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PRQR

ProQR Therapeutics

$6.18 /

+0.22 (+3.69%)

06:08
10/10/19
10/10
06:08
10/10/19
06:08

ProQR Therapeutics' sepofarsen shows vision improvement in Phase 1/2 results

ProQR Therapeutics announced 'positive' top-line results from the PQ-110-001 study, a Phase 1/2 dose range finding, first-in-human trial of sepofarsen in patients with Leber's congenital amaurosis 10 due to the p.Cys998X mutation in the CEP290 gene. "We reported today that patients receiving sepofarsen had a clinically meaningful improvement in vision, and in some cases the patient's vision improved to a level that could be deemed life changing (...)" said Stephen Russell, Principal Investigator at the University of Iowa. "LCA10 is a severe inherited retinal disease that leads to blindness, and for which there is currently no treatment." David Rodman, ProQR's Executive VP of R&D, said, "(...) The top-line data from this study strengthen our confidence in the design of the ongoing Phase 2/3 trial, which could be the sole registration trial for the sepofarsen program (...) " Top-line data is expected in the first half of 2021. The 12-month top-line results from the Phase 1/2 trial confirm durable activity of sepofarsen for up to one year in patients with LCA10. Results also support assumptions . The target registration dose was associated with a clinically meaningful and statistically significant improvement in vision and had a favorable benefit/risk profile, a six-month dosing frequency, as used in Illuminate, was associated with durable improvements in vision, the response observed at 12 months in the target registration dose was equal to or greater than the response at the 3-month interim analysis, and the study population were more likely to respond to treatment with sepofarsen. Sepofarsen was observed to be well-tolerated with manageable safety findings.

  • 11

    Oct

PRQR ProQR Therapeutics
$6.18 /

+0.22 (+3.69%)

10/09/19 Cantor Fitzgerald
ProQR shares could more than double on tomorrow's data, says Cantor Fitzgerald
03/27/19 Cantor Fitzgerald
ProQR Therapeutics spin-out 'smart, but not surprising,' says Cantor Fitzgerald
02/20/19 Citi
Citi opens 90-day catalyst watch on ProQR Therapeutics
01/14/19 Cantor Fitzgerald
Cantor highlights BioMarin, ProQR after JPMorgan Healthcare Conference

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