Business
ProQR Announces Fourth Quarter and Full Year 2019 Results and Provides Business Update
Substantial progress in 2019 with final data from Phase 1/2 trial of sepofarsen for Leber’s congenital amaurosis 10 showing rapid, significant and durable
About this update from Proqr Therapeutics N.v.
[{"type":"text","content":"Substantial progress in 2019 with final data from Phase 1/2 trial of sepofarsen for Leber’s congenital amaurosis 10 showing rapid, significant and durable improvements in vision; Phase 2/3 pivotal trial Illuminate initiated with data expected in H1 2021Phase 1/2 Stellar trial initiated for QR-421a for Usher syndrome type 2A exon 13; on track to report first clinical data for QR-421a in late Q1 2020Initiated a first-in-human trial for the third inherited retinal disease program, QR-1123 for autosomal dominant retinitis pigmentosa€48.6 million net proceeds from public offering in 2019 extended cash runway into 2022 \n LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 26, 2020 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today reported its financial results for the fourth quarter and full year ended December 31, 2019 and provided a business update. “We made significant progress in 2019 as we advanced our portfolio of investigational RNA therapies for patients that suffer from inherited retinal diseases and are in a strong position as we enter 2020,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “In 2019 we shared positive Phase 1/2 data for sepofarsen in LCA10 and started the pivotal Phase 2/3 Illuminate trial for this program. We advanced two additional programs to Phase 1/2 clinical testing – QR-421a for Usher syndrome and non-syndromic RP, and QR-1123 for adRP. We strengthened our Supervisory Board and Leadership Team, and extended our cash runway into 2022 with an upsized public offering in October.” Mr. de Boer continued, “We expect to build on this momentum with a number of anticipated events in 2020. We are on track to report the first clinical data from the Phase 1/2 Stellar trial for QR-421a in late Q1. This interim analysis will include data from the first two dosing cohorts and is intended to inform next steps in development. We also plan to provide an update from the InSight open label extension study of sepofarsen in H2 2020, including data from the contralateral eye treatment. In anticipation of the pivotal data readout from the Illuminate trial of sepofarsen in H1 2021 we are preparing for the planned NDA submission and continue to build our commercial capa...