Eloxx aims to take lead can­di­date in­to PhI­II for rare dis­ease

Eloxx Phar­ma­ceu­ti­cals said it’s hop­ing to take its lead can­di­date in­to a Phase III clin­i­cal tri­al for an ul­tra-rare dis­ease af­ter the same drug failed a Phase II study in cys­tic fi­bro­sis.

The RNA biotech an­nounced Wednes­day that it is plan­ning on tak­ing small mol­e­cule can­di­date ELX-02 in­to a piv­otal tri­al for pa­tients with Al­port syn­drome with non­sense mu­ta­tions, a rare ge­net­ic con­di­tion char­ac­ter­ized by pro­gres­sive loss of kid­ney func­tion. The com­pa­ny says there are about 9,400 to 13,000 pa­tients who have this form of Al­port syn­drome, for which there are no ap­proved treat­ments.

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