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.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER