Eloxx re­ports PhII cys­tic fi­bro­sis fail as the biotech weighs its op­tions

A rare dis­ease biotech has fall­en short in a clin­i­cal tri­al — and in­vestors are none too hap­py about it.

Eloxx Phar­ma­ceu­ti­cals re­port­ed Wednes­day that its small mol­e­cule can­di­date ELX-02 com­bined with iva­caftor failed a small Phase II tri­al. The com­bi­na­tion tri­al test­ed the two drugs in pa­tients with Class 1 cys­tic fi­bro­sis and at least one “non­sense mu­ta­tion.” Iva­caftor is one-half of Ver­tex’s cys­tic fi­bro­sis block­buster drug Orkam­bi.

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