Challenging rare eye indication sinks a second modality

A Phase II/III miss for ProQR’s antisense oligonucleotide therapy sepofarsen marks the second blow in less than a year for patients with LCA10, a rare eye disease that is proving to be a challenging proof-of-concept indication for new modalities.

ProQR Therapeutics N.V. (NASDAQ:PRQR) lost $313 million in market cap, its shares falling 75% to $1.39 on Friday, after sepofarsen missed the primary and secondary endpoints in the Illuminate study in 36 patients with LCA10 caused by mutations in the CEP290 gene...