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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