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ProQR's lead product candidate successful in early-stage CF study; shares ahead 33% premarket

Published 10/27/2016, 08:56 AM
ProQR's lead product candidate successful in early-stage CF study; shares ahead 33% premarket
  • Thinly traded micro cap ProQR Therapeutics is up 33% premarket on increased volume in response to its announcement of successful results in a Phase 1 proof-of-concept study, PQ-010-002, assessing lead product candidate and Fast Track-tagged QR-010 in homozygous deltaF508 cystic fibrosis (CF) patients. Top-line data will be presented today at the North American Cystic Fibrosis Conference in Orlando, FL.
  • The study met its primary endpoint (exploratory efficacy) of total chloride response as measured by nasal potential difference (NPD) from baseline through the end of the study (baseline, Week 2, Week 4 and three weeks after treatment). Specifically, in seven CF patients homozygous for the deltaF508 mutation treated with QR-010, the average change from baseline in NPD was -4.1 mV (p=0.0389). The finding was supported by a change in sodium channel activity and other sensitivity analyses of the NPD measurements which indicated restoration of CFTR activity.
  • QR-010 was safe and well-tolerated.
  • Additionally, all four single-dose cohorts have been completed in another early-stage study, Phase 1b PQ-010-001. The multiple-dose cohorts are ongoing. Top-line data on safety, tolerability and exploratory efficacy are expected in mid-2017.
  • CF is caused by mutations in a protein called CFTR. QR-010 is an inhaled therapy designed to repair the genetic defect in RNA which will result in the synthesis of a normal healthy CFTR protein.

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