­store CFTR func­tion in pa­tients with CF due to ∆F508, the most com­mon mu­ta­tion. Our proof-of-con­cept NPD study did ex­act­ly that in CF pa­tients ho­mozy­gous for ∆F508; it demon­strat­ed that CFTR pro­tein chan­nels are ac­tive in this co­hort fol­low­ing ad­min­is­tra­tion of QR-010 as mea­sured by the to­tal chlo­ride re­sponse. Hav­ing achieved this ma­jor step, we have in­creased con­fi­dence in QR-010’s po­ten­tial to make a mean­ing­ful clin­i­cal im­pact for pa­tients and will move for­ward with an ag­gres­sive de­vel­op­ment plan.”

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