ProQR decimated as sepofarsen misses goal

Dutch rare diseases biotech firm ProQR Therapeutics (Nasdaq: PRQR) saw its shares plummet more than 74% to $1.45 today, as it announced that its pivotal Phase II/III Illuminate trial of sepofarsen for the treatment of CEP290-mediated Leber congenital amaurosis 10 (LCA10) did not meet its primary endpoint of best corrected visual acuity (BCVA) at month 12.

“Given the results observed in earlier studies of sepofarsen, the Illuminate trial results are unexpected and disappointing, especially for people living with LCA10,” said Daniel de Boer, founder and chief executive of ProQR Therapeutics. “ProQR was founded with the goal of developing RNA therapies for patients with high unmet medical need, and we will continue to advance our robust pipeline of therapies for genetic eye disease,” he added.

In 2019, the European Medicines Agency (EMA) granted PRIority MEdicines (PRIME) status to a drug as a potential treatment for LCA10), as well as Rare Pediatric Disease (RPD) designation from the US Food and Drug Administration.